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The University of Texas MD Anderson Cancer Center announced on Nov. 8, 2024 that it is creating the Institute for Cell Therapy Discovery & Innovation for the development of cell therapies for cancer, autoimmune diseases, and infections. The institute will bring scientists and clinicians together to provide insight on immunology and cell engineering with $80 million support from philanthropic organizations and institutions.

Led by Katy Rezvani, vice president and head of the Institute for Cell Therapy Discovery & Innovation, the new institute will integrate MD Anderson’s different research areas with the goal of accelerating discovery and development of new cell therapies from preclinical through clinical phases. This effort includes the James P. Allison Institute and Institute for Data Science in Oncology. External researchers and bio/pharmaceutical companies will also be part of the collaborative effort.

“Cell therapies have delivered profound results for patients with hematologic cancers, but there remain too many patients that do not benefit. It is critical to our mission to bring these promising therapies forward as options for patients with cancer and other conditions,” Rezvani said in a press release (1). “Through our discoveries, we aim to provide many more patients with effective cell therapies that are available as off-the-shelf treatment options, limiting the need for lengthy hospital stays and associated costs.”

“MD Anderson clinicians and researchers have been instrumental in shaping the cell therapy field, and we are proud to take this important step into the future,” said Peter WT Pisters, president of MD Anderson, in the release. “The Institute for Cell Therapy Discovery & Innovation is a key piece of our institutional strategy, as we focus on cancer breakthroughs to improve patient outcomes. Our world-class facilities, exceptional talent and unique research environment will enable our teams to deliver transformational new medicines and to further our efforts to maximize our impact on humanity to finally end cancer.”

Cell therapies use a patient’s immune cells as building blocks to create treatments. MD Anderson has performed clinical trials on FDA-approved chimeric antigen receptor (CAR) T cell therapy products targeting CD19 and BCMA for B-cell lymphomas, leukemias, and multiple myeloma. The institute also advanced CAR natural killer (NK) cells into clinical studies patients with lymphoid malignancies, based on cord blood-derived CD19 CAR NK cell therapies developed by Rezvani and her team. Additional research by the group has been done for cell therapies in solid tumors, including adoptive T-cell receptor therapy targeting MAGE-A4 for patients with multiple solid tumor types.

“For years, our talented scientists and clinicians have been at the forefront of discovery, understanding and development of novel cell therapies,” said Giulio Draetta, chief scientific officer at MD Anderson, in the release. “By integrating with experts across the research and clinical enterprise, the Institute for Cell Therapy Discovery & Innovation will be able to create important new treatments, tailored to unmet patient needs, in ways only possible here.”

MD Anderson. MD Anderson Launches Institute for Cell Therapy Discovery & Innovation to Deliver Transformational New Therapies. Press Release Nov. 8, 2024.

Articles

The new Institute for Cell Therapy Discovery and Innovation will utilize expertise to develop cell therapies for cancer, autoimmune diseases, and infections.

MD Anderson Launches Cell Therapy Institute

Felicity Thomas is the former Associate Editorial Director of Pharmaceutical Technology, Pharmaceutical Technology Europe, BioPharm International.

Flexibility and adaptation concept. Arrows on cubes following a flexible path | Image Credit: © Cagkan - stock.adobe.com

Flexibility in pharmaceutical manufacturing is becoming an increasingly pertinent requirement. Various industry trends, such as the development of increasingly complex and novel therapies, a greater focus on orphan drug designation, and the use of more highly potent ingredients, are leading to demand for adaptable and versatile processing equipment.

“For decades, large-scale drug production was the industry standard. However, the rise of rare diseases now demands increasingly expensive medications, which, in turn, require greater research and development efforts, including innovations in machine design,” remarks Andreas Mattern, vice-president Strategy and Global Product Management Pharma at Syntegon. “For example, there is now a need for machines capable of processing small and micro batches with greater flexibility. At the same time, new drugs like anti-obesity medication are sustaining the demand for high-performance equipment that can handle large batch volumes.”

“There have been significant advances in flexible manufacturing to accommodate the rapidly changing therapeutic landscape, particularly in response to the rise of personalized medicine, biologics, gene and cell therapies, and the need for rapid responses to health crises (like the COVID-19 pandemic),” specifies Caterina Funaro, Competence Center manager at IMA Active. “These advances are helping the pharmaceutical and biotech industries adapt to smaller batch sizes, varied production processes, and the need for faster, more adaptable production cycles. Key innovations in processing equipment, facility design, and digital technologies are enabling greater flexibility in manufacturing.”

Specialized treatments are certainly driving new equipment developments, confirms Mattern, who points out that personalized therapies are becoming a focus for future treatment breakthroughs. “Many types of cancer and genetic disorders can only be treated with highly specialized drugs,” he says. “These [drugs], in turn, require production in ever smaller batch sizes.”

New therapeutic modalities, such as cell and gene therapies, require flexible equipment that can handle fast changeovers from one product batch to the next, Mattern continues. “A wide variety of products must be filled into different container sizes and types such as vials, syringes, or cartridges. Hence, it comes as no surprise that the market for ready-to-use (RTU) containers has been growing at a rapid pace over the past years,” he says.

Benefits of RTU containers include reduced time-to-market, lower cost of ownership, increased flexibility, and greater integrity of the drug product, explains Mattern. “While RTU containers have been the de-facto standard for pre-filled syringes for some time, they are now also increasingly being used for vials and cartridges in both bulk and small batch applications,” he states.

A general move away from traditional approaches to manufacturing has been trending for a while, comments Funaro. “Continuous manufacturing (CM) has gained traction as an alternative to traditional batch production,” she adds. “[CM] provides greater flexibility by allowing production to run continuously, without the need for lengthy shutdowns between batches, which is especially beneficial for both small- and large-scale manufacturing.”

“The industry has made significant progress in adopting continuous processes, which are progressively replacing traditional batch processes, particularly in the pharmaceutical, chemical, and food processing sectors,” explains Funaro. “Continuous processes offer enhanced efficiency, higher throughput, better product consistency, and improved energy use, driving their adoption across industries.”

For pharmaceuticals, manufacturers are employing continuous processes to API production, granulation, mixing, tableting, and coating, remarks Funaro. Through using continuous processes for these tasks, companies are being able to reduce lead times, improve product consistency, and facilitate any potential scale changes that may be required to meet therapeutic demand, such as increased production of vaccines or biologics, she confirms.

Within the oral solid dosage (OSD) space, CM has been trending for a few years, Mattern adds. “Higher flexibility, shorter development times with minimum API usage, and a direct transfer from development to production without scale-up are among the primary requirements of pharmaceutical manufacturers,” he says. “The main challenge with traditional solutions is the precise dosing of the starting materials in a constant mass flow rate of milligrams per second.” However, as the pharmaceutical industry is highly regulated, adopting newer processes can take time, Mattern specifies.

“FDA has endorsed continuous processes, recognizing their potential for faster production and more consistent product quality,” asserts Funaro. “As a result, manufacturers are investing in equipment such as continuous coaters and continuous mixers.”

Additionally, while investment in continuous processes grows, advances are also being seen in real-time data monitoring tools, which are vital in helping to control variables in CM, such as temperature, pressure, and product quality, continues Funaro. “Real-time analytics are increasingly being integrated with machine learning (ML) algorithms to allow for self-regulating systems that automatically adjust process parameters to maintain optimal efficiency and output quality,” she says.

“The shift from batch to continuous processes is well underway, but ongoing advances in processing equipment will further accelerate the transition,” asserts Funaro. “As technologies such as real-time monitoring, modular designs, AI [artificial intelligence] integration, and energy-efficient systems mature, industries will be able to harness the full potential of continuous processes, resulting in lower costs, higher productivity, and improved sustainability.”

“Automation is playing an increasingly critical role in processing equipment across industries such as pharmaceuticals, food and beverage, chemicals, and biotechnology,” explains Funaro. “The current trends in automation are transforming manufacturing processes by enhancing productivity, improving consistency, ensuring quality, and reducing human error.”

Through the use of automation, manufacturers can achieve a multitude of benefits. For example, Funaro points out that automated material handling, packaging, and processing reduces human intervention and speeds up cycle times. Additionally, she alludes to the fact that automation reduces variability in production processes, which ensures consistent quality.

For Funaro, the use of digital twins, which are virtual replicas of physical processes or equipment, will become more widespread within industry over time. “Digital twins allow manufacturers to simulate process changes, predict how equipment will behave under different conditions, and optimize production before making physical adjustments,” she says.

“Predictive maintenance is becoming a standard practice, leveraging AI, IoT [Internet of Things], and analytics to predict when equipment will fail, and schedule maintenance before a breakdown occurs. This reduces unplanned downtime and extends the lifespan of processing equipment,” adds Funaro. “As predictive maintenance becomes more advanced, it will shift toward prescriptive maintenance, where AI not only predicts failures but also suggests the optimal maintenance actions to take, further minimizing disruption and cost.”

While digitalization is producing large amounts of data in production every day, it will mean nothing if it cannot be viewed or used, Mattern warns. “New cloud-based software solutions, such as Synexio from Syntegon, provide a remedy by enabling the acquisition, evaluation, and visualization of equipment and production data,” he says. “[These solutions] allow for real-time monitoring of machine conditions, helping manufacturers make data-based decisions and optimize operations.”

“In sectors like pharmaceuticals, where regulatory compliance and precision are critical, automation ensures that products meet exacting standards,” continues Funaro. “Automated quality control systems, including automated visual inspection and process analytical technology (PAT), are reducing the risk of human error and ensuring that processes stay within defined limits.”

Reducing human intervention in processes is particularly helpful in high-risk environments, such as dealing with hazardous chemicals or highly potent ingredients, asserts Funaro.

“The use of robotics is taking the industry a huge step further towards even higher product protection and quality, as it eliminates the main cause for contamination (i.e., human intervention),” Mattern remarks. “This is further fueled by [the European Union’s] Annex 1 [guidance].”

In August 2023, the updated EU good manufacturing practice (GMP) Annex 1 guidance (1) came into operation (except for point 8.123), which has profound implications for sterile manufacturing across the global pharmaceutical sector, Mattern specifies. “One of the key challenges presented by the new Annex 1 is the requirement to separate the aseptic processing area from the operator’s environment. As a result, pharmaceutical manufacturers now need at least restricted access barrier systems (RABS) to gain approval for new products,” he says.

After the updated guidance was published, industry saw a surge in upgrades to existing cleanroom lines to incorporate RABS, Mattern notes. “Manufacturers must choose whether to upgrade their current cleanroom line for a new product or invest in new filling equipment with isolator technology or RABS,” he specifies.

“It is expected that isolator technology will become the new standard, given the added safety provided by automatic bio-decontamination processes and the pressure differential from the operator environment,” Mattern notes. “[Whereas], RABS will likely remain a preferred option for more frequent product changes and experienced operators.”

The second chapter of the revised Annex 1 guidance places a focus on automation and robotic systems, highlighting these as “Appropriate Technologies” (1), Mattern reveals. “These technologies aim to reduce, or even eliminate, the need for gloves and human intervention in barrier systems,” he says.

“Gloves pose several challenges, which are addressed in dedicated paragraphs of the [Annex 1] document,” Mattern continues. “For instance, during longer production campaigns, [gloves] must be tested in operation—a process that is difficult to achieve with current glove testing equipment without compromising the sterile environment. The shift toward gloveless systems, utilizing robotics for these tasks, is viewed as the ideal solution.”

There are several potential future trends that are set to impact processing equipment and pharmaceutical manufacturing in general, asserts
Funaro. “These trends are being driven by technological innovation, sustainability goals, and the need for enhanced efficiency, safety, and compliance,” she says.

Notably, automation and Industry 4.0 are set to shape the future, explains Funaro. For example, employing predictive maintenance to anticipate equipment failures before they happen, utilizing AI and robotics for fully autonomous processing systems, integrating advanced sensors and machine vision systems for greater precision and control, and analyzing vast amounts of data with ML and AI, she summarizes

“Future equipment will increasingly incorporate self-learning systems that can adjust their operation based on past performance data. This will enable greater flexibility and adaptability in production lines, particularly in industries that need to switch between different products or processes frequently,” says Funaro. “As demand for customization and flexibility grows, processing equipment will increasingly be designed with modularity in mind.”

“A persistent concern within the pharmaceutical industry and its suppliers is the need for more sustainable equipment and processes. Unfortunately, resource consumption in the sector continues to surpass that of many other industries,” stresses Mattern. “In response, drug manufacturers are intensifying efforts to reduce their carbon footprint, with the support of manufacturing companies offering services such as lifecycle assessments for their equipment.”

Adoption of more energy-efficient equipment and an overall reduction of environmental footprint is becoming a greater focus for manufacturers, confirms Funaro. “Recyclable and reusable components in machinery, as well as the use of environmentally friendly materials, are becoming more widespread,” she says.

“Given its longevity, equipment usually contributes to overall emissions over several decades,” remarks Mattern. “By continuously optimizing their machines and developing new, more energy-efficient solutions, equipment providers can help pharmaceutical manufacturing companies achieve their sustainability goals.”

“These future advances will fundamentally change how industries approach processing equipment, offering greater flexibility, efficiency, and sustainability while adapting to the needs of a rapidly evolving marketplace,” concludes Funaro.

1. EC. Annex 1 Manufacture of Sterile Medicinal Products. 

Felicity Thomas is associate editorial director for 

®
Vol. 48, No. 11
November 2024
Pages: 9–11

When referring to this article, please cite it as Thomas, F. Increasing Processing Flexibility to Meet Demand. 

Evolving industry demands are driving the need for greater flexibility and innovation in processing equipment for pharmaceutical manufacturing.

Increasing Processing Flexibility to Meet Demand

Feliza Mirasol is the science editor for 

On Nov. 6, 2024, Johnson & Johnson (J&J) announced that the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom has granted marketing authorization for Balversa (erdafitinib) to be used as a monotherapy in treating adults with unresectable or metastatic urothelial carcinoma (UC), which is the most common form of bladder cancer (1,2). This approved indication specifically covers eligible patients harboring susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations, and who have previously received at least one line of therapy containing a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor in the unresectable or metastatic treatment setting.

MHRA’s authorization is based on results from Cohort 1 of a Phase III study (THOR). The study was randomized, open-label, and spanned multiple clinical treatment centers. The study evaluated the efficacy and safety of erdafitinib (n=136) versus chemotherapy (n=130) in patients with advanced or metastatic UC with select FGFR alterations who have progressed on or after one or two prior treatments, where at least one of those treatments included a PD-1 or PD-L1 inhibitor. The study’s primary endpoint was overall survival (OS), and secondary endpoints were progression-free survival (PFS), objective response rate (ORR), and duration of response (DOR), according to a company press release.

“Patients living with this advanced stage of bladder cancer, and whose tumors harbor FGFR3 alterations, require access to innovative precision therapies that can target the specific characteristics of their disease,” said Professor Alison Birtle, consultant oncologist and honorary clinical professor, Lancashire Teaching Hospitals National Health Service (NHS) Foundation Trust, in the press release. “Unfortunately, until now, there have been limited treatment options available for this group of patients, which may affect not only their prognosis, but their wellbeing and quality of life. Today’s authorization of erdafitinib, a targeted therapy that has been shown to significantly improve overall and progression-free survival for patients with FGFR3 alterations, will come as welcome news to eligible patients, and highlights the importance of integrating biomarker testing into the treatment pathway for people with urothelial cancer, so that genetic alterations such as FGFR3 can be detected as early as possible.”

Erdafitinib is administered orally as a once-daily, FGFR kinase inhibitor. It works by inhibiting the activity of FGFR3 alterations in cancer cells and has demonstrated an ability to extend overall survival compared to chemotherapy in the second-line setting (3).

In June 2023, the Phase III study (THOR) was stopped based on the recommendation of an independent data safety monitoring committee, following a review of efficacy and safety data of the study at interim analysis (3). All patients who were randomized to chemotherapy (docetaxel or vinflunine) were offered an opportunity to receive erdafitinib as crossover therapy, and results at the data cut-off timepoint showed that median OS of over one year was achieved in those patients receiving erdafitinib. These results marked a significant improvement compared to the patients in the chemotherapy arm (12.1 months vs. 7.8 months; hazard ratio, 0.64; 95% confidence interval, 0.44 to 0.93; P=0.005) (1).

Moreover, treatment with erdafitinib showed an improvement in median PFS compared to chemotherapy of 5.6 months versus 2.7 months and a confirmed ORR of 48 out of 136 patients (35.3%) versus 11 out of 130 patients (8.5%) (1).

“We are delighted that the MHRA has recognized the value that erdafitinib could bring to eligible patients with metastatic urothelial cancer,” said John Fleming, country medical director, Johnson & Johnson Innovative Medicine UK, in the release. “This milestone reflects J&J’s long-standing dedication to getting in front of cancer and delivering the most innovative precision therapies to patients in need. We look forward to progressing with HTA [health technology assessment] submissions for erdafitinib in the coming months, with the view to enabling eligible patients to access erdafitinib through the NHS as soon as possible.”

In the UK, approximately 10,500 people are diagnosed with bladder cancer each year, equating to 29 people every day (4). Of those diagnosed with advanced or metastatic bladder cancer, roughly 20% have FGFR3 alterations, which can drive the growth of cancer cells (5).

1. MHRA. Electronic Medicines Compendium, Balversa Summary of Product Characteristics. 

 (accessed November 6, 2024).
2. Leslie, S.W.; Soon-Sutton, T. L.; Aeddula, N. R. Bladder Cancer. In 

; StatPearls Publishing, Aug. 15, 2024.
3. Loriot, Y.; Matsubara, N.; Park, S. H.; et al. Erdafitinib or Chemotherapy in Advanced or Metastatic Urothelial Carcinoma. 

 (21),1961–1971.
4. Cancer Research UK. Bladder Cancer Statistics. 

cancerresearchuk.org/health-professional/cancer-statistics/statistics-bycancer-type/bladder-cancer

 (accessed Nov. 6, 2024).
5. Xiao, J. F.; Caliri, A. W.; Duex, J. E.; Theodorescu, D. Targetable Pathways in Advanced Bladder Cancer: FGFR Signaling. 

Balversa (erdafitinib) marks the first and only bladder cancer therapy to target FGFR3 alterations, with a demonstrated increase in overall survival from 7.8 months to 12.1 months. 

J&J’s Balversa Gains MHRA Marketing Authorization as Monotherapy for Bladder Cancer

Members of academia and industry in the United Kingdom are highlighting the need for training within the pharma manufacturing field after Chancellor of the Exchequer, Rachel Reeves, announced in the Autumn Budget that there will be £520 million (US$675 million) made available to develop drug manufacturing capabilities in the UK (1). The budgetary funding allocation will begin with an increase in manufacturing capacity so that the UK can better respond to healthcare emergencies. But some are saying that the funds would be better suited in training of personnel.

“This announcement in the Budget to support life sciences is a welcome step for the UK medicines manufacturing sector,” said Professor Ivan Wall, co-director of Resilience, the UK’s first Medicines Manufacturing Skills Centre of Excellence, in a press release (2). “However, there is an acute skills shortage that must be addressed to ensure we have trained staff ready to work in the sector.”

Resilience, which was created to address the shortage of skills in the pharmaceutical industry within the UK, is a two-year project funded by the Office for Life Sciences in the UK Government’s Department for Science, Innovation & Technology that is managed through Innovate UK. The center’s interest, as would be expected, is to ensure that talent is being focused on. Resilience will be providing industry training courses that address issues such as digital technology, artificial intelligence, data analysis, and environmental sustainability. The center will be partnering with universities across the UK to develop the program, which will include in-person and remote training courses for advanced laboratory and manufacturing skills at the academic level.

“A major consideration for success in building this capability will be in ensuring a strong supply of talent to maximize productivity across new facilities,” said Wall in the release. “Talent is in short supply across the UK Life Sciences sector and that is where projects like Resilience can help, by building the ecosystem to maintain a strong, internationally competitive sector and ensure patients have access to the best drugs through the NHS [National Health Service].”

Sustainability is also a goal of the center. “There is a great opportunity to design with environmental sustainability principles embedded,” Wall said. “Around 25% of NHS emissions are in the supply chain, so our approach using VR [virtual reality] training will reduce plastics and other waste, helping the industry deliver net zero medicines manufacturing.”

Autumn Budget 2024: Fixing the Foundations to Deliver Change

2. Resilience. UK’s Medicines Manufacturing Skills Centre Welcomes Budget’s £520 million for Manufacturing but Says the Priority Must be Skills Development. Press Release. Nov. 6, 2024.

A £520 million investment for manufacturing capacity was announced by Chancellor of the Exchequer, Rachel Reeves, but academic and industry leaders stress the money should be used to train personnel.

UK Medicines Manufacturing Skills Centre Stresses Skill Development after Budget Announcement

Communication between the manufacturing plant and retail stores. | Image Credit: © Cagkan - © Cagkan - stock.adobe.com

Bespak, a contract development and manufacturing organization that specializes in inhalation products, announced on Nov. 6, 2024 that it is partnering with Orbia Advance Corporation, S.A.B. de C.V (BMV: ORBIA) Four & Energy Materials (Orbia Fluor & Energy Materials) to transition to climate-friendly inhalers. As part of the collaboration, Orbia, a supplier of fluoroproducts and technologies, will provide Bespak with its low global warming potential (GWP) propellant, Zephex 152a for use in Bespak’s pressurized metered dose inhalers (pMDIs).

Bespak is making the transition to the GWP propellant as part of the overall pMDI industry’s commitment to sustainable solutions. The company verified the use of producing pMDI formulations with GWP propellants with the assistance of its network of partners and its good laboratory practice (GLP) lab-scale testing, as well as its clinical- and commercial-scale good manufacturing practice (GMP) capabilities. Bespak will also utilize Orbia’s clinical-scale GLP and GMP capabilities.

The two companies will work closely at their locations in North West, England, with support from Bespak’s King’s Lynn, United Kingdom, location, which has conducted research on a range of valves and actuators.

“We are committed to leading the transition to low GWP propellants in pMDIs, and in collaboration with Orbia Fluor & Energy Materials, we will enable customers to progress their inhaler portfolios utilizing Zephex 152a,” said Chris Hirst, CEO of Bespak, in a press release (1). “Our goal is to convert as many formulations as possible to maintain patient access to pMDIs as global regulation evolves, and we look forward to working with Orbia Fluor & Energy Materials to provide customers with a pathway to commercialization at our Holmes Chapel site.”

“This is another step forward in bringing pMDIs using our low GWP medical propellant Zephex 152a to the market,” said Markus Laubscher, general manager of Orbia Fluor & Energy Materials Pharma Business Unit, in the release. “We are excited to extend our industry collaborations to Bespak who bring the needed capabilities to the table, from their expertise in valves and actuators, to their demonstrated commitment to clinical and commercial-scale product manufacturing.”

Bespak has made recent investments in its development services through its market approval services for all pMDI formulations and propellants. It has also increased capacity for commercial supply. In October 2024, the company announced that, in connection with H&T Presspart, it had added a GMP pilot-scale filling line for pMDIs to produce GWP propellants HFDA-152a and HFO-1234ze (2). The new filling line provide access to small-scale GMP capabilities for development programs and clinical trials.

“This new low GWP pilot-scale filling capability addresses the short-term market need for clinical trial supply and will help pharma companies to advance their low GWP development programs towards commercialization,” Hirst said in the press release. “When added to our rapidly expanding low GWP commercial-scale capability, it is clear that we can now offer customers a complete transition pathway.”

“H&T Presspart is committed to supporting the industry in transitioning to low GWP propellants,” said Christian Krätzig, president of H&T Presspart, in the release. “In our partnership with Bespak, we are now leveraging our combined expertise to create the infrastructure needed for small-scale manufacturing with low GWP propellants. The launch of a GMP pilot-scale filling line is an important step forward, and we are excited about future collaborations that will help to create a more sustainable pMDI platform for the next generation.”

Bespak. Bespak and Orbia Fluor & Energy Materials Unite to Accelerate Transition to Climate-friendly Inhalers. Press Release. Nov. 5, 2024.

Bespak. Bespak & H&T Presspart Announce Availability of GMP Pilot Line for Low GWP pMDIs. Press Release. Oct. 8, 2024.

The companies will work to create climate-friendly pMDIs using Orbia’s low global warming potential propellant.

Bespak and Orbia Fluor & Energy Materials Collaborate on Climate-Friendly Inhalers

Aditum Bio announced on Nov. 7, 2024 that it has formed a new company with Leads Biolabs, called Oblenio Bio. Oblenio Bio was formed in conjunction with an exclusive option and license agreement that the companies have entered into for the development of LBL-051, a first-in-class CD19xB-cell maturation antigen (BCMA)xCD3 tri-specific T-cell engager antibody, for treating autoimmune diseases. A US-based biotech venture firm, Aditum Bio will fund Oblenio Bio. In collaboration with Leads Biolabs, a clinical-stage biotechnology company based in Nanjing, China, the companies aim to rapidly progress LBL-051 to clinical studies.

Under the agreement, Leads Biolabs will grant the newly formed Oblenio Bio an exclusive option to develop, manufacture, and commercialize LBL-051 worldwide. In return, Leads Biolabs is eligible to receive up to $35 million in upfront and near-term payments, $579 million in development, regulatory, and commercialization milestone payments, and royalties on sales. Leads Biolabs is also entitled to receive an equity stake in Oblenio Bio.

“By targeting both CD19 and BCMA in autoimmune disorders, LBL-051 has the potential to achieve a complete immune reset and superior efficacy and durability, compared to single targeting of either CD19 or BCMA alone”, said Joe Jimenez, co-founder and managing director of Aditum Bio, in the release.

Based on recent clinical data (1,2), CD19 and BCMA targeted therapies have shown compelling efficacy in difficult-to-treat autoimmune diseases, according to a company press release. Despite the promising results, however, there remains a continued unmet need for increased efficacy and durability; thus, by targeting both CD19 and BCMA, LBL-051 offers the potential to deliver stronger and more durable responses. The therapeutic is designed to deplete a broader range of pathological B-cell populations across a wide spectrum of antibody-mediated autoimmune diseases, the companies stated in the press release.

LBL-051 is a novel tri-specific T-cell engager developed from the LeadsBody Platform. Each target binding domain—CD19, BCMA, and CD3—was engineered for the express intent of enhancing safety while optimizing efficacy. These domains have been finely tuned to enhance their individual relative potency.

“LBL-051 offers a differentiated approach to treating certain autoimmune conditions and has the potential to be a Pipeline in a Product. We are pleased to establish this partnership with the high caliber team at Aditum to bring this innovative therapy to patients around the world,” said Xiaoqiang Kang, MD, PhD, founder, chairman, and CEO of Leads Biolabs, in the release.

Oblenio Bio marks the thirteenth company launched by Aditum Bio as part of the company’s missioin to give large patient populations access to important medicines. Aditum Bio fosters an incubator model, which aims to speed time-to-market and focuses on the translational phase of drug development. The company also in-licenses promising drug candidates and spins out individual companies dedicated to progressing each candidate through to early clinical trials.

Leads Biolabs focuses on the discovery and development of innovative therapies that address significant unmet medical needs in oncology, autoimmune diseases, metabolic disorders, and other serious conditions. The company’s R&D pipeline includes more than 20 novel candidates across various modalities, including monoclonal antibodies, multi-specific antibodies, T-cell engagers, and antibody-drug conjugates.

1. Zhang, W.; Feng, J.; Cinquina, A.; et al. Treatment of Systemic Lupus Erythematosus using BCMA-CD19 Compound CAR

, 2120–2123. DOI: 10.1007/s12015-021-10251-6
2. Wang, W.; He, S.; Zhang, W.; et al. BCMA-CD19 Compound CAR T Cells for Systemic Lupus Erythematosus: A Phase 1 Open-label Clinical Trial. 

In the collaboration, the new company, Oblenio Bio, has been granted an exclusive option to license LBL-051, a tri-specific T-cell engager antibody, from Leads Biolabs.

Oblenio Bio Created by Aditum Bio and Leads Biolabs to Develop Therapeutic Antibody for Treating Autoimmune Disorders

The training function in a pharmaceutical facility is vital to site operations. The objective of training is straightforward; training is responsible for employee learning. Site training programs must be effective to accomplish employee learning. Effective training is difficult; the broad scope of topics, trainer communication skills, trainee motivation, logistics, documentation, maintenance of training quality, and other considerations may challenge effective training. Training is a cost; training in some organizations may be restricted by budget, time, facilities, conflicting priorities and criticalities, or other limitations. Identifying problematic aspects in training and appropriately addressing these considerations is crucial to accomplishing effective employee learning. 

Submitted: Aug. 4, 2024
Accepted: Aug. 29, 2024

This manuscript discusses the application of quality risk management (QRM) concepts within a site training program. QRM application helps to identify potential problematic aspects in training; these potential issues may then be addressed by following the QRM process. ICH Q9(R1) is the fundamental document describing QRM (1). Hazard identification (i.e., potential problem identification) is the first step in the QRM process. This paper focuses on problem identification within a comprehensive training process. Specific application of QRM to training and education is identified in ICH Q9(R1).

QRM application in training provides a systematic approach that complements traditional training—a thought process distinct from usual training. Typical training involves information, actions, and procedures (i.e., how to do things); training should also address understanding (i.e., why are things done). QRM identifies potential problems during training—QRM addresses what might go wrong. QRM thus improves training and makes training content more relevant. QRM application facilitates poka-yoke, mistake-proofing, defensive design, or other approaches intended to prevent problems—QRM proactively facilitates problem prevention. Adult learners in pharma are practical in orientation; QRM problem identification is harmonious with adult learning (2). Examples of potential training problems are presented throughout the following discussion:

 Training functions within pharma may be organized in different ways. Training may be the responsibility of each technical function (e.g., engineers train other engineers, chemists train other chemists, etc., within function departments); each function accomplishes their individual personnel training. Centralized training with varying levels of activities is also possible; some centralized functions may be responsible for minimal administrative activities (e.g., personnel training records); others may manage training requests, training content documents, schedule sessions, and periodically monitor training quality—a much more comprehensive responsibility. Some training organizations may be linked to corporate human resource (HR) systems to integrate government-mandated training within employee files. The following discussion addressing QRM in training utilizes a centralized function with responsibility for individualized training and administrative activities. The point of this discussion is the identification of potential problems adjunctive to typical training regardless of organizational structure.

 The role of training management is critical to training program success. Training management is responsible for actual training as well as training function management. Quality systems guidance documents (3–5) identify expectations of management; QRM considerations are also mentioned in these documents. Training management may need to mediate conflicting training time/costs vs. site criticalities with other site functions. Other management training responsibilities are also crucial for success. Site training programs often rely on personnel in other departments to accomplish training objectives; training management must ensure that these individuals know and understand their responsibilities supporting training. Training management also provides an unspoken message to trainees during training; conscientious effort in training with thorough and well-designed training content conveys a message of quality and compliance to employees. A common thread in deficient training is a lack of visible management involvement; one cannot demonstrate commitment by staying in the office and going to meetings. Training management must be “on the floor” when and where training happens (i.e., management by walking around [MBWA]) (6). Current workplace trends in Zoom management exacerbate management lack of visibility. An ongoing commitment to training excellence demonstrated by management visibility helps to develop a quality culture in the organization.

 This discussion is structured according to the FDA lifecycle approach to process validation (7). The process validation model provides a comprehensive approach to training. Process validation is designed, executed, and continually maintained; training activities can likewise follow this same approach. 

 describes three stages in a training lifecycle akin to three stages in process validation. Training stage 1 describes the design of the training topic including content, presentation, and associated elements; stage 2 comprises actual training performance by trainers and trainees; stage 3 addresses the ongoing maintenance of training (i.e., effectiveness, content accuracy, and compliant performance). A basic tenet of process validation is the identification and control of variation; training problems may include performance variation by trainers, uncontrolled logistics, and other causes. Training management should develop a training master plan akin to a validation master plan containing training strategies, approaches, procedures, documentation, and commitments. The following continues previous discussions correlating training with process validation including validation application overview, training content development, trainer qualification, trainee prerequisites, the training process, performance monitoring, management overview, and documentation (8–15). 

QRM terminology may often confuse readers based on common language within their pharma settings—specifically the words ‘hazard’ and ‘risk’. Hazard and risk are often assumed to describe dangerous workplace situations (e.g., fall, slipping, electric shock, overhead machinery, others). Per ICH Q9(R1), ‘hazard’ and ‘risk’ are not synonymous (16,17). Hazard is a source of harm; after a hazard is identified, its risk of causing harm (high, medium, low, or descriptive numeric) is assessed using risk assessment tools or subjective methods. Risk assessment describes the probability of harm occurrence and the level of harm severity; some common risk analysis methods include failure mode and effect analysis (FMEA), preliminary hazard assessment (PHA), and fault tree analysis (FTA); these tools are identified in ICH Q9(R1) Annex I. FMEA integrates frequency, severity, and detection in its Risk Priority Number (RPN), which quantifies risk. 

 more completely defines terminology as explained in Q9(R1) training modules and used in the following discussion. Q9(R1) changed the initial step in the risk assessment process to ‘hazard identification’ from ‘risk identification’. Although this change enabled consistency with the definition of hazard in other regulatory documents, it may cause confusion in common word usage. Hazard identification in training is the primary objective of the following discussion. 

 Quality risk management terminology. Hazard is a potential source of harm. Risk analysis quantifies the harm.

Stage 1 comprises the design, planning, and development of training to be executed in actual employee training. Training design must comprehensively address the topic to be trained. Stage 1 design addresses new training as well as evaluating and integrating training changes identified during subsequent training stages. When the training process is designed, conscious thought about what might go wrong is integrated with the how and why being trained. Training design is the most important activity in the training process and may be complex depending on the subject topic. The following briefly describes elements of training designed in stage 1:

Content, presentation, testing, and process

. Numerous content categories (e.g., policies, procedures, equipment operation, analytical, etc.) are required for pharma employee training. Content presentation describes the method of delivering content; computer delivery (e.g., one-way delivery), virtual reality, Zoom interactions, classroom training, and skill delivery (i.e., on-the-job [OJT] training with a subject matter expert [SME]) are general categories. Testing to verify effective training is related to content complexity; “read and sign,” multiple choice, and skills demonstration to an SME are options.

 Supporting elements in training design include the trainer, trainee, and logistics. Background, technical expertise, and communication skills of trainers must be consistent with training complexity. Trainees must be capable of competent performance post-training. Training logistic considerations such as facilities and equipment, number of trainees per session, scheduling, internet access, and other relevant elements are specified in training design.

 All of the above must be recorded in an approved controlled document for use in future actual training. This document serves as an ongoing record of training subject matter. Document changes are controlled via change management; content is modified as experience is gained, and risks are evaluated. Supplementary information used in training to emphasize key content is appended.

 Training management is ultimately responsible for training design and related elements. Establishment of training standards for content, presentation, testing, trainers, trainees, logistics, and related elements is required. Standards must be documented. Standards are the backbone of the training effort that helps to establish a quality culture in the organization. Training compliance with standards should provide a quality message to trainees. A Training Master Plan listing training standards, approved Training Content Records, and other function information is recommended.

 Stage 1 hazards are identified during the design and development of the training content by the training author. Hazards are identified concurrently with the ‘what’ and ‘why’ of training; authors address ‘what might go wrong’ along with the what and why in subject matter. Addressing potential hazards within training leads to more effective content, presentation, and testing. Design personnel must anticipate potential problems during training design. Illustrations, videos, checklists, and other aids addressing problems should be incorporated into training design. Trainers must verbally emphasize critical content in their training presentation. Testing should emphasize questions on critical content. Addressing training problems through different types of learning (e.g., visual, auditory, reading/writing, kinesthetic, etc.) is recommended; employees learn in different ways. The greater the anticipated risk to product, process, or organization, the greater the potential problems, and the greater the effort that is required in training—a principle of ICH QRM. 

 provides examples of stage 1 hazards based on experiences and observations. 

 Examples of hazards in Stage 1 Training Design. P&ID is piping and instrumentation diagram.

Case study #1: deficient training design—machine setup.

 Customer complaints and returned product were received on a new prescription sedative product. Distinct white spots on the blue tablet surface were observed. Recall letters were sent and product was back-ordered with availability, financial, and regulatory ramifications. Engineers determined that milling equipment was incorrectly set up causing large white-spot particulate. Performance by manufacturing technicians was the hazard in this case; incorrect machine-part assembly was directly correlated to ineffective training. Improvements in training content and illustrations highlighting correct setup were developed. Trainees were required to demonstrate correct equipment assembly to an SME to successfully pass training.

Stage 2 training performance comprises the executed training of a trainee by a trainer and review of the training process. Stage 2 activities confirm the stage 1 design and identify necessary modifications—actual stage 2 training experiences between trainer and trainee may differ from stage 1 expectations. Stage 2 observation also affords opportunity to confirm trainer–trainee focus on critical aspects of the training subject matter:

 The actual training process executes the planned training designed in stage 1.

 Stage 2 training is evaluated; trainer, trainee, training management, training designer, and other interested individuals are involved. Overlooked or unanticipated problems are noted. Trainer emphasis on critical parameters is reviewed. Trainee engagement is evaluated.

Documentation—Personnel Training Record.

 The Personnel Training Record documents completion of training for employees. When an auditor asks to see the training record for a specific employee, this document will be provided. Training documentation contains dates and times of training sessions; these must be consistent with training design. If training is not clearly documented, it didn’t happen.

 Observation and evaluation of the training process by management provides an opportunity to enhance training design and performance. If changes are needed, the stage 1 Training Content Record is modified through the site change management process.

 Stage 2 hazards that were not anticipated in stage 1 design but become apparent during actual training are identified. New hazards noted in stage 2 are evaluated per ICH Q9(R1) methods and integrated per change management. Actual performance variation in trainers and trainees not anticipated in stage 1 design is noted. Training content, testing, logistics, and other considerations are modified in the Training Content Record via change management. 

 and case studies provide examples of potential stage 2 hazards. 

 Quality risk management (QRM) hazards in Stage 2 Training Performance. GMP is good manufacturing practice.

Case study #2a: training logistics—training in the auditorium. 

GMP) training for more than 200 site employees was conducted in response to a regulatory audit observation. The site auditorium was reserved, and a two-hour session was scheduled for 3 pm to include first and second shift employees. The session discussed data integrity, original data recording, error corrections, signature/date verification, “white-out” use, “post-dating,” and numerous other documentation topics. A few days after the training was completed, a site engineer was observed recording original data on scrap paper for later transfer to his notebook. Other technicians were observed writing data on “Post-it” notes instead of batch records. Continuing “white-out” use was also observed. Many key topics addressed in the training were not understood—training was an obvious failure. Poor training logistics was the fundamental hazard that caused ineffective training. Failure modes included too many people in the same room, a lengthy session at the end of the workday (i.e., sleeping employees), uncomfortable room temperature, smartphone distractions, and too many topics without adequate discussion. Trainer communication was also identified as a hazard; supplementary illustrations, lack of hardcopy handouts; and no Q&A with trainer were deficiencies. Training was poorly designed—-an efficient training session became more important than employee learning. Training was disingenuous; management just wanted it done to claim accomplishment of the regulatory commitment. Training was not given appropriate time, effort, and priority to accomplish employee learning. New sessions with smaller groups and improved presentation of content were developed and rescheduled. Large group auditorium training sessions were eliminated in future training.

Case study #2b: training logistics—computer fatigue. 

A site training program for “read and sign” training was managed through a training computer system. Employees were assigned training materials to be read; computer links were provided. Employees were allowed a 30-day time limit to complete the training; completion was self-paced. Employees would routinely give training a low priority and delay training until the deadline. This approach was not acceptable for new employees assigned an excessive number of training modules; more than 50 documents were often “read and sign” in one day—training was ineffective. Trained performance was the hazard identified in this case. The training computer system was modified to allow completion of not more than five training modules in one day.

A senior SME operator was the designated training person for a non-sterile manufacturing area. He always accomplished training of new personnel in one demonstration session. Some procedures required several hours of demonstration. The trainee and trainer then signed documentation that the trainee was qualified to perform the task without actual trainee demonstration of competency. When the newly qualified trainee was assigned manufacturing tasks, the senior person would be nearby to help as needed. Training deficiencies became apparent when the trainee was working alone. When the trainer was asked about his training methods, he responded that his manager told him to complete training as quickly as possible so that the trainee can begin working. Deficient technician performance was the hazard in this case. The trainer should have adjusted training repetition and emphasis based on trainee understanding and complexity of training content. After adequate repetition, the trainee should have been required to independently demonstrate trained skills. Training could not be firmly standardized due to the variation in trainee skills; experienced operators could be adequately trained by observation; inexperienced operators required additional repetition. Management did not give the priority to training due to production criticalities; production criticalities were more important than training. The manager had no incentive to make training a priority vs. his manufacturing responsibility; what the manager did undermined or undercut training. Future training required complete skills demonstration to an independent SME.

Continued training verification comprises the periodic review of training content and performance. Activities in stage 3 enable continuous improvement in training and maintenance of training quality. Training periodic reviews are specified in a Training Master Plan or other scheduling commitments:

 Training effectiveness is evaluated by review of experiential documents (e.g., deviations, non-conformances, investigations, corrective and preventive action [CAPA]), stakeholder interviews, trainer/trainee discussions, and other reviews that critically evaluate training performance. Investigations citing training deficiencies as causes of process deficiencies indicate the need for training improvements. Problem trends should be noted and addressed.

 Training content in the Training Content Record is reviewed for accuracy. Changes in equipment, processes, cleaning methods, and other variables may have occurred without concurrent changes in training. Supportive documents (e.g., flow diagrams, piping and instrumentation diagram [P&ID], sampling protocols, etc.) relevant to training may also be obsolete, inaccurate, or incomplete.

 Performance of trained procedures may also have been modified in response to equipment changes or improved efficiency without concurrent changes in training. Employee shortcuts in performing trained procedures are a common occurrence. Non-compliance with procedures are among the highest frequency of FDA-483 citations (18)

Documentation—Training Periodic Review.

 Performance of the Training Periodic Review must be documented.

 Completion of periodic training reviews as identified in the Training Master Plan or other commitment is the responsibility of management. If new hazards are identified, these must be submitted for evaluation through the QRM process and change management for implementation if appropriate.

 Stage 3 hazards may be identified in periodic review activities for a variety of reasons. New hazard identification may occur years after training was successfully completed. Stage 3 periodic review provides the opportunity to review ongoing routine performance of trained activities vs. approved training content, supporting materials, and procedures. Changes may have occurred in any of the above, making training inconsistent with aforementioned materials. For example, utilities may have been modified, supporting sampling diagrams may have changed, procedures may have changed, or practices may have changed without concurrent changes in training. Stage 3 hazards are also evaluated and integrated in stage 1 design per change management. 

 and case studies provide examples, experiences, and observations of potential stage 3 problems. 

 Quality risk management (QRM) hazards in Stage 3 Continued Training Verification. SME is subject matter expert. CAPA is corrective and preventive action. P&ID is piping and instrumentation diagram.

Case study #4: low tablet target potency. 

Periodic review of a tablet compressing process was conducted. The manufacturing technician was an excellent machine operator (“Best Technician Award”). His manufactured products always met acceptance specifications and product yields were always above average. Further evaluation of product potency data made under his responsibility indicated a slightly low average potency (97–98%) compared to lots made by other personnel. The manufacturing supervisor observed his machine setup and operation; tablet weight setup was within specifications, but always at a theoretical weight of less than 100%. When questioned why tablet weight was not adjusted to 100%, the technician responded that he always set a slightly low weight so that he would have a high product yield—management wants high yield and more profit—this is why he was “Best Technician.” Technician performance was the hazard in this case. GMP allows product release within specification (usually 95-105%), but product must be formulated to contain 100% unless otherwise justified. The technician was retrained on GMP requirements. Training documentation was revised to emphasize 100% formulation target requirements. Recognition procedures were modified to eliminate product yield as criterion for employee performance.

Case study #5: supporting documentation. 

Periodic review of site utility systems was conducted. Documents reviewed included procedures, supporting documents, and associated training. Supportive drawings and training documents were incomplete and not updated. Hazard in this case was inaccurate documentation. Investigation of discrepancies between documents indicated that final changes on drawings and training were not completed due to competing work priorities; responsible personnel were assigned other projects with higher priority. Review of other systems (e.g., compressed air, ethanol, and other utilities that had been recently modified) had similar inconsistencies—engineers were never allowed to completely finish a project before being reassigned by management to higher priority work.

Case study #6a: training culture—cleaning process review. 

Internal review of a product cleaning process was conducted in advance of a regulatory audit. Technical, Manufacturing, and quality assurance (QA) supervisors observed technician performance. Several discrepancies compared to approved procedures were noted. The amount of cleaning agent was modified; specific steps were changed to increase efficiency; an unspecified organic solvent was used to remove stains. When questioned about discrepancies, the manufacturing technician expressed site reality—the approved cleaning procedure was ineffective. Management advised to fix the cleaning problems without involving the technical group. The site culture was identified as the hazard in this case. The above occurrence demonstrated problems with interpersonal relationships, interactions between groups, function silos, and general disrespect for approved policies and procedures; technicians proudly stated, “We do whatever it takes to get the job done.” Management leadership and attitude were also identified as contributing to this hazard. The site quality culture begins with management (19). Several corrective actions were initiated within various functions at the site. Personnel learned to follow procedures and not “to do whatever it takes”.

Case study #6b: training culture—aseptic compounding. 

A sterile compounding facility required technician training and annual re-qualification on aseptic techniques. Testing required handwashing, garbing, work area sanitization, technique demonstrations, aseptic transfer of sterile media between vials, finger impressions testing, and related activities. Personnel observing testing noted unusually high frequencies of hand sanitization and surface sanitization by technicians; supervisors were continually reminding trainees to sanitize their hands. Most troubling was hand sanitization immediately prior to finger impression testing. The donning of new sterile gloves or spraying 70% alcohol immediately before finger-impression testing does not challenge aseptic technique and does not represent actual work performance. The site culture was identified as the hazard in this case. Technicians explained that the department manager told them, “There shall be no problems with qualification testing—do whatever necessary to prevent failures.” A deficient quality culture at the site translated into substandard employee behavior..

This paper discusses applications of QRM principles in a site training quality system. QRM applications enable several significant benefits to training.

 QRM in training focuses on identification of training problems. QRM application provides a distinct and comprehensive approach that complements traditional training. Training fundamentally comprises prospective activities (i.e., how to do things and why things are done). QRM provides additional relevant considerations to training—what can go wrong. QRM thus helps to describe training reality in the workplace. Hazard identification facilitates problem evaluation and subsequent action as needed.

 This discussion is structured according to the lifecycle approach as used in process validation. A lifecycle approach to training is described that affords opportunities to review and continually improve the training process. Three stages of QRM activities are identified, and QRM hazards are identified in each stage:

Stage 1 training design comprises planning and relates to hazards that are associated with training content, trainers, trainees, and the training process; stage 1 hazards are identified when new training is originally developed.

Stage 2 training performance relates to hazards associated with actual training activities when trainers present content to trainees; observation of stage 2 training may identify new hazards and necessitate modifications to content, trainer, or other designed elements.

Stage 3 continuous training monitoring (i.e., training periodic review) relates to hazards associated with the ongoing performance of trained procedures—training effectiveness, content accuracy, employee performance, consistency with related documentation, and compliance with approved procedures. Observation of training content and/or performance in stage 3 may also necessitate modifications to training content.

 illustrates the respective stages of the training lifecycle. Stages of the training lifecycle (black print) anchor the overall training process; hazards are originally identified in stage 1 when training is developed. If new hazards are identified in stage 2 or stage 3 (red print), these are forwarded to stage 1 for evaluation and integration with training content, presentation, testing, and other elements; training quality is maintained and continually improved by this process. 

 Quality risk management (QRM) integration into Training Quality System. Training lifecycle comprises Stages 1, 2, and 3 including management support. New training hazards identified in Stages 2 and 3 are evaluated and integrated within training as needed.

 Training management has significant responsibility and influence on performance of site training as follows:

 Training management is responsible for all training activities in all stages as well as for the general training function administration. Training is a cost; training management must balance accomplishing training objectives within site budgetary constraints. Numerous QRM hazards may be associated with management responsibilities. Management must integrate QRM considerations to maintain and improve training content. Management in this context includes training management as well as other site function management involved in training.

 A site training program often utilizes individuals from other site functions as trainers, SMEs, training content authors, or other contributors. These individuals also have direct responsibility for employee training. Training management must ensure these contributors know and understand their responsibilities, including QRM considerations, and that they are accountable for successful employee learning.

 Training management significantly influences the attitudes and performance of trained employees. Employee training is among the first activities of new employees; this relationship continues throughout the employee career with additional training, re-training, and other interactions. Management commitment to training excellence, including QRM considerations, must be visible and sets the tone for employee performance. Management actions influence the development of a quality culture in the organization.

Helpful discussions with Kevin O’Donnell, PhD, Health Products Regulatory Authority (HPRA) Ireland; Alan M. Mancini, RPh; and Richard Poska, RPh, managing director, Flexo CMC Consulting, Nokomis, Fla., are greatly appreciated.

1. ICH. Q9(R1) Quality Risk Management, Final version (January 2023).
2. Pappas, C. Eight Important Characteristics of Adult Learners. elearningindustry.com, May 8, 2013.
3. ICH. Q10 Pharmaceutical Quality System, Step 4 version (June 2008).
4. FDA. Guidance for Industry, Quality Systems Approach to Pharmaceutical CGMP Regulations (CDER, CBER, September 2006).
5. FDA. CDER Quality Management Maturity. fda.gov (accessed July 24, 2024)
6. Belyh, A. Management by Walking Around (MBWA)—The Essential Guide. cleverism.com, July 28, 2020.
7. FDA. 

Guidance for Industry, Process Validation: General Principles and Practices

 (CDER. CBER, January 2011).
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(6).
9. Pluta, P. L. Application of Validation Principles to the Training Quality System. Part 2—Technical Training Modules. 

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10. Pluta, P. L. Application of Validation Principles to the Training Quality System. Part 3—Trainers. 

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11. Pluta, P. L. Application of Validation Principles to the Training Quality System. Part 4—Trainees. 

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12. Pluta, P. L. Application of Validation Principles to the Training Quality System. Part 5—Training Process. 

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13. Pluta, P. L. Application of Validation Principles to the Training Quality System. Part 6 —Continued Training Verification. 

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14. Pluta, P. L. Application of Validation Principles to the Training Quality System. Part 7—Training Management Overview. 

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15. Pluta, P. L. Application of Validation Principles to the Training Quality System. Part 8—Training Process Documentation. 

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16. ICH. Quality Risk Management, ICH Q9(R1) Training Slides Hazard Identification—Part 1. 

 (accessed July 25, 2024).
17. ICH. Quality Risk Management, ICH Q9(R1) Training Slides Hazard Identification—Part 2. Consideration in Relation to Identification of Failure Modes, in the Pharmaceutical Environment. 

 (accessed July 25, 2024).
18. Hoare, A. The Most Common FDA 483 Observations. 

, April 18, 2023.
19. Friedman, R. Establishing a Culture of Quality. 

Paul L. Pluta, PhD, RPh, is a pharmaceutical scientist with experience in pharma industry, university academia, pharmaceutical journal editorship, and community and hospital pharmacy practice.

®
Vol. 48, No. 11
November 2024
Pages: 20–26

When referring to this article, please cite it as Pluta, P. L. Application of Validation Principles to the Training Quality System: Part 9, Quality Risk Management Hazard Identification. 

Pharmaceutical training programs are enhanced by integrating ICH quality risk management considerations.

Application of Validation Principles to the Training Quality System: Part 9, Quality Risk Management Hazard Identification

Aptamer Group announced on Nov. 7, 2024 that one of its customers has advanced use of the company’s Optimer binders for the commercial phase of its product. The customer has used the Optimer delivery vehicle for the product’s development phase and Aptamer shipped test materials to the customer in October 2024.

According to Aptamer, the customer has validated the performance of the delivery vehicles, indicating the vehicles can target the required cell type. The vehicles have the potential to be used as part of a precision therapy. This would create downstream licensing revenue for Aptamer.

The customer also found that Optimer showed binding to the target cells from a panel of animal species, which makes the delivery vehicle desirable for preclinical development. “Due to the targeting specificity, multiple species binding, and reproducibility of the results, the customer has committed to progressing to the final commercial development stage for the therapeutic delivery vehicle,” Aptamer stated in a press release (1).

According to Aptamer, targeting genetic medicines to specific cell types is a challenge. Highly selective delivery vehicles address this challenge by targeting these cell types, which has been difficult in the past. The company is developing delivery vehicles that target fibrotic liver cells, creating progress for genetic medicine payloads.

A genetic medicines customer has selected the company’s binders for commercial development of the customer’s product.

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