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Rare diseases, medical concept surrounded by pills and medical supplies on gray background. Top view. | Image Credit:  ©maicasaa -stock.adobe.com

Rare diseases, defined in the United States as conditions affecting fewer than 200,000 patients, collectively represent one of the largest areas of unmet medical need in modern healthcare. More than 7000 rare diseases affect an estimated 25–30 million people in the US and more than 400 million globally (1,2). Despite decades of scientific progress, however, only approximately 5% of rare diseases currently have an FDA-approved therapy (2). The clinical, economic, and societal burden of these conditions is substantial: on a per-patient basis, rare diseases generate healthcare costs nearly 10 times higher than non-rare diseases, driven largely by delayed diagnosis, fragmented care, and avoidable morbidity and mortality (3).

For much of the pharmaceutical industry’s history, these realities did not translate into meaningful investment in drug development. Small patient populations, limited disease understanding, and uncertain reimbursement made rare diseases appear commercially unattractive. Instead, capital flowed toward large, prevalent indications such as cardiovascular disease, diabetes, and oncology, where scale and drug pricing justified risk. Even when scientific hypotheses existed, investors and manufacturers alike questioned whether development costs could ever be recovered in ultra-small markets (4).

That perception has changed fundamentally. Over the past four decades, rare diseases have shifted from a marginal pursuit to a core pillar of biopharmaceutical innovation and growth. Today, roughly one-third of FDA-approved drugs carry an orphan designation, and rare disease assets are sought-after in business development and licensing (BD&L) transactions (2,5). This transformation was not accidental. It emerged from a repeatable formula built on the convergence of three forces: supportive policy incentives, scientific breakthroughs, and durable and consistent commercial validation. These elements reshaped the risk–reward profile of rare disease development and established a model that continues to attract capital and strategic focus.

This article, Part one of a four-part rare disease thought-leadership series, examines how that winning formula emerged and why it continues to underpin rare disease development and investment. Part two will explore the pricing and access landscape of rare disease assets in the US and key global regions.

Which policy incentives were the catalyst for rare disease development?

The modern rare disease era began with a deliberate policy intervention. The US Orphan Drug Act (ODA) of 1983 fundamentally altered the economics of developing therapies for small patient populations by introducing a package of incentives designed to reduce financial and regulatory risk (6). These included seven years of market exclusivity, tax credits for clinical research, and waivers of certain FDA fees. At the time, the legislation was viewed as an experiment, an attempt to correct a market failure by encouraging investment where traditional commercial development had fallen short for patients with the highest unmet need (

Growth of orphan disease development in the United States from the enactment of the Orphan Drug Act in 1983 until present also showing the enactment year of the Biologics Price Competition Innovation Act (BPCIA) and the establishment of the Rare Pediatric Disease Priority Review Voucher (PRV) program. During this period, the first monoclonal antibody (mAB; Orthoclone OKT3), the first enzyme replacement therapy (ERT, Ceredase), the first gene therapy (GTx, Glybera), and the first chimeric antigen receptor T-cell (CAR-T, Kymriah) were all approved. (Figure courtesy of the author)

The impact was profound. By lowering barriers to entry, the ODA enabled small biotechnology companies and academic spinouts to pursue programs that previously had been considered commercially untenable. Importantly, the ODA did not operate in isolation. Over time, additional regulatory mechanisms further accelerated rare disease development. FDA’s Accelerated Approval pathway allowed therapies to reach patients based on surrogate endpoints reasonably likely to predict clinical benefit, particularly relevant in rare disease where large, long-duration trials are impractical (7). The Biologics Price Competition and Innovation Act (BPCIA) later extended exclusivity for biologics to 12 years, reinforcing incentives for novel modalities (8).

Supplementary programs, such as the Rare Pediatric Disease Priority Review Voucher (PRV), provided additional financial upside by allowing companies to monetize expedited FDA review of future assets, often for sums exceeding $100 million on the secondary market, and further justifying unknown clinical development pathways (9). Although PRV was allowed to sunset in 2024, as of Jan. 27, 2026, the Mikaela Naylon Give Kids a Chance Act, which would reinstate the PRV, has passed the House of Representatives and is awaiting Senate approval (10). 

US leadership also catalyzed global adoption. Japan implemented orphan drug incentives in the early 1990s, followed by the European Union’s orphan regulation in 2000 (11). Today, more than 50 countries have some form of orphan drug legislation, creating a broadly harmonized global framework that supports rare disease innovation across regions.

Collectively, these policies did more than stimulate development volume. They reset expectations around what constituted an acceptable development risk and signaled long-term regulatory commitment to rare disease innovation. Without this foundation, many subsequent scientific breakthroughs would likely have remained confined to academic interest rather than translating into approved therapies.

Development realities in rare diseases: smaller trials, different risks

Rare disease development is often assumed to be more expensive and riskier than development in common indications. In practice, the picture is more nuanced. Clinical trials in rare diseases are smaller by necessity, frequently rely on surrogate endpoints, and increasingly employ adaptive or single-arm designs. These features reduce enrollment requirements but introduce distinct operational challenges related to patient identification, recruitment, and endpoint validation.

Analyses comparing orphan and non-orphan drugs show that, on average, clinical development costs for orphan drugs are lower, not higher. Estimates place the average cost per approved orphan drug at approximately $166 million, compared with $291 million for non-orphan drugs, with capitalized costs following a similar pattern (12). For new molecular entities, orphan development costs are roughly half those of non-orphans. Longer trial durations, driven by recruitment challenges, partially offset these savings, but overall economics remain favorable.

Market research with industry experts further highlights that development difficulty varies meaningfully across rare disease therapeutic areas (

). Experts ranked challenges related to trial execution, particularly patient identification and recruitment, are consistently ranked as both the most difficult and most costly barriers 

. Scientific uncertainty and lack of validated endpoints add complexity, while manufacturing and logistics emerge as disproportionately costly relative to perceived difficulty. These findings underscore that rare disease development is not monolithic; execution risk depends heavily on disease biology, diagnostic infrastructure, and therapeutic modality.

Market research analysis of industry experts (n=31) showing rare disease drug development costs by stage of development from pre-clinical to Phase III. Analysis of development costs was analyzed by therapeutic area. Experts were also asked to rank order therapeutic areas by difficulty to develop as shown here. (Figure courtesy of the author)

Innovations in trial design and evidence generation continue to mitigate these risks. Decentralized trials, expanded access programs, and the increasing use of real-world data have all played growing roles in regulatory submissions, particularly where randomized controlled trials are infeasible (13,14). In several orphan approvals, data from expanded access programs contributed pivotal efficacy evidence, further illustrating regulatory flexibility in high-unmet-need settings.

Commercial validation: proving the model works

Ultimately, it was commercial success, not policy or science alone, that cemented rare diseases as a sustainable strategic focus. A growing number of rare disease therapies have achieved blockbuster status, disproving the notion that small patient populations inherently limit revenue potential. Two complementary commercial models have emerged: high-chronic-revenue therapies, such as Spinraza, and ultra-premium, potentially curative therapies, such as Zolgensma.

Beyond spinal muscular atrophy, multiple rare disease therapies have crossed the $1 billion annual sales threshold, including Soliris for PNH and Cerezyme for Gaucher disease. More recently, gene therapies such as Elevidys for Duchenne muscular dystrophy are emerging as blockbuster candidates despite ongoing safety scrutiny, reflecting strong physician demand in high-unmet-need populations 

(data not shown, accessed from Global Data, September 2025)

Revenue analyses of orphan-designated drugs approved between 2000 and 2024 show that approximately 14% achieve blockbuster status, with an additional 12% reaching high-tier revenues of $500 million to $1 billion annually (

). While the majority of orphan drugs remain mid-tier or niche products, many still generate positive lifetime returns due to limited competition and reduced exposure to generic or biosimilar erosion.

 Peak annual revenue analysis of orphan-designated drugs approved in the United States between 2000 and 2024 (n=398). Drugs are categorized by peak annual sales into Blockbuster (≥$1B), High-tier ($500M–<$1B), Mid-tier ($250M–<$500M), Small ($100M–<$250M), and Niche (<$100M). Revenue estimates may include ex-U.S. sales. This analysis highlights that while a minority of orphan drugs achieve blockbuster or high-tier revenue, a substantial proportion of therapies remain mid-tier or niche, reflecting the diverse commercial potential within the rare disease space. (Figure courtesy of the author)

Comparative analyses of early post-launch performance further illustrate the advantage of orphan strategies. Drugs approved exclusively for orphan indications demonstrate steady growth in the first three years post-launch, whereas non-orphan drugs approved over the same period show declining early revenues, reflecting crowded markets and intense competition 

. These patterns reinforce the strategic appeal of rare diseases as a way to achieve earlier and more predictable commercial traction.

Commercialization, however, is not without cost. Industry expert research indicates that rare disease launches require a consistently high-touch model, with significant investment in patient services, diagnostics, field teams, and evidence generation (data not shown). Notably, commercialization spending converges across therapeutic areas, reflecting shared structural challenges rather than indication-specific dynamics. Over a five-year window surrounding launch, total commercialization investment often approaches $250 million, underscoring the importance of disciplined portfolio planning and sequencing.

What is the rare disease treatment winning formula?

Taken together, four decades of experience reveal a consistent formula underlying rare disease success. Policy incentives catalyzed initial investment. Scientific advances proved feasibility. Flexible development pathways reduced execution risk. Commercial validation attracted further capital. Each element reinforced the others, creating a self-sustaining cycle that continues to define the sector today (

Over four decades of rare disease drug development reveals a consistent formula for success: The development of national and now international policy incentives, breakthrough advances in science and technology and expanded clinical development results not only in the approval but also widespread commercial successes for rare disease therapeutics. (Figure courtesy of the author)

Patient advocacy has played a central role throughout this evolution. Advocacy organizations generate natural history data, facilitate trial enrollment, and influence regulatory and reimbursement decisions, effectively reducing development risk and accelerating adoption. In rare diseases, patient centricity has moved from aspiration to operational necessity.

This model has also shaped industry structure. Small and mid-cap biotech companies typically drive early discovery and proof-of-concept, while large pharmaceutical companies scale manufacturing, commercialization, and global access through partnerships or acquisitions. Capital markets have reinforced this sequencing: orphan designation announcements are associated with positive abnormal returns, and orphan-focused assets consistently command valuation premiums in M&A transactions.

Looking ahead: from success to sustainability

What began as a policy experiment has matured into a cornerstone of biopharmaceutical growth. Yet the rare disease model now faces new questions. Rising launch prices, expanding multi-indication strategies, increasing payer scrutiny, and an emerging value-access paradox where regulators and payers are requiring different evidence standards, raise concerns about long-term sustainability. These issues will be addressed in Part 2 of this series, which examines the global pricing and access landscape for rare disease therapies. The rare disease winning formula remains intact, but its next phase will be defined not by whether it works, but by how responsibly and strategically it is applied.

Schieppati, A.; Henter, J. I.; Daina, E.; and Aperia, A. Why Rare Diseases Are an Important Medical and Social Issue. 

Han, Q.; Fu, H.; Chu, X.; Wen, R.; Zhang, M.; You, T.; Fu, P., Qin, J.; and Cui, T. Research Advances in Treatment Methods and Drug Development for Rare Diseases. 

Andreu, P.; Karam, J.; Child, C.; Chiesi, G.; Cioffi, G. The Burden of Rare Diseases: An Economic Evaluation. Chiesi Global Rare Diseases White Paper. 

Miller, K. L. Do Investors Value the FDA Orphan Drug Designation? 

Chambers, J. D.; Clifford, K. A.; Enright, D. E.; and Neumann, P. J. Follow-On Indications for Orphan Drugs Related to the Inflation Reduction Act. 

H.R.5238 - 97th Congress (1981-1982): Orphan Drug Act. (

, January 4). https://www.congress.gov/bill/97th-congress/house-bill/5238

FDA. Accelerated Approval Program. NDA and BLA Approvals. United States Food and Drug Administration. 

FDA. Biologics Price Competition and Innovation Act. Center for Drug Evaluation and Research (CDER). FDA, 

Rare Pediatric Disease Priority Review Voucher. Food and Drug Administration Safety and Innovation Act. 

. 21 U.S.C. ch. 9; Pub. L. 112-114. 126 Stat. 993-1132.

Mikaela Naylon Give Kids a Chance Act. 2025. 119

Costa, E., Vijayalekshmi Ajith, Al, A. F., Antonella Isgrò, Lee, K. J., Riccardo Luigetti, Pasmooij, A. M. G., Stoyanova-Beninska, V., Trachsel, E., Vaillancourt, J., & Steffen Thirstrup.  Addressing Global Regulatory Challenges in Rare Disease Drug Development. 

Jayasundara, K.; Hollis, A.; Krahn, M.; Mamdani, M.; Hoch, J. S.; and Grootendorst, P. Estimating the Clinical Cost of Drug Development for Orphan versus Non-Orphan Drugs. 

Polak, T. B.; van Rosmalen, J.; and Uyl-de Groot, C. A. Expanded Access as a Source of Real-World Data. 

Moore, J.; Goodson, N.; Wicks, P.; and Reites, J. What Role Can Decentralized Trial Designs Play to Improve Rare Disease Studies? 

Figures 1 and 3: Analysis was conducted using data accessed from Global Data (September 2025), following the subsequent methodologies. Total Orphan Designations reflects the total count of unique drugs that received an FDA Orphan Designation for any indication, organized by the year in which their first designation was received; excludes drugs which pursued any indication in which they did not receive an Orphan Designation. Orphan Approvals by Year reflects the total count of unique drugs that received an FDA Orphan Designation for any indication and were approved in that indication, organized by the year in which their first approval was received; also excludes drugs which pursued any indication in which they did not receive an Orphan Designation. Further sub-divided based on molecule type was performed using the dichotomous classification of Small Molecule and Biologic based on manufacturer reported mechanism of action and drug classification. The total number of unique drugs that received an FDA Orphan Designation for any indication and were subsequently approved in that indication between the years 2000 and 2024; excludes any drugs for which sales data was not available. This sample is further subdivided by peak annual sales into Blockbuster (≥$1B), High-tier ($500M–<$1B), Mid-tier ($250M–<$500M), Small ($100M–<$250M), and Niche (<$100M) and represented as the number of drugs within each category of peak annual sales as a percentage of the overall sample.

Figure 2: Data was collected through an online survey designed to capture quantitative insights into the clinical development and commercialization of products in the rare disease space. Respondents were required to meet specific qualification criteria, including holding a senior position title, having a minimum of 5 years industry experience, and possessing strong or extensive knowledge of the costs associated with clinical and commercial development. The survey comprised 15 closed-ended questions utilizing multiple choice, matrix tables, and ranking formats. The survey was conducted between September 19 and September 25, 2025, and included a final sample of 31 qualified respondents (n=31). All data shown represents averages across respondents.

 is a global, full-service boutique consulting firm that partners with biopharma companies to navigate complexity and support growth. We combine deep therapeutic expertise, real-world insights, and end-to-end commercialization capabilities with a people-first approach. Our mission is simple: deliver strategies that create measurable results and lasting impact for our clients, our people, and the communities we serve.

Articles

Rare diseases, affecting fewer than 200,000 patients in the US, represent a significant unmet medical need, with only 5% having FDA-approved therapies. Historically, rare diseases were commercially unattractive due to small patient populations and uncertain reimbursement. However, policy incentives like the Orphan Drug Act, scientific breakthroughs, and commercial success have transformed rare diseases into a core focus for biopharmaceutical innovation. Despite challenges in trial design and commercialization, rare disease therapies have achieved significant revenue, proving the model's viability and attracting further investment.

In part one of this four-part series regarding rare diseases, the author explores how rare disease treatment development evolved from a niche area to a cornerstone of biopharma growth. 

Rare Disease Therapies: From Niche Experiment to Strategic Growth Engine

With a potential US government shutdown looming, Megha Sinha, Kamet Consulting, discusses the impacts on pharma manufacturing and development.

How a Government Shutdown Causes FDA Inspection Bottlenecks and Backlogs

In this part 1 of a 2-part video interview on the impacts of a government shutdown on pharmaceutical manufacturing and development, Megha Sinha, CEO, Kamet Consulting Group, discusses the complex logistical challenges teams face during a resulting FDA shutdown. She clarifies that a shutdown is typically a slowdown rather than a total stop, but one in which predictability drops significantly, creating downstream delays for new submissions and manufacturing changes.

Sinha categorizes the immediate risks to the industry into three specific areas: inspection schedules, agency responsiveness, and backlog behavior. She notes that while mission-critical safety work and core reviews funded by user fees often continue, the "operational glue"—such as meeting coordination and administrative processing—becomes severely constrained. According to Sinha, "The fastest way these type of shutdowns can turn into supply risk is through inspection and response bottlenecks."

What Are the Manufacturing and Clinical Constraints?

For manufacturers and clinical trial sponsors, Sinha says the challenges are often physical rather than administrative:

• Onsite Inspections: Facility readiness and site travel become the primary "gating factors" for approvals.

• Prioritization: Routine inspections are often reduced to prioritize high-risk or "for-cause" activities, leaving those waiting for pre-approval inspections in a state of uncertainty.

• Lot Release: Even for high-priority items like biologics and vaccines, the processing cadence slows as staffing and time become constrained.

What Are the Industry Impacts Upon Reopening?

The most significant takeaway from Sinha’s analysis is that the end of a shutdown does not mean an immediate return to normal. Sponsors will likely experience a loss of cycle time and friction in regulatory communication that persists long after funding is restored.

As Sinha summarizes, "the net is that the biggest manufacturer risk isn't that the FDA disappears. It's the queue that will start to form, and the recovery period really after reopening can… be as disruptive as the shutdown itself." This backlog creates a "ripple effect" that can impact approval decisions and supply chains for months.



Watch Part 2 of this interview about treating FDA disruptions as predictable risks by protecting milestones and early readiness!

Editor's note: This transcript is a lightly edited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.



Hi, I'm Megha Sinha, CEO of Kamet Consulting Group. At Kamet, we support life sciences teams through high-stake changes, so acquisitions and divestiture transformation and technical advisory. I bring 17-plus years across industry and consulting, including eight years at PWC.

Yeah, so I think we need to think slow down, not total stop, but the predictability is going to drop. So, many ongoing reviews that FDA has can continue because that really heavily relies on user fees and carryover funding for core review work. But the really big breakpoint will be that US FDA generally would not accept new fee-bearing submissions during a lapse in appropriations, which will form a queue that can cause downstream delay

For clinical trials, missions critical safety work will continue, but really the non-urgent interaction—so think of, you know, any setups of the meetings, follow up—that can be constrained as the staffing shifts.

And then for manufacturing changes, the risk is really less about the paperwork, but what is really needed to close it out. So, if there is any onsite inspection required, facility readiness verification, site travel, that will become really the gating factor for manufacturers.

So, for approvals really in progress, a short shutdown may be absorbed, but really the shutdown creates this backlog, and that's the effect you'll see after reopening. And you know, I think as you may be seeing in the news, right, there's some bills that are already passing, so there might be still funding available; it's just, you know, which part of the FDA gets that funding, which will be the high-critical items.

So, the fastest way these type of shutdowns can turn into supply risk is through inspection and response bottlenecks. I would really categorize the immediate risk clusters into three pieces. So, it's the inspection schedule, it's the agency responsiveness, and it's the backlog behavior.

First, in case of inspection, in a shutdown scenario, FDA staffing and travel can be constrained. So, the routine inspection may be reduced while the higher-risk or for-cause activity will be prioritized. And this can create uncertainty around pre-approval inspection or follow ups, which can be a critical path for certain approvals and for sure manufacturing changes. So that's going to start to impact the manufacturers who are waiting for that.

Secondly, regulatory communication. So even when the review work is continuing under the user fee carryover, the operational glue—so, think of meeting coordination, really rapid back and forth with the agency, administrative processing—these will all get constrained. So, this is where sponsors will start to feel real friction because you start to lose cycle time and delay in approval decisions.

Third, with respect to lot release and release-related interaction, for certain biologics and vaccines, lot release activities are obviously higher priority than routine work, but because of the staffing issues, the processing cadence can still slow down, because staff and time gets constrained.

So, really the net is that the biggest manufacturer risk isn't that the FDA disappears. It's the queue that will start to form, and the recovery period really after reopening can just be as disruptive as the shutdown itself.

Videos

A government shutdown impacts pharmaceutical manufacturing and development by creating logistical challenges, particularly with the FDA. While critical safety work continues, the predictability of operations decreases, leading to delays in new submissions and manufacturing changes. Key risk areas include inspection schedules, agency responsiveness, and backlog behavior. Manufacturers face physical constraints, such as facility readiness and site travel, affecting approvals. Even after reopening, the backlog and disrupted regulatory communication can prolong recovery, causing a ripple effect on approval decisions and supply chains for months.

International day of Women and Girls in science is observed every year on February 11, The day recognizes the critical role women and girls play in science and technology. 3D Rendering | Image Credit: ©Waseem Ali Khan -stock.adobe.com

International Day of Women and Girls in Science

 (1). This day focuses on the gender gap in science, technology, engineering, and mathematics (STEM) fields and emphasizes the need to close gender gaps in STEM through supporting girls in targeted programs and promoting actions and policies surrounding inclusion.

In recognition of the International Day of Women and Girls in Science, 

will be interviewing industry experts on the innovations and successes women have achieved in the pharmaceutical industry. We will also be highlighting some recent news about notable CEOs, scientists, and innovators impacting the industry and improving patients’ lives.

The following are some of the women we will be highlighting in the weeks to come:

Jennifer Levin Carter, MD, MPH, MBA is Founder & CEO of Medzown. Dr. Carter is a globally recognized leader in precision medicine, with a proven track record of building transformative companies at the intersection of data, technology, and patient care. Dr. Carter will be discussing lessons she’s learned from founding her own company in the biotech field. Watch Part One of our interview with Dr. Carter now, in which she discusses the differences between personalized medicine and precision medicine. Check back in February for more with Dr. Carter.


https://www.pharmtech.com/view/personalized-medicine-vs-precision-medicine

Elisabeth Gardiner, CSO of Tevard Biosciences

to explain how transfer RNA is impacting the development of gene therapies for diseases with unmet needs. In an upcoming interview, Elisabeth will discuss how women working in STEM fields uniquely contribute to areas such as rare disease treatment development.


https://www.pharmtech.com/view/gene-therapy-rare-diseases-and-transfer-rna

 to talk about her work as director of Clinical Research at 

, which is an early-phase oncology clinical trial unit located within General Hospital San Pedro in Logroño in northern Spain. Check back on February 11 to watch the interview.

, CEO of IMUNON, which is advancing a novel non-viral, DNA-based immunotherapy for women with advanced ovarian cancer and experts from Symbiosis, Quotients Sciences, and more.

 on February 11 for all of our coverage of the International Day of Women and Girls in Science.

UN. International Day of Women and Girls in Science, 11 February. UN.org. 

https://www.un.org/en/observances/women-and-girls-in-science-day

The International Day of Women and Girls in Science, observed on February 11, highlights the gender gap in STEM fields and promotes inclusion. PharmTech will feature interviews with industry experts, showcasing the achievements of women in the pharmaceutical industry. Notable figures include Jennifer Levin Carter, a leader in precision medicine; Elisabeth Gardiner, who discusses transfer RNA in gene therapies; and María de Miguel, who leads oncology clinical trials. The coverage aims to celebrate women's contributions to science and inspire future generations to pursue STEM careers.

Visit PharmTech.com for upcoming coverage of the International Day of Women and Girls in Science on February 11.

Innovations from Women in STEM 

WuXI collabs with HanchorBio | Created with Canva - Partnership image courtesy of WuXi Biologics.

HanchorBio, a clinical-stage biotechnology company, and WuXi Biologics, a global Contract Research, Development and Manufacturing Organization (CRDMO), established a strategic collaboration for the development and manufacturing of next-generation bi-functional and multi-functional fusion protein programs from HanchorBio’s pipeline according to a WuXi Biologics press release.¹ Focusing on multiple assets derived from a proprietary Fc-Based Designer Biologics (FBDB) platform, this platform is designed to activate both innate and adaptive immune pathways to address unmet needs in oncology and autoimmune diseases.

Why is technical scalability vital for complex fusion proteins?

In the specialized field of fusion protein development, maintaining execution efficiency in Chemistry, Manufacturing, and Controls (CMC) is essential for successful clinical translation. The collaboration is structured to support the transition of molecules from HanchorBio’s FBDB platform into scalable global clinical development. Under the terms of the agreement, the partnership provides end-to-end services including cell line development, drug product formulation, and 

Good Manufacturing Practice (GMP) manufacturing

.¹ The robustness of these processes is critical for managing capital efficiency and maintaining flexibility across multiple clinical assets.

Dr. Scott Liu, founder, chairman, and chief executive officer of HanchorBio, commented in the press release¹, “This partnership with WuXi Biologics strengthens our ability to translate platform-driven innovation into high-quality clinical and commercial assets. As we advance multiple next-generation fusion protein programs, execution speed, manufacturing reliability, and scalability are critical. WuXi Biologics’ proven expertise in complex biologics and global development makes them a strong strategic partner as we build a differentiated, multi-asset immunotherapy pipeline.”

How do integrated platforms address development timelines for novel modalities?

The manufacturing landscape is increasingly dominated by complex modalities. As of late 2025, approximately 60% of the 945 integrated projects supported by WuXi Biologics involved bi-specific antibodies, multi-specific antibodies, and fusion proteins.¹ To manage this complexity, the industry relies on integrated platforms that offer specialized technologies, such as targeted integration-based cell line development and high-throughput formulation tools designed to enhance quality and scalability. These technical capabilities, combined with a workforce of over 12,000 employees and a global quality system, are intended to shorten the journey from concept to commercialization.

Dr. Chris Chen, chief executive officer of WuXi Biologics, said: “We are excited to embark on this collaboration with HanchorBio, which underscores the strong trust they placed in WuXi Biologics’ comprehensive, end-to-end capabilities for developing next-generation biologics, especially complex molecules. By leveraging our industry-leading technology platforms, proven expertise, and unwavering commitment to quality, we strive to accelerate the development of HanchorBio’s innovative bi-/multi-fusion proteins and help bring transformative therapies to patients worldwide.”¹

This alignment of discovery-stage innovation with globally compliant GMP manufacturing execution reflects a broader industry strategy to advance complex, platform-derived pipelines with increased speed and reliability.¹ By utilizing a single-source CRDMO model, the partnership seeks to streamline the technical requirements of CMC development while supporting the expansion of innovative immunotherapy portfolios.

How else is WuXi Biologics bolstering its manufacturing capabilities?

Beyond the initial focus on platform-driven innovation, the capacity to meet diverse international standards is a prerequisite for global commercial success. This is evidenced by recent regulatory milestones, such as the UK MHRA granting GMP certifications for commercial manufacturing of an ophthalmic biologic to WuXi Biologics.²

Dr. Chris Chen, Chief Executive Officer of WuXi Biologics, noted in a press release, “we are delighted to receive GMP certification from MHRA, a testament to our steadfast commitment to the highest global quality standards. Quality is fundamental to ensuring safety and efficacy. Maintaining a 100% success rate for regulatory inspections reflects our relentless pursuit of excellence and efficiency in commercial manufacturing.”²

WuXi Biologics continues to maintain a strong regulatory track record, including 46 successful inspections by late 2025,² providing the reliability necessary to navigate complex global supply chains. By integrating high-capacity manufacturing with specialized delivery technologies and a focus on green technology innovation, developers can better ensure that platform-derived innovations reach patients with speed and compliance.


WuXi Biologics and HanchorBio Enter Strategic Partnership to Advance Next-Generation Bi- and Multi-Functional Fusion Protein Pipeline

WuXi Biologics Obtains GMP Certification from UK MHRA for Commercial Manufacturing of an Ophthalmic Biologic

HanchorBio and WuXi Biologics have formed a strategic partnership to develop and manufacture next-generation bi-functional and multi-functional fusion proteins. This collaboration leverages HanchorBio's Fc-Based Designer Biologics platform to activate immune pathways for oncology and autoimmune diseases. WuXi Biologics will provide end-to-end services, including cell line development and GMP manufacturing, to ensure scalability and reliability. The partnership aims to streamline CMC development and accelerate the commercialization of innovative therapies. WuXi Biologics' global quality system and regulatory success bolster its manufacturing capabilities for complex biologics.

WuXi Biologics and HanchorBio partner to accelerate multi-functional fusion protein translation via end-to-end CMC and GMP services.

WuXi Biologics Collabs with HanchorBio on Next-Generation Fusion Protein Pipelines

Laine Mello Industry Outlook | Created with Canva

Laine Mello, director of Marketing at Ecolab for its bioprocessing business, shares her insights on the digital transformation of the pharmaceutical sector, the shift from batch to continuous processing, and the economic drivers shaping the industry’s future into 2026.

PharmTech: How is AI reshaping the drug discovery and manufacturing landscape?

 We are seeing AI fundamentally change the drug discovery timeline. Researchers are now able to design new molecules in weeks rather than months, which is an incredible shift that helps identify promising candidates with precision that wasn't possible even two years ago. This acceleration has massive implications for how quickly therapies can reach patients. On the manufacturing side, the impact is just as significant. Predictive maintenance tools are now catching equipment issues before they cause batch failures, and real-time analytics are allowing for much faster quality decisions. Digital monitoring is even making continuous processing more viable by removing the uncertainty that previously made manufacturers hesitant to move away from batch production.

What primary hurdles prevent companies from full adoption?

Adoption isn't always straightforward because regulatory frameworks are still catching up. There is an understandable need for transparency in how AI makes critical decisions, especially when patient safety is involved. Many facilities are struggling to integrate cutting-edge digital tools with legacy systems that might be 10-15 years old. We also have to address data governance and cybersecurity; these are not solved problems yet, and they cannot be afterthoughts when you are dealing with sensitive manufacturing data.

What benefits does continuous manufacturing offer over traditional methods?

It’s been a game-changer, we’ve seen facilities cut production cycles by 30%-40% while actually improving consistency. Shifting away from batch processing isn't just about increasing speed, it fundamentally changes how quality control and scale-up work. Once a facility can run continuously, it opens the door to optimizing operations in ways that simply weren’t possible before.

How is the industry responding to increasing margin pressures and the need for better process economics?

The industry is responding to margin pressure by investing heavily in next-gen therapies, such as biologics and advanced modalities that offer clinical benefits that generic competitors cannot easily replicate. This shift creates a demand for sophisticated purification solutions. For example, high-capacity chromatography resins are helping manufacturers maximize yield and recovery. These advanced resins enable intensified processing, which translates directly into cost savings and faster throughput. For biologic manufacturers dealing with complex molecules, these improvements in purification efficiency can be the difference between a commercially viable process and one that struggles economically.

What digital technology and sustainability shifts did you see in 2025?

In 2025, digital technology stopped being a "nice to have". Companies that had been piloting AI or the IoT for years suddenly found themselves racing to implement these tools across R&D and manufacturing to avoid falling behind the competition. At the same time, sustainability moved beyond simple compliance to become a genuine competitive differentiator. Companies are discovering that reducing water and energy consumption isn't just good for the planet, it improves their bottom line and makes their operations more resilient.

What will be the defining priorities for bioprocessing in 2026?

The reduction of the cost of goods will be the defining priority in 2026. Pricing pressures are intensifying, particularly for complex biologics. Manufacturers need to squeeze more productivity out of every single process step. We expect continuous and intensified bioprocessing to move from being innovative to becoming a standard practice, utilizing modular facilities and multi-column chromatography to deliver flexibility without sacrificing efficiency. We are also focused on solving technical challenges like stability and aggregation for high-concentration biologics and mRNA platforms that push the limits of current manufacturing.

How is the relationship between pharmaceutical companies and their suppliers changing?

We are seeing a shift where pharma companies are no longer looking for transactional relationships. Instead, they expect suppliers to act as strategic partners. This involves co-creating solutions, sharing technical expertise, and collaborating on process innovations. Using collaborations and acquisitions to access new technologies quickly is becoming essential for staying agile in a market where competitive advantages can erode very quickly.

The pharmaceutical sector is undergoing a digital transformation, with AI significantly accelerating drug discovery and enhancing manufacturing processes. Continuous manufacturing is replacing traditional batch methods, offering improved efficiency and quality control. However, regulatory challenges and legacy systems hinder full digital adoption. Economic pressures are driving investment in advanced therapies and purification solutions. By 2025, digital technology and sustainability became crucial competitive factors. In 2026, reducing costs and enhancing bioprocessing efficiency will be priorities, with a shift towards strategic partnerships between pharmaceutical companies and suppliers to foster innovation and agility.

Laine Mello, director of Marketin, Ecolab, details how AI, continuous manufacturing, and Pharma 4.0 drive bioprocessing efficiency and cost savings.

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Geneva / Switzerland - February 23, 2020: sign and logo of The World Health Organization (WHO/OMS) and UNAIDS | Image Credit: © hectorchristiaen - stock.adobe.com

United States’ withdrawal from the World Health Organization (WHO) on January 22, 2026

 marks a significant shift in global health governance. While WHO expressed regret over the decision,

 the move is rooted in U.S. allegations of organizational failure and politicization. This withdrawal is not merely a political event; it signals potential disruptions to global regulatory alignment, supply chain stability, and international research collaborations. “From a pharmaceutical manufacturing and development perspective, a US withdrawal from WHO would primarily be felt through regulatory fragmentation and loss of alignment, rather than any immediate operational shock,” notes Henrik Johanning, senior vice president, Quality & Strategy, Epista Life Sciences.

WHO responded to the US notification by highlighting the country's historical role as a founding member that contributed to the eradication of smallpox and progress against threats like polio, HIV, and antimicrobial resistance.

 WHO countered specific US claims with the following points:

• Refuting "Failures" in COVID-19: The US cited "WHO failures" and the "concealed" obstruction of information during the pandemic as a primary cause for exit. WHO defended its record, stating it activated its emergency system on December 31, 2019, and alerted the world through formal channels by January 11, 2020.

• Impartiality vs. Politicization: The US claimed the WHO pursued a "politicized, bureaucratic agenda."

 WHO maintained that it remains an impartial UN agency governed by 194 member states, existing to serve all countries without "fear or favour."

• Sovereignty: Addressing claims that it compromised US independence, WHO noted that while it recommended tools like vaccines and masks, it never recommended mandates or lockdowns, leaving those decisions to sovereign governments.

WHO warned that this withdrawal makes both the US and the world "less safe."

 The decision will be reviewed by the WHO Executive Board in February 2026 and the World Health Assembly in May 2026.

Why US Withdrawal From WHO Matters to Pharma Professionals?

Drug development, manufacturing, regulatory compliance, and supply chain management all rely on international stability and standardized frameworks to bring therapies to market efficiently. “WHO actually plays a critical, but often under-appreciated, role in providing global reference standards, technical guidance, and convergence frameworks, particularly for GMP, quality systems, pharmacovigilance, and essential medicines,” Johanning adds. “For industry, that translates into increased complexity: parallel regulatory narratives, more conservative development strategies, duplicated justifications, and higher friction when moving products across markets.”

 on pharmaceutical registration. The US withdrawal could lead to a divergence between US standards and those set by WHO and other international bodies. Such fragmentation complicates 

, as manufacturers may need to adhere to 

A critical concern for drug discovery and R&D teams is the proposed Pathogen Access and Benefit Sharing system.

 This system, part of the WHO Pandemic Agreement, is designed to ensure the rapid sharing of pathogens with pandemic potential to facilitate the development of vaccines and therapeutics. By withdrawing, US-based pharmaceutical companies might lose streamlined access to global pathogen data, potentially slowing down the 

 processes that the industry is currently prioritizing to 

The pharmaceutical industry is already sensitive to 

; even the threat of tariffs can rattle drug supply chains. The US exit from a central global health body adds a layer of geopolitical uncertainty that can complicate 

 for APIs and excipients. “In highly regulated environments, uncertainty itself becomes a cost driver,” says Johanning.

WHO coordinates global efforts against antimicrobial resistance and infectious diseases.

 For companies specializing in anti-infectives or vaccines, the loss of US involvement in WHO-led initiatives may shift the landscape for clinical trial sites and international procurement through organizations like WHO.

Industry professionals should view the US withdrawal as a catalyst for potential regulatory and operational volatility. As the industry shifts toward "agentic AI" and "sustainable efficiency," the loss of a unified global health framework may require firms to invest more heavily in independent regulatory intelligence and diversified supply chain strategies to manage the fallout.

“In the short term, most companies will continue to operate against FDA, EMA, and ICH requirements as before,” concludes Johanning. “The longer-term concern is whether reduced US engagement weakens global harmonization efforts at a time when supply chains, development programs, and manufacturing networks are more interconnected than ever. Interdependency and interconnections are key here; fragmentation anywhere ultimately creates friction everywhere.”

WHO Statement on Notification of Withdrawal of the United States

The United States' withdrawal from the World Health Organization (WHO) marks a pivotal change in global health governance, potentially disrupting regulatory alignment, supply chain stability, and international research collaborations. WHO countered US allegations of organizational failure and politicization, emphasizing its impartiality and historical contributions. The withdrawal could lead to divergent standards, complicating quality assurance and control in the pharmaceutical industry. It may also hinder US-based companies' access to global pathogen data, affecting drug discovery and development. The industry must prepare for increased regulatory and operational volatility.

US WHO withdrawal triggers pharma regulatory and supply chain risks while impacting global R&D collaborations.

US Withdrawal From WHO Creates New Pharma Logistics Vulnerabilities

Raj Puri Industry Outlook | Created with Canva

In a rapidly shifting pharmaceutical environment, the role of CDMOs has become increasingly complex. 

, oversees operation of two distinct divisions: one for life science and diagnostic products and another for aseptic drug product filling. In this interview, Puri discusses the economic, regulatory, and human factors shaping the industry's future.

PharmTech: In an industry often defined by high-tech equipment, how does a CDMO set itself apart from other firms?

 While anyone with sufficient capital can build a state-of-the-art facility, the only long-term sustainable advantage we have as a CDMO is our staff. Our executive team prioritizes getting and keeping the "right people on the bus.” As a midsize organization, we offer a dynamic environment where staff can work across various functional areas. This cross-training, where an operations professional understands the demands of Quality Assurance and vice-versa, creates an organization that truly understands the floor-level challenges of manufacturing. Our team’s intellectual curiosity drives us to adopt the latest technologies and techniques to remain on the cutting edge.

How are tariffs and massive manufacturing investments affecting the industry?

 has been the biggest and most unwelcome surprise of 2025, particularly those applied to equipment and consumables from non-US manufacturers. For Argonaut, which is completing a three-year investment plan for a new site, we were hit with a seven-figure tariff on a key piece of equipment. These costs have a chilling effect on investments because they make ROI calculations for multi-million dollar projects incredibly difficult.

We are also seeing north of $370 billion in announced investments for US manufacturing. If realized, this massive influx will likely extend construction timelines and increase costs significantly, disrupting the typical supply and demand curve. Simultaneously, if the government forces down pharmaceutical pricing, companies will be desperate to reduce their COGS. This creates an operational tightrope for CDMOs, we must find ways to lower costs while the FDA maintains its high expectations for product safety and manufacturing robustness.

How has the global climate changed how pharma companies view supply chains?

Geopolitical shocks and the lessons of COVID-19 have underscored the vital importance of redundancy. We are seeing a shift where biotech and pharma companies are looking to establish secondary manufacturing sites that are both geographically and economically segregated from their primary sites.

There is significant pressure to get products to market faster. What are the risks and rewards of compressed timelines?

Speed is critical for patient access and commercial viability. However, compressed timelines force companies to take a financial gamble, often requiring them to start mass production at the time of submission before receiving any indicative guidance from regulatory agencies.

I’ve seen cases where a Phase 3 study was discontinued early due to overwhelming efficacy. While this is a fantastic outcome for patients, it created a 6-8 month gap for data analysis and submission, leading to patient access issues before commercial approval. It also resulted in a smaller data set, leading to regulatory labels that required clinicians to use unfamiliar clinical trial severity scores. Ultimately, these compressed timelines require a robust contingency strategy and close coordination with regulatory bodies to negotiate allowances like rolling data submissions. They may even force companies to prioritize one jurisdiction, such as the FDA, over others due to differing requirements.

In the evolving pharmaceutical landscape, CDMOs face increasing complexity due to economic, regulatory, and human factors. The key differentiator for CDMOs is their workforce, emphasizing cross-functional training and intellectual curiosity. Tariffs and substantial US manufacturing investments impact ROI calculations and operational costs, challenging CDMOs to balance cost reduction with regulatory compliance. Geopolitical events and COVID-19 have highlighted the need for supply chain redundancy. Compressed timelines, while beneficial for patient access, pose financial risks and require strategic planning and regulatory coordination to manage potential data and approval challenges.

Raj Puri, CCO, Argonaut Manufactoring, provides key insight on navigating tariffs, supply redundancy, and the risks of compressed timelines.

Navigating the Operational Tightrope in Pharmaceutical Manufacturing

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