Christopher Cole

Maggie Saykali, Cefic, reviews how the EU’s proposed Critical Medicines Act aims to curb supply dependence and shortages by reforming drug procurement.

The CMA: Transforming Procurement to Ensure API and Excipient Supply Chain Resilience

In this part 1 of a 3-part interview regarding the presentation “The Critical Medicines Act: Game-Changing Opportunity for a Sustainable and Resilient Supply Chain?” at 

, held Oct 28-30 in Frankfurt, Germany, Maggie Saykali, Director – Specialty Chemicals, Cefic (European Chemical Industry Council), provides insight into the origins and development of the 

European Union’s proposed Critical Medicines Act

 (CMA). The push for the CMA originated during the 2020–2022 pandemic, during which medicine shortages underscored Europe's deep dependency on other regions—particularly Asia—for critical medicines, a situation aggravated by years of pharmaceutical offshoring. The core goal of the legislation is to identify the root causes of these shortages and drastically reduce them, explains Saykali.

The European Commission, working with major stakeholders, including API manufacturers, generics and branded medicine producers, patient associations, and member states, first developed a methodology to define what constitutes a "critical medicine." This methodology relies on such factors as a medicine’s supply chain structure, therapeutic family, and the number of suppliers. The concept of the CMA itself emerged in 2023, modeled after similar European legislation focusing on raw materials or semiconductors. This process involved the formation of a Critical Medicines Alliance, which included approximately 200 stakeholders. Published in March 2025 by the Directorate General for Health (DG SANTE), with assistance from the directorates for industry and medical emergencies, the act is now undergoing consultation with the European Parliament and Council, with a final vote anticipated by the end of this year or the first quarter of 2026, according to Saykali.

A primary focus of the CMA is reforming procurement practices. Currently, procurement is often based solely on price, resulting in a "one winner takes the whole tender" system that lacks guarantees regarding quality, security of supply, or adherence to environmental and social norms. This setup is detrimental to European manufacturers who must comply with strict EU regulatory frameworks. By reforming procurement criteria, introducing multi-winner tenders, and embedding environmental standards, the CMA seeks to ensure that compliance with stringent requirements becomes an advantage rather than a penalty for European producers. Furthermore, vulnerabilities must be addressed throughout the entire pharmaceutical supply chain. Saykali emphasized the often-overlooked components:

"We should not forget excipients because a lot of the critical medicines, generics or branded, actually depend on the excipients for their mode of action,” says Saykali. “And if we have a shortage and excipients, we… cannot make this medicine."

 and Part 3 of this interview and access 

*Editor’s Note: This transcript is a direct, unedited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.



My name is Maggie Saykali. I am the director of the European Fine Chemicals Group, and that is the trade association that represents the European manufacturers of active pharmaceutical ingredients, excipients. Intermediates and the key inputs, in fact. So our members are mostly CDMOs or some of them also make finished dosage forms, but mostly they are CDMOs and manufacturers of excipients or active pharmaceutical ingredients.

So the Critical Medicines Act is something that took a long time to elaborate. It originated during the pandemic, the idea of it. During the pandemic, we were witness to quite a few shortages that illustrated actually the existing situation that we all knew about, that for some critical medicines and with the offshoring that has been going on for years from Europe to Asia due to different factors that we were very dependent on other regions for the supply of some of our critical medicines. And this is more acute in generics of course, so we can go into the numbers behind that, But so, during the pandemic in 2020 until 2022, it was very obvious that the situation was exacerbated and that the solution must come. The European Commission worked with major stakeholders, meaning us as active pharmaceutical ingredient manufacturers, the generics manufacturers, and the branded medicines manufacturers mainly, but also patients associations, pharmacists, and member states to come up with a solution, first of all to identify the root causes for the shortages, and then, of course with the purpose of finding solution to alleviate them or to reduce them drastically.

So that started with a methodology to determine what is actually a critical medicine, what makes for the criticality. And the commission came up with a methodology with different factors based on the structure of the supply chain of a medicine. It's therapeutic a family factors like the number of suppliers, et cetera.

So there was a rather complex matric that would then identify what is a critical medicine and what is and differentiated from medicines that might be short but not qualify for the criticality. After that, in 2023, the idea came that we need a critical medicines act for Europe on the same model as the one for the chips or for raw materials, et cetera.

And this idea originated with a few member states that pushed it really hard together with the commission, European Commission, and there was a Critical Medicines Alliance formed. With the stakeholders, there was about 200 stakeholders, but the critical alliance board, steering board was these associations that I mentioned, plus representatives of patients, pharmacists, et cetera, and member states.

And we worked on what were the possible solutions. And then the commission went to work, put this all together. And in March, 2025, the Critical Medicines Act was published, and it was published by the director General for Health (DG SANTE). So the directorate in the European Commission that is responsible for everything that concerns health, but it was also helped by the directorate for industry and the one for medical emergencies.

So since then the Critical Medicine Act entered the European decision-making process, which involves consultations with the European Parliament and the council, meaning the member states, the European member states, and this is where the process is now. The council, the commission, and the parliament are discussing, and the final vote will happen we think, at the end of this year or first quarter of 2026. And then it'll be it'll have legal value.

First of all what we see today is a situation, for instance, in procurement that is completely based on cost, a price only meaning one winner takes the whole tender and it's only based on price. Meaning there are no guarantees on quality, on security of supply, on any other factors on the fact that it's produced, respecting environmental norms or social norms, all of that doesn't enter into account.

And is actually detrimental to European manufacturers because we have a set of standards, environmental, social that we have to comply with. That's normal. These are the European re regulatory frameworks. And some of the other suppliers from other world regions are not abiding by these, and they have different cost structures.

By addressing the issue of the criteria for procurement, this might give European manufacturers better possibilities to be competitive. Plus in putting environmental criteria in there as we asked for, that will give value. To the fact that we produce according to a very strict and very tight ESG requirements.

And this means that we will not be penalized because of it, but that will become rather an advantage for us. Plus then we are asking for not to go for the one one winner takes it all. But to have multi-winner tenders, which will put at an advantage Also people who manufacture in Europe.

Be it finished dosage, forms, active pharmaceutical ingredients excipients, et cetera. We should not forget excipients because a lot of the medicines critical or sorry, generics or branded actually depend on the excipients for their mode of action. And if we have. Shortage and excipients. We are at the same point that we cannot make this medicine.

So the role throughout the supply chain, we have to make sure that there are no causes for vulnerability and no possibilities for shortages or delivery of faulty or not up to standard quality ingredients.

Videos

Eva-Maria Hempe, NVIDIA, explains that accelerated computing, data governance, and “lab in the loop” are key to bio/pharma’s AI strategy for transforming drug discovery and R&D.

AI and Accelerated Computing Propel the Biopharma Ecosystem Forward

In this part 1 of a 3-part interview regarding the presentation “The State of AI in Next-Generation R&D” at CPHI Europe 2025, held Oct 28-30 in Frankfurt, Germany, Eva-Maria Hempe, Head of Healthcare & Life Sciences, NVIDIA, discusses the pivotal role of artificial intelligence (AI) and accelerated computing in transforming the bio/pharmaceutical ecosystem.

A core challenge and competitive strength in this sector stems from the prevalence of siloed and proprietary data. Leading organizations are tackling this by adopting advanced AI platforms designed to integrate, process, validate, and harmonize these complex, disparate data sources, Hempe explains. She adds that specialized tools operate atop these platforms, including Parabricks for genome sequencing, MONAI for medical imaging, and BioNeMo for biological language models, all aimed at accelerating analysis and extracting rich insights from often unstructured data.

According to Hempe, success in AI hinges directly on data quality and integrity, adhering to the principle that "garbage in garbage out is just a matter of fact." This necessity drives the implementation of robust data governance frameworks, such as ALCOA (attributable, legible, contemporaneous, original, accurate), alongside rigorous automated validation routines and training, she says. These AI platforms support safe model development practices by allowing the tracing of data provenance.

Hempe notes that the single greatest operational opportunity identified is what is termed the “lab in the loop”—the seamless integration of the wet lab (experimental work) and the dry lab (computational analysis). By automating wet lab processes, high-quality, repeatable data is generated, complete with necessary metadata. This reliable data then feeds computational models, which in turn predict the optimal next set of experiments to run, thereby accelerating the entire research workflow, she states. This systematic, data-driven exploration is essential given the immense scope of chemical possibilities. Speaking to this scale, Hempe notes, "This is more than sand grains in the universe." Furthermore, foundation models, such as Evo 2, are being deployed to deepen the understanding of genetic mutations at scale, predict their functional impact, enhance candidate selection, and encourage crucial cross-disciplinary collaboration.

Check out 



I'm Eva-Maria Hempe and I lead Healthcare and life Sciences for NVIDIA in Europe, Middle East, and Africa. What this means is we're working with the whole ecosystem, so both healthcare and life sciences. I sometimes joke I do everything from genomics over direct discovery, surgical robots all the way to helping physicians and nurses to do their job better. But that's a bit the span we're covering. We're working with companies of all different sizes. We're working with both established companies as well as startups and really helping them to use AI to the fullest and avoid reinventing the wheel and really tapping into the potential of accelerated computing.

It's one of the big strengths or big competitive advantages actually in this field is that there is, you can call it siloed, you can call it proprietary data, but of course it is something that has to be managed. And in general, the way we see is that leading organizations have advanced AI platform, which really allows them to integrate the data, to process the data, and to validate the data.

And then on top of such a platform, we're offering specialized tools like, for example, Parabricks for genome sequencing or MONAI for medical imaging or BioNeMo or biological language models, as well as some kits for agentic AI that then enable accelerating data analysis and really reducing the manual input and you really get this rich insights from these complex data sets, which are just, they're just a matter of fact, this ius nothing we're going to be changing in a way, that is the richness of this field, is the unstructured data, is the multitude of data. And so these platforms are then really supporting centralization without really having to repackage everything and the harmonization of those disparate data sources.

The question of data quality is actually really important, and integrity. So, the old proverb of garbage in garbage out is just a matter of fact. So, it's really about enforcing compliance at the data lifecycle and having a really robust data governance framework. So we're seeing concepts like ALCOA (attributable legible, contemporaneous, original, accurate) or other frameworks being rolled out. Then it's really about having the right processes on audits, training, and as well automated validation routines. And we can help with that by having, again, a platform that allows us to trace data provenance and to really have safe model development practices that you can rely on as as a basis for the more behavioral aspects, which I just outlined.

I think the greatest opportunity really is what we call the lab in the loop or bridging the wet lab and the dry lab, and that has kind of AI challenges on both sides. In the web lab, it's really about automation, so you want to have data, which is, as you said before, high quality. So you have to have the right metadata, and you want to have a really repeatable loop, a really repeatable workflow here. So we are seeing companies working in that space who are, automating the entire wet lab, like all the processes of transferring plates between different machines and capturing all that data in a harmonized form. And that data then provides the basis for better predictions of what experiments we should be running, and then you're basically closing the loop. So you're having the wet lab, which is the validation, which gets you the real data in a good quality thanks to automation, and that data quality then feeds models which can predict which experiments to do next. I think that's once this flywheel, you get to go, it can really accelerate things because the chemical space is just so huge. I mean, this is more than sand grains in the universe. And so far we've just been looking at where we felt comfortable in a way, looking at where we've been always looking. And with this more data-driven approach, we can really explore that space in a much more structured and systematic way.

And I think there's another opportunity. So these are foundation models like Evo 2, that's to really dive deeper into genetic mutations at scale and understand and predict the functional impact of that. And then, again, this allows better candidate selection and, again, drives cross-disciplinary collaboration.

How 100% Pharmaceutical Tariffs Will Impact Domestic Manufacturing and Supply Chains

A new trade policy from President Donald Trump introduces a significant tariff on imported pharmaceuticals, directly impacting manufacturing strategies and supply chains for companies serving the United States market (1). This development adds another layer of complexity to an industry already navigating supply chain pressures, regulatory changes, and capital market demands (2-7).

In a statement released on Thursday via Truth Social, the president detailed a plan to impose a 100% tariff on all imported branded or patented pharmaceutical products, effective October 1, 2025 (1). This move follows months of discussion around potential sectorial tariffs, with rates previously suggested to be as high as 250% to incentivize domestic production. The president stated his reasoning clearly, noting, "because we want pharmaceuticals made in our country".

"The details of the 232 tariffs on pharmaceutical products and APIs have yet to be released, but President Trump’s social media post last night indicates that the Department of Commerce is preparing to announce 100% tariffs on what is expected to be a broad swath of drugs and ingredients," adds Jason Waite, international trade lead at Alston & Bird.

The policy includes a critical provision for pharmaceutical developers: the tariff can be avoided if a company has initiated the construction of a manufacturing facility within the US (1). "There will, therefore, be no Tariff on these Pharmaceutical Products if construction has started," the president wrote. In other words, as Waite states, "Significantly, it appears as if generic drugs may escape the tariffs, and that companies building plants in the United States may enjoy some form of exemption." This ultimatum to onshore production presents a pivotal challenge for industry leaders, from those managing outsourcing and supply chains to executives making long-term capital investment decisions. 

It remains unclear if this new 100% rate will be applied in addition to a previously announced 15% tariff on pharmaceuticals imported from the European Union (1). This lack of clarity introduces further uncertainty for global manufacturing and logistics planning. Indeed, Waite points out that "there are many questions about exactly how companies will be able to avail themselves of [the aforementioned] exemption, but, overall, the President’s announcement suggests a recognition of the complexities of the pharma market and manufacturing, and the impending emergence of a novel tariff regime intended to meet some of the challenges that industry has brought to the Administration’s attention over the past five months."

How are industry and investment trends responding?

For professionals tracking major industry moves and investment strategies, the tariff announcement crystallizes a trend that has been building for months (1-23). Many major pharmaceutical companies, anticipating such policy shifts, have already committed to substantial investments in US-based manufacturing infrastructure.

AstraZeneca announced a $50-billion investment in late July, expressing confidence in its ability to produce nearly all its drugs for the US market domestically (24). Other significant commitments have come from Johnson & Johnson ($55 billion), Roche ($50 billion), Bristol Myers Squibb ($40 billion), Eli Lilly ($27 billion), Novartis ($23 billion), and Sanofi (at least $20 billion) (1). Eli Lilly recently broke ground on two new plants as part of its investment.

While these multi-billion-dollar projects signal a clear direction, questions remain about whether these new facilities will have the capacity and technical capability to manufacture every patented product currently marketed in the US (1). For those in drug development and manufacturing, this raises critical considerations about technology transfer, scalability, and the complexities of establishing new production lines for diverse product portfolios.

 editorial advisory board member Eric Langer, managing partner at BioPlan Associates, says "In the near term, the lack of clarity around how these tariffs will be implemented is likely to increase uncertainty. Uncertainty creates roadblocks to investments, and bio/pharma investments are not quickly executed. So, the investment announcements may be more aspirational or speculative, and the near term impacts minimal."

Langer notes that this invest-or-tariff model could also be used by other countries in the longer-term. "This could result in zero net gains in terms of expanding US pharma manufacturing," he points out. "Worse, it could end up creating inefficient manufacturing sites that are built simply to avoid tariffs in major markets."

Building plants in the US will take time, notes 

editorial advisory board member Rory Budihandojo, an independent GMP consultant. "And the finished drug product cost will be potentially higher than manufacturing them in other countries; the increased production cost will be passed on to consumers."

Linda Girgis, MD, a primary care physician with a private practice in South River, NJ, agrees with Budihandojo. "Raising tariffs 100% can hurt patient already being squeezed by the high cost of pharmaceuticals," she says. Many patients are already unable to afford certain medications and are forced to take cheaper alternatives that may not be as effective, or no medications at all." 

What are the regulatory and supply chain implications for bio/pharma?

This policy shift intersects directly with ongoing industry-wide conversations about securing supply chains and navigating the regulatory landscape (3). The vulnerability of established drug supply chains has been a recurring theme, and this tariff serves as a powerful catalyst for onshoring initiatives (3,25,26).

To support this domestic manufacturing push, FDA recently launched its PreCheck initiative (27). This program is designed to streamline the process for companies seeking to build new manufacturing sites in the US. For regulatory affairs professionals, understanding and leveraging such initiatives will be crucial for managing timelines and ensuring compliance, echoing the industry's interest in programs like the European Commission's efforts to streamline drug lifecycle management.

The ultimatum effectively ties market access to domestic investment, forcing companies to re-evaluate their global manufacturing footprint. This will have far-reaching effects on everything from the development of small molecules and active pharmaceutical ingredients to the sourcing of excipients, areas already facing significant supply chain pressures (3).

Dr. Girigs notes that disruptions to the drug supply chain can have disastrous health effects for many patients, adding that "medications for chronic medical problems need to be taken on a daily basis and, if they become unavailable, can lead to medical complications and/or death."

What about pharmaceuticals that aren't fully generic or entirely new?

"We should discuss drugs approved under 505(b)(2)—a category that is neither fully generic nor entirely new," says 

 editorial advisory board member Aloka Srinivasan, PhD, principal and managing partner, Raaha LLC, and an expert on generics. "Generics, which are pharmaceutical equivalents of innovator products, are approved under 505(j). New chemical entities, which require the full spectrum of nonclinical and clinical studies, are approved under 505(b)(1)."

Srinivasan adds that the 505(b)(2) pathway, however, applies to pharmaceutical alternatives to existing products. "These drugs are approved with a limited set of data and may or may not be under patent protection," she explains. "For example, if the innovator product is a 10 mg tablet, a company might develop a 10 mg capsule or an oral solution. By demonstrating equivalence to the original tablet through minimal studies like in case of generics, the company can establish safety and efficacy and obtain FDA approval under the 505(b)(2) pathway. Most 505(b)(2) have some patent protection, though they are not as extensive as new chemical entities."

Although these products are technically considered "new" by the FDA, according to Srinivasan, they are very close to generics and can offer advantages, such as improved patient compliance. "Additionally, they may help reduce costs of drugs and give patients better choices," she continues. "It will be interesting to observe how such products are impacted in the market and whether 100% tariff on such products affects the broader drug landscape."

Budihandojo does think it will make more sense to manufacture generics in the US than overseas "due to the quality and data integrity issues we are seeing in overseas drug production in India and China (where most generics are being produced). The lower profit margin for manufacturing generics  potentially leads to manufacturers trying to cut corners to save cost, impacting quality." He adds that generic manufacturing in the US makes it easier for FDA to "oversee" production quality and compliance.

What is boils down to, says Srinivasan, is that nobody is able to clearly understand the 100% tariff. "President Donald Trump announced Thursday that brand-name or patented pharmaceutical products will be subject to a 100% tariff starting October 1 – unless the drugmaker is building a manufacturing plant in the US," she notes. "The definition is not clear, there are several brand named products with no patents; will they be exempt? Also, several products may have patents but not a 'brand name.' We need more clarity around these."

Trump Will Slap Tariffs on Imported Drugs, Trucks and Household Furnishings

Ask the Expert: Questions about Real-World Evidence and Supply Chain Security

Drug Digest: Small Molecules, APIs, and Excipients—Trends, Challenges, and Opportunities

Stakeholders React to FDA's Push for Quality, Integrity, and Uniformity

Behind the Headlines Episode 25: Major Big Pharma Moves, Pressures of Capital Markets at Intersection with Autologous Versus Allogeneic

CDC Panel’s Vote Separates MMRV Shot and What That Means

FDA Abandoning Expert Reviews: Implications for Drug Developers and Quality Professionals

Tariffs & Trade Policy: Results of a Bio/Pharma Peer Survey

Understanding the Operational Impacts of the 15% US-EU Pharma Tariff Cap

Bio/Pharma's Tariff Response: Key Insights from Industry Survey Results

Onshoring’s Unexpected Benefits: Quality, Efficiency, and Supply Chain Resilience

How Pharma Can Build Resilient Supply Chains Amid Trade and Tariff Shifts

Trade Policy Shifts: Compliance and Cost Strategies for Pharma

Tariffs & Trade Policy: What to Watch for, Cost Impacts, and Supply Chain Strategies

What Pharma Manufacturers Need to Know About US Trade Policy Changes

9 Implications of Trump’s Tax-Cut and Spending Bill for the Bio/Pharmaceutical Industry

Key Leaders at BIO 2025 Respond to Continuing Political Shifts

FDA Refines Regulatory Pathway for Canadian Drug Importation

Trump Issues Executive Order Aimed at Reducing Prescription Drug Prices for US Patients

Trump Directs FDA, EPA to Reduce Regulatory Barriers to Domestic Pharma Manufacturing

Industry Outlook 2025: Political Implications for Pharma

Industry Outlook 2025: The Impact of Politics on Pharma

Pharmaceutical Tariffs Are Imminent: How Industry is Bracing for Impact

AstraZeneca to Invest $50 Billion in US Manufacturing and R&D. 

Acetaminophen and Autism: Industry Impacts of the White House Statement

Pfizer’s $4.9 Billion Acquisition of Metsera Enhances Obesity Drug Pipeline

FDA Moves to Strengthen Drug Supply Chain with PreCheck Program. 

European Commission Streamlines Drug Lifecyle Management.

Articles

President Trump's new trade policy introduces a 100% tariff on imported branded or patented pharmaceuticals, effective October 2025, to encourage domestic production. Companies can avoid the tariff by starting construction of US manufacturing facilities. This policy adds complexity to the pharmaceutical industry, already facing supply chain and regulatory challenges. Major companies like AstraZeneca and Johnson & Johnson have announced significant investments in US manufacturing. The FDA's PreCheck initiative aims to streamline domestic manufacturing site approvals, further influencing industry strategies and supply chain dynamics.

The 100% tariff on imported drugs will pressure pharma companies to build manufacturing sites in the US or face significant costs.

How 100% Pharmaceutical Tariffs Will Impact Domestic Manufacturing and Supply Chains 

In the first-ever “Two-Minute Mysteries: BioPharma Stories,” Agilent’s Ken Boda shares the mystery of a low reading.

Two-Minute Mysteries: BioPharm Stories - Episode 1: The Mysterious Cause of a Low Reading



In this inaugural episode of the “Two-Minute Mysteries: BioPharma Stories” video series--created in partnership with our sister publication

--Ken Boda, a dissolution product specialist at Agilent Technologies, shares the story of a mysterious low reading.

Ken Boda
Dissolution Product Specialist
Agilent Technologies

Ken works primarily with customers in the US and Canada. He has a wide knowledge of analytical methods, method development, validation, and regulations in the pharmaceutical environment. He enjoys educating people on the science of dissolution and helping customers work through their method challenges.

Specialties: Dissolution Method Development and Validation, Dissolution Failure Investigations, HPLC, UV, Physical Testers, well-versed in USP, FDA, and ASTM regulations in regard to pharmaceuticals, nutritional supplements, and medical devices.

Find Ken on 



Hi, my name is Ken Boda. I'm a dissolution product specialist with Agilent Technologies. I wanted to share a little, scientific mystery with you. Oftentimes, when we're working with dissolution and we have a failure, these can present as little mini mysteries to try to do a failure investigation, determine what's going on.

One example of a quick mystery is a customer of ours who had a dissolution with baskets and a disintegrant dosage form, and they had unexplained low results on a particular dissolution bath. They asked us to come out and take a look at it. We brought our 280-DS mechanical qualification system to the site to evaluate the system.

We found that the alignment of the system was good. However, we did note that there was some interesting spikes in vibration over time, and it turned out that the vibration was coming from a refrigerator that was on the same bench top as the dissolution. So what was going on? What was causing those low results?

Well, when we have spikes in vibration in a disintegrant dosage form in basket, we can shake that basket out and we end up having the powder land on the bottom of the vessel, and at the bottom of the vessel, we don't have much mixing. And so, as a result, we have a large amount of drugs sitting at the bottom of the vessel where it can't dissolve.

And that leads us to having low results. This is really important to monitor vibration and dissolution, because it can have a lot of interesting effects. We usually think of vibration causing higher and more variable results, but that's not always the case as evidenced by this basket study. So, thank you, and hope you enjoy the rest.

We highlight the week’s industry news in an easy-to-consume, fun format.

PharmTech Weekly News Roundup - Week of September 15

In this PharmTech video feature, we highlight the week’s industry news in an easy-to-consume, fun format. New roundups will drop every Friday, so be sure to come back each week. And be sure to check the links below for more in-depth coverage.

Collectively, the week’s coverage discusses the pharmaceutical industry’s recent developments and current regulatory landscape. Read on for recaps of each story we’ve covered.

ABC Farmaceutici to Celebrate Centennial at CPHI Frankfurt

ABC Farmaceutici will commemorate its 100th anniversary

 at CPHI Frankfurt from October 28–30, 2025, unveiling a special logo and hosting an event at its booth. The company, now part of ICE Pharma, has expanded globally into over 50 countries and developed expertise in active pharmaceutical ingredients (APIs), dermo-cosmetics, and ursodeoxycholic acid (UDCA), a treatment for a rare autoimmune liver disease.

CPHI Frankfurt Announces 2025 Pharma Awards Finalists

 have announced finalists across 14 categories, highlighting innovations in synthetic biology, artificial intelligence and sustainable packaging. The awards underscore the industry's shift towards digital tools and green practices, with winners to be revealed at the CPHI Frankfurt opening ceremony on October 28.

Cancer Research UK and Nxera Launch Phase IIa Trial for Immuno-Oncology Drug

Cancer Research UK and Nxera Pharma have begun a Phase IIa clinical trial for HTL0039732

, an investigational small-molecule immunotherapy for advanced solid tumors. The drug targets the EP4 receptor to address tumor-mediated immunosuppression, a different mechanism from traditional checkpoint inhibitors, and will be tested in cancers with high unmet needs, including colorectal and prostate cancers.

Competition Intensifies in MASH Treatment with New Drug Approvals

market for metabolic dysfunction-associated steatohepatitis (MASH) is expanding rapidly

 following the FDA's approval of Madrigal Pharmaceuticals' Rezdiffra and Novo Nordisk's Wegovy. With the MASH market projected to reach $31.8 billion by 2033, major pharmaceutical companies like Eli Lilly, GSK, and Boehringer Ingelheim are advancing their own innovative therapies, creating a highly competitive landscape.

FDA Issues Warnings Over Misleading Drug Promotions

FDA issued warning letters to Eli Lilly, Novo Nordisk, and Hims & Hers Health

 for misleading promotional communications regarding popular weight-loss drugs. The warnings specifically criticized a television program for omitting and minimizing significant risk information for drugs like Mounjaro and Ozempic, signaling heightened regulatory scrutiny of advertising practices across all media platforms.

FDA Approves Enbumyst, a Nasal Spray for Edema

, a bumetanide nasal spray for treating edema associated with congestive heart failure, liver, and kidney disease. Designed as a self-administered outpatient therapy, the nasal spray offers rapid absorption comparable to IV diuretics, potentially reducing hospitalizations and healthcare costs for the 6.7 million Americans with heart failure.

The week's pharma news includes FDA warnings for Lilly and Novo, new drug approvals for MASH and edema, and a look ahead at CPHI Europe.

Conceptual stop sign with stormy background. Warning, caution and danger sign | Image Credit: © Rechitan Sorin - stock.adobe.com

The pharmaceutical industry stands at a critical juncture, as a recent report suggests FDA is considering a significant departure from its long-standing practice of utilizing external expert advisory committees for new drug applications (1). This potential shift, initially signaled by George Tidmarsh, head of FDA’s Center for Drug Evaluation and Research, has profound implications for regulatory strategy, transparency, and internal quality systems, directly impacting the core concerns industry professionals.

Historically, FDA advisory committees, established in their current form in 1972, have served a crucial role by convening external experts to review evidence and vote on the approval of drugs, vaccines, and medical devices, particularly when the agency faces complex or difficult decisions (2). Tidmarsh, however, argues that these panels are redundant and time-consuming, involving "a tremendous amount of work for the company and for the FDA." He suggests that the agency should instead focus its efforts on "big questions," such as evaluating appropriate endpoints for different drug classes.

A key part of the FDA's rationale for this potential change is the recent initiative to publish Complete Response Letters (CRLs) in real time (3). These letters, which previously required Freedom of Information Act requests, provide developers with "early insight into regulatory deficiencies to improve development planning." Tidmarsh posits that releasing CRLs promotes a level of transparency "akin to the advisory meetings’," suggesting the letters make expert committee meetings on individual drugs redundant (1).

However, this perspective is met with considerable pushback from former FDA officials, academics, and industry experts. Critics argue that abandoning expert reviews would "shield the agency’s decisions from public scrutiny” (1). Robert Califf, former FDA Commissioner, finds Tidmarsh’s reasoning "hard to follow," emphasizing that it is "extremely useful for people inside FDA to find out what other experts think before they make their final decisions" and essential for public understanding (1).

How do CRLs and expert review committees differ?

Experts highlight several critical differences between CRLs and advisory committee meetings:

• Public Input and Dialogue: Advisory committees allow experts to ask questions of the company or FDA that might not have been considered, and provide a vital public forum for comment on FDA decisions (1). A disclosed CRL informs the public 

 an application was rejected, while an advisory meeting seeks 

 a decision is made, a crucial distinction.

• Internal Consensus and Scrutiny: Committees often help settle disagreements within the FDA itself, providing an external, unbiased perspective that can strengthen decisions (1).

• Rigorous Evaluation: While the FDA is not obliged to follow committee votes, their input is particularly valued for "important new types of medications or when a decision is especially tricky because of high demand for a product that may have limited value," as exemplified by the controversial approval of Biogen's Aduhelm despite a "no" vote from its advisory committee (4).

For industry professionals, this potential shift carries significant implications across various domains:

Companies will need to closely monitor how drug approval pathways might evolve. If external expert validation becomes less common, drug developers may need to anticipate heightened internal FDA scrutiny or adjust their data submission strategies to preemptively address potential concerns that would typically be vetted in a public forum.

Quality systems and compliance excellence

A less transparent or externally validated regulatory environment could place even greater emphasis on robust, holistic internal quality systems to ensure compliance excellence and mitigate risks without the benefit of public expert debate (5,6). Ensuring data integrity and quality of data will become even more paramount for regulatory compliance and patient safety (7).

The pharmaceutical industry operates under intense public scrutiny. A move away from public advisory committees could, as critics suggest, reduce transparency and potentially erode public trust in drug approvals (1). This would necessitate companies to be even more proactive in communicating their commitment to quality and patient safety.

While Tidmarsh indicates committees would still be consulted on general regulatory issues, the absence of detailed, specific drug reviews could alter how industry and the FDA collaboratively address emerging scientific and manufacturing challenges (1).


Evidence of this shift is already emerging, with the FDA holding only seven advisory committee meetings since the current administration entered the White House, compared with 22 in the same period last year (1). While some initially viewed this reduction as temporary, others perceive it as a "strategic" move to "consolidate power in the agencies" and reduce accountability to outside experts or the public. Concerns have also been raised about the potential for replacing public, vetted committees with "handpicked scientists" who may align with specific views.

As the FDA navigates these changes, industry professionals, from drug development and manufacturing professionals to those in quality systems and regulatory affairs, must remain vigilant. Understanding the evolving regulatory landscape, proactively strengthening internal quality and compliance frameworks, and upholding the highest standards of transparency will be crucial for success in this new era of drug approval.

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The FDA is contemplating a shift away from using external expert advisory committees for new drug applications, a move that could significantly impact regulatory strategy, transparency, and internal quality systems. George Tidmarsh, head of the FDA’s Center for Drug Evaluation and Research, argues that these committees are redundant and time-consuming. Critics, however, warn that this could reduce public scrutiny and transparency. The potential change emphasizes the need for robust internal quality systems and proactive communication to maintain public trust and ensure compliance in the pharmaceutical industry.

This potential shift has profound implications for regulatory strategy, transparency, and internal quality systems, directly impacting the core concerns of industry professionals.