Richard Jaenisch surveys pharma's biggest recent AI deals

Manufacturing Intelligence: Sizing Up Pharma's Biggest AI Deals, Part One

Three regulatory actions are converging to move biologics, gene therapy, and the ATMP platform forward quickly

Regulatory Actions Impacting Biologics

June saw regulators look back at their achievements, take stock of drug shortages, and advance treatments for rare diseases.

Regulatory Actions for June 2026

The use of artificial intelligence is facing harder questions of governance and data quality

PharmTech Weekly Roundup—July 17, 2026

How evolving regulatory expectations are reshaping pharma development and manufacturing.

Drug Digest April 2026

Genglycos (pariglasgene brecaparvovec-opnr), a gene therapy for adult and pediatric patients eight years and older with glycogen storage disease type Ia recieved accelerated approval from the FDA.

Ultragenyx Pharmaceutical received accelerated approval from the FDA for Genglycos (pariglasgene brecaparvovec-opnr), a gene therapy for adult and pediatric patients 8 years and older with glycogen storage disease type Ia (GSDIa), the company announced on August 19, 2026.

 The approval marks the company's fifth FDA approval and its first for a gene therapy product.

GSDIa is an ultra-rare, inherited metabolic disorder caused by pathogenic variants in the gene encoding glucose-6-phosphatase, an enzyme needed to release glucose from the liver into the bloodstream.

 Without it, patients face a constant risk of severe, potentially life-threatening low blood sugar and must follow an around-the-clock regimen of raw cornstarch intake to maintain stable glucose levels. The condition affects an estimated 1500 to 2500 patients in the United States and 6000 to 8000 worldwide in commercially accessible regions.

The approval rests on data from the phase 3 GlucoGene study, a 48-week randomized, double-blind, placebo-controlled trial that enrolled 46 participants.

 Those who received the therapy showed a statistically significant reduction in daily cornstarch requirements compared with placebo. Following the initial 48-week period, eligible participants crossed over to receive the alternate treatment, with follow-up analyses planned at 96 and 144 weeks to track longer-term outcomes.

“Day-to-day management of GSDIa requires a relentless regimen of raw cornstarch and strict dietary management that can be extraordinarily demanding for patients and families. Even with meticulous adherence to this regimen, patients must be perfect. Any missed cornstarch puts patients at risk of severe hypoglycemia, seizures, and even death,” said David Weinstein, MD, MMSc, a longtime researcher in the GSDIa field, in a press release.

 “The approval of Genglycos represents a major step forward for the GSDIa community and reflects almost 30 years of work and scientific progress aimed at improving safety and the quality of life of people living with this disease.”

How Does This Approval Affect Pharma Development and Manufacturing Broadly?

Since the therapy was cleared under the accelerated approval pathway, continued marketing authorization depends on confirmatory evidence of clinical benefit.

 Ultragenyx has committed to a post-marketing disease monitoring program that will follow 50 treated patients and 20 untreated control patients, the latter excluded from treatment due to preexisting antibodies to the viral vector, over a projected 10-year period. That structure illustrates how sponsors of ultra-rare disease gene therapies are increasingly relying on long-term, real-world monitoring to satisfy regulators when trial populations are too small to generate conventional statistical power.

The therapy also carries a boxed set of warnings common to adeno-associated virus vector gene therapies, including hypersensitivity reactions, immune-mediated liver toxicity, and adrenal insufficiency tied to the corticosteroid regimen used to manage hepatic reactions.

 Distribution will run through a national network of qualified treatment centers trained to administer the therapy, an access model increasingly used for specialized cell and gene therapy products that require infusion oversight and long-term patient monitoring beyond what a typical infusion center provides.

“As our first gene therapy approval, Genglycos represents an important achievement for our company and the realization of the promise of a powerful new tool to deliver transformative medicines for people living with rare diseases,” said Eric Crombez, MD, chief medical officer, Ultragenyx, in the press release.

The product is manufactured entirely at Ultragenyx's gene therapy manufacturing facility in Bedford, Massachusetts, a detail relevant to ongoing conversations in the sector about internalizing viral vector production rather than depending on external capacity.

 As more gene therapy sponsors weigh build-versus-buy manufacturing decisions, cases like this one offer a data point on how in-house facilities can support both scale-up and consistent quality control for accelerated-approval products.

Why Do Therapy Sponsors Build Their Own Manufacturing Capacity Instead of Outsourcing?

Ultragenyx's decision to manufacture Genglycos entirely in-house reflects a build-versus-buy calculation that has become increasingly common across the gene therapy sector.

 Viral vector production, particularly for AAV-based therapies used in vivo, requires specialized biosafety containment, nucleic acid workflows, and scale-up expertise that many contract development and manufacturing organizations (CDMOs) still struggle to deliver quickly. Industry reporting has noted that CDMO wait times for viral vector production slots can exceed 18 months, a delay that larger, well-resourced sponsors often choose to avoid by internalizing production. Building in-house capacity also allows a company to retain control over its manufacturing process and intellectual property, an advantage particularly relevant for orphan drug programs where development timelines are already compressed. For sponsors without the capital or technical experience to construct a dedicated facility, however, outsourcing, despite its scheduling constraints, often remains the only practical path to commercial supply.

Ultragenyx announces U.S. FDA approval of GENGLYCOS™ gene therapy, the first-ever FDA-approved treatment designed to treat the underlying cause of glycogen storage disease type Ia (GSDIa). Press Release. Ultragenyx Pharmaceutical Inc. August 19, 2026. 

https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-us-fda-approval-genglycostm-gene-therapy

Strategies for viral vector manufacturing. Press Release. Cytiva. February 16, 2025. 

https://www.cytivalifesciences.com/en/us/insights/strategies-for-viral-vector-manufacturing

Articles

Ultragenyx secured FDA accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), an AAV gene therapy for patients ≥8 years with glycogen storage disease type Ia due to glucose-6-phosphatase deficiency. Approval is supported by the 48-week, randomized, double-blind, placebo-controlled phase 3 GlucoGene trial (n=46), demonstrating a statistically significant reduction in daily raw cornstarch requirements versus placebo. Ongoing authorization hinges on confirmatory evidence via long-term monitoring, alongside class boxed warnings and specialized center-based distribution. Manufacturing is fully in-house in Bedford, Massachusetts.

The FDA grants accelerated approval to Ultragenyx's Genglycos, the first gene therapy for GSDIa, raising manufacturing and post-market monitoring questions.

GSDIa Gene Therapy Approval Signals New Path for Rare Disease Manufacturing

Pasatru (garetosmab-grts) has been approved by the FDA to treat symptoms of fibrodysplasia ossificans progressiva

The FDA has approved Pasatru (garetosmab-grts) to reduce the formation of new heterotopic ossification lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP), Regeneron announced on August 19, 2026.

 The approval marks the first therapy demonstrated to reduce new lesion formation in a placebo-controlled trial for this ultra-rare genetic disorder, according to the company.

FOP is characterized by abnormal bone formation that progressively infiltrates muscles, tendons, ligaments, and other connective tissues.

 When this bone growth affects the jaw, spine, hip, or rib cage, it can impair speaking, eating, walking, or breathing. Approximately 900 people worldwide are diagnosed with the condition, and most patients lose the ability to walk by age 30, with a median survival age of 56.

The therapy is a fully human monoclonal antibody that blocks Activin A, a protein identified through the manufacturer's research as a driver of abnormal bone development in patients with the disorder.

 Dosing is weight-based, starting at 10 mg/kg administered intravenously over 60 minutes once every 4 weeks, with a reduced 3 mg/kg dose available for patients who do not tolerate the higher amount. The treatment can be administered across multiple care settings, including home infusion where appropriate.

Approval rested on data from a phase 3, multi-center trial enrolling 63 adults with active FOP.

 Participants were randomized to receive 1 of 2 doses of the therapy or placebo every 4 weeks for 56 weeks. At that point, both the 10 mg/kg and 3 mg/kg arms met the trial's primary endpoint, reducing new lesions by 90% and 94%, respectively, compared with placebo, as measured by CT scans. Clinician-assessed flare-ups, a key secondary endpoint, dropped 88% in the higher-dose group, though the reduction was more modest, at 15%, in the lower-dose group. Patient-reported flare-up rates did not differ significantly between treatment and placebo groups. Serious treatment-emergent adverse events were infrequent and similarly distributed across all 3 arms. The most common adverse reactions, occurring in at least 10% of treated patients, included abscess, acne, excessive hair growth, eyebrow loss, oral ulcers, nosebleeds, folliculitis and nail infection.

“For people living with FOP, every irregular new bone formation is a step toward disability and potential loss of mobility,” said Dr. Kathryn Dahir, a primary investigator for the trial and professor, Department of Internal Medicine, Vanderbilt University, in a press release.

 “With the ability to reduce the number of new bone lesions and flare-ups, we now have a new treatment that can positively affect patients.”

Michelle Davis, executive director, International FOP Association, said in the press release,

 “This approval is monumental for our community, providing a vital new therapy that can have a significant impact on the life of someone with FOP.”

What Did the Earlier Phase 2 Trial Reveal About Safety Trade-offs?

Garetosmab's path to approval included a phase 2 trial, LUMINA-1, that surfaced safety questions the phase 3 program would later need to address.

 Five deaths occurred during that trial's open-label extension, a rate investigators called high for a study of this size, though causality was not established. The trial also missed its original primary endpoint, reduction in existing lesion activity, prompting researchers to redesign the primary analysis around new lesion formation instead. That pivot shaped how efficacy was ultimately measured and demonstrated in the later, larger trial supporting approval.

This approval illustrates the path a targeted biologic can take from mechanistic discovery to regulatory clearance in an ultra-small patient population, built on a trial of just 63 participants.

 It also underscores the growing role of imaging-based endpoints, in this case whole-body CT scans, in demonstrating efficacy where clinical outcome measures are difficult to standardize across a small, heterogeneous population. A pediatric trial is already planned, and a European regulatory submission is under review, both signals worth tracking for teams managing global development timelines and manufacturing capacity planning for orphan-designated biologics.

Pasatru (garetosmab-grts) first and only FDA-approved treatment demonstrating reduction in new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in a placebo-controlled trial in adults with fibrodysplasia ossificans progressiva (FOP). Press release. Regeneron Pharmaceuticals, Inc. August 19, 2026. 

https://investor.regeneron.com/news-releases/news-release-details/pasatrutm-garetosmab-grts-first-and-only-fda-approved-treatment

Raje N, Hochberg Z, Hsiao EC, et al. Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial. 

 2023;29(10):2614-2623. doi:10.1038/s41591-023-02561-8. September 8, 2023. 

https://www.perplexity.ai/search/233aad98-6f38-4a25-b345-ae2ee1f193f8

FDA approval of Pasatru (garetosmab-grts), a fully human anti–Activin A monoclonal antibody, provides the first placebo-controlled evidence for reducing new heterotopic ossification in adult fibrodysplasia ossificans progressiva. In a 63-patient phase 3 trial, weight-based IV dosing every 4 weeks cut new lesion formation by ~90%–94% on CT and reduced clinician-assessed flare-ups, with dose-dependent effects. Safety was generally comparable to placebo, despite earlier phase 2 mortality concerns that influenced endpoint selection and development strategy.

The FDA approves first therapy shown to reduce new bone lesions and flare-ups in fibrodysplasia ossificans progressiva (FOP), based on a 63-patient trial using CT-scan efficacy endpoints.

How a 63-Patient Trial Delivered an FDA Approval

Susan J. Schniepp is a fellow at Regulatory Compliance Associates, Inc. and a member of Pharmaceutical Technology's Editorial Advisory Board.

Siegfried Schmitt, PhD, is Vice President Technical at Parexel International, Siegfried.Schmitt@parexel.com.

Ask the Expert: Complying with Standards

Editor’s note: If you have a question you would like our experts to answer, please submit it to 

 and add Ask the Expert in the subject line.

In this episode of Ask the Expert, Susan J. Schniepp, distinguished fellow with Nelson Labs, and Siegfried Schmitt, PhD, vice president, Technical, with Parexel answer the question of whether pharmaceutical companies must comply with pharmacopeias, such as the 

Schniepp explains that pharmacopeial monographs, which are specifications and test procedures, are validated, recognized by regulators under good manufacturing practices (GMPs). In the United States, USP defines a monograph as “a written document that reflects the quality attributes of medicines approved by the US Food and Drug Administration” that provide quality expectations and include tests and information regarding identity, strength, purity, and performance.

However, "general information chapters" offering broader guidance are generally considered advisory rather than mandatory. Companies may deviate from these recommendations, but must document and justify how their alternative approach still achieves GMP compliance.

Schmitt notes that the USP is developed by a non-government organization; however, the European and Japanese pharmacopeias are government-issued, making them more directly enforceable. He points out that there are other voluntary standards (e.g., GAMP, ISO, ANSI, and NIST) and organizations like the Parenteral Drug Association operate within an ANSI-accredited framework, issuing technical reports and white papers as best-practice guidance where official standards don’t exist.

Schmitt and Schniepp stress that merely citing a standard (e.g., "we followed GAMP") doesn't guarantee compliance. According to Schmitt, there have been cases where companies claimed adherence but lacked the required documentation such as validation plans.

Schniepp adds that adopting a compendial method still requires labs to perform their own verification, because original validation data come from the submitter (government or innovator), not the adopting lab. Variations from lab to lab, such as equipment differences (e.g., high-performance liquid chromatography vs. ultra-high-performance liquid chromatography), requires that labs must confirm the method works on its own systems, essentially a mini robustness check.

Schmitt brings up an interesting point manufacturers should consider. In China, companies must maintain the original-language pharmacopeia and demonstrate someone can actually read it, because translations may be inaccurate.

Compendial compliance is nuanced, requiring companies to interpret regulations, verify methods locally, and document deviations rather than assume automatic compliance.

Click on the above video to watch Sue and Siegfried answer the following question:

“We have just acquired the latest versions of the 

. We are not clear if it is mandatory to comply with these pharmacopeias. Do we need to comply?”

An overview of USP monographs. USP. Accessed August 5, 2026. 

https://www.usp.org/about/public-policy/overview-of-monographs

Susan J. Schniepp is distinguished fellow at Nelson Labs and a member of 

Siegfried Schmitt is vice president, Technical at Parexel and a member of 

Videos

In this episode of Ask the Expert, Susan J. Schniepp, distinguished fellow with Nelson Labs, and Siegfried Schmitt, PhD, vice president, Technical, with Parexel, clear up some of the confusion about what does and does not need to be complied with regarding standards set by international pharmacopeias.

FDA concept | Concept of the Food and Drug Administration. Magnifier glass, folder and clock | Image Credit © Sviatlana - stock.adobe.com

President Donald Trump has nominated Heidi Overton, deputy director of the White House Domestic Policy Council, to serve as the next commissioner of the FDA.

 The nomination fills a post that has been without permanent leadership since 

Overton, a physician with a doctorate from Johns Hopkins University, has served as deputy assistant to the president during Trump's second term, advising on health policy issues including prescription drug pricing.

 She has appeared alongside Trump at several White House health policy events, including the recent executive order signing addressing childhood vaccine recommendations. In that setting, she voiced support for reducing the childhood immunization schedule and emphasized what she described as gold-standard science alongside restoring vaccination decisions to parents. She has also expressed anti-abortion views.

Before joining the administration, Overton held leadership roles at the America First Policy Institute's Center for a Healthy America, serving as both vice chair and chief policy officer.

 That organization was previously led by Agriculture Secretary Brooke Rollins, and several other current administration officials, including Education Secretary Linda McMahon, held positions there before returning to government service.

Why Does This Nomination Pose Potential Change?

Any transition in FDA leadership carries direct implications for organizations navigating regulatory review timelines, drug approval pathways, and compliance expectations.

 The agency's approach may signal broader shifts in how evidence is weighed across the agency's review functions, not only in immunization policy. Sustained leadership instability at the agency has practical consequences: acting leadership can slow decision-making on pending applications, complicate long-term regulatory planning, and create uncertainty around enforcement priorities during periods of transition.

The nomination follows reporting that Acting Commissioner Kyle Diamantas, who has led the administration's response to the current outbreaks and been described as central to its food policy work, was offered the permanent role multiple times and declined it.

 That detail underscores the difficulty the administration has faced in securing sustained leadership commitment for one of its most consequential health posts, a dynamic the industry has already had to account for since Makary's departure.

Overton's confirmation is not guaranteed.

 As a Senate-confirmed position, her nomination will proceed through hearings expected to probe her alignment with the president's health agenda. One source familiar with the administration said, in a press release,

She's gonna stay in line because she's tested. She's a loyalist to the president. That's kind of his thinking behind all of this, to keep loyalists in these top jobs.”

For an agency that has already seen a 20% staffing reduction during this administration, professionals should be watching the confirmation process closely, both for what it signals about continuity in review operations and for early indications of how a new commissioner might approach enforcement, safety communication, and agency staffing decisions going forward.

How Is Industry Reacting to the Nomination?

Early reaction from health policy analysts has been sharply divided.

 Chris Meekins, a Raymond James analyst and former Trump health official, stated in an Axios report,

 “Overton has degrees but seemingly no real experience running anything. Her claim to fame is having been a Makary protege who has bought further into anti-science and anti-vaccine messaging of elements of MAHA.”

Others disagree: Brian Blase, president, Paragon Health Institute, 

 “Heidi has the highest character and is dedicated to improving American well-being and helping American patients get the best possible care.” 

Senate confirmation, including potential resistance from health committee members, remains uncertain.

Jones A. Trump picks White House aide Dr. Heidi Overton for top FDA job. ABC News. August 19, 2026. 

https://abcnews.com/Politics/trump-picks-dr-heidi-overton-top-fda-job/story?id=135777387

Owens C. Trump to tap Heidi Overton as FDA commissioner. Axios. Published August 19, 2026. 

https://www.axios.com/2026/08/19/trump-heidi-overton-fda-commissioner

President Trump nominated Heidi Overton, deputy director of the White House Domestic Policy Council, as FDA commissioner, ending a prolonged period without permanent leadership since Marty Makary’s May resignation. A Johns Hopkins–trained physician, Overton has advised on health policy and publicly supported reducing the childhood immunization schedule while emphasizing parental decision-making; she also holds anti-abortion views. Her background includes senior roles at the America First Policy Institute. Confirmation is uncertain amid FDA staffing reductions and concerns about regulatory continuity, enforcement priorities, and evidence standards.

The Trump administration nominates Heidi Overton for FDA commissioner amid leadership instability, raising questions for drug developers on review timelines and oversight.

What Does Overton's FDA Nomination Mean for Drug Reviews?

Silver Spring, MD, USA - June 25, 2022: The FDA White Oak Campus, headquarters of the United States Food and Drug Administration, a federal agency of the Department of Health and Human Services (HHS) | © Tada Images - stock.adobe.com (521699065)

On August 19,2026, the FDA accepted Teva Pharmaceuticals' New Drug Application (NDA) for ecopipam, an investigational treatment for pediatric patients with Tourette syndrome.

 The application has been granted Priority Review along with Orphan Drug designation, and the agency has set a targeted action date for late in the first quarter of 2027.

Ecopipam is an investigational therapy designed to block dopamine signaling at the D1 receptor.

 This mechanism addresses a specific hypothesis in Tourette syndrome pathology, that D1 receptor hypersensitivity may drive the repetitive and compulsive behaviors characteristic of the condition. If approved, ecopipam would represent the first new treatment option for pediatric Tourette syndrome patients in more than a decade, and the first therapy built on a novel mechanism of action in more than 50 years.

The NDA acceptance rests on data from phase 2b and phase 3 trials.

 The phase 2b study enrolled 153 pediatric participants across 68 sites in North America and Europe in a 12-week randomized, double-blind, placebo-controlled design. Patients receiving ecopipam showed statistically significant and clinically meaningful reductions in tic severity compared to placebo at week 12. A subsequent open-label extension followed 121 of those participants for up to 12 months and supported the durability of that effect.

The phase 3 program took a randomized withdrawal approach: 216 pediatric and adult participants entered an open-label stabilization period, after which 104 responders were randomized to continue ecopipam or switch to placebo.

 For the primary endpoint, pediatric responders on ecopipam had a 53% decreased risk of relapse over 12 weeks compared to placebo. 

Across the phase 2b, open-label extension, and phase 3 studies, researchers reported no clinically meaningful changes in body weight, metabolic or laboratory parameters, cardiac measurements, movement-disorder scales, or psychiatric comorbidity measures.

What Did the Trial's Design Reveal About Ecopipam's Durability?

The phase 3 randomized clincal trial ecopipam was titrated to a target dose of 1.8 mg per kilogram per day.

 Only participants who achieved at least a 25% improvement in the Yale Global Tic Severity Scale Total Tic Scoreby week 12 were randomized into the double-blind withdrawal phase, a design intended to isolate maintenance of effect from initial response.

Among pediatric participants, relapse occurred in 68.1% of those switched to placebo compared with 41.9% of those who continued ecopipam.

 The most frequently reported adverse events were somnolence, anxiety, headache, insomnia, tic, and fatigue, and the trial recorded no clinically meaningful changes in body mass index, metabolic markers, or drug-induced movement measures. This safety profile is notable given that antipsychotics, currently the only approved Tourette syndrome medications, carry known risks of weight gain and movement-related side effects that often drive treatment discontinuation.

Tourette syndrome affects an estimated 100,000 children and adolescents in the United States, yet only about half receive prescription treatment, and just 20-30% remain on therapy after a year.

 That gap reflects a long-standing reliance on medications developed for other indications, many of which carry tolerability limitations. An indication-specific therapy targeting an underserved pediatric population illustrates how orphan drug and priority review pathways continue to shape sponsors' regulatory and development strategies in areas of high unmet need.

Eric Hughes, MD, PhD, executive vice president, Global R&D and Chief Medical Officer, Teva, said in the press release,

 “If approved, ecopipam would be the first new therapy for Tourette syndrome in more than 10 years and the first novel mechanism of action in more than 50 years, offering patients and families a long-awaited new treatment option.”

The FDA's targeted action date puts a potential approval decision in the first quarter of 2027, a milestone worth tracking for its implications on pediatric neuroscience development pathways more broadly.

US FDA accepts Teva’s new drug application and grants priority review for ecopipam, a first-in-class investigational therapy for pediatric patients with Tourette syndrome. News release. Teva Pharmaceuticals. August 19, 2026. 

Gilbert DL, et al. Efficacy and safety of ecopipam for Tourette syndrome. 

 Published online August 19, 2026. doi:10.1001/jamaneurol.2026.3063

FDA grants Priority Review to Teva's ecopipam new drug application, backed by phase 3 data. Ecopipam is a first-in-class D1 receptor therapy for pediatric Tourette syndrome.

Ecopipam's D1 Receptor Approach Signals New Pediatric Neuroscience Pathway

Glowing neural network inside a transparent antibiotic capsule surrounded by a large language model on a black background. Illustration of the concept of AI assistance in pharmacy and drug discovery | Image Credit: ©Dragon Claws -stock.adobe.com

For the past 2 years, the conversation about artificial intelligence (AI) in pharmacovigilance (PV) has largely revolved around the question, can the system explain itself? Can it show its reasoning, cite its sources, produce an audit trail? These are genuinely important qualities. But 2026 has been the year the industry, and its regulators, started asking a harder question: not what the system can explain after the fact, but what it prevents from happening in the first place, and how that is governed over time.

That shift matters most in post-marketing changes: the ongoing stream of signal detection, safety labeling updates, and case-processing decisions that don’t stop once a product reaches market. This is where AI’s promise is most real, and where the cost of getting governance wrong is most direct.

An aircraft’s black box and its altitude alarm serve different purposes. The recorder lets investigators reconstruct what happened after an incident, valuable, but too late for anyone on board at the time. The altitude alarm intervenes before a threshold is crossed, while there is still a chance to change the outcome. Most of the AI governance conversation in PV has been built around the recorder. Comparatively, little attention has gone to what it would take to build-in the ability to intervene before a missed or delayed signal becomes a patient safety issue.

Two enforcement actions illustrate exactly what that gap looks like in practice. In March 2026, an FDA warning letter to Novo Nordisk

 documented case-handling failures, including a report excluded from mandatory reporting on assumed causality grounds, and a patient death case invalidated for a missing identifier that the FDA’s own inspectors later found present in the company’s source documents. Notably, the system involved wasn’t AI-based at all. A conventional, rule-based process failed because nothing was actually enforcing the rule, and no one was monitoring closely enough to catch it.

In April 2026, the FDA issued its first warning letter to explicitly name AI overreliance as an inspection finding,

 this time against a drug manufacturer that had used AI agents to generate compliance documents without human review. When investigators found the company hadn’t conducted required process validation, the response was that the AI agent had never flagged it as necessary.

Read together, these 2 letters trace the full arc of governance failure in post-marketing change management: rules that exist on paper but aren’t enforced, and accountability that gets quietly delegated to a system incapable of holding it.

Regulators Are Converging Faster than the Market Has Noticed

This isn’t a scattered set of national concerns. Three threads illustrate just how aligned regulators now are, despite different mandates and methods.

First, none of them believe a single point-in-time approval is sufficient anymore. The Council for International Organizations of Medical Sciences (CIOMS) Working Group XIV report on AI

 in pharmacovigilance, the product of 3 years of work across regulators, industry, and academia, sets out 7 core principles for AI in PV, explicit that explainability is one principle among 7, not the standard on its own, and that human oversight must be risk-calibrated and architecturally enforced.

The European Medicines Agency’s (EMA’s) Network Data Steering Group has translated that same direction into dated commitments

: new AI-enabled signal detection capabilities for authorized products rolled out in the second quarter of 2026, with AI-based suspected unexpected serious adverse reactions (SUSAR) screening for investigational products following in 2027, alongside a life cycle management principle in the 

EMA-FDA Guiding Principles of Good AI Practice

 published in January 2026, requiring scheduled monitoring and periodic re-evaluation to catch data drift throughout an AI system’s life, not just at launch.

The United Kingdom’s National Commission into the Regulation of AI in Healthcare reached the identical conclusion from a public-facing consultation of more than 700 respondents:

 between 61% and 68% of every stakeholder group, patients, clinicians, providers, and industry alike, said current post-market surveillance requirements are not sufficient for how AI systems actually behave once deployed.

Second, all 3 sources agree that oversight has to scale with risk and context rather than apply uniformly. The EMA-FDA principles pair a risk-based approach with a requirement for a clearly defined context of use, on the basis that risk cannot be assessed in the abstract, only against a bounded purpose. The UK Commission’s evidence shows broad support for that same logic, while also surfacing where consensus breaks down: 77% of patients and the public described the current regulatory framework as too loose, while 65% of industry respondents described it as too restrictive. Everyone wants proportionality. Nobody agrees yet on which direction the dial needs to move, which is exactly the kind of ambiguity PV teams should expect to navigate rather than wait out.

Third, transparency and human oversight are treated as the mechanism for accountability, not a nice-to-have. The EMA-FDA principles call for plain-language disclosure of an AI system’s context of use, performance, and limitations, addressed to the intended audience rather than buried in technical documentation. The UK Commission’s evidence found the same expectation from the public side: 77% to 88% of respondents across every group said the current liability framework has significant gaps, tied directly to how difficult it is to trace accountability through an AI system whose reasoning isn’t transparent. Regulators are drawing a straight line from explainability to who is answerable when something goes wrong.

The FDA and EMA have also formalized a standing cluster on AI in pharmacovigilance,

 meeting every 2 to 3 months with Japan’s Pharmaceuticals and Medical Devices Agency and Health Canada as observers, working through use cases spanning the full PV workflow from adverse event extraction through signal evaluation. Regulators are deploying AI in the same workflows they will be inspecting industry for. As an MHRA spokesperson put it earlier this year, the agency’s consistent position is that AI should augment expert judgement, not replace it.

The Risk Explainability Quietly Makes it Worse

There’s a subtler problem worth naming directly. When AI produces fluent, well-explained output, human reviewers tend to scrutinize it less, not more. A confident, well-formatted recommendation creates an illusion of verification: the reviewer feels they’ve checked something they’ve actually just approved. In a PV environment where a case manager may be processing hundreds of cases a day with AI assistance, that’s a foreseeable consequence of deploying explainable AI without controls on reviewer behavior, not a hypothetical one. Complete governance has to monitor what happens to human judgment when the system is working well: approval velocity, override rates, whether a required review step actually happened, not just whether it was scheduled.

What This Means for How Post-Marketing Changes Actually Get Made

Put these threads together and a practical picture emerges. Effective AI governance for post-marketing change management treats deterministic and probabilistic processing as a deliberate choice, not a default. Decisions that need the same correct answer every time, whether a report meets a reporting threshold regardless of assumed causality, for instance, are handled by fixed logic that cannot be configured around. Judgment calls that genuinely require interpretation are handled by AI reasoning, scoped tightly to the question at hand, with confidence thresholds that trigger escalation rather than confident silence when uncertainty is high. Governance itself has to be evidenced across the full lifecycle, design, validation, and live monitoring, not documented once and assumed to hold.

This is consistent with conversations the author has had with customers and echoes a pattern seen repeatedly the author’s experience at a large biopharma safety organization. The appetite has shifted noticeably: less patience for AI framed purely as incremental case-processing efficiency, more urgency around faster, more defensible signal detection and inspection-readiness that’s built in rather than retrofitted. Several enterprise sponsors have independently raised interest in standardizing signal detection methodology across the industry, precisely so regulatory acceptance doesn’t have to be re-litigated company by company. That’s where the practitioners doing this work every day are already heading.

None of this means explainability is unimportant. It’s necessary. But it answers only 1 part of the question that post-marketing change management now demands. The more useful measure of readiness isn’t whether a system can explain itself when asked. It’s whether anyone would have noticed if it were no longer able to.

Warning Letter to Novo Nordisk Inc., MARCS-CMS 717576 / FDA Ref. 26-HFD-45-03-01. March 5, 2026. FDA. fda.gov.

FDA, Warning Letter to Purolea Cosmetics Lab, FEI 3011669383 / Ref. 320-26-58. April 2, 2026. fda.gov

CIOMS Working Group XIV, Artificial Intelligence in Pharmacovigilance. Council for International Organizations of Medical Sciences. December 4, 2025. cioms.ch

EMA/HMA. Network Data Steering Group Workplan 2026–2028. Adopted February 2026. ema.europa.eu

Joint FDA/EMA document: Guiding Principles of Good AI Practice in Drug Development. FDA. Accessed August 19, 2026 

https://www.fda.gov/about-fda/artificial-intelligence-drug-development/guiding-principles-good-ai-practice-drug-development

National Commission into the Regulation of AI in Healthcare: research, engagement and call for evidence findings. 2026. Medicines and Healthcare products Regulatory Agency (MHRA). Accessed August 19, 2026. 

https://www.gov.uk/government/groups/national-commission-into-the-regulation-of-ai-in-healthcare

Guiding principles for the EMA–US FDA cluster teleconferences on artificial intelligence in pharmacovigilance. EMA/422032/2023. European Medicines Agency and US FDA. Published March 2, 2026. Accessed August 19, 2026. 

https://www.ema.europa.eu/en/documents/other/guiding-principles-ema-us-fda-cluster-teleconferences-artificial-intelligence-pharmacovigilance_en.pdf

Beena Wood is chief product officer at Qinecsa Solutions, where she leads product strategy across the company’s pharmacovigilance technology portfolio.

Regulatory and industry scrutiny of AI in pharmacovigilance has shifted from post hoc explainability toward prevention, enforcement, and longitudinal governance in post-marketing change management. Recent FDA warning letters highlight two ends of the same failure mode: unmonitored rule-based processes and undocumented delegation of compliance judgment to AI agents. Regulators are converging on lifecycle monitoring, risk-calibrated human oversight, and plain-language transparency to anchor accountability. Explainable outputs can paradoxically reduce reviewer skepticism, necessitating controls on human behavior and operational metrics. Readiness is defined by detectability of degradation, not narrative quality.

Post-marketing governance must be evidenced across the full lifecycle, design, validation, and live monitoring, not documented once and assumed to hold.

How AI Can Assist with Post-Marketing Changes

VP Operational Delivery, PPD FSP Solutions, part of the PPD clinical research business of Thermo Fisher Scientific

As sponsors face mounting pressure to accelerate timelines while managing costs and expanding portfolio complexity, many are moving past the binary choice between functional service provider (FSP) and full-service outsourcing (FSO) models. 

spoke with Samantha Hadfield, VP Operational Delivery, PPD FSP Solutions, part of the PPD clinical research business of Thermo Fisher Scientific, about how mixed FSP/FSO strategies are reshaping clinical development operations. Hadfield discusses common triggers for adopting blended models, governance structures that support accountability across outsourcing relationships, and the internal capabilities sponsors need before pursuing this approach.

PharmTech: How Do You Define FSP, FSO, and Mixed FSP/FSO Models, and What Distinguishes Each Approach?

 A FSP model provides dedicated resources to support specific clinical trial functions, such as clinical operations, pharmacovigilance (PV), or medical writing, as a few examples. These resources operate as an extension of the sponsor’s team, working within established processes, systems, and governance structures to deliver deeper functional expertise while supporting team continuity and visibility into day-to-day activities, aligned with the sponsor’s goals and ways of working.

A FSO model covers a broader scope of work, potentially including an entire study or program, supported by a CRO [contract research organization] that manages delivery across functions. This model provides sponsors with access to broader capabilities and operational scalability while shifting greater responsibility for execution, resource management, and delivery outcomes to the CRO.

A mixed FSP/FSO model combines FSP and FSO approaches to create a tailored outsourcing strategy. Sponsors may integrate FSP and FSO solutions from the outset or add targeted FSP capabilities to an existing FSO partnership as needs evolve. This flexible model can support individual studies, portfolios, or enterprise-wide programs, enabling organizations to scale resources, expand into new regions or therapeutic areas, retain oversight of strategic functions and data, preserve continuity across programs, and balance flexibility with operational execution throughout clinical development.

What Are the Most Common Trigger Events That Push Sponsors to Adopt a Mixed FSO/FSP Model?

The most common trigger is a change in portfolio needs. As sponsors expand their pipelines, enter new therapeutic areas, or integrate assets through M&A, they often need specialized capabilities quickly without the time or investment required to build them internally. Shifting clinical trial volume and increasing study complexity are also common catalysts, as is the need for greater access to data and real-time transparency into clinical trial outcomes to enable faster, more data-led decisions. A 

 found that 84% of sponsors say accelerating development timelines is their top organizational priority, while 72% cite managing costs.

Balancing these competing priorities is increasingly leading sponsors to adopt mixed FSO/FSP models that allow them to retain dedicated expertise where it matters most while scaling support across broader programs and evolving portfolio needs.

As Sponsors Move Away from Treating FSO and FSP Models as a Binary Choice, What Does a Well-Designed Mixed Model Look Like in Practice?

A well-designed mixed FSP/FSO model is not a fixed operating structure, but an approach that evolves alongside a sponsor's portfolio. As programs mature, geographic footprints expand or resource demands shift, sponsors should be able to adjust how work is allocated between FSP models and FSO without disrupting delivery.

In practice, responsibilities are often assigned at the function, program, or regional level rather than through a single enterprise-wide outsourcing strategy. Sponsors may initially rely more heavily on FSO support to launch a new program and gradually transition specific functions or regions to an FSP model as demand becomes more consistent or deeper integration with internal teams is needed.

The final component is clear governance and accountability. Successful mixed FSP/FSO models require defined ownership of strategic decisions, including when a function should transition between FSO and FSP support based on changing priorities, operational requirements, or portfolio needs. Without ongoing evaluation and active management, the model can become static rather than continuing to deliver the flexibility and efficiency it was designed to provide.

What Internal Capabilities or Infrastructure Does a Sponsor Need to Have in Place Before a Mixed FSP/FSO Model Can Succeed?

The foundation for a successful mixed model is organizational alignment. Sponsors need clinical, operational, and financial teams working from the same assumptions around demand, capacity, timelines, and cost, rather than planning independently. Just as importantly, they need governance that is designed to manage both FSP and FSO relationships as parts of a single operating strategy instead of separate outsourcing models. A clear technology roadmap is also critical, including the right infrastructure and investment to enable data sharing, transparency, and connectivity across the different delivery models.

Sponsors are increasingly adopting mixed FSP/FSO outsourcing to balance speed, cost control, and rising portfolio complexity. FSP embeds dedicated functional experts within sponsor processes, while FSO delegates broader program delivery to a CRO; blended models dynamically allocate work by function, program, or region as needs evolve. Common triggers include pipeline expansion, new therapeutic areas, M&A integration, variable trial volume, and demands for real-time data transparency. Success depends on aligned cross-functional planning, integrated governance, shared reporting infrastructure, clear decision rights, and an accountable owner to continuously rebalance the model.

Samantha Hadfield, PPD, explains how mixed FSP/FSO models help sponsors balance speed, cost, and complexity in clinical trial outsourcing.

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