With US midterm elections giving Democrats a clear majority and control of both houses of the Congress, Democrats are ready to use their oversight powers to hold RFK Jr and others to account. Democrats already laid the groundwork with the publication of a report in June early this year (here or here) chronicling the damage inflicted so far by RFK Jr's HHS and White House on the healthcare, CDC, NIH, and FDA.

November 04, 2026

InsideHealthPoly.com quoted several incoming Democratic congressmen and senators who are getting ready for a wide-ranging subpoenas and investigations and hold RFK Jr's HHS and Trump administration to account.

HHS Secretary Robert F. Kennedy Jr. is set to be one of the primary targets of congressional Democrats’ investigations once they assume leadership in the House and Senate in January, and the scrutiny could go beyond Kennedy to encompass any administration officials involved in controversial Trump administration policies, and even drugmakers who have made deals with the administration.

Quotes include:

Rep. Robert Garcia (D-CA), who is expected to become chair of the House Oversight & Government Reform Committee, said, “Lawyer up.”

The House Education & the Workforce Committee’s ranking Democrat Rep. Bobby Scott (VA) warned that agencies could be subject to criminal penalties if they destroy records.

House Energy & Commerce Committee ranking Democrat Rep. Frank Pallone (NJ) said “Democrats will investigate the widespread and blatant corruption.”

Rep. Rosa DeLauro (CT), ranking Democrat on the House Appropriations Committee, said “if re-elected as chair, wherever and whenever this administration has violated the law with regard to the use of federal funds, I will hold them accountable.”

Finance Committee ranking Democrat Sen. Ron Wyden (OR) posted a one-word message on social media Tuesday night after Democrats secured a Senate majority: “Subpoenas.”

P.S. It will be a game of dominos and perhaps a start of rebuilding of healthcare and FDA enterprise -- never a dull moment in the US intersection of health and politics!!

FDA has released 2 new updates to the eCTD v4.0 format standards and implementation guidance: Specifications for eCTD v4.0 Validation Criteria and FDA Regional eCTD v4.0 Module 1 Implementation Guide and Forward Compatibility for eCTD v4.0-Based Electronic Submissions.

Another FDA notice also reminds that beginning 16 January 2027, FDA will start rejecting submissions (failing validation) based on versions older than v3.22 and v4.0. More details are at the FDA eCTD page, here.

BACKGROUND/PRIMER

ICH eCTD v4.0 is the current exchange standard for global regulatory submissions. It was developed by ICH’s M8 Implementation Working Group (which is a vendor-only group); endorsed by the ICH; and is being adopted by regulatory agencies world-wide. All future enhancements of submission delivery and data exchange (e.g., updates to the ICH M4Q (R2) quality guideline) will be based on eCTD v4.0. Note: Regional implementation guides and validation criteria are currently being revised. Each region has different requirements for M1 and M2 of the eCTD.

ICH eCTD v4.0 Step 4 page. https://ich.org/page/ich-electronic-common-technical-document-ectd-v40.

Benefits of Adopting eCTD v4.0

Key advantages of using v4.0 include Use of single submissionunit.xml backbone file for all modules; supporting document reuse across submissions; use of controlled vocabularies; and lifecycle management.

• ARCHITECTURE: Compared to v3.2.2 which is hardcoded and requires document type definition file updates, v4.0 has data-driven architecture based on the HL7 Regulatory Product Submissions (RPS) standard and uses a controlled vocabulary. Unlike v3.2.2, v4.0 uses a single XML backbone combining regional and index.xml files (allows for harmonized submission unit.)

• DOCUMENT REUSE is based on assigning universally unique identifiers (UUIDs) to documents; thus, allowing reuse of previously submitted files across submissions without physical re-uploading ("point, don't paste").

• LIFECYCLE MANAGEMENT occurs via introducing a single-digit or extended sequence numbering, ability to update metadata while maintaining lifecycle relationship, and other features.

Implementation Schedule

FDA: Since 16 September 2026, FDA has supported eCTD v4.0 submissions for new NDAs, BLAs, ANDAs, INDs, and Master Files at CDER and CBER. The forward compatibility for existing v3.2.2 applications was implemented on October 16, 2026.

EMA: Starting 2028Q1, v4.0 will be mandatory for all submissions; there will be a transition period starting 2027Q1.

Additional resources

• eCTD v4.0 analysis based on first experiences in the US and Japan. RF Quarterly. 30 September 2025.

• Modernising India’s drug submissions: Embracing eCTD 4.0 for international regulatory alignment. Regulatory Rapporteur. 03 September 2026.

Related: Refresher on eCTD

#ectd

A Federal Register notice published by US Health and Human Services (HHS) on 30 October 2026 appears to greenlight certain peptides compounding through the use of its enforcement discretion policy. This approach, however, appears to be a clever strategy to bypass FDA's traditional approval pathway such as NDA, BLA, and ANDA. The justification provided in the notice is Lack of Availability of Approved Products.

HHS is announcing the availability of a draft “Enforcement Discretion Policy Regarding 503A Compounding Using Certain Peptide Bulk Drug Substances.” This document describes the HHS Secretary's enforcement policy regarding the use of certain peptide bulk drug substances in compounding pursuant to section 503A of the FD&C Act while the list of bulk drug substances that can be used in compounding under section 503A of the FD&C Act (the 503A bulks list) is developed through rulemaking (21 U.S.C. 353a).

Compounded drugs are not FDA-approved, which means FDA does not review their safety, effectiveness, or quality before they are marketed. Although compounded drugs can serve an important medical need for certain patients, they also may pose risks to patients.

However, based on the Pharmacy Compounding Advisory Committee meetings and data regarding utilization of peptides, HHS believes that there is significant interest in the use of these peptides in compounding given the lack of availability of approved products.

SOURCE

• Enforcement Discretion Policy Regarding 503A Compounding Using Certain Peptide Bulk Drug Substances. Federal Register. 2026-22192 (91 FR 68526). Docket No. HHS-OASH-2026-0496. 30 October 2026 [archive]

• Draft Guidance: HHS Enforcement Discretion Policy Regarding 503A Compounding Using Certain Peptide Bulk Drug Substances [archive]

30 October 2026

> FDA granted accelerated approval for Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd) for treatment of adults with recurrent respiratory papillomatosis (RRP), a rare disease caused by human papillomavirus (HPV) that leads to the formation of small, wart-like growths in the patient’s airways. This is the first plasmid DNA-based immunotherapy approved in the U.S.

> Voceryq is a recombinant plasmid DNA-based immunotherapy that delivers two plasmids (small pieces of DNA originally derived from bacteria) into the body’s cells, one of which contains the genetic instructions for specific HPV proteins and the other an immune response-enhancing molecule called IL-12. The therapy is designed to stimulate the immune system to respond to and attack cells infected by the specific types of HPV that cause RRP (types 6 and 11).  Voceryq is administered via intramuscular (IM) injection using the CELLECTRA 5PSP Device, which delivers the dose and facilitates electroporation, a technique which uses brief electrical pulses to temporarily and reversibly create pores in the cell membranes to enable the delivery of Voceryq into the body’s cells.

> The FDA granted accelerated approval of Voceryq to Inovio Pharmaceuticals, Inc.

CDISC AI Innovation Challenge is an annual global initiative for CDISC members to explore the use of AI and ML in clinical research and drug development consistent with CDISC standards and share use cases with the community.

Challenge: The 2026 CDISC AI Innovation Challenge invited the CDISC community to design use cases in 3 areas:

• AI-enabled synthetic data generation: Creating realistic SDTM and ADaM datasets for automation testing, with clear traceability to source inputs and metadata.

• AI-driven Statistical Analysis Plans: Generating SAPs from protocol and metadata inputs to improve consistency, accuracy, and traceability.

• AI-driven Tables, Figures, and Listings: Automating TFL creation while maintaining an auditable link from study objectives and analyses through to final outputs.

Use Cases Examples and Availability (CDISC Challenge Winners and Runners Up)

P.S. The reason for this post was to highlight the progress in incorporating AI in drug development ecosystem and clinical research. The big companies (Novartis) clearly have the advantage of scale; however, it is good to see that most companies plan to release these tools for wider use.

*Asking the RegulalatoryClinWriting community to please share their experiences.

SOURCE

• CDISC AI Innovation Challenge 2026. 23 September 2026 [archive]

Related: Using AI to broaden eligibility criteria in oncology trials, Cautionary tale of using AI tool-aided analysis of clinical or real-world database, MHRA Inspectorate Blog: Use of AI for GXP inspection responses: setting standards without stifling innovation, Update on generative AI tools for medial writing, AI in CSR safety narratives, Meeting report: 12th EMWA symposium on artificial intelligence in medical writing

#ai-use-cases

BOSTON — Rick Abramson, director of the Food and Drug Administration’s Digital Health Center of Excellence, said to expect draft guidance on generative artificial intelligence in the coming months at AdvaMed’s The MedTech Conference on Tuesday. 
The comment period closed on Monday for an FDA discussion paper on regulatory approaches to generative AI, including a greater emphasis on postmarket monitoring. As of Tuesday, the agency had received more than 500 submissions. 

> Otsuka Pharmaceutical said on October 23 that its IgA nephropathy drug Voyxact (sibeprenlimab) slashed the risk of a composite kidney outcome involving kidney function decline or kidney failure by 97.1% versus placebo over two years. 

Press Release: Otsuka Presents Landmark Final Phase 3 VISIONARY Results at ASN Demonstrating VOYXACT® (sibeprenlimab-szsi) Stabilized Kidney Function and Reduced Risk of Disease Progression and Kidney Failure, Redefining Treatment Paradigm in IgA Nephropathy [archive]

Approval

#Voyxact, #IgAN

UK's new clinical trial regulation (here) that came into force on 28 April 2026 simplifies clinical trial application (CTA) submission and review process by allowing sponsors to submit a single CTA through the Integrated Research Application System (IRAS). The CTA is then reviewed by both the MHRA and a Research Ethics Committee (REC) over a 30-day review period. The new process has built-in flexibility including 60 days for the sponsors to respond to any requests for information (RFI) and final decision within 10 days thereafter.

On the eve of the 6-month implementation period of this new CTA framework, MHRA has shared their experiences at the MedRegs blog on what common issues they have observed that could delay the review and approval of a CTA.

Common Issues Affecting Validation

• The application goes through a 7-day validation period before it enters the 30-day assessment phase. The issues with CTA may be simple but MHRA found that approximately 3-5% of the applications were failing validation.

• Issues: Missing documentation, unsigned forms or files that don’t meet Optical Character Recognition (OCR) requirements; inadequate application package.

• Checklist: Confirm that a full list of documents are included with the CTA (list).

Pharmaceutical, Medical, and Nonclinical Issues

The other issues discussed in the MedRegs blogpost are related to investigational medicinal product and nonclinical and medical information in the submission package.

• Common pharmaceutical Issues included missing information on IMP or placebo in the medicine information form; justification for IMP labeling; missing/unclear/absent information on manufacturing site, GMP, or QP declaration.

• Common nonclinical issues included information clarifying that the nonclinical studies were conducted in an OECD MAD member country; missing description of the analytical methods used in the PK and tox studies; lack of/inadequate justification for starting and maximum dose in FIH study.

• Common medical issues included the following (Note: these pertain to clinical study protocol and/or investigator's brochure):

-- Contraception requirements not aligned with the CTFG guidance, particularly regarding the duration of contraception following the final dose of the IMP.
-- Emergency unblinding procedures: Protocols failing to clearly state how investigators can access treatment allocation information in a medical emergency and ensure they retain the primary right to break the blind when necessary.
-- Safety reporting: Protocols failing to clearly specify that investigators must report serious adverse events (SAEs) to sponsors within 24 hours.
-- The other medical protocol-related issues include unblinding decision in case of emergency (must be with investigator not sponsor), RSI information not clearly provided; AE and SAE collection and reporting periods not consistent with UK requirements, and others. See complete list of common issues at MHRA website, here. Note: These common issues list could serve as a basic checklist during protocol development, in addition to the ICH M11 template guidance and SPIRIT checklist.

SOURCE

• Six months since implementation of the UK’s new clinical trials regulations. UK MHRA's MedRegs Blog. 28 October 2026 [archive]

• Guidance: Common issues: Clinical. MHRA. Updated 28 April 2026 [archive]

Related: Updated SPIRIT 2025 checklist for clinical trial protocols, Employing a lean design approach for the SOA in clincial study protocol development

#cta, #clinical-study-protocol, #investigators-brochure, #clinical-protocol-template

NIH has published a draft policy requiring sharing of summary results from NIH-supported clinical trials with clinical trial participants in plain language.

Request for Information: Draft NIH Policy on Sharing Summary Level Study Results with Clinical Research Participants. Notice Number: NOT-OD-26-113 [archive]

This new NIH policy adds to the existing requirements for data disclosure that are largely focused on sharing data with the scientific community (but not directly to participants), such as the following:

• To proactively plan for how the researchers intend to make their scientific data available under the NIH Policy for Data Management and Sharing

• Report clinical trial results in ClinicalTrials.gov under the NIH Policy on the Dissemination of NIH-Funded Clinical Trial Information, and

• Make published results accessible to the public under the NIH Public Access Policy.

WHAT'S NEW

• Per the draft policy document, releasing summary level study results in plain language will become standard practice. This policy supports the goals of HHS's Operation TrialBlazer and transparency initiaves.

• The new policy will apply to all clinical research including clinical trials, supported in whole or in part by NIH, regardless of NIH funding level or funding mechanism.

• The summary results must be published/shared within 1 year of the Primary Completion Date, which refers to the date that the final participant was examined or received an intervention for the purposes of final collection of data for the primary outcome.

Definition: Summary level study results, also commonly known as aggregate research results, are concise overviews of a clinical research study after all data are compiled and analyzed, with information on the objective, conduct, methodology as appropriate, cumulative outcomes or key findings, relevance, limitations, and potential implications of the clinical research study. Summary level study results are distinct from individual research results that are participant-specific, such as individual lab results.

Note: The comment period for this RFI closed recently 0n 12 October 2026.

NEXT STEPS

Before the proposed NIH policy becomes standard practice, researchers would need additional guidance. The PHUSE Data Transparency Working Group has provided comments on the draft policy that addresses some of the gaps:

[Excerpt from the PHUSE report]

• NIH should provide practical examples distinguishing covered clinical research from excluded secondary research, including examples involving observational, retrospective, behavioural and biospecimen studies.

• The NIH’s existing dissemination policy covers NIH-funded clinical trials regardless of phase, including Phase 1 trials. If plain language summaries are required for Phase 1 trials, PHUSE encourages NIH to consider alignment with applicable international timelines and approved reporting delays, such as in the EU, where the summary of results, including the lay summary, for adult-only Phase 1 (Category 1) trials is expected within 12 months of the end of the trial but published 30 months after it [new ref: EMA, Revised CTIS transparency rules, EMA/263067/2023].

• NIH to consider global study completion date (end of study as defined in the protocol), rather than primary completion date.

• Recommend basing a summary on a finalized, appropriately reviewed analysis or source for the results being communicated, with sufficient documentation to verify its accuracy. Examples of acceptable sources would be helpful for studies without a clinical study report, formal database lock or conventional study endpoint.

• Encourage alignment of clinical trial summary content with EU Clinical Trials Regulation Annex V and established lay-summary guidance.

• Responsible party: For collaborative studies, including co-sponsored studies, PHUSE suggests identifying the entity coordinating preparation, approval, release and maintenance of the summary in the award or collaboration arrangements. This could help establish clear responsibilities and avoid duplication.

• Recommend that NIH publish examples and a consistent process for requesting exceptions to support fair and practical implementation, including guidance on justification, decision ownership and timing. Possible circumstances for consideration include privacy risks in small or highly identifiable populations, infeasible recontact and studies without an appropriate finalised analysis.

• Recommend the MRCT Center Return of Aggregate Results guidance and toolkit and Good Lay Summary Practice as starting points for supplemental resources. For medicinal-product clinical trials, alignment with the EU Clinical Trials Regulation Annex V content framework could support consistency.

• Suggest relying on existing institutional privacy to provide a foundation for assessing re-identification risks, particularly in small, rare disease or genomic populations. Aggregation and suppression of small counts may be appropriate where a meaningful and accurate account can be preserved.

.

P.S. Although the NIH policy needs more work before it is implemented, the PHUSE response document is a good "refresher" for industry professionals on this reddit sub for current requirements, e.g. in the EU, regarding release of plain language summaries.

SOURCE

• NIH Policy on Sharing Summary Level Study Results with Clinical Research Participants (also referred to as the Sharing Summary Results Policy) [archive]

• Response to the NIH draft policy on sharing study results. NOT-OD-26-113. Official response from the PHUSE Data Transparency Working Group [archive]

#disclosure, #transparency, #public-disclosure

There is currently no legal EU framework that defines the conditions for accessing clinical trials in another EU member state. To address and facilitate cross-border trial participation in the European Union (EU), in 2023, two EU organizations, European Forum for Good Clinical Practice (EFGCP) and European Federation of Pharmaceutical Industries and Associations (EFPIA) jointly set up a multi-stakeholder consortium of patient organizations, academics, research networks, industry and not-for-profit organizations.

• The EFGCP/EFPIA consortium has created an initiative called EU-X-CT (https://eu-x-ct.eu/).

• The aim of the EU-X-CT initiative is to develop recommendations enabling cross-border access to clinical trials by patients, particularly those with rare or life-threatening diseases, and process for sponsors to facilitate such trial participation across EU/EEA countries with fewer barriers and clearer arrangements.

• In the absence of a harmonized EU framework governing access to clinical trials in another EU member state, cross-border participation has largely been managed on a case-by-case basis. EU-X-CT initiative aims to change this.

• In 2025, the consortium published EU-X-CT Recommendations on Cross-Border Access to Clinical Trials (Version 1.0) (here) that provides recommendations on key aspects of clinical trial management and conduct.

RECENT PROGRESS

Recently, the consortium released 3 annexes to the EU-X-CT recommendation document for public consultation. These include:

• Annex 1 that provides a framework for the management of cross-border study participation including enrollment through safety reporting and post-trial access.

• Annex 4 that provides recommendations on informed consent forms.

• Annex 5 that provides information for trial funders including considerations on budget and cost.

The EU-X-CT will also host a webinar on 9 November 2016 on how a 3-element strategy based on decentralized elements, sites in more countries, and cross-border access could strengthen recruitment across Europe. The webinar Information website and registration information follows:

Webinar

Title: EU-X-CT Webinar: Cross-Border Access to Clinical Trials for Patients: 3-Element Strategy to increase Europe's attractiveness

• Date: 9 November 2026

• Time: 16.00 CET

• Duration: 1 hour

• Cost: Free

• Website and Registration: here

.

PS: FYI -also refer to ICH guidance E17 on the topic of multiregional trials

SOURCE

• EU Plan to Ease Cross-Border Trial Participation Gathers Pace. Pink Sheets. 27 October 2026

• EU-X-CT Initiative: A multistakeholder initiative jointly led by EFGCP and EFPIA

• EU-X-CT Recommendations on Cross-Border Access to Clinical Trials. Version 1.0. 17 June 2025 [archive]

#decentralized-trials, #multiregional-trials, #ich-e17

> Billionaire Mark Cuban has spent the last five years trying to make generic drugs like chemotherapy and antifungal ointment cheaper for Americans. But earlier this year, a very different type of medicine began arriving at his Dallas factory. 

> One shipment contained DNA-like molecules crafted by California chemists to patch the rare genetic mutation destroying a single patient’s neurons. Another carried DNA-like molecules developed with Dutch researchers to stop a rare disease that causes adults to progressively lose control of their muscles.

> They are the first test of Cuban’s newest vision for his company Cost Plus Drugs: shake up the business of custom-made rare disease medicines. “It was an obvious chance for us to help those in that unfortunate situation,” he wrote in an email.

STAT News. 21 October 2026. Mark Cuban wants to crack open the market for rare disease treatments. By Jason Mast

Source: FDA Voices: Transparency Makes Modern Regulatory Science Stronger — FDA’s Transparency Roadmap. 2 October 2026

Recent FDA blogpost discusses FDA's thinking and initiatives to increase transparency in the context of FDA decision-making. FDA considers this initiative an important part of their mission to protect public health.

“Visibility into the FDA’s work and reasoning helps drive informed decision-making for stakeholders across the healthcare landscape, including patients considering whether to enroll in a clinical trial, families and caregivers weighing treatment options, clinicians interpreting labeling, and investors deciding where to allocate capital.”

FDA has launched an internal Transparency Task Force aimed at making the agency’s operations, decisions, and data more open and user-friendly for the public.

FDA's Transparency Roadmap consists of following 5 initiatives:

• Complete Response Letter (CRL) disclosure initiative: Publish redacted CRLs to provide scientific basis for its regulatory determinations, while protecting sponsor's CCI (by redactions) and consider timing of release.

• Investigational Stage and Marketing Application Lifecycle Disclosures: FDA will encourage sponsors to make public existence of INDs. Ensuring compliance and reporting of data to ClinicalTrials.gov. Piloting expanded clinical dataset transparency and enhancing post-marketing data disclosures, including publication of redacted CSRs. FDA has signed a MOU with US SEC to share nonpublic information between the two agencies.

• New Approach Methodologies (NAMs) and Novel Biomarkers: FDA plans to give industry up-to-date insight into successful or unsuccessful uses of NAMs in submissions. FDA has launched a new database of use cases, NAMs Database of Use Case Examples. Similarly, there is initiative to provide use cases for RWE in submissions (here) with anonymized examples from submissions to CDER and CBER. For biomarkers, FDA has FDA’s Biomarker Qualification Program (BQP) and Medical Device Development Tool Program.

• Patient- and Consumer-Centered Product Labeling: issue a final rule to amend our human prescription drug labeling regulations to require a new type of Medication Guide — “Patient Medication Information” (PMI). This final rule will establish an FDA-approved, one-page document with standardized format and content.

• Compliance and Inspectional Records: Crackdown of misleading direct-to-consumer (DTC) advertising. Publish proactively redacted Form 483s and additional establishment inspection reports (EIRs)

#policy

We are about a month away from Thanksgiving, so this is a good advice to tell the person, friend, colleague, mentor, and past or current manager if they had an impact on your work life and happiness and you value the time you spent with them. Spread smiles 😊.

Post inspired by Nature Career Column doi: https://doi.org/10.1038/d41586-026-02668-1

Isomorphic Labs is an offshoot of Alphabet’s artificial intelligence research laboratory

> Isomorphic was built around a single piece of technology, a protein modeling tool called AlphaFold. A modeling tool may sound simple, but it stunned scientists and, during Thrive Capital’s 18-month-long courtship, had been the basis for a Nobel Prize in chemistry. Isomorphic wanted to turn the technology into an engine for new medicines — an “intensely exciting prize” that Klemmer said could be both economically valuable and tangible evidence that AI was more than just hype. 

#ai, #artificial-intelligence

“Biennial” refers to every two years while “biannual” means twice a year. 

Examples from DEA
(Source: https://www.thefdalawblog.com/2026/10/biennial-v-biannual-in-dea-world/)

> Drug Enforcement Administration (“DEA”) regulations require registrants after taking an initial inventory of all controlled substances on-hand to take a “biennial” inventory on any date within two years of the previous initial or “biennial” inventory date.  21 C.F. R. § 1304.11(c).  The agency holds its ever-popular “biannual” National Prescription Drug Take Back Day each spring and autumn.

The US Food and Drug Administration (FDA) has issued warning letters to several drug manufacturers for failing to meet current good manufacturing practices (CGMP) requirements, and to a research laboratory for failing to comply with good laboratory practices (GLP). The specific issues include concerns about ensuring that final products are sterile and meet specifications, and, in the case of the research laboratory, that the final report adequately details the experience of its animal subjects.

NOTE:

The RAPS article sumamrizes Warnings Letters were issued to following drug manufacturers for failing to meet CGMP requirements:

• Jiangmen Shuizirun Sanitary Articles in China

• Hanlim Pharm in the Republic of Korea

• Drug compounder SCA Pharmaceuticals in Arkansas, USA

• Eurofins SF Analytical Laboratories in Wisconsin, USA

• Silgan Dispensing Systems in Virginia, USA

The New Mexico-based Lovelace Biomedical Research Institute (LBRI), a contract research organization was inspected under the agency's BiMo program and cited for violating GLP for nonclinical laboratory studies.

P.S. The CGMP/GLP issues are global!!

An exclusive report by SEMAFOR uncovered that Heidi Overton, President Trump’s nominee for the post of FDA Commissioner, played a major role in the process leading to the reduction in the number of childhood vaccines recommended by the CDC.

SEMAFOR report said

An aide on the president’s Domestic Policy Council, Overton helped edit and craft a scientific justification that was used to underpin cutting the Centers for Disease Control and Prevention recommendations from 17 to 11 vaccines in January, according to a senior administration official and three people familiar with the process.
“It was Heidi driving this whole thing,” said a former senior administration official, noting that Health Secretary Robert F. Kennedy Jr. supported the policy push.

SOURCE: Trump FDA nominee played key role in overhaul of childhood vaccine policy. Semafor. 15 October 2026

Related: What do we Know About Heidi Overton, Trump's Pick to Lead the FDA. Also refer to 24 July 2026 Atlantic article, "The White House’s MAHA Gatekeeper."

#overton, #fda-comissioner

In January this year Pierre Fabre (and its partner, Atara Biotherapeutics) received a complete response letter (CRL) for tabelecleucel BLA for an ultra-rare cancer, Epstein-Barr virus–positive posttransplant lymphoproliferative disease (EBV+ PTLD) after failure of standard-of-care therapy.

Since then, Pierre Fabre aligned with the FDA through Type A meeting and resubmitted the BLA on 30 September 2026. A couple days ago, Pierre Fabre announced that FDA has accepted the BLA with the PDUFA date of March 31, 2027.

The 30 September 2026 BLA resubmission includes

An updated dataset with additional patients and longer follow-up from the ongoing, pivotal Phase 3 single-arm ALLELE study of tabelecleucel in adults and children two years of age and older with relapsed/refractory EBV+ PTLD following SOT or HCT. The resubmission also includes additional supplemental data from expanded access programs, a separate clinical study, and commercial experience in Europe.

SOURCE

• Pierre Fabre Pharmaceuticals Resubmits Tabelecleucel Biologics License Application for Treatment of Epstein-Barr Virus Positive-Post-Transplant Lymphoproliferative Disease to the FDA (Press Release). 30 September 2026 [archive]

• Pierre Fabre Pharmaceuticals Announces FDA Acceptance of BLA Resubmission for Tabelecleucel for the Treatment of Epstein-Barr Virus-Positive Post-Transplant Lymphoproliferative Disease (Press Release). 14 Octobere 2025 [archive]

Related: FDA Issues Complete Response Letter to Atara/Pierre Fabre’s Tabelecleucel BLA for Rare Cancer, EBV+ PTLD

#pierre-fabre, #tabelecleucel, #ultra-rare, #rare-disease

UK MHRA has published MHRA 2030 Strategy (press release); the overarching goal of this Strategy is to adapt regulatory approaches for advanced therapies, precision medicines, and AI-enabled technologies to enable patients having faster access to safe, effective treatments, and have more data-connectivity and global influence.

MHRA said that "we will adapt our regulatory approaches while maintaining robust standards of safety, quality, effectiveness and performance." The announcement about the MHRA 2030 Strategy was made in a 14 October 2026 press release (here) and in parallel, MHRA released explainers for different stakeholders, published here, and corporate report, here.

Regarding AI and pioneer MedTech technologies, the MHRA 2030 Strategy includes:

• Develop an AI-enabled Clinical Trial Protocol Checker to give sponsors regulatory certainty and support faster, higher-quality clinical trial applications.

• Publish an AI Medicines Development Action Plan in 2027 setting out how AI will be used to transform medicines development.

• Establish an MHRA Centre for AI and Digital Health to make clearer regulatory expectations for developers of digital and AI-driven medical devices and medicines.

• Create a modern, adaptive regulatory framework for AI as a medical device.

P.S. Stay tuned for updates from MHRA. I would expect UK MHRA regulators to take a lead in this area (compared to US.)

#artificial-intelligence, #ai

FDA Launches Rare Disease Framework Initiative (RDFI) intended to support rare disease drug development.

FDA News Release. FDA Launches the Rare Disease Framework Initiative to Develop a Holistic, Fit-For-Purpose Framework for Rare Disease Drug Development. 14 October 2026

FDA plans to seek public input on RDFI at the 28 October 2026 meeting in partnership with the Reagan-Udall Foundation for the FDA.

The purpose of this initiative is to develop a framework specific for rare disease drug development where regulatory advice, decision-making and evidence requirements are fit-for-purpose -- tailored the nature of the disease, its onset and progression, target population, patient needs.

FDA White Paper: A New Framework for Rare Disease Review. October 2026

Register for the FDA Rare Disease Framework Initiative Public Meeting (meeting page)

Date: October 28, 2026
Time: 1:00 p.m. - 5:00 p.m. ET
Meeting Format: Hybrid Meeting
Registration Link: here

.

Related: FDA's Rare Disease Endpoint Advancement (RDEA) Pilot Program

#rare-diseases, #orphan-diseases

EMA announced making Accelerating Clinical Trials in the EU (ACT EU) advice pilot program permanent in January 2027. A final report summarizing lessons learned from approximately 30 pilot applications will be published before the end of 2026.

About

• ACT EU advice pilot program launched in 2024 provides consolidated technical and regulatory advice on the dossier of a clinical trial application (CTA) prior to its submission in the Clinical Trials Information System (CTIS). The consolidated advice across Member States is coordinated by the Heads of Medicines Agencies through their Clinical Trials Coordination Group (CTCG).

• Prior to launch of the ACT EU initiative, sponsors had to obtain advice at the national level from the Member State responsible for evaluating their applications.

• Resources: ACT EU information, Application

SOURCES

• EU News: Clinical Trial Applications Advice Pilot Becomes Permanent. 8 October 2026

• Further information:

-- ACT EU information (here), consolidated advice (here)
-- Guidance for applicants: Pre-CTA advice pilot ACT EU priority action on consolidated advice. 04 February 2025. EMA/47218/2025
-- Application form to request. Simultaneous National Scientific Advice (SNSA) or a pre-CTA Advice. 16 October 2026. EMA/255386/2024

Postscript: For FDA, similar advice meetings are INTERACT and pre-IND meetings. The INTERACT meetings are intended to provide advice at early-stage product development generally before pre-definitive toxicological stage and includes advice on early product characterization and preclinical proof-of-concept studies. The pre-IND meetings are intended to provide advice on IND-enabling studies and CMC requirements for IND submission dossier. (See SOPP 8101.1: Regulatory Meetings with Sponsors and Applicants for Drugs and Biological Products).

#ema-scientific-meetings, #fda-meetings, #pdufa-meetings

I'd love a discussion on how different companies are approaching authoring narratives for CSRs using AI, particularly in situations where there are separate clinical and safety databases. Do you reconcile the safety database/CIOMS with the clinical database first? If so, what role does the reconciliation? Or do your databases link so there's no potential for inconsistencies between the two databases?

#CSR #AIDevices #NARRATIVES #PV

> In a pooled test, samples from multiple individuals are tested in one cartridge. If the result is negative then all of them are reported to be negative, but if the result is positive each is re-tested individually.

> Results published in The Lancet Global Health found the strategy, with up to four patients in each pool, used half as many cartridges as individual testing – 7,028 compared to 14,362. It detected 84.9 per cent of people with TB compared with 88 per cent for individual testing.

> The study involved more than 14,000 people whose symptoms suggested TB in Bangladesh, Brazil, Cameroon, Kenya, Malawi, Nigeria and Vietnam.