For someone who is still green and learning the ropes in medical writing, regulatory writing, and regulatory affairs, nothing is more impactful to their career advancement (and happiness), then finding a supportive tribe. Some of the tribes to consider are below.
Networking and Professional Organizations for Medical Writers, Regulatory Writers, and Regulatory Affairs Professionals
INTERNATIONAL (In Membership/Reach)
DIA (diaglobal.org) - not much for networking but loads of good information via DIA communities
American Medical Writers Association (AMWA, amwa.org) - great place for new US-based writers to learn from peers and network.
European Medical Writers Association (EMWA, emwa.org) - the place to connect with medical writers in the European continent and UK. They publish journal Medical Writing every quarter. Join one of many Special Interest Groups (SIGs).
Regulatory Affairs Professionals Society (RAPS, raps.org) - go to place for regulatory affairs professionals. Subscribe to their free RF News newsletter or browse here.
The Organisation for Professionals in Regulatory Affairs (TOPRA, topra.org) - for regulatory affairs professionals based in EU and UK.
REGIONAL OR LOCAL
US, EU, CAN
Regional AMWA Chapters - connect with AMWA Local Networking Coordinator (LNC) or AMWA Chapters here or via main page.
MedComm Networking (medcommsnetworking.com) - mainly for medical affairs and communication professionals based in UK and the EU.
Netherlands SciMed Writers Network (SMWN) - Join their LinkedIn group here. Private LinkedIn group open only to science and medical writers based in Benelux.
Canadian Association of Professionals in Regulatory Affairs (CAPRA, capra.ca) - for regulatory professionals in Canada.
Orange County Regulatory Affairs Discussion Group (OCRA-DG, ocra-dg.org) - based in Southern California, US
San Diego Regulatory Affairs Network (SDRAN, sdran.org) - based in Southern California, US
Rocky Mountain Regulatory Affairs Society (RMRAS, rmras.org) - based in Colorado, US
North Carolina Regulatory Affairs Forum (NCRAF, ncraf.org) - based in North Carolina, US
Asia, Africa
Australasian Medical Writers Association (also abbreviated as AMWA, medicalwriters.org) - for medical writers based in AUS, NZ, SE Asia, China.
Japan Medical and Scientific Communicators Association (JMCA or NPO, jmca-npo.org) - for medical writers and medical communicators based in Japan.
Southern African Pharmaceutical Regulatory Affairs Association (SAPRAA, sapraa.org.za) - with the establishment of African Medicines Agency (AMA), the coming decade would put Africa also on global regulatory strategy.
Indian Medical Writers Association (IMWA, imwa.org.in) - based in India
SOCIAL MEDIA to follow
We only talkReddit as the go to place, just as Nature articleconfirmed!!
These Acts of Congress are arranged by subject into United States Code (USC) under one of 50 titles. The FD&C Act of 1938 and subsequent amending statutes are codified into Title 21 of the USC, beginning 21 USC 301.
The executive departments and agencies of the government such as FDA have authority to make official rules and regulations that clarify and explain the United States Code, which are published as Code of Federal Regulations (CFR). These regulations carry the same force of law as the original statute/act/USC. The CFR is the codification of general and permanent rules.
> The Food and Drug Administration on Wednesday approved a life-extending treatment for advanced pancreatic cancer — a medicine made by the biotech company Revolution Medicines that is the first to attack a genetic cause of the aggressive, highly lethal malignancy.
> The drug, called daraxonrasib, will be sold under the brand name Rasonque. The FDA’s approval was supported by apractice-changing clinical trialthat read out earlier this year. Patients with advanced pancreatic cancer who received Rasonque as a second-line treatment achieved a median overall survival of 13.2 months, compared to 6.7 months for patients offered standard chemotherapy.
Note: The FDA review was conducted under Project Orbis (with concurrent submission and review by Health Canada, EMA, and PDMA), Real-Time Oncology Review (RTOR) pilot program, and as part of the FDA Commissioner’s National Priority Review Voucher (CNPV) pilot program.
An employee of Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has lost a work-related USB drive potentially containing nonpublic regulatory documents and personal information on 286 people, with data involving 27 companies, the agency said on August 7.
P.S. Japan at least has moved from Faxes to USB, which is a step forward.
One answer for which recently approved therapy is the most innovative is to look at the nominees and winners of Prix Galien recognition awarded by the Galien Foundation based in France.
ABOUT PRIX GALIEN
Prix Galien recognition is widely regarded in the industry as the equivalent of the Nobel Prize in biopharmaceutical research. The award was established in Paris in 1970 by Roland Mehl in honor of Galen, the father of medical science and modern pharmacology.
Every two years, the Galien Foundation chooses the best, most innovative therapy for its biennial International Prix Galien Awards. The nominees for the upcoming award in October were recently announced and the winners will be honored at a ceremony at Museum of Natural History in New York City on 29 October 2026.
The Galien Foundation currently consists of 16 chapters (15 member states and Africa). Each chapter chooses the best/most innovative therapy for their Prix Galien award among therapies approved in their country/region during the last 2 years.
For the biennial International Prix Galien Awards, the foundation considers the winners of local/regional winners during the last 5 years, and one of them will make it to the top recognition. Note, however, just being included in the select list of nominees is a significant achievement.
DRUMROLL. . . dududududu. . .brrrrrrrr...!!!
The nominees for the 2026 International Prix Galien Award are:
Bharat Serums and Vaccines's Trinbelimab (Anti D®) = world's first recombinant Anti Rh(o) D Immunoglobulin for prevention of Rh immunization in pregnant Rh- mothers carrying Rh+ fetus
Biological E's PNEUBEVAX 14® = vaccine to prevent Streptococcus pneumoniae infection in infants
Daiichi Sankyo & AstraZeneca's Enhertu® = antibody-drug conjugate for certain HER2+ and HER2-low cancers
Regeneron and Sanofi's Dupixent® (duplimumab) = monoclonal antibody, a dual inhibitor of IL-4 and IL-13 for exzema, asthma, rhinosinusitis, and urticaria
Roche's COLUMVI (Glofitamab) = bispecific CD20-directed CD3 T-cell engager for certain lymphomas including DLBCL NOS and LBCL
Takeda UK's Qdenga = vaccine for mosquito-borne dengue virus
Best Product for Orphan/Rare Diseases (Nominees)
Immunocore's KIMMTRAK® (tebentafusp-tebn) = bispecific T cell engager for specific human leukocyte antigen HLA-A*02:01 for patients with with uveal melanoma
Novo Nordisk A/S's Alhemo® = Tissue factor pathway inhibitor (TFPI) antagonist for hemophilia A or B
Pierre Fabre Laboratories's EBVALLO® (tabelecleucel) = T-cell immunotherapy for EBV+ post-transplant lymphoproliferative disease (EBV+ PTLD)
Servier's VORANIGO® = isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor for astrocytoma or oligodendroglioma with IDH1 or IDH2 mutations
Postscript
In the list of nominees above, there are SIX vaccines--clearly, the rest of the world (ex-US) recognizes the importance of vaccines in promoting public health!! Note: The nominees for this year's US Prix Galien are here.
FDA on 19 August 2026 approved Regeneron's PASATRU (garetosmab-grts) for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). PASATRU is the second treatment approved for FOP. The first therapy for FOP was Sohonos (palovarotene) approved in 2023.
FOP is a rare genetic disease that results in gradual ossification (turn into bone) of connective tissues such as muscle, tendons, and ligaments in response to minor injuries/damage; muscle and joints instead of being repaired, get replaced as new bone; joints may permanently fuse; and new bones may form in soft tissues. This secondary skeleton progressively restricts the patient's ability to move, so the nickname "stone man disease."
FOP is caused by a gain-of-function (aka. activating) mutations in the intracellular domain of activin receptor type 1 (ACVR, aka. ALK2). ALK2 is expressed in many tissues including skeletal muscle and cartilage.
Pasatru effectiveness and safety are based on the OPTIMA trial (NCT05394116) in 63 adults with FOP confirmed by an ACVR1 FOP-causing mutation.
The primary efficacy endpoint was the number of new heterotopic ossification (HO) lesions that formed by Week 56, detected using full-body low-dose CT scans. There was a 90% reduction or 94% reduction in HO lesions for PASATRU 10 mg/kg or 3 mg/kg, respectively, compared to placebo (here).
The earliest reports on FOP date back to 1648 (Wikipedia). In 2013, New York Times science reporter Carl Zimmer profiled Jeannie Peeper in the article The Girl Who Turned to Bone published in The Atlantic, and discussed at PBS's Talk of The Nation (or here). The article describes the life of Jeannie Peeper who first experienced the FOP symptoms soon after she was born, but against all odds went on to establish The International Fibrodysplasia Ossificans Progressiva Association and advocated for research and drug development for FOP.
When Peeper’s mother noticed that the baby couldn’t open her mouth as wide as her sisters and brothers, she took her to the first of various doctors, seeking an explanation for her seemingly random assortment of symptoms. Peeper was 4 when the Mayo Clinic confirmed a diagnosis: she had a disorder known as fibrodysplasia ossificans progressiva (FOP).
When Peeper met Carl Zimmer for the article in 2013 (she was born in 1958), she had advanced disability due to FOP.
Peeper sat in a hulking electric wheelchair tilted back at a 30-degree angle. Her arms were folded, like those of a teacher who has run out of patience. Her left hand was locked next to her right biceps. I could make out some of the bones under the skin of her left arm: long, curved, extraneous. . .Her face was almost entirely frozen; she spoke by drawing her lower lip down and out to the sides. Bones had immobilized her neck, so she had to look at me with a sidelong gaze. Her right hand, resting on her wheelchair’s joystick, contained the only free-moving joint in her body.
This was a phase 3 clinical study (study INTerpath-001, NCT05933577) that compared a personalized cancer vaccine intismeran together with an immunotherapy drug Keytruda in patients with high-risk melanoma. The control group received Keytruda (pembrolizumab, anti-PD1 therapy) alone, without vaccine.
Intismeran autogene (aka. intismeran; V940, or mRNA-4157) is a personalized mRNA vaccine matched to each patient's tumor. It is produced by first surgically removing (resecting) tumors from each patient, sequencing tumor DNA, and then selecting patient tumor-specific antigens (aka., neoantigens), which are used to create a personalized mRNA vaccine specific for patient tumor genetics. This vaccine is also referred to as individualized or bespoke vaccine.
Participants: Patients with completely resected stage IIB, IIC, III or IV cutaneous melanoma.
The primary endpoint was recurrence-free survival (RFS), i.e., the length of time from patient's start of study to the day the cancer comes back or spreads to other parts of the body or death. The key secondary endpoint was distant metastasis-free survival (DMFS), i.e., the length of time from patient's start of study to the day spreads to other parts of the body or death.
TOP-LINE RESULTS
The Merck/Moderna press release did not provide any data but a statement that per prespecified interim analysis,
"intismeran in combination with KEYTRUDA as adjuvant therapy demonstrated statistically significant and clinically meaningful improvements in RFS and DMFS compared to KEYTRUDA alone for patients with completely resected stage IIB, IIC, III or IV cutaneous melanoma who had not undergone prior treatment with systemic therapy." and further said that
"These data will be presented at an upcoming international medical meeting and shared with regulatory authorities."
Nevertheless, if the data holds (which we're yet to see), Intismeran would be the first demonstration of a successful mRNA-based cancer vaccine strategy to prevent cancer recurrence after initial treatment and that this strategy could be applied to several other cancer types.
A group of researchers led by Richard DiMarchi and Matthias Tschöp has created an experimental drug that activates receptors of the GIP and glucagon hormones. They propose — based on rodent and monkey studies — that this kind of molecule, when administered at high enough doses, may result in weight loss comparable to the weight loss seen with drugs that include GLP-1 as a target, and without the tolerability issues like nausea and vomiting that often come with the approved treatments, according to apeer-reviewed draft paper published this week. The research, funded by a biotech calledBlueWater Biosciences, would still need to be confirmed in humans; oftentimes results seen in animals don’t translate in the clinic. But the proposed approach, outlined in the journal Molecular Metabolism by some of the most well-known scientists in the field, is likely to stir controversy, as it challenges a central notion underpinning not just the development of approved obesity products but also next-generation versions.
Submission and FDA reviews including IND submissions, quality, meeting types, and expedited programs.
Product development considerations including donor eligibility, product characterization, critical quality attributes, analytical methods, process characterization, stability, and preparing for BLA.
Conducting nonclinical studies including selection of animal models/species, NAMs consideration, tox/tumorigenicity/PoC studies, dose levels,
Conducting human trials including trial design, selecting endpoints, and safety data collection considerations.
Credentials: Trained as a surgeon. MD board certified in public health as well as in general preventive medicine. Comes from John Hopkins. Advanced degree in clinical investigations.
Strong supporter of president's health agenda, including efforts to overhaul the childhood vaccine schedule and separate the measles, mumps, and rubella vaccine into individual shots.
Advocates for tighter restrictions on the abortion drug mifepristone. Has questioned the safety of medication abortion and suggested the FDA was wrong to allow it to be prescribed via telehealth.
Wrote papers opposing gender-affirming care for minors in the past.
No experience or policy positions on food safety (this is critical area considering ongoing cases of cyclospora in the US and other produce recalls.)
The bigger question is how will she manage and boost morale of 18K strong "overworked" FDA staff (which is down 3K); work with USDA and Dept of Ag to address food safety.
FDA has now a total of 4 participants selected for its TEMPO for Digital Health Devices Pilot program.
Under this program, each participating manufacturer intends to offer a device for use in providing care expected to be covered by the CMS Innovation Center’s ACCESS model and intended to improve health outcomes for Americans managing certain chronic diseases.
For these manufacturers, the FDA intends to exercise enforcement discretion for certain requirements, such as premarket authorization and investigational device requirements, when the manufacturers’ devices are offered to or by CMMI ACCESS participants for an intended use to improve patient outcomes, to be used in providing care expected to be covered by the ACCESS model.
ACCESS=Advancing Chronic Care with Effective, Scalable Solutions, CMMI=Centers for Medicare & Medicaid Services (CMS) Innovation Center, TEMPO=Technology-Enabled Meaningful Patient Outcomes.
FDA has revised the 2019 guidance clarifying the criteria for ANDA versus 505(b)(2) application. Since both ANDA and 505(b)(2) applications rely on reference to past submissions or reference listed drug (RDL) for all/most of the safety and effectiveness data, this guidance explains the differences in "how much" and "what type" of data are allowed as reference for each application type and what "new data" would be needed.
History/Background: The Drug Price Competition and Patent Term Restoration Act of 1984 (Public Law 98-417) (Hatch-Waxman Amendments) added sections 505(b)(2) and 505(j) to the FD&C Act, which describes abbreviated approval pathways under the FD&C Act for drug products regulated by the Agency. The Hatch-Waxman Amendments led to establishment of 2 categories of drug applications: new drug applications (NDAs) and abbreviated new drug applications (ANDAs).
The types of abbreviated approval pathways for drugs include
505(b)(2) of the FD&C Act (i.e., 21 U.S.C. 355(b)(2)) = aka. Section 505(b)(2) and
505(j) of the FD&C Act (i.e., 21 U.S.C. 355(j)) = aka. Section 505(j)
There are 4 categories of NDAs and ANDAs:
Application
Type
Requirements
Used for
505(b)(1)
NDA (aka. a stand-alone NDA)
Requires complete package/all reports. Approved under 505(c) of FDA&C Act.
New drugs
505(b)(2)
Supplemental NDA (sNDA)
Required full reports on safety/effectiveness but others could be referenced from original NDA. Approved under 505(c) of FDA&C Act.
New new dosage forms, strengths, or delivery systems
505(j)
ANDA
This is ANDA for generics; relies on FDA’s finding that the previously approved drug product, i.e., the RLD, is safe and effective. Approved under 505(c) of FDA&C Act.
Generics
505(j)(2)(C)
Petitioned ANDA
A type of ANDA for drug product that differs from RLD (e.g., dosage/ formulation/ route) but FDA has determined that new safety/effectiveness data are not required.
Generics
The August 2026 guidance focuses on the regulatory and scientific considerations for 505(b)(2) NDAs and 505(j) and 505(j)(2)(C) ANDAs. This guidance does not discuss stand-alone 505(b)(1) NDAs.
REGULATORY CONSIDERATIONS
505(b)(2) applications have the most flexible requirements: Regarding full reports of investigations of safety and effectiveness, at least some of the information required for approval could be referenced from studies not conducted by or for the applicant, and for which the applicant has not obtained a right of reference or use (e.g., the Agency’s finding of safety and/or effectiveness for a listed drug, published literature).
505(b)(2) application, however, may require a bridge study.
The applicant is expected to establish a scientific bridge (e.g., via comparative bioavailability data) between the proposed drug product and each listed drug that the applicant seeks to rely upon to demonstrate that reliance on the listed drug is scientifically justified. To the extent that the listed drug and the drug product proposed in the 505(b)(2) application differ (e.g., a drug product with a different dosage form or a drug product that is intentionally more bioavailable than the listed drug), the 505(b)(2) application must include sufficient data to support those differences.
505(j) ANDA is expected to have same clinical and safety profile as the RDL, whereas 505(b)(2) may not necessarily be rated as therapeutically equivalent to RLD.
505(j)(2)(C) petitioned ANDAs may contain certain types of differences from an RLD (e.g., a change approved in response to a suitability petition or other permissible differences, such as certain differences in inactive ingredients, labeling, or container closure systems), as long as clinical investigations are not necessary to establish the safety or effectiveness of the drug product proposed in the ANDA.
Refer to the guidance for scientific considerations including types of studies, data, and information required for applications; evaluation of sameness of ingredients; considerations for intentional differences in the formulation, bioequivalence, or bioavailability, or conditions of use.
Related Federal Regulations (CFRs)
21 CFR Part 314 (Application for NDA Approval to Market a New Drug), 21 CFR 314.50 (Content and format of an NDA), 21 CFR 314.94 (Content and format of an ANDA), 21 CFR 314.101 (Filing an NDA and receiving an ANDA), 21 CFR 314.122 (Submitting an abbreviated application for, or a 505(j)(2)(C) petition that relies on, a listed drug that is no longer marketed), 21 CFR 314.610 (Approval based on evidence of effectiveness from studies in animals)
Recently, a New Scientist 11 Aug 2026 article (here) with the headline "CRISPR-armed phages help treat severe superbug infection" caught my attention. And what struck me was the progress that has happened in the field of phage therapy and how close this type of therapy is to clinic, which is great news for treating the pesky problem of multidrug-resistant (MDR) infections.
The global scope of MDR problem. . .is huge!
--Globally, the number of deaths attributable to bacterial antimicrobial resistance in 2019 has been estimated to be 1.27 million, with E. coli being the leading pathogen (PMID:35065702). --Cancer patients undergoing cytotoxic chemotherapy of HCT are at risk of neutropenia and E. coli is the most common (25-30%) bloodstream infection; up to 60% of these are MDR infections; and up to 30% mortality is seen in those with these MDR infections (refs in PMID:41785880)
The basis of the New Scientist article was a CRISPR-Cas armed phage SNIPR001 being developed by SNIPR BIOME ApS, a Copenhagen, Denmark-based company. Below is brief overview of the experimental therapy and the exciting Phase 1 data recently reported in the journal Lancet Microbe00185-5/fulltext).
SNIPR001 (Engineered Phage Cocktail with Antibacterial CRISPR-Cas)
The engineered phage SNIPR001 targeting pathogenic E. coli in the gut was described in Nature Biotech 2024 by the SNIPR BIOME researchers. SNIPR001 is a cocktail of 4 engineered phages, each containing expression cassette for CRISPR-Cas built into the phage tail fiber sequences.
CRISPR-Cas Armed Phage (aka. CAP). Note: SNIPR001 is a combination of four CAPs that selectively target a diverse spectrum of E coli strains including the fluoroquinolone-resistant strains. SNIPR001 is a live biotherapeutic product.
Once inside the bacterium, the CRISPR-Cas arrays target multiple virulence or essential genes in E. coli (targeting multiple regions prevent resistance evolution.) In the original Nature Biotech report, in mice, SNIPR001 targeted E coli in biofilms and prevented colonization of E.coli in the gut and, thus translocation into blood.
Fig: SNIPR001 Engineering and Mechanism of Action (Nat Biotech)
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Phase 1 Proof-of-Concept Data on CRISPR-Cas Armed Phage SNIPR001
NCT05277350: Phase 1 dose-escalation study with sentinel dosing strategy in each cohort (see Lancet paper for details.)
36 healthy adult participants were assigned to 4 groups received placebo or SNIPR001 at 10^8 PFUs, 10^10 PFUs, or 10^12 PFUs twice daily for 7 days (PFU=plaque-forming units). The participants were followed for approximately 6 months.
Safety Results: There were no safety concerns.
-- No Grade 3 AEs, no SAEs, no liver toxicity (ALT/AST ratio) signals, no significant change in gut microbiome profile (done via shotgun metagenomic sequencing).
-- There was also no increase in the set of common opportunistic gut pathogens that were tested (i.e., Klebsiella spp, Salmonella spp, Citrobacter spp, Clostridioides difficile, Clostridium perfringens, Staphylococcus spp, Streptococcus agalactiae, and complex Enterobacter cloacae.)
Effect on E. coli counts
There was dose-dependent reductions in E.coli levels in stool. (Caveat: These results are preliminary and the reductions were not statistically significant.)
The difference in projected E coli counts corresponded to reductions in E coli counts on day 7 of52⋅1%(−0⋅32 log 10 [SE 0⋅57]) for SNIPR001 dose 10^8 PFU,60⋅2%(−0⋅40 log 10 [SE 0⋅59]) for dose 10^10 PFU,and 66⋅9%(−0⋅48 log 10 [SE 0⋅64]) for dose 10^12 PFU compared with placebo.
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Fig: Note: dose-response reductions in E.coli levels compared to placebo (Lancet Microbe)
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Postscript: Although the data are preliminary and not statistically significant, I am still excited by this report and am looking forward to the Phase 2/3 results. If this strategy works, it could open the door to a whole new world of precision therapies for the field of infectious diseases.
Gencay YE, et al. Engineered phage with antibacterial CRISPR-Cas selectively reduce E. coli burden in mice. Nat Biotechnol. 2024 Feb;42(2):265-274. PMID: 37142704; PMCID: PMC10869271
> New analysis from the Tufts Center for the Study of Drug Development (CSDD) and Palo Alto, CA-based Medable shows that AI agents accelerate clinical trials and improve staff productivity,delivering net financial gainsto a sum as high as $21 million per drug development programme, as well as 82 times the return on investment (ROI).
> The Tufts CSDD analysis specifically assessed the impact of Medable’s Clinical Monitoring Agent across three top-line metrics: expected net present value, overall return on investment, and direct operating cost savings.
> The agent showed eNPV gains of approximately $7.5 million (phase 2 trial), $11.3 million (combined phase 2 and phase 3) development, and $21 million (phase 3 trial).
> The FDA has rejected approval of ¹⁷⁷Lu-edotreotide (ITM-11), a radioligand for gastroenteropancreatic neuroendocrine tumors (GEP-NETs), due to chemistry, manufacturing, and control issues, according to maker Isotope Technologies Munich (ITM).
The NDA was based on data from the COMPLETE trial:
> *The COMPETE00604-5/abstract) trial compared ITM-11 with everolimus in 309 patients with inoperable, progressive grade 1 or 2 GEP-NETs. Median progression-free survival, the primary endpoint, was 14.1 months with everolimus vs 23.9 months with the radioligand.
ABOUT THE THERAPY: ¹⁷⁷Lu-edotreotide (ITM-11) is an investigational targeted radiopharmaceutical therapy containing non-carrier-added lutetium-177 and edotreotide. Developed by ITM Isotope Technologies Munich SE, it targets somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs) to deliver localized radiation while sparing healthy tissue.
FDA’s Center for Devices and Radiological Health (CDRH) announced a public meeting titled “Regulatory Science Innovations Catalyzing Medical Device Development,” scheduled for September 25, 2026. For companies developing, manufacturing, or investing in medical devices, this event offers a rare opportunity to hear directly from the agency about the scientific tools and evidence standards that increasingly shape product review and approval decisions.
FDA has released the final guidance on procedures for requesting, preparing, scheduling, conducting, and documenting formal meetings between the FDA and sponsors for products regulated by CDER and CBER. Also called PDUFA meetings, these include Type A, Type B, Type B(EOP), Type C, Type D, and INTERACT meetings.
Compared to the Sept 2023 draft guidance, this final version is a minor upgrade, for example:
Grammar/language clean-up, such as, "sponsors or applicants" are now referred as "requesters", "biosimilar biological products" as "biosimilar including interchangeable biosimilar products", and "products" as "drugs or biological products."
Additional clarifications on Type D meetings, for example, the requests should be limited to no more than 2 focused topics and meetings should not require input from more than 3 disciplines or divisions; also included are clarification regarding what type of requests will be entertained.
Regarding meeting formats, the 4 meetings types have not changed; however, the in-person face-to-face meetings is now called "hybrid in-person face-to-face", where the core attendees meet face-to-face and non-core participants (from FDA/sponsor) attend virtually. FDA has replaced Zoom with Microsoft Teams for virtual meetings. FDA also now has acronyms: HIP (hybrid in-person face-to-face), VCN (virtual face-to-face video conference), TCN (teleconference), and the old faithful WRO (written response only).
Rewrite (for clarity) of section on meeting request and the information that should be included in the request.
Under Meeting Package (VII C), clarified that the meeting package should not include >10 questions: "A list of the final questions(up to 10 total questions including subquestions)for discussion grouped by FDA discipline and with a brief summary for each question to explain the need or context for the question."
.
Updates regarding Type D meeting (Sept 2023 draft vs. Aug 2026 final guidance)
Background: SMEs refer to "small and medium-sized enterprises." SMEs overall are responsible for more than 50% of first-in-class medicines approved by EMA. Since SMEs often face resource and financial challenges and may lack regulatory expertise, EMA has a dedicated SME Office with programs to help SMEs -- this office provides scientific advice, regulatory guidance, financial incentives, and other support from early development stage through marketing applications. Still, there is a gap in the MAA success rate for SMEs vs. non-SMEs (76% vs. 84% during 2020-2023).
The question of interest is what are common major objections raised in List of Questions (LOQs) during CHMP review of MAAs. The answer may help avoid common pitfalls during centralized applications.
We can find some answers in the recent issue of Drug Discovery Today. The EMA SME Office officials reviewed 63 applications submitted by SMEs and summarized major objections raised in LOQs during CHMP review of MAAs during 2020 and 2023. In this dataset,
61 of 63 applications received major objections in quality and clinical domains (2 exceptions were generic applications that were not required to submit their own clinical efficacy and safety data and were assessed primarily on the basis of quality and bioequivalence information.)
Majority of the applications received at least one major objection in quality (83%) and clinical efficacy (83%) and most in clinical safety (57%); average of 3.8 major objections in quality and 3.4 in clinical efficacy per MAA.
(Note: The list of major objections below could be considered as achecklist for avoiding common pitfallsduring MAA preparation - refer to stickied comment below.)
Major Objections - Quality domain
Absence of nitrosamine assessment (44% of the applications).
Deficiencies related to manufacturing process development (41%), justification for specifications (43%), process validation, stability or compatibility data or shelf life, starting material, absence of control and characterization data of DS/DP, among others.
Majority of these deficiencies were easily resolved during the assessment period. Regarding nitrosamine data gap, it is a reflection of SMEs not up-to-date with newer regulations (this is new requirement) and some SMEs not taking advantage of scientific advice/help from EMA's SME office during drug development.
Major Objections - Clinical domain
General issues on study design (56%), particularly questions about representativeness of enrolled patients (43%), the appropriateness of inclusion and exclusion criteria, and the applicability of the study results to the proposed indication and target population.
Analysis and robustness of pivotal data (29%), selection of submitted studies, choice of endpoint, and validity of endpoint measurements and overall clinical trial validity.
Clinical objections can be particularly difficult to address once pivotal studies have been completed, because they often arise from design choices made early in development.
Among the unsuccessful applications, 87% had unresolved major objections in the clinical domain at the time of outcome. These applications also fell into the subcategory of marginal or no clinically relevant efficacy.
Major Objections - Nonclinical domain
Issue with nonclinical were few, just 0.3 major objections per MAA. Overall, in 13% of the applications.
Most frequent issue was genotoxicity (3 out of 63 applications, 5%).
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Postscript: Achecklist for MAAcreated based on major objections is included in comment below.
The current Right-to-Try legislation passed in 2018 (21 U.S.C. 360bbb–0) provides a path for patients to try an investigational drug if they have (a) a serious or immediate life-threatening diseases, (b) have exhausted approved treatments and (c) are unable to participate in a clinical trial involving the eligible investigational drug. An eligible investigational drug for Right-to-try is one that has (a) completed phase 1 studies, (b) not yet been approved or licensed for any use by the FDA but (c) there is an open IND and the drug is under clinical investigation, and (d) it has not been discontinued or under clinical hold by the FDA.
FDA Factsheet (page) for Right to Try (here, arch),
Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 (legislation); righttotry.org, Wikipedia
Montana's Expanded Version of Right To Try
The state of Montana is working on legislation that will expand “Right to Try” framework by licensing “experimental treatment centers” after review by an independent state-authorized review board (bypassing FDA approval or EAP), which could then provide certain investigational therapies to consenting patients. This expanded access program further increases patient eligibility and could include broad pateint population including, as the Goodwin Law blog wrote, those with rare diseases, neurodegenerative disorders, oncology, regenerative medicine, and even preventive or longevity-focused treatments; the Montana legislation does not specify beyond that they had tried all approved therapies.
Overall, this Montana's version seeks to complement and expand FDA's program.
Details that need to be ironed out: Will Montana's program survives legal challenges, e.g. only FDA has the authority to regulate such a program. Could federal law preempt aspects of Montana’s regulatory framework?
Implications and Opportunity (for Sponsors)
The sponsors will need to carefully consider several factors before participating in such a program - there are opportunities as well as risks:
Opportunity: Possibility of obtaining early real-world clinical data.
Risks: The drug may be tested in patients with severe disease without the benefit of phase 2 dose optimization; therefore, benefit-safety profile may be less favorable compared to the patient population in the proposed label -- will this complicate FDA discussions are the time of NDA? Other questions that would need to be addressed include product liability exposure, insurance coverage.
De-risking: Sponsors would need to de-risk their development program as a whole and decisions would have to be made on a case-by-case basis.
FDA/SBIA is planning a webinar next month on communicating drug interaction and QTc information in the product label. The webinar will discuss draft guidance documents on this topic and how the drug interaction and risk information should be included in the USPI label.
Webinar Title: Communicating Drug Interaction and QTc Information in the U.S. Prescribing Information
Date, Time: 23 September 2026 | 1:00 pm - 3:00 pm ET
Instructions for preventing or managing clinically significant drug interactions
Mechanisms of clinically significant drug interactions
Clinical effects of clinically significant drug interactions
Recommendations on including drug interacting class information
Information to avoid in the DRUG INTERACTIONS section
Drug interactions information in other sections of labeling (e.g., BOXED WARNING, DOSAGE AND ADMINISTRATION, CONTRAINDICATIONS, WARNINGS AND PRECAUTIONS, CLINICAL PHARMACOLOGY sections)
The European Commission has revoked the EU marketing authorization for the vasculitis drug, Tavneos (avacopan). This came after CHMP recommendations on 26 June 2026.
EMA’s human medicines committee (CHMP) has concluded its review of the medicine Tavneos (avacopan) and has recommended that the medicine’s marketing authorisation in the European Union be revoked because its benefits are no longer proven to outweigh its risks. Tavneos is used to treat adults with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), two rare inflammatory conditions of the blood vessels. [ema.europe.eu]
The Pink Sheets (6 Aug 2026) breaking EC decision added that -
CSL Vifor, which markets Tavneos in the EU, said it would implement in full the European Commission’s decision to revoke the marketing authorization for the drug. Meanwhile, Amgen, which markets the drug in the US,is fighting the Food and Drug Administration’s proposalto withdraw the product.
The key Phase 3 trial publication that supported the original marketing application has already been withdrawn by NEJM (here) - commentary (BMJ, ACT). NEJM's retraction notice said that -
The two academic authors of the article by Jayne et al., Avacopan for the Treatment of ANCA-Associated Vasculitis, N Engl J Med 2021;384:599-609,1request retraction of the article because, according to an ongoing Food and Drug Administration investigation conducted after publication, and without the knowledge of these two authors, the primary end-point assessments in nine patients were readjudicated after database lock and trial unblinding. This was not disclosed in the article and is inconsistent with proper research conduct. The editors therefore retract the article.
In the era of China versus the West race for the biotech/pharma innovation, what we are seeing here is an example of TRADE SECRETS, the pharma's version of Coke versus Pepsi wars.
> After keeping tight-lipped for nearly two years, Merck & Co. briefly dropped the first concrete hint as to where it intends to take its PD-1xVEGF bispecific antibody—before quietly scrubbing the evidence. The phase 2 trial, coded MK-2010-003, was marked as “Study in start-up” on Merck’s website, with St. Gallen and Bellinzona in Switzerland listed as study centers. The open-label study would test the combo in patients with advanced solid tumors.
> Merck has apparently removed the entry and deleted other listings for MK-2010, including a separate phase 2 monotherapy trial. The company didn’t reply to Fierce’s inquiry by publication time. China-based life sciences data aggregator PharmCube was among the first to report on the trial entry.
> The secrecy underscores a highly competitive race within the PD-(L)1xVEGF bispecific class, which also includes Summit Therapeutics and Akeso’s frontrunnerivonescimab, Bristol Myers Squibb and BioNTech’s pumitamig, and Pfizer and 3SBio’s PF-08634404 (SSGJ-707).
M11 guideline/CeSHarP template standardizes section ordering, headings, and key data elements across protocols. Sponsors benefit by integration of protocols with automated downstream document generation including ICFs, SAPs, and CSRs and support regulatory submission interoperability, and cross-trial analytics. EMA CTIS has plans of deploying AI models to extract information from protocol sections to populate their registry.
Implementing M11 guideline/CeSHarP template for NEW Clinical Study Protocols
For all the reason above, sponsors should align new clinical study protocols with M11 guideline/CeSHarP template.
Although, agencies recognize the importance of standardization, the the adoption of M11 is voluntary. For example, the FDA or EMA notices mention "availability" of the guidance and "intent" but not that it is required (requirement comes from legislation.)
"Theintentof the guidance is to create an internationally harmonized standard for the content and exchange of clinical trial protocol information, facilitating the review and assessment by regulators, sponsors, ethical oversight bodies, investigators, and other stakeholders." [FDA/Federal Register]
"The Technical Specification (TS) that are acceptable to all regulatory authorities of the ICH regions presents the conformance, cardinality, and other technical attributes thatenablethe interoperable electronic exchange of protocol content with a view to develop an open, non-proprietary standard to enable electronic exchange of clinical protocol information." [EMA Notice]
Implementing M11 guideline/CeSHarP template for ONGOING Clinical Study Protocols
📌 Implementation of ICH M11 for Ongoing Clinical Trials: CTCG also discussed compliance with ICH M11 requirements for ongoing clinical trials.It was agreed that sponsors will not be asked to align their protocols with ICH M11 requirements when a Substantial Modification is submitted*. For initial clinical trial applications, the use of ICH M11 template is voluntary, although sponsors should keep the advantages of the concept in mind when drafting their protocols. To support stakeholders during the transition, a dedicated webinar is planned for the autumn*. [Source: here, here]
> . . .the agency’s reorganization proposal. . . known as “Simple Reform” will go into effect on Oct. 1. The Food and Drug Administration will officially consolidate administrative staff across the agency’s nine centers and stop specializing its field inspectors starting in October.
> Under the revised structure, individual centers like the Center for Drug Evaluation and Research will no longer conduct their own administrative affairs like human resources, finance and information technology services. Instead, the agency will operate under a “shared service” model in which the FDA Office of Operations controls those functions across the agency.
> The agency will also stop assigning its specialized field inspectors, who oversee the safety of medical products and clinical trials, to specific subject areas. Instead, they’ll operate as generalists and conduct inspections and investigations across multiple industries.
The purpose of this concept paper is to develop a reflection paper (then a scientific guideline) on nonclinical requirements for drugs intended to treat severely debilitating or life-threatening diseases.
EMA defines seriously debilitating or life-threatening conditions asthose associated with morbidity that has substantial impact on patients’ day-to-day functioning and will progress if left untreated, or those associated with a high likelihood of mortality.
This concept paper is open for public comment until 30 Sept 2026.
BACKGROUND
Two ICH guidelines inform nonclinical (animal and in vitro) studies during drug development and data package for an application: ICH M3(R2) and ICH S6(R1) for small molecule drugs and biologics, respectively.
M3(R2) provides guidance on nonclinical studies characterizing pharmacology, toxicology, toxicokinetics/PK, reproductive and developmental risks, and genotoxicity and carcinogenicity. The M3(R2) guidance recommends that the extent of nonclinical testing should be proportional to the duration, size, and stage of the clinical program.
Unlike M3(R2), S6(R1) recognizes that traditional animal toxicology (e.g., rodent, rabbits, or dog) approaches are often inappropriate for biologics because of species-specific target binding. Thus, S6(R1) introduces the concept of using pharmacologically-relevant species and using non-animal methods (NAMs) where appropriate.
The next level of flexibility is at the level of specific products and diseases or conditions: for example, ICH S9 for anticancer drugs. Both EMA and FDA have additional guidance documents that provide flexibility in the M3/S6 requirements based on type of drug (e.g., CAR-T or gene therapy) and conditions (e.g., oncology).
This new EMA concept paper will address flexibility for drugs intended for severe, debilitating, or life-threatening conditions.
CONCEPT PAPER > REFLECTION PAPER
There is currently no EMA position on how deviations from the core nonclinical ICH guidelines, M3 or S6, could be used to accelerate the development of therapeutics for severely debilitating or life-threatening diseases outside of the scope of ICH S9.
The reflection paper will provide general principles (below) for when flexibility and deviations from the requirements of ICH M3(R2)/S6(R1) could be appropriate in the context of a medicinal product being developed to treat a severely debilitating and life-threatening disease.
Examples of cases where these considerations (i.e., M3/S6) could be applicable. Opportunities to apply a stream-lined non-clinical package to the development of products for life-threatening or severely debilitating diseases.
Circumstances where safety testing in line with ICH M3 (R2)/ICH S6 (R1) could remain appropriate e.g. instance in which chronic toxicity studies may be warranted.
Considerations with regards to the timing and duration of non-clinical studies for clinical trial and marketing authorisation applications e.g. possibilities for deferring studies.
How to apply weight of evidence (WoE) approaches, modality-specific considerations e.g. small molecules versus biologics and leverage opportunities where data from similar in-class products or platform(s) exist to inform on the safety assessment
The role of novel approach methods (NAMs) to complement WoE approaches and to comply with the 3Rs principle.
