What we expect to be talking about in August and September
- The end of the fiscal year (and a budget showdown or shutdown): The last day of the government’s fiscal year is September 30, which will mark an especially busy period for the FDA. The agency has many deadlines associated with the end of the fiscal year (and in particular those associated with its various user fee programs). In addition, FDA’s Congressionally appropriated funding runs out at midnight on September 30, requiring Congress to pass a new budget. For various reasons, that currently seems unlikely, but it is perhaps more likely that Congress will pass a short-term continuing resolution to fund the federal government for a few months, as it often does.
- Happy 40th Birthday, generic drugs: September 24 will mark the 40th anniversary of the 1984 signing of the Hatch-Waxman Act, which created a formal pathway for the approval of generic drugs. While generic drugs technically pre-dated the law (look up “Paper NDAs” if you’re interested), the law turbocharged the ability of generics to come to market. We expect the FDA to mark the occasion, especially since drug pricing continues to be such a potent issue in the Presidential election.
- Rare Pediatric Disease Priority Review Voucher Program Expiration Threat: The FDA’s popular voucher program for rare pediatric diseases is currently scheduled to expire as of September 30, 2024 unless it is renewed by Congress. While legislators have expressed interest in doing so, there are very few days between now and the end of September when Congress will be in session. That raises the chances that there will be at least a short-term lapse in the program. Luckily, that won’t prevent any companies that have already been declared eligible for a voucher (i.e., companies with pending applications for approval) from being granted one.
- Good Guidance Practices Report: We had been expecting to receive a major and interesting report on the FDA’s Good Guidance Practices FDA by June 29. An earlier iteration of that report detailed how the agency wanted to change some of its practices surrounding the publication of guidance documents. However, the draft document was somewhat FDA-focused, and industry asked the agency to incorporate other ideas into the final version. However, while FDA missed it statutory deadline to release the report, we’re still expecting it soon.
- The Summer Slowdown: For regulatory professionals, August is an especially slow period. There are relatively few meetings scheduled for August, and Congress will mostly be in recess until just after Labor Day.
Things FDA expects to do in August and September
This list is comprised of specific actions and the dates by which FDA has said it plans to accomplish them.
| Date | What’s Happening | Explanation | Source |
| September 24 | Anniversary of the signing of the Hatch-Waxman Act | The Drug Price Competition and Patent Term Restoration Act, better known as the Hatch-Waxman Act, turns 40 in September. The law created the entire generic drug pathway, and we expect that the FDA (and Congress) will do something to mark the occasion. | Wikipedia |
| September 30 | Rare Pediatric Disease Priority Review Voucher program expires unless renewed | The Rare Pediatric Disease Priority Review Voucher Program is set to expire at the end of the fiscal year unless Congress extends it. While this would not affect existing vouchers, it would prevent FDA from issuing new ones after September 2026. | FDA |
| September 30 | Deadline for submission of Advancing RWE Pilot Program projects | A semi-annual deadline for FDA’s Advancing Real-World Evidence Program is set for the end of the fiscal year. | FDA |
Regulations and guidance under OIRA review as of August
The White House’s Office of Information and Regulatory Affairs (OIRA) is the regulator of regulators, tasked with ensuring that all federal policies and regulations adhere to laws, existing regulations, federal policies and the wishes of the President. This is what OIRA is currently reviewing. (Note: If a link no longer works, it is likely because OIRA has since cleared the document.)
| Title | Type | Date Received by OIRA | Legal Deadline |
| Expedited Program for Serious Conditions –Accelerated Approval of Drugs and Biologics | Draft Guidance | July 9 | Yes |
| Conducting Clinical Trials With Decentralized Elements | Draft Guidance | July 25 | Yes |
| Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice | Draft Guidance | July 25 | None |
Regulations and guidance awaiting immediate publication in August
The following documents have cleared review by the White House’s Office of Information and Regulatory Affairs (OIRA) and may be released by the FDA at any time. (Note: If a link no longer works, it is likely because OIRA has since deleted it.)
| Title | Type | Date Cleared by OIRA | Legal Deadline |
| None |
Notable FDA Comment Periods Closing in August and September
FDA comment periods are typically open for 30-60 days, unless they are extended.
Meetings that FDA has planned for August and September
These are FDA-hosted events set to take place over the next two months.
Third-party regulatory meetings being held in August and September
These are notable industry events related to regulation, including those at which FDA speakers are confirmed or likely to speak.
| Date | Group | Event | Notable FDA Speakers |
| August 5 | Sentinel | Overview of CDER’s Real-World Evidence Demonstration Projects | |
| August 12-14 | Pharma Conference Education | GMP By The Sea | Peter Marks, Emily Thakur, Alonzo Cruse, Numerous others |
| August 20 | Duke-Margolis | Continual Improvement of CDER BLA Submission, Assessment, and Facility Readiness/ Inspection: CMC for Biologics & Biosimilars | |
| August 22 | HL7 | REMS Public Meeting | |
| September 4 | Reagan-Udall Foundation for the FDA | Opportunities for Global Regulatory Convergence | |
| September 4 | TransCelerate | Webinar: In Comparable Terms: Driving Efficiencies to Simplify the Ability to Detect Class Effects in Automated Manner through the Use of SEND | Stephanie Leuenroth-Quinn |
| September 6 | UMaryland | ADEPT-9: Public Workshop on Enhancing Diversity in Therapeutics Development for Pediatric Patients | Hilary Marston, Mathilda Fienkeng, Christine Lee, Ann McMahon, Dionna Green, Carla Epps, Lynne Yao |
| September 10-11 | MDIC | Annual Public Forum | Suzanne Schwartz, Robert Califf, Numerous others |
| September 8-11 | Healthcare Distribution Alliance | 2024 Annual Board and Membership Meeting | |
| September 9-11 | Critical Path Institute | C-Path 2024 | Peter Stein, Michelle Campbell, |
| September 17-18 | CHPA | Regulatory, Scientific & Quality (RSQ) Conference | Robert Califf, Angela Granum, Theresa Michele, David Strauss, Peter Stein, Cara Welch, Shontell Wright |
| September 17-19 | RAPS | RAPS Convergence 2024 | Numerous; AgencyIQ |
| September 19-20 | 3D Symposium | Dartmouth Device Development Symposium | TBD |
| September 19-20 | Pharma and Biopharma Outsourcing Association | PBOA Annual Meeting and Conference | Jacqueline Corrigan-Curay, Alonza Cruse, Elizabeth M. Kelley |
| September 20 | MDMA | 2024 MDMA Medical Technology Executive Forum Agenda | Michelle Tarver |
| September 23-24 | ASGCT | ASGCT Policy Summit | Nicole Verdun |
| September 26 | United Nations | UN General Assembly High-Level Meeting on antimicrobial resistance 2024 | |
| September 26-27 | Global Genes | 2024 RARE Advocacy Summit | |
| September 26-27 | Critical Path Institute | Complex In Vitro Model: Qualification Framework Public Workshop | |
| September 27-30 | Heart Failure Society of America | Annual Scientific Meeting 2024 | |
| September 27-29 | SOCRA | 2024 Annual Conference |
PDUFA Dates expected in August and September
PDUFA dates represent the expected date of a regulatory decision by the FDA on a New Drug Application or Biologics License Application. The following PDUFA dates were obtained from publicly available sources.
| Date | Company | Drug | Indication |
| August 4 | Adaptimmune | Afami-cel | Treatment of Advanced Synovial Sarcoma |
| August 10 | Humacyte | Human Acellular Vessel | Treatment of Vascular Trauma |
| August 13 | Citius Pharma | LYMPHIR | Treatment of patients with persistent or recurrent CTCL |
| August 14 | Ascendis Pharma | TransCon PTH | Adults with hypoparathyroidism |
| August 14 | Gilead Sciences | Seladelpar | Treatment of Primary Biliary Cholangitis |
| August 20 | Servier | Vorasidenib | Treatment of IDH-mutant diffuse glioma |
| August 22 | Regeneron | Linvoseltamab | Relapsed/Refractory Multiple Myeloma |
| August 28 | Incyte, Syndax Pharmaceuticals | Axatilimab | cGVHD |
| September 5 | Travere Therapeutics | FILSPARI | Conversion from Accelerated Approval to Full Approval |
| September 7 | Avadel Pharmaceuticals | LUMRYZ | Treatment of pediatric narcolepsy |
| September 18 | Vanda Pharmaceuticals | Tradipitant | Treatment of Gastroparesis |
| September 21 | Zevra Therapeutics | Arimoclomol | Treatment for Niemann-Pick Disease Type C |
| September 25 | Merck | Keytruda | First-line treatment of patients with unresectable advanced or metastatic malignant pleural mesothelioma |
| September 26 | Syndax | Revumenib | Treatment of Relapsed/Refractory KMT2Ar Acute Leukemia |
| September 26 | Karuna/BMS | Xanomeline-trospium | Treatment of schizophrenia in adults |
| September 27 | Sanofi | Sarclisa | Treatment of patients with transplant-ineligible newly diagnosed multiple myeloma (NDMM) |
User Fee Deliverables expected in August and September
FDA has generally been meeting its commitments under its various new user fee programs, and typically weeks or months ahead of schedule. The following commitments are due within the next two months, minus any commitments the FDA has already met (to our knowledge).
| Letter | Program Tag | Commitment | Due Date |
| MDUFA | International Harmonization | Commencing with FY 2024, publish an annual assessment of the international harmonization activities described the strategic plan due by the end of FY 2023. | September 30, 2024 |
| PDUFA | Novel Approaches to Development of Cell and Gene Therapy | Convene a public meeting to solicit input on methods and approaches (e.g., use of RWE, registries) for capturing post-approval safety and efficacy data for cell and gene therapy products. | September 30, 2024 |
| PDUFA | Novel Approaches to Development of Cell and Gene Therapy | Issue a Q&A draft guidance based on frequently asked questions, and commonly faced-issues identified by sponsors or by public-private partnerships | September 30, 2024 |
| PDUFA | Enhancing the Incorporation of the Patient’s Voice in Drug Development and Decision-Making | Workshop 1, Enhancing the Incorporation of the Patient’s Voice in Drug Development and Decision-Making | September 30, 2024 |
| PDUFA | Advancing Model-Informed Drug Development | Issue a Request for Information (RFI) to elicit public input for identifying priority focus areas for future policy or guidance development and stakeholder engagement | September 30, 2024 |
| PDUFA | Advancing Utilization and Implementation of Innovative Manufacturing | Issue a draft strategy document for public comment that outlines the specific actions the agency will take over the course of PDUFA VII to facilitate the utilization of innovative manufacturing technologies, including addressing barriers to their adoption | September 30, 2024 |
| MDUFA | Patient Science and Engagement | By the end of FY 2024, hold a public meeting to explore ways to use patient-generated health data to help advance remote clinical trial data collection and support clinical outcome assessments. | September 30, 2024 |
Upcoming (or overdue) legislative requirements due as of August and September
Congress often asks the FDA to release or hold guidance documents, regulation, reports, meetings, hearings or pilot programs as of specific dates. Many of these requirements will be met weeks or months before the actual due date. The following legislative requirements are due within the next two months:
| Legislation | Requirement | Due Date |
| FDORA Section 3606 | Decentralized clinical studies: FDA must issue final guidance on decentralized clinical studies no later than one year after the close of the public comment period for draft guidance. | August 1, 2024 |
| FDORA, Section 2512 | Shortages: FDA must review its policies related to drug or biologic expiration dates and issue draft guidance or revise existing guidance on stability testing data in drug and biological product submissions. The guidance will include FDORA, Section recommendations FDORA, Section on the inclusion of the “the longest feasible expiration date supported by such data” in a drug’s label. | 12/29/2023 |
| FDORA, Section 3202 | Rare Diseases: GAO to release a report to Congress assessing the policies, practices, and programs of the FDA with respect to the review of applications for approval of drugs and biologics intended to treat rare disease, with a focus on the effectiveness of FDA’s policies and challenges encountered by sponsors. | 6/29/2024 |
| FDORA, Section 3210 | Accelerated approval: FDA must publish draft guidance on how sponsor questions about novel surrogate or intermediate endpoints can be answered earlier in the development process, as well as other factors related to the accelerated approval process. | 6/29/2024 |
Overdue and pending regulatory actions
This list, based on the federal government’s Unified Agenda, contains all the regulations that FDA has said it intended to release, or plans to release in the coming months. In our experience, the FDA does not meet its own intended deadlines for the release of these actions in more than half of all cases (sometimes due to delays at the White House reviewing regulations). We’ve also included documents that are actively under White House review – a process which often takes several weeks (or months) to conclude. [ Read the full list of documents on FDA’s Unified Agenda here.]
To contact the author of this piece, please email Alec Gaffney (agaffney@agencyiq.com)