The Advocate

Volume 3, Issue 1


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January 2023

In This Issue:

Read Our Comments to FDA on Ethical Considerations for Pediatric Subjects
Omnibus Spending Package Passes with ASGCT-Supported Provisions
Apply to Join a Committee Through Jan. 31
Events & Opportunities
Policy News

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ASGCT Advocacy


Read Our Comments to FDA on Ethical Considerations for Pediatric Subjects

ASGCT submitted comments in December to FDA on the draft guidance: Ethical Considerations for Clinical Investigations of Medical Products Involving Children. ASGCT supports the protection of human subjects in FDA-regulated clinical investigations through adherence to current clinical trial regulations and robust Institutional Review Board (IRB) processes and commended practices that ensure vigorous safeguards for children involved in clinical research.

Pediatric research principles, including the ethical questions addressed in the guidance, are of particular relevance for product sponsors, academic researchers, and interested patient groups currently engaged in bringing the next generation of current gene and cell therapy products to market given the pipeline’s focus on pediatric indications. The cell and gene therapy pipeline offers a unique opportunity to address pediatric diseases, many of which do not have available treatment options. In comments, ASGCT advocated for:

  • Pediatric non-clinical data sources to be used when adult data is not available or otherwise unethical

  • Additional safeguards around informed consent, including unique mechanisms of action, potentially durable treatment effect, uncertain oncogenic risk profile, and the ethics of control arms in rapidly degenerative diseases

  • IRBs to consider the innate differences between CGT and small molecule products when offering guidance on pediatric risk-benefit considerations

The Society is committed to the safety and protection of pediatric and adult patients in clinical trials and will continue to engage on these issues as additional regulatory opportunities arise.

Omnibus Spending Package Passes with ASGCT-Supported Provisions

Last month Congress passed a $1.7 trillion government funding package for the 2023 Fiscal Year (FY23) which included many of the PDUFA VII provisions left on the table after the continuing budget resolutions passed in the fall. Throughout this legislative cycle, ASGCT has been active in supporting the efforts of our members in a variety of ways. See our recent blog post for full details on the individual provisions and past ASGCT comments included in the bill.

Broadly, the bill includes robustly increased funding for FDA, the National Institutes of Health (NIH), and the Advanced Research Projects Agency for Health (ARPA-H). Previously ASGCT  joined other leading healthcare organizations asking Congressional leadership to complete the FY23 appropriations process promptly, and the Society is pleased that they have now done so.

Non-funding provisions included reforms to the accelerated approval pathway, which notably did not incorporate product labeling requirements, though it did include changes to confirmatory trial and post-approval study timelines. Clinical trial design provisions included a new requirement for diversity plans prior to entering phase III studies, with a forthcoming guidance on this policy to be released this year. The bill also contains the creation of a new review pathway for advanced manufacturing technologies to more efficiently approve advanced therapies, and calls for the creation of a Platform Technology Designation Program, under which platform technologies would receive additional support from the Agency for products that use a single technology across multiple products. These new regulatory offerings will help streamline the review process and help support innovating cell and gene therapy development.

For more information on these individual points, reference our website to read previous regulatory comments, sign-on letters, and blog posts on previous accelerated approval bills, user fee reauthorization, the FY23 omnibus, and more!

Apply to Join a Committee Through Jan. 31

Applications are now open through Jan. 31 for the 2023 committee cycle for anyone interested in getting more involved with ASGCT! Our volunteer committee members serve as subject matter experts on topics spanning hematology, immune response, global outreach, ethics, government relations, and more, on 33 standing and scientific committees. Committee appointees must be ASGCT members and submit a short excerpt on their relevant experience. More information on the appointment process and committee expectations can be found here.

Events & Opportunities


Save the Date for the 2023 Policy Summit!

ASGCT’s fifth annual Policy Summit will take place September 18-19 at the Westin Washington, DC Downtown in a hybrid format – attendees will be able to join either in person or online. The program will feature presentations and panels diving into the key issues in regulatory and government affairs policy for gene and cell therapies. Information on the agenda and registration is coming soon! In the meantime, catch up with recaps of previous Policy Summits.

Register Now for the 26th Annual Meeting

Register now for the ASGCT 26th Annual Meeting which will be held as a hybrid event May 16 – 20. You can book your hotel during the registration process. Keynote speakers include Jennifer Doudna, David Liu, Crystal Mackall, and Stanley Riddell. More information on our agenda and speakers are coming soon – watch this newsletter for information about our regulatory and policy sessions. We hope to see you in Los Angeles or online!

Next Week: Free Immune Responses to AAV Vectors Workshop

As part of FDA’s efforts to improve communications with sponsors, ASGCT is partnering with the Office of Tissues and Advanced Therapies (OTAT) for a two-day workshop, Immune Responses to AAV Vectors, Jan. 24-25 from 10 a.m. – 2 p.m. (CT) each day. Registration is free and open to the public.

Attend NASEM-FDA Workshop on Accelerated Approval

The National Academies of Sciences, Engineering, and Medicine (NASEM) will be holding a two-day virtual workshop with FDA on the accelerated approval process for new pharmaceuticals, taking place Jan. 30-31. Check out the full agenda listing speakers and daily topics.

Join OTAT Town Hall on Gene Therapy for Rare Disease

On Feb. 7, OTAT is hosting a virtual town hall to answer stakeholder questions related to the clinical development of gene therapy products for rare diseases (excluding hematology/oncology products). Register for the event here.

Policy News


  • The 118th Congress was sworn in this month, and ASGCT will be on the Hill educating elected representatives and their staff, as well as sharing the work our members are doing to advance the science of genetic therapies.

  • Research funding may be negatively impacted by newly adopted spending rules in the House of Representatives.

  • HHS, FDA, CMS, and NIH will begin working on promised reforms in food, drug, and public health departments following product bottlenecks and regulatory uncertainty this past year. AGSCT will be watching as omnibus provisions are implemented, NIH leadership is finalized, and Medicaid expansion moves forward.

  • Transcripts are now available from the Patient-Focused Drug Development Listening Meeting — Patient Perspectives on Gene Therapy Products hosted by CBER this past November.

  • FDA Commissioner Robert Califf emphasized the need for extended, product- and disease-appropriate long-term follow-up requirements for gene therapies at the recent JP Morgan Chase conference. Califf also noted that the need for patient follow up should not be a deterrent from new, accelerated review pathways for these therapies.

  • The Center for Medicare and Medicaid Innovation (CMMI) has released their 2022 Report to Congress. This annual report assesses the impact of new rulemaking on patients and the public payor system, and quantifies spending and quality under Medicare and Medicaid programs. 

2023

26th Annual Meeting

May 16-20 | Los Angeles, CA

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