The Advocate

September 2024: Volume 4, Issue 9

In This Issue:

The Policy Summit Starts Monday!
Letters to CMS Address Payment Policy Considerations
Society Calls for Streamlined Platform Technology Designation Process
Community Quick Takes: Genetic Testing’s Role in Clinical Trial Access
Government Funding to Be Determined as Sept. 30 Deadline Approaches
Congressional RFI Seeks Feedback on Biomedical Supply Chains
Events
Policy News

ASGCT Advocacy

The Policy Summit Starts Monday!

ASGCT's Policy Summit starts this Monday, Sept. 23, in Washington, D.C. and online. You can still register and view the full speaker lineup. The Summit presents a unique opportunity to advance science-based, patient-focused policymaking for cell and gene therapies (CGTs). Featured sessions include:

Past and Present Regulatory Perspectives
Understanding the nuances of regulatory strategy is key to advancing CGT products toward commercialization. At the Policy Summit, attendees will gain invaluable insights through sessions featuring current and former regulatory leaders. From global submission strategy to FDA policy updates under PDUFA VII, these talks offer a wide-ranging view of the regulatory landscape. Participants will learn about international harmonization efforts, evolving guidelines, and practical strategies for navigating challenges. By exploring case studies and current trends, attendees will develop a deeper understanding of how regulatory considerations shape CGT development.

Payment Policy and Access
Ensuring patient access to innovative CGTs requires navigating the complex landscape of payment and reimbursement policy. Sessions at the Policy Summit will provide a detailed overview of current and future payment policy discussions, including public and private payer coverage for CGTs. Attendees will learn about value-based arrangements, state Medicaid approaches to coverage, and the impact of federal policy decisions on patient access. Attendees will come away with a better understanding of the financial and policy dynamics affecting CGT products following FDA approval.

Hot Topics in Clinical Development
Following the current trends and best practices in clinical development is vital for advancing CGTs. This session will provide a deep dive into the risk-benefit analysis of CGTs, focusing on the use of surrogate endpoints, biomarkers, patient experience data, and the challenges of novel technologies. It’s an invaluable opportunity for professionals to stay updated on the latest developments in clinical trials.

Join your colleagues for these critical discussions, and enhanced networking opportunities, in Washington, D.C.

Letters to CMS Address Payment Policy Considerations

As part of ASGCT’s ongoing efforts to improve patient access to CGTs, the Society recently submitted comment letters to the Centers for Medicare & Medicaid Services (CMS). These letters address proposed payment policies for 2025 in both the Medicare Physician Fee Schedule and the Hospital Outpatient Prospective Payment System (OPPS).

In comments on a potential change to the Physician Fee Schedule, ASGCT voiced support for CMS's proposal to establish separate payments for four new CPT codes related to CAR-T cell therapy administration. This proposal recognizes the complex, multi-step process involved in administering these innovative therapies. Comments also encouraged CMS to carefully consider feedback from clinicians when finalizing the work Relative Value Units (RVUs) and Practice Expense values for these codes, ensuring that the final values accurately reflect true costs and unique characteristics of CGTs.

Regarding OPPS, ASGCT strongly endorsed CMS's proposal to exclude qualifying CGTs from Comprehensive Ambulatory Payment Classification (C-APC) packaging. This exclusion is crucial, as it acknowledges that CGTs are not merely ancillary to other services but are often the primary treatment themselves. Comments urged CMS to extend this exclusion policy indefinitely beyond 2025, recognizing the unique nature of these therapies and the need for payment methodologies that don't unfairly penalize providers offering these services. ASGCT also joined the CAR T Working Group’s sign on to reinforce our key asks.

By engaging directly with CMS on these critical issues, we aim to shape a reimbursement landscape that supports the continued development and availability of these therapies. For those interested in diving deeper into these issues, we encourage you to visit our website for a complete list of our policy positions and to read the full text of our comment letters.

Society Calls for Streamlined Platform Technology Designation Process

ASGCT recently submitted comments to FDA on the Platform Technologies Designation Program for Drug Development draft guidance. The Society is been supportive of platform regulatory approaches to streamline access and approval for CGTs. However, the current draft does not fully embrace the spirit of the program as set forth by Congress, and could be improved to better serve the CGT field. ASGCT’s comments stressed the need for:

Streamlining post-approval changes: ASGCT urges the FDA to embrace the spirit of the law and allow for a more streamlined process for post-approval changes to platforms across products. They argue that the current draft guidance seems to require multiple individual applications rather than a single, streamlined application for changes across all products using the designated platform.
Issues with the Biologics License Applications (BLA) and Master Files rule: ASGCT criticizes the requirement that all information on the platform must be submitted with each BLA and cannot cite a Drug Master File (DMF). They argue this diminishes the value of the designation and keeps the regulatory burden high.
Lack of guidance for companies without approved products: ASGCT notes that many cell and gene therapy (CGT) developers are small companies or academic institutions without approved products. They request more guidance on how these entities can leverage the principles of Designated Platform Technologies in their initial programs.
Resubmission of platform information: ASGCT suggests that it should not be necessary to resubmit platform information for follow-on products if the platform has already been designated. They argue this requirement reduces potential regulatory efficiencies.
Clarification on "well-understood" technology and “sufficient efficiencies”: ASGCT requests clarification on how the FDA will interpret the statutory definition of "well-understood" technology, as it's crucial for determining eligibility. The Society also asked for examples of how “sufficient efficiencies” will be measured.

Platform technologies can accelerate the development of CGTs by providing standardized, scalable methods for key processes such as gene editing, cell culture, and vector production. To read ASGCT’s past comments across FDA guidances, be sure to check out all our policy positions online.

Community Quick Takes: Genetic Testing’s Role in Clinical Trial Access

The recent ASGCT Community Quick Takes module on genetic testing and clinical trial eligibility provides crucial insights for patients and families affected by genetic disorders such as Duchenne muscular dystrophy (DMD), giant axonal neuropathy (GAN), and Angelman Syndrome. Experts discuss how specific genetic variants influence trial eligibility, and how immune modulation strategies can be tailored based on genetic profiles to enhance the safety and effectiveness of gene therapies.

We encourage you to share this information among your networks, along with our past takes on informed consent, recent CGT approvals, and patient access

Government Funding to Be Determined as Sept. 30 Deadline Approaches

With Congress back in session, lawmakers are working to finalize FY2025 appropriations for major agencies before the September 30 fiscal year deadline. Key bills currently moving through the House and Senate include the Labor-HHS bill, which covers critical funding for the National Institutes of Health (NIH), and the Agriculture-FDA bill, which includes FDA funding. Both bills face hurdles as lawmakers will attempt to reconcile differences between the House and Senate proposals.

Negotiations are underway for a continuing resolution (CR) to keep the government funded past November’s election, as it is unlikely at this stage that Congress will pass all of its 12 appropriations bills. A CR would temporarily extend FY2024 funding levels into the next fiscal year; if no agreement is reached, a government shutdown is a possibility.

Congressional RFI Seeks Feedback on Biomedical Supply Chains

Representatives Wenstrup, Moore, Pfluger, and Green have issued a Request for Information seeking input on strengthening domestic medical supply chains in the United States. This initiative aims to reduce dependence on foreign adversaries like China and Russia for critical medical supplies and manufacturing. The RFI, open until October 4, 2024, invites feedback from experts, stakeholders, and industry leaders on various aspects of medical supply chain security. Topics include economic obstacles to onshoring, lessons from supply chain diversification efforts, priority areas for onshoring or friendshoring, regulatory barriers, workforce challenges, and potential policy solutions.

Events

Register for CBER Patient and Care Partner Listening Meetings

FDA is holding listening sessions to engage with patients and caregivers on their experiences, perspectives, and informational needs regarding the short-term and long-term risks of approved gene therapies. These meetings will help inform patient-centered protocols for long-term post-market studies of these transformative treatments. Registration is open for the first meeting on Sept. 20; the second meeting details are TBD.

This Fall: Inaugural Events on Muscular Dystrophy and Cancer Innovation

Don't miss out on two exciting ASGCT events this fall! Explore and dig into advancements in muscular dystrophy and cancer with these new hybrid events. Register today to join leading experts and stay ahead in the field with ASGCT:

Advancing Gene and Cell Therapies for Cancer: Oct. 16-17 | Philadelphia + Virtually

Breakthroughs in Muscular Dystrophy: Nov. 19-20 | Chicago + Virtually

NORD Breakthrough Summit to Focus on Inclusion and Access

The National Organization for Rare Disorders (NORD) is hosting their hybrid Rare Disease and Orphan Products Breakthrough Summit, in Washington, D.C. Oct. 20-22. With a theme of “Equitable Access to Innovation,” this event will feature thought-provoking discussions and actionable strategies on the latest advances in clinical trials, gene therapy, medical devices, AI and patient-focused drug development with an underlying focus on inclusion and equal access for all. Review the full agenda on their website and register!

Register for NCATS Director's Strategic Plan Roundtable

Join NCATS Director Dr. Joni Rutter in a roundtable discussion on Sept. 30. This event is an opportunity for our community to hear about the new 2025 – 2030 Strategic Plan and help NCATS as they translate the plan into action. Registration is now open.

Save the Date for Empowering Patients 2025: A Cell and Gene Therapies Summit

Coming next spring: A new, free virtual event hosted by ASGCT on March 12-13, 2025. This event will be two half-days of educational material geared toward foundation leaders, patients, and caregivers to provide timely information to support their CGT journies. It will feature educational sessions and engaging discussions on the CGT landscape, exploring the unique challenges impacting patient communities at various stages of the CGT pipeline. Registration for this event will open later this fall.

FDA-Partnered Workshop Will Cover AAV Class Considerations

ASGCT and FDA will host a virtual scientific workshop over two half days on January 22 and 23, 2025. This free, public event will explore key immunology-related topics that have implications across the class of AAV gene therapies, including immune responses, antibody testing, and redosing strategies. The workshop will provide valuable insights for professionals in both academia and industry as it addresses pressing challenges in AAV gene therapies and new approaches. Registration for this event will open in early November.

Policy News

A new glossary of cell and gene therapy terms was released on the ASGCT Patient Education site. Freely available online, it aims to guide individuals through complex CGT topics and increase consistency of messaging these concepts to patients and the public.
FDA’s Office of Therapeutic Products (OTP) released a new series of webpages on available meeting types and interactions with OTP. This website reorganization is intended to provide a more streamlined resource for sponsors.
Expert Volunteer Opportunities:
- The National Academies provided new recommendations to the FDA and Congress on the topic of rare disease regulatory policy. The report was called for by Congress in a 2022 law, and addresses topics such as global alignment on evidence-based approaches and the inclusion of pediatric population.
- Contribute your expertise and collaborate with global scientific leaders to shape quality standards and solutions that safeguard public health by applying to become an Expert Volunteer with the United States Pharmacopeia (USP). 
The National Center for Advancing Translational Sciences (NCATS) released its 2025-2030 strategic plan. NCATS Director Joni Rutter will host a webinar on the new strategic plan on September 30.
The BIOSECURE Act, aimed at decoupling American biotechnology and biomedical research from foreign manufacturing threats, passed the House of Representatives. The bill also focuses on improving regulatory processes and providing additional funding for U.S. biotech and research initiatives.
The National Academies provided new recommendations to the FDA and Congress on the topic of rare disease regulatory policy. The report was called for by Congress in a 2022 law, and addresses topics such as global alignment on evidence-based approaches and the inclusion of pediatric population.