Patient-Focused Policy Positions

Activity Types

• Requests for Information (RFI) - Responses to congressional stakeholders seeking background information on cell and gene therapy (CGT) development, patient access, and related topics. These inform legislative and policy decisions.
• FDA Comments - Formal responses to FDA draft guidance documents, highlighting positive change and suggesting specific recommendations to ensure regulations effectively serve CGT stakeholders.
• Congressional Testimony - Oral and written statements delivered to Congress regarding proposed legislation affecting the CGT field. Includes expert presentations, Q&A from Members of Congress, and detailed written submissions prepared in advance.
• Spotlight Article- Peer-reviewed documents outlining ASGCT's stance and recommendations on key CGT issues. These provide background analysis and propose solutions based on expert input.

Clinical Trials and Research

Congressional Testimony: Legislative Proposals to Support Patients with Rare Diseases

• Key Takeaways: This information empowers patients and advocates to push for changes that accelerate access to new treatments.
• Summary: ASGCT’s Vice President discusses the exciting pipeline of new therapies and the hurdles that delay patient access, such as complex clinical trials, regulatory barriers, and insurance coverage issues. We advocate for ongoing research funding, more flexible regulations, and policies that ensure insurance covers these therapies as soon as they are FDA-approved.

FDA Comment: Diversity Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Trials

• Key Takeaways: These comments promote greater access to clinical trials and therapies for underrepresented groups, an important goal for patients and advocates.
• Summary: ASGCT supports diversity in clinical trials to ensure all patient populations benefit from new therapies. Comments on the FDA’s diversity plans emphasize setting appropriate diversity goals, reducing participation barriers, and ensuring racial and ethnic categories are relevant.

FDA Comment: Ethical Considerations for Clinical Investigations of Medical Products Involving Children

• Key Takeaways: Patients and advocates may find these comments helpful in understanding the issues facing pediatric trials.
• Summary: These regulatory comments focus on the need for robust informed consent processes and sufficient data to assess risks and benefits for children in clinical trials. This ensures that children have access to potentially life-saving therapies, while maintaining ethical standards and patient-focused development.

Request for Information: National Center for Advancing Translational Science Strategic Plan

• Key Takeaways: Patients and advocates may find these comments helpful in understanding the challenges in developing cell and gene therapies for rare diseases.
• Summary: ASGCT responded to NCATS’ draft strategic plan for 2024-2027, emphasizing the use of real-world data and natural history studies for CGT development in rare diseases. They called for shorter diagnostic journeys, increased clinical trial diversity, and strategies to accelerate CGT delivery through innovative trials and public-private partnerships.

Access and Patient Support

Request For Information: Facilitating the Early Diagnosis and Equitable Delivery of Gene-Targeted Therapies

• Key Takeaways: Patients and advocates can use this information to push for policies that improve early diagnosis and access to advanced treatments.
• Summary: ASGCT’s response to this RFI highlights ways to ensure timely and equitable access to gene-targeted therapies. Comments support initiatives like newborn screening for early diagnosis, efforts to increase clinical trial diversity, innovative payment models to improve access, and enhanced collaboration among stakeholders.

Coverage and Reimbursement

Request For Information: Employee Retirement Income Security Act

• Key Takeaways: Patients and advocates can use this information to understand how employer-sponsored insurance can better support access to advanced therapies.
• Summary: ASGCT recognizes the challenges employers face in providing coverage for high-cost cell and gene therapies. The response to this RFI highlights the need for innovative payment models, such as value-based arrangements, to make these therapies more accessible. Comments also emphasize the importance of transparency in coverage policies to avoid delays in patient access.

Request For Information: Improving Americans’ Access to Gene Therapies

• Key Takeaways: This is crucial information for patients and advocates aiming to improve public health insurance programs to ensure everyone can benefit from CGTs.
• Summary: ASGCT is working to eliminate the barriers that prevent patients on Medicaid and Medicare from accessing gene therapies. The response to this RFI shares findings on inconsistent state Medicaid policies and calls for efforts to align coverage with FDA-approved uses, support value-based payment models, and speed up access at the federal level.

Spotlight Article: Medicaid Coverage Practices for Approved Gene and Cell Therapies

• Key Takeaways: This paper is a valuable resource for patients and advocates seeking to understand and improve Medicaid coverage for advanced therapies, and to use as evidence of these cross-state issues.
• Summary: ASGCT’s position paper outlines the difficulties state Medicaid programs face in covering expensive gene and cell therapies and suggests federal solutions to ensure full coverage. They highlight how some state policies restrict access to these potentially curative therapies and propose ways to ensure all patients can get the treatments they need.

Regulatory Reform

Request For Information: Scientific Challenges and Opportunities to Advance the Development of Individualized Cellular and Gene Therapies

• Key Takeaways: Patients and advocates can use this information for more comprehensive advocacy around personalized medicine.
• Summary: This is a broad response covering clinical and nonclinical barriers to development, challenges with limited patient populations for rare disease. Comments advocate for alternative approval pathways and regulatory flexibility in data interpretation.

Letter to Congress: 21^st Century Cures 2.0 [introduced]

• Key Takeaways: This is an important read for patients and advocates who want to support legislative efforts that accelerate the development and availability of new therapies.
• Summary: This is a comprehensive comment letter that covers many CGT issues. ASGCT provides recommendations to Congress on increasing clinical trial diversity, improving FDA’s review capacity for cell and gene therapies, leveraging real-world evidence, and enhancing coordination between FDA and CMS.

Request for Information: Enacted Cures 2.0 Act

• Key Takeaways: This RFI response is a good overview of many legislative and regulatory issues, and presents a snapshot of the current policy landscape for biomedical research and patient care.
• Summary: ASGCT comments support ongoing legislative and regulatory efforts to enhance clinical trial diversity, streamline post-market surveillance, and improve Medicaid coverage consistency for CGTs. The response also highlights the need for flexible manufacturing guidelines, enhanced FDA-CMS coordination, and novel payment models to reduce patient access barriers. This advocates for additional federal guidance, structural reforms, and support mechanisms to further these goals, ensuring that CGTs reach the patients who need them most.

Manufacturing, Quality, and Data

FDA Comment: Real World Data: Assessing Registries to Support Regulatory Decision-Making Guidance

• Key Takeaways: This information helps patients and advocates understand how real-world evidence can speed up the availability of safe and effective new therapies.
• Summary: ASGCT works to ensure that real-world data is of high quality and representative enough to be used in regulatory decisions, which can increase patient access to therapies. Their comments on this FDA guidance highlight the importance of robust data to support the approval of new treatments.