The Advocate

June 2024: Volume 4, Issue 6

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In This Issue:

Read Comments on FY2025 IPPS Proposed Rule
Society Responds to NCATS Strategic Plan
FDA Releases Guidance on Platform Technology Designation Program 
ASGCT Members Share Expertise With House Budget Committee 
Society Supports Comprehensive Care for Sickle Cell Disease  
RFI from NIH Seeks Input on New Policies to Support Commercialization  
FDA Rare Disease Pilot Selects CGT Participants  
Policy News


ASGCT Advocacy

Read Comments on FY2025 IPPS Proposed Rule

ASGCT provided comments to the Centers for Medicare and Medicaid Services (CMS) on the FY2025 Inpatient Prospective Payment Systems (IPPS) Proposed Rule. The Society voiced support for CMS's proposal to increase the New Technology Add-on Payment (NTAP) percentage from 65% to 75% for gene therapies treating sickle cell disease to improve patient access. 

However, ASGCT expressed ongoing concerns about policy changes that could restrict NTAP eligibility for future cell and gene therapies. The Society urged CMS to establish multiple NTAP review periods per year, increase the NTAP cap to 100% of costs, and maintain flexibility on criteria for creating new MS-DRGs for rare disease products.  

The letter highlighted the importance of predictable, innovative payment models to support adoption of transformative cell and gene therapies. ASGCT has also joined with other patient advocacy organizations to voice similar collective concerns.  


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Society Responds to NCATS Strategic Plan

In keeping with the mission to advance the clinical application of genetic and cellular therapies, ASGCT recently responded to the National Center for Advancing Translational Sciences’ (NCATS)  draft strategic plan for the years 2024-2027. The Society's comments highlighted key areas where NCATS can help propel the development and delivery of transformative CGTs, accelerate rare disease development, and help promote health equity. 

Amongst other items, the plan focused on facilitating the development of treatments for rare diseases, and patients with unmet needs. ASGCT emphasized the unique challenges of CGT development and urged leveraging real-world data and natural history studies to meet regulatory data requirements and overcome issues with small sample sizes. Additionally, the Society called for initiatives to shorten the diagnostic odyssey, ensure diversity in clinical trials, and efforts to address disproportionate health impacts on minority populations. ASGCT also supported strategies to accelerate CGT delivery through platform development, innovative trial designs, and public-private partnerships.  

By providing this input, ASGCT aims to support safer and more efficient CGT development and delivery through robust translational science pathways. The Society is pleased to see NCATS’ focus on rare disease and equitable access and will continue to engage in these areas. 

FDA Releases Guidance on Platform Technology Designation Program 

At the end of 2022, Congress mandated the Food and Drug Administration (FDA) to create a new pathway for the review of drugs that are based on the same "platform" as a previously approved drug. This "Platform Technology Designation" program aims to expedite the development and review of drugs that utilize well-understood and reproducible technologies, such as nucleic acid sequences, molecular structures, delivery methods, mechanisms of action, vectors, or a combination of any such technologies that the FDA deems appropriate. 

FDA has released its draft guidance for implementing this program. The guidance outlines the eligibility criteria, potential benefits, submission process, and review timelines for sponsors seeking a platform technology designation. The draft guidance has a 60-day comment period, and ASGCT plans to submit a response. 

As noted by the Agency, there are several potential benefits for drug companies that receive a platform technology designation. These include having early and additional interactions with the FDA, as well as the ability to leverage information previously submitted in support of the designation in subsequent submissions, such as new drug or biologics license applications.  

ASGCT Members Share Expertise With House Budget Committee 

ASGCT was invited to host a briefing for the US House of Representative’s Budget Committee called Enabling Access to Cell and Gene Therapies.  

The briefing included an overview of gene and cell therapy (CGT) applications, the development pipeline, and access challenges. Presenters included ASGCT’s vice president, Matt Porteus, MD, PhD with Stanford University Medical School and vice president of ASGCT, and Rayne Rouce, MD from Baylor College of Medicine. The conversation centered around the lifetime costs savings of gene therapies compared to the standard treatment of care, and the challenges of manufacturing and delivering innovative products. 

The Society strives to find solutions to patient access challenges and inform policymakers about challenges. ASGCT published a peer reviewed white paper detailing access challenges under the Medicaid system. The full article identifies barriers and proposes federal solutions to ensure full coverage to label.   

Society Supports Comprehensive Care for Sickle Cell Disease  

National Sickle Cell Awareness Day, observed on June 19, aims to increase public knowledge and understanding of sickle cell disease, a genetic blood disorder that affects millions worldwide. This day is crucial for highlighting the challenges faced by those with the condition and the need for continued research and support. ASGCT is committed to supporting the development of innovative therapeutics for individuals affected by this condition. 

ASGCT has joined with fellow scientific societies and patient advocacy organizations in supporting the Sickle Cell Disease Comprehensive Care Act (HR 7432). This critical legislation aims to ensure that individuals with SCD have better access to comprehensive, high-quality outpatient care, including recommended clinical, mental health, ancillary, and support services, by allowing states to establish Medicaid Health Home programs with SCD as the single qualifying condition for eligibility. This bill represents a unified effort to enhance the quality of life for SCD warriors by facilitating access to the latest advancements in therapies and specialized care. As the bill makes its way through Congress, ASGCT will continue to advocate for policies that support people living with SCD. 

ASGCT hosted a series of briefings with legislators in the House and Senate last fall, with the goal of educating policymakers on the potential of CGTs to treat SCD. The Society has also been closely monitoring the rollout of CMMI’s CGT Access Model – aimed at enhancing patient access to SCD treatments.  

RFI from NIH Seeks Input on New Policies to Support Commercialization  

In May, NIH issued a Request for Information (RFI) on a proposed policy for the Intramural Research Program. The policy would require patent licensing arrangements to include plans outlining efforts to promote access to products derived from taxpayer-funded inventions, though pricing information would not be mandated. There is a 60-day public comment period on this proposal. 

In 2022 the Biden-Harris Administration released an executive order instructing agencies to consider prescription drug costs specifically. In December 2023, the National Institute of Standards and Technology (NIST) released a “Draft Interagency Guidance Framework” outlining factors for considering the exercise of march-in rights. The Bayh-Dole Act, enacted in the 1980s, grants intellectual property rights for federally funded inventions to the technology’s inventors. The legislation aimed to address the rights of individuals or other entities who generate inventions with federal financial support, then allow private entities to commercialize them. As a guardrail, the Act also enables the federal government to invoke march-in rights and compel licensing under specific conditions. The proposal outlined in NIH’s RFI does not go as far as to invoke march-in-rights, but does reflect themes related to licensing arrangements, access, and drug pricing. 

FDA Rare Disease Pilot Selects CGT Participants  

FDA has taken a significant step to facilitate rapid development of novel rare disease therapies  through the launch of the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. On May 29, FDA notified seven participants of their acceptance into the innovative initiative. The five companies who have publicly announced their participation are Denali Therapeutics, Larimar Therapeutics, Grace Science, Neurogene, and Myrtelle. The participants work on rare disease treatments, including several gene therapies, for conditions such as NGLY1 Deficiency, MPS IIIA, Friedreich's ataxia, Rett syndrome, and Canavan disease. 

Announced last September, the START program is collaboratively conducted by FDA's Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER). Those selected to participate will gain access to enhanced rapid communication channels with FDA to address critical development hurdles specific to their programs. This includes assistance on clinical trial design, control group selection, refining target patient populations, leveraging nonclinical data, and product characterization. 

For ASGCT members pioneering rare disease treatments, especially in the cell and gene therapy arena, the START program represents a valuable opportunity to utilize FDA's expertise earlier in development. This collaborative approach could help propel transformative therapies efficiently through the pipeline and into the clinic for patients.


Community Quick Take Looks at Patient Access Issues Beyond Approval 

AGSCT released a 'Community Quick Take' unit with four short talks touching on critical access issues faced by the cell and gene therapy patient community.  Experts in the field discussed the various barriers patients may encounter, such as disease eligibility, financial constraints, provider referrals, and logistical challenges within clinical settings. 

This quarterly Community Quick Take emphasizes the complex journey patients face. These barriers will require multistakeholder, collaborative solutions to ensure the right treatments are meeting the right patients in a timely manner. Be sure to watch and share these quick insights to keep up with the current access landscape for CGTs.  

Policy News

  • Reps. Larry Bucshon (R-IN) and Diana DeGette (D-CO) released an RFI seeking input on the 21st Century Cures Act, focusing on the law's current effectiveness, potential policy gaps, and needed reforms or incentives. Comments can be submitted by August 2. 

  • The Head of the European Medicines Agency, Emer Cooke, recently authored a blog post detailing how her Agency works with FDA. The article highlighted convergence opportunities and challenges ahead, with areas of cooperation spanning scientific advice, inspections, international engagement, and tackling global issues like supply chain resilience. 

  • The European Commission has finalized rules to pool expertise across the EU for assessing new drugs' effectiveness compared to existing ones, aiming to enhance collaboration and efficiency in health technology assessments. These rules outline coordination with the European Medicines Agency and set timelines for joint assessments, starting with cancer and advanced therapy medicines in January. 

  • In April, the FDA warned about the risk of secondary cancers in patients undergoing CAR-T cell therapy. However, a large Stanford Medicine study has found that the risk is low and not linked to the CAR-T cells themselves. 



Register for the ASGCT Policy Summit

September 23-24, 2024 | Washington, D.C.

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