Collaboration and Innovation at the Policy Summit: Paving the Way for Patient Access to CGTs
Daniela Drago, PhD - October 25, 2024
The 2024 ASGCT Policy Summit, held in Washington, D.C., brought together key stakeholders from across the cell and gene therapy (CGT) landscape. The central theme? Collaborative innovation.
From regulatory convergence to payment models, the discussions at the Policy Summit tackled the challenges that come with bringing transformative therapies to patients while maintaining a sharp focus on ensuring equitable access. (Above photo, from left: Angela Whatley, PhD; Sol Ruiz, PhD; Yasuhiro Kishioka, PhD; Sarah Wassmer, PhD; and Nicole Verdun, MD)
Global Regulatory Convergence: One World, One Set of Standards?
A major highlight of the Summit was the ongoing push for global regulatory alignment. Regulators from the United States, Canada, Japan, and the European Union convened to discuss how international harmonization efforts can streamline approval processes for CGTs. Collaboration aims to reduce uncertainty for companies and expedite patient access to life-changing treatments. Notable initiatives like Project Orbis took center stage, showcasing their role in reducing the burden on developers while accelerating patient access to life-saving therapies. Dr. Nicole Verdun, Director of FDA’s Office of Therapeutic Products, emphasized the importance of continually reassessing regulatory requirements, noting, “there may be less requirements at one agency versus another... We need to have these conversations and figure out, is this [requirement] really needed? ... What we said 5, 6, 7 years ago may not still stand.”
Raynce Rouce, MD (left) talks with Clare Pierce-Wrobel
Dr. Verdun was joined by Dr. Sol Ruiz from the Spanish Agency of Medicines and Medical Devices, Dr. Yasuhiro Kishioka with the Pharmaceuticals and Medical Devices Agency in Japan, and Dr. Sarah Wassmer from Health Canada. While challenges remain in aligning regulatory interpretations across countries, both formal and informal collaboration efforts between regulatory bodies are proving essential in addressing the complexities of product approvals across jurisdictions. With such collaborations already yielding positive outcomes, the conversation hinted at a future where regulatory divergence could be minimized, making it easier for companies to navigate the global CGT landscape.
New Approaches to Payment Policy: Affordability in Focus
As CGTs come with high upfront costs, the Summit also explored innovative payment models aimed at addressing these financial hurdles. Discussions centered around value-based arrangements (VBAs) and outcomes-based payments (OBPs), which tie reimbursement to the success of a treatment. These models allow for more manageable cost distribution over time while sharing risk between manufacturers and payers. Additional strategies discussed included pooling to aggregate financial risk across larger patient populations, and reinsurance to provide a safety net for payers.
A particular focus was placed on the CMMI CGT Access Model, which aims to improve access to gene therapy for Sickle Cell Disease for patients insured under Medicaid. Presenters were excited to see the outcomes of initial negotiations between CMS and manufacturers this December.
Centering health equity and what those state partners are going to carry out is going to be critical ... It’s not just about access to the therapy at the end, it’s about how patients are interacting with the entire health care system.
Clare Pierce-Wrobel, White House Domestic Policy Council
The conversations highlighted the need for creative solutions that balance the interests of patients, payers, and innovators, such as providing assistance to states and offering support for states’ participation.
Addressing Manufacturing/CMC Challenges: From Bottlenecks to Breakthroughs
Manufacturing remains a bottleneck for many CGTs, especially those that require personalized production. Each batch is often produced for a single patient, involving complex and time-sensitive supply chains, variability in starting materials, and strict quality control requirements. The Policy Summit emphasized the importance of public-private partnerships in addressing these challenges. Initiatives include the Bespoke Gene Therapy Consortium (BGTC), which aims to establish standardized processes for manufacturing viral vectors and create a rare disease development playbook.
Conversations at the event also addressed regulatory efforts to resolve manufacturing hurdles. Speakers discussed the Platform Technology Designation, which focuses on well-understood, reproducible technologies that can be used across multiple products. Dr. Joni Rutter, Director of NCATS, shared her excitement for using these types of platform approaches. “If it fits it ships,” she said, referring to the idea that well-understood, reproducible technologies could be applied across different products, potentially reducing duplication in regulatory submissions and making gene therapies more accessible.
While the intent behind the Platform Technology Designation Program is welcomed by the field, presenters and attendees noted that the recent guidance could be further developed to maximize its utility. Notably, sessions touched on the Advanced Manufacturing Technologies Designation, which encourages early adoption of advanced manufacturing methods that improve product quality, reduce human intervention, and expedite the regulatory review of CGTs, thus enhancing the scalability and reliability of manufacturing. Speakers stressed that the success of CGTs is highly dependent on the ability to efficiently produce these therapies at scale.
Rare Disease: Accelerating Therapies for the Most Vulnerable
The Summit also spotlighted the FDA’s Rare Disease Innovation Hub, which aspires to act as a central point of engagement for the rare disease community, enhance inter-center collaboration to address common product development challenges, and advance regulatory science through innovative approaches such as novel endpoints, biomarkers, and real-world evidence.
Legislative efforts, such as the Rare Pediatric Disease Priority Review Voucher (PRV) Program, were recognized as essential tools in incentivizing innovation for rare diseases. The PRV program’s short-term extension through December 2024 brought renewed hope for companies focusing on rare pediatric conditions, though a long-term extension is also needed. The program offers a voucher for priority review of a future product upon approval of a rare pediatric disease therapy, which companies can use or sell, representing an important incentive to support rare disease R&D.
Investment Strategies: De-risking the CGT Landscape
Attendees chat during a networking break.
High R&D costs and lengthy timelines create significant barriers for investors in CGT. To mitigate these challenges, speakers at the Summit discussed potential ways to de-risk investments through leveraging large genomic data sets, conducting natural history studies, and developing improvedmetrics to guide investment decisions. The involvement of Dr. Daria Fedyukina, a program manager from the Advanced Research Projects Agency for Health (ARPA-H), shed light on a unique approach to funding breakthrough technologies aimed at reducing CGT production costs and scaling manufacturing capabilities.
The conversations highlighted how real-world evidence (RWE) and patient registries could be better leveraged to offer insights into long-term efficacy and safety, potentially supporting regulatory decisions and payer negotiations. The discussions during this session further demonstrated the need for more collaborations between researchers, clinicians, regulators, and investors to develop standardized methods for collecting and reporting data that can de-risk investments and prove the long-term value of CGTs.
Looking Forward: The Path to Progress
The 2024 Policy Summit reaffirmed the importance of multi-sector collaboration to drive forward the next generation of CGTs. By fostering the harmonization of regulatory frameworks, developing flexible payment models, overcoming manufacturing hurdles, and leveraging long-term data, stakeholders can create a sustainable path for these groundbreaking therapies. The Summit laid the foundation for continuing progress: nurturing partnerships between regulators, legislators, industry leaders, patient advocacy groups, and investors, plus advancing the dialogues that will ensure CGTs can fulfill their promise of personalized medicine for patients worldwide.
Dr. Drago is a partner with NDA Partners and a Member of ASGCT’s Regulatory Affairs Committee.
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