Exploring Barriers to Accessing Gene and Cell Therapy Treatments

Patients accessing cell and gene therapies can face barriers in development, eligibility, coverage, referrals, and logistics. ASGCT’s latest Community Quick Takes module highlights the multifaceted meanings of “access,” with the module encompassing approved products, eligibility criteria, provider knowledge and how cell and gene therapies (CGTs) excite families compared to standard of care options. Our speakers shed light on the critical factors shaping the field and impacting individual access to CGTs. Check out the highlights below. 

Mechanisms Behind Current CGT Developments 

The field of CGT is evolving rapidly, with many new mechanisms being used for further development. Approved products are the result of extensive research, technological advancements, and clinical trials. For example, the use of adeno-associated viral (AAV) vectors plays a significant role, although AAV vectors come with limitations like the size constraints on the genetic material they can carry. This restricts their application to certain diseases, creating a need for alternative approaches for broader disease coverage. 

Impact of Eligibility Criteria 

Eligibility criteria significantly influences who can receive approved CGTs. Factors such as age, disease progression status, and prior treatments fall into two main categories: delivery problems and health care system factors. Delivery issues include gene size limitations and the presence of neutralizing antibodies, while health care system factors pertain to prescription guidelines and insurance coverage. These criteria can exclude segments of the patient population, highlighting the need for inclusive efficacy data to secure broader insurance coverage. 

Lessons from CAR-T Approvals 

The approval of CAR-T therapies has provided valuable insights applicable to newly approved CGTs. Key lessons include the necessity of robust efficacy data and the importance of decentralized studies and real-world evidence (RWE) in demonstrating treatment effectiveness. Such data is crucial for gaining insurance approval and ensuring that more patients can access these life-changing therapies. 

Provider Knowledge 

Providers' knowledge about new treatments is pivotal in facilitating patient access to CGTs. As the pipeline for gene, cell, and RNA therapies continues to grow, with 3,951 therapies in development as of 2023, providers must stay informed about emerging treatment options. Continued education and training opportunities are essential for health care professionals to confidently refer eligible patients to receive advanced therapies. 

Patient and Family Excitement Around CGTs 

Communities eligible for CGTs are excited about the potential quality-of-life improvements these treatments offer. For conditions like sickle cell disease (SCD), CGTs can alleviate the fear of health crises, allowing individuals to live more active and fulfilling lives without frequent hospital visits. Patients and families should engage in open discussions with health care providers about the viability and accessibility of new treatments. 

Get Involved and Stay Informed 

ASGCT members aim to navigate the complexities of CGTs to improve patient access. Addressing eligibility criteria, delivery method limitations, and health care system preparedness are critical steps. Additionally, advocating for legislative support and sharing patient experiences can drive positive changes in access to CGTs. By understanding and addressing these barriers, we can enhance access to transformative treatments, ultimately improving the lives of those in need.  

To learn more about gene and cell therapy, consider becoming an ASGCT member or attending an upcoming ASGCT event. Stay updated on legislative proposals and coverage issues by subscribing to The Advocate. For more information on current conditions with approved CGT treatments, visit the ASGCT Patient Education site.  

Alexis Starosta is ASGCT's Advocacy Programs Specialist.

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