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Access to Approved Cell and Gene Therapies

Patients accessing cell and gene therapies (CGTs) can face barriers in development, eligibility, coverage, referrals, and logistics. ASGCT experts discuss approved products, eligibility criteria, CAR-T lessons, provider knowledge, and how CGTs excite families compared to current sickle cell care.

A person considering a gene or cell therapy treatment may experience barriers to accessing the treatment. Our experts will explore the various meanings of the term “access.”  This includes the development of therapies, eligibility to receive an FDA-approved treatment, coverage, provider referral, and being able to navigate the logistics of treatment within a clinic.  Each expert touches on aspects that shape the field and impact an individual’s access to gene and cell treatments.

Listen to Quick Takes from experts in the community on the following questions (Time: 25 min):

  • What are the mechanisms behind some current developments in CGTs, including approved products?
  • How do certain eligibility criteria impact the ability of patient communities to access CGTs?
  • What are some lessons learned from CAR-T approvals that might be relevant to newly approved products?
  • What knowledge do providers have about these new treatments and how does this relate to access?
  • What aspects of CGTs excite families more than standard care options when considering potential treatments (for SCD)?

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Key Takeaways

There are many moving pieces that affect patient access to gene and cell therapies, and the speakers explored a few of these issues:

  • Eligibility criteria, including age range, status of disease progression, and past treatments, significantly influence who can receive approved gene and cell therapies. These criteria often fall under one of two main categories: delivery or scientific issues (i.e., gene size for vector and neutralizing antibodies) and the healthcare system (how they are prescribed and insurance coverage).
  • Not all methods for delivering genetic material are viable for every disease or disorder. Many vectors, such as AAV (adeno-associated virus), have size limitations on the genetic material they can carry. This restricts the ability to address certain diseases using standard CGT approaches.
  • The healthcare system is often unprepared to distribute new therapies effectively. Once a condition has an approved therapy, it does not mean all individuals with that condition will be eligible. There is a label that indicates who the treatment is approved for use in, which may exclude a segment of the population with the condition. Without wider efficacy data (to show the treatment works well in many people), insurance companies may deny coverage for the treatment.  Considering the often-small patient populations, alternative study designs – including decentralized studies, use of RWE (Real World Evidence), and patient experience data – are crucial in advancing research and understanding of gene and cell therapies.
  • In 2023, the gene, cell, and RNA therapies pipeline grew by 6%. As a result, there are 3,951 such therapies in development, ranging from preclinical through pre-registration (filing for regulatory approval). The gene and cell therapy pipeline offers a unique opportunity to address many of these diseases for which there are currently no viable treatment options. To keep pace with the robust development pipeline, providers will require additional opportunities to learn about new and emerging treatment options, so they become more comfortable referring patients to receive CGTs if eligible. Advocacy at the legislative level is also important, with patients and families sharing how life-changing these treatments can be.
  • Communities eligible for CGT treatments are excited about potential quality-of-life improvements. These treatments can alleviate the constant fear of health crises, allowing individuals to work and be active without the worry of frequent hospital visits (i.e. for those with sickle cell disease). Patients should be encouraged to discuss the viability, accessibility, and availability of new treatments with their healthcare providers.

By understanding and addressing some of these areas, ASGCT members hope to better navigate the complexities of gene and cell therapies and improve access for those in need.

Conditions with Approved Products

Check out some of the current conditions with approved CGT treatments on the ASGCT Patient Education site:

Call to Action

As more approved CGT products come to market, multiple stakeholders will be involved in the discussions and decisions surrounding patient access. If you want to learn more about gene and cell therapy and want to further connect with those working to change the way individuals access treatments for rare diseases, consider becoming an ASGCT member or attending an upcoming ASGCT event. If interested in receiving monthly updates about legislative proposals and coverage issues, subscribe to The Advocate.

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Last Updated: 06/05/2024

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