Editor’s note: The attached comment letter was submitted to the Senate HELP committee on January 19, 2024 in response to a request for information from stakeholders about improving access to cell and gene therapies for ultra-rare diseases.
For more than a decade, researchers at the USC Schaeffer Center for Health Policy & Economics have developed and evaluated innovative pricing structures to ensure access to breakthrough therapies while incentivizing future innovation. Cell and gene therapies for ultra-rare diseases are the next frontier in biomedical advances, offering the potential for substantially improving the lives of many individuals suffering from debilitating conditions. However, the long-term benefits and risks of these new treatments are largely unknown, making financing these treatments precarious for payers and the pharmaceutical companies developing them.
Though data are still accumulating, patient access to gene therapy has been spotty at best. Payers have imposed both explicit and hidden barriers to patient access, an arguably rational response given the still-uncertain benefits of many of these new treatments. At this stage, federal policymakers could play a leadership role in increasing access to therapies through several tools, including:
- Providing the legal framework to allow the creation of financial intermediaries – publicly managed or private, third-party companies – to develop financial instruments to manage risk;
- Developing updated regulatory frameworks that encourage and facilitate outcomes-based or value-based contracts;
- Ensuring generous coverage coupled with predictable and transparent evaluations of value, which will safeguard access and incentives for future innovation.
The full letter can be read here.
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