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A roadmap for affordable genetic medicines.

Melinda Kliegman1, Manar Zaghlula2, Susan Abrahamson2

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The first CRISPR gene-editing therapy for sickle cell disease, CASGEVY, is approved but costly. Recommendations for sustainable alternatives aim to reduce prices tenfold, improving access to genetic therapies.

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Area of Science:

  • Biotechnology
  • Genetics
  • Medical Economics

Background:

  • The US Food and Drug Administration has approved twenty genetic therapies, including the first CRISPR-based gene-editing therapy for sickle cell disease, CASGEVY.
  • These advanced therapies offer significant promise for treating genetic disorders and cancers but are associated with extremely high costs, exceeding $4 million per patient.

Purpose of the Study:

  • To address the high cost and limited accessibility of newly approved genetic therapies.
  • To propose sustainable, alternative models for the development and commercialization of genetic therapies from discovery to market.

Main Methods:

  • Convening a multidisciplinary task force to develop recommendations.
  • Proposing a new pricing structure and a business model for genetic therapy development.
  • Outlining strategies involving academic licensing, manufacturing innovation, and regulatory support.

Main Results:

  • A proposed pricing structure could potentially reduce per-patient costs by tenfold.
  • A new business model distributing responsibilities and leveraging diverse funding sources is outlined.
  • Strategies for cost reduction through academic licensing, manufacturing, and regulation are presented.

Conclusions:

  • Sustainable alternatives are crucial to enhance affordability and access to transformative genetic therapies.
  • Implementing proposed pricing and business models, alongside supportive regulations and manufacturing innovations, can broaden patient access to treatments like CASGEVY.