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The value of the accelerated approval pathway: real-world outcomes associated with reducing the time between

William Wong1, Stacey Kowal1, Tu My To1

  • 1Genentech, Inc., South San Francisco, CA 94080, USA.

Future Oncology (London, England)
|January 23, 2024
PubMed

Insights

The accelerated approval (AA) pathway significantly increases survival gains for innovative cancer therapies. Earlier access to these treatments via the AA pathway resulted in substantial life-years gained across three solid tumors.

Area of Science:

  • Oncology
  • Health Economics
  • Regulatory Science

Background:

  • The accelerated approval (AA) pathway aims to expedite patient access to innovative therapies.
  • Evaluating the impact of shortened innovation timelines on patient outcomes is crucial.

Purpose of the Study:

  • To investigate the effect of the accelerated approval pathway on patient survival in three solid tumors.
  • To quantify the population-level impact of earlier access to innovative treatments.

Main Methods:

  • Real-world analysis of patients receiving sequential AA-approved innovations.
  • Estimation of outcomes attributable to AA and extrapolation to the US population.
  • Case studies focused on three specific solid tumors.

Main Results:

  • Survival gains were 2.3-3.8 times higher with the AA pathway.
  • Approximately 8000 life-years were gained at the US population level across the three tumor types.
  • Earlier access to innovations via AA enhanced the value of existing therapies.

Conclusions:

  • The AA pathway enhances survival benefits by providing earlier access to innovative cancer treatments.
  • Earlier access to AA innovations should be considered in program evaluations.
  • This pathway is particularly valuable in fields with rapid clinical development.
Keywords:
accelerated approvalscancerinnovationreal option valuereal-world datatherapeutic bridging

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