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Expensive Drug Prices for Rare Cancers: Are Patients Truly Benefitting?

Kristina Jenei1, Bishal Gyawali2

  • 1Research Associate, School of Population and Public Health, University of British Columbia, Vancouver, BC.

Healthcarepapers
|January 24, 2023
PubMed

Insights

Cancer medicines for rare diseases are often expensive. Regulators must ensure these drugs provide real patient benefit, especially those fast-tracked for approval.

Area of Science:

  • Oncology
  • Pharmacoeconomics
  • Health Policy

Background:

  • Cancer medicines represent a significant portion of expensive drugs for rare diseases (EDRDs).
  • The US Orphan Drug Act (ODA) incentivizes development for rare diseases, shifting focus to rare cancer subtypes.
  • While some ODA-approved medicines have transformed cancer care, many lack demonstrated added therapeutic benefit over existing treatments.

Purpose of the Study:

  • To evaluate the therapeutic benefit of cancer medicines approved via the US Orphan Drug Act.
  • To inform Canadian regulatory and payer practices regarding fast-tracked cancer medicines.

Main Methods:

  • Analysis of cancer medicines approved through the US Orphan Drug Act.
  • Review of regulatory approval pathways and therapeutic benefit assessments.

Main Results:

  • Approximately half of the cancer medicines approved via the ODA do not show added therapeutic benefit compared to current alternatives.
  • The ODA has successfully redirected cancer medicine development towards rare subtypes.

Conclusions:

  • Canadian regulators must ensure that fast-tracked cancer medicines provide tangible benefits to patients.
  • Payers should consider ongoing reassessment and renegotiation strategies for these high-cost medications throughout their lifecycle.

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