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Prescription, Nonprescription and Orphan Drugs01:02

Prescription, Nonprescription and Orphan Drugs

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Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
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Area of Science:

  • Pharmacoeconomics
  • Drug Policy
  • Rare Disease Research

Background:

  • The Orphan Drug Act (ODA) of 1983 aims to incentivize the development of drugs for rare diseases.
  • Recent policy discussions question the ODA's incentives due to concerns about approvals for common diseases and multiple indications.

Purpose of the Study:

  • To investigate the approval pathways and indications of orphan drugs approved under the ODA.
  • To analyze the prevalence of orphan drugs approved for rare-only, rare and common diseases, and pediatric rare diseases.

Main Methods:

  • Analysis of orphan drug approval data from 1990 to 2022.
  • Examination of drug indication expansion, including for pediatric populations.
  • Assessment of revenue data for top-selling orphan drugs.

Main Results:

  • 491 novel orphan drugs were approved between 1990 and 2022.
  • 20% of these orphan drugs received approvals for both rare and common diseases.
  • 10% of orphan drugs gained subsequent approval for pediatric rare disease populations.

Conclusions:

  • A significant proportion of orphan drugs are approved for common diseases, raising questions about the ODA's focus.
  • Modifying ODA incentives could potentially reduce the development of orphan drugs for pediatric rare diseases.