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Systematic review of comparative effectiveness data for oncology orphan drugs

Mindy M Cheng1, Scott D Ramsey, Emily Beth Devine

  • 1Pharmaceutical Outcomes Research and Policy Program, University of Washington, Seattle, WA 98195-7630, USA.

The American Journal of Managed Care
|March 23, 2012
PubMed
Abstract

Insights

This review assessed clinical and economic evidence for US oncology orphan drugs. Findings show variable evidence quality and a lack of economic data, necessitating new approaches for decision-making.

Area of Science:

  • Oncology
  • Pharmacoeconomics
  • Health Services Research

Background:

  • Orphan drugs are crucial for rare cancers but often face challenges in evidence development.
  • Assessing both clinical efficacy and economic value is essential for informed healthcare decisions.

Purpose of the Study:

  • To systematically evaluate the clinical and economic evidence base for oncology orphan drugs in the US.
  • To identify challenges and opportunities in developing robust evidence for this drug category.

Main Methods:

  • A systematic literature search of Medline and Embase databases was performed for studies published before June 2010.
  • The quality of clinical evidence was assessed using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) method.
  • Economic evidence quality was evaluated using the Quality of Health Economic Studies (QHES) criteria.

Main Results:

  • 60 randomized controlled trials and 21 cost-effectiveness analyses were identified for 47 oncology orphan drugs.
  • Clinical evidence quality varied: 21 drugs had moderate/high quality, 11 had low/very low quality, and 15 lacked sufficient evidence.
  • Cost-effectiveness analyses demonstrated high quality, with a mean QHES score of 85.

Conclusions:

  • Oncology orphan drugs in the US exhibit diverse clinical evidence levels and a notable scarcity of economic evidence.
  • There is a need for innovative analytical and policy strategies to create a decision-making framework.
  • This framework should align with evidence-based medicine and comparative effectiveness research principles.

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