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[How shall we set priorities among experimental treatment methods?]
O F Norheim1, O Ekeberg, S A Evensen
1Cand Med, Senter for medisinsk etikk, Oslo.
Summary
Prioritizing investigational medicine requires balancing evidence quality, patient benefit, and risks. Scarce resources necessitate careful evaluation of clinical protocols, using criteria like cost and patient population size.
Area of Science:
- Clinical Research
- Oncology
- Health Economics
Context:
- Growing patient demand for novel treatments for severe diseases.
- Norway's new public funding for large-scale clinical investigations of treatment effect and safety.
- Establishment of a funding principle for investigational medicine.
Purpose:
- To examine criteria for prioritizing clinical protocols in investigational medicine.
- To address the challenge of allocating scarce resources effectively.
- To apply prioritization criteria to a controversial case: high-dose chemotherapy for metastatic breast cancer.
Summary:
- Key criteria for prioritization include quality of evidence, magnitude of expected benefit, risk-benefit balance, protocol quality, cost, and patient population size.
- These criteria provide a framework for making difficult decisions in investigational medicine funding.
- The article uses high-dose chemotherapy with hematopoietic stem cell support for metastatic breast cancer as a case study.
Impact:
- Provides a framework for evidence-based decision-making in investigational medicine.
- Aims to optimize resource allocation for promising, yet unproven, treatments.
- Facilitates informed prioritization of clinical research protocols in healthcare systems.