Evidence and values: requirements for public reimbursement of drugs for rare diseases--a case study in oncology.
BACKGROUND
Doubts have been expressed about whether standard methods of health technology assessment are suitable for the evaluation of drugs for rare diseases. Under conditions of rarity, it may be more difficult to conduct large randomized trials in order to gather adequate evidence on efficacy, and the standard methods of economic evaluation may not adequately reflect societal preferences for the treatment of serious and/or life-threatening rare diseases.


METHODS
A roundtable was held at the University of Toronto Joint Centre for Bioethics on February 18, 2008 to address these issues. While the focus was on evaluation and reimbursement decision-making for rare cancers, the discussion was broadened to consider the place of evidence and values in considering public reimbursement of drugs prescribed for rare disorders more generally.


CONCLUSIONS
This paper explores the relevant issues in more detail, using the example of a new drug for treatment of renal cell carcinoma.


CONCLUSIONS
There should be a greater commitment by reimbursement agencies to a fair and transparent decision-making process with appropriate community input. Criteria should be developed to validate surrogate markers for rare diseases. It should also be acknowledged that the traditional measures of benefit in economic studies do not incorporate all elements of social value. The need should be recognized to balance equity with an efficient use of resources.
Drummond
Michael
M
University of York, Canada.
Evans
Bill
B
LeLorier
Jacques
J
Karakiewicz
Pierre
P
Martin
Douglas
D
Tugwell
Peter
P
MacLeod
Stuart
S
D000970:Antineoplastic Agents / Q000191:economics / Q000627:therapeutic use*; D001557:Benzenesulfonates / Q000191:economics / Q000627:therapeutic use*; D002292:Carcinoma, Renal Cell / Q000188:drug therapy* / Q000401:mortality; D002986:Clinical Trials as Topic / Q000191:economics; D003362:Cost-Benefit Analysis; D016527:Drug Costs / Q000331:legislation & jurisprudence; D019317:Evidence-Based Medicine; D033161:Government Regulation; D006291:Health Policy; D006292:Health Priorities / Q000191:economics; D006297:Health Services Accessibility / Q000191:economics; D006301:Health Services Needs and Demand / Q000191:economics* / Q000941:ethics / Q000331:legislation & jurisprudence; D006801:Humans; D007349:Insurance, Health, Reimbursement / Q000941:ethics / Q000331:legislation & jurisprudence; D007680:Kidney Neoplasms / Q000188:drug therapy* / Q000401:mortality; D009536:Niacinamide / Q000031:analogs & derivatives; D010671:Phenylurea Compounds; D011725:Pyridines / Q000191:economics / Q000627:therapeutic use*; D012109:Research Support as Topic; D012945:Social Values; D000077157:Sorafenib; D016019:Survival Analysis; D016896:Treatment Outcome
D000970:Antineoplastic Agents; D001557:Benzenesulfonates; D010671:Phenylurea Compounds; D011725:Pyridines; D009536:Niacinamide; D000077157:Sorafenib