An evaluation framework for funding drugs for rare diseases.

@article{Winquist2012AnEF,
  title={An evaluation framework for funding drugs for rare diseases.},
  author={Eric William Winquist and Chaim M. Bell and Joe T. R. Clarke and Gerald Evans and Janet M. Martin and Mona Sabharwal and Anita Gadhok and Helen Stevenson and Doug Coyle},
  journal={Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research},
  year={2012},
  volume={15 6},
  pages={982-6}
}
OBJECTIVES For rare diseases it may be difficult to generate data from randomized trials to support funding of a drug. Enzyme replacement therapies for diseases of inherited metabolic enzyme deficiency provide an example of this dilemma. The Ontario Public Drug Programs convened the Drugs for Rare Diseases Working Group to develop a policy for assessing these drugs. METHODS The Drugs for Rare Diseases Working Group developed terms of reference expecting that the ideal policy product would be… CONTINUE READING
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Evaluating and Valuing Drugs for Rare Conditions: No Easy Answers.

Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research • 2018
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Economic aspects of rare diseases.

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