From rationing to rationality: an n-of-1 trial service for off-label medicines for rare (neuromuscular) diseases (WC2010-064)


Starting date: 15/11/2010


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In the Netherlands, expensive medicines are not reimbursed for off-label use unless there is sufficient evidence of efficacy. Patients with rare diseases are at a disadvantage because the burden of proof is difficult to meet. There are obstacles both for industry and academia to performing traditional randomized, controlled trials. Moreover, the reimbursement rules discourage doctors from prescribing expensive medicines off-label, even to small groups of patients. Thus, evidence will not even accrue from case series.

This project proposes to develop an n-of-1 trial service, integrated in the Dutch health care (assessment) system. The aim of the n-of-1 trial service is to promote equitable pharmaceutical care for patients with rare diseases while at the same time generating evidence on the efficacy of promising, off-label drugs.
Examples of reimbursement problems with off-label medicines are known from many rare disease areas, e.g. oncology, movement disorders, neuromuscular diseases and more. This project focuses on neuromuscular disorders, as a pilot group. If successful, it may serve many other groups of patients with serious, chronic, rare disease as well.

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