Why orphan drug coverage reimbursement decision-making needs patient and public involvement

Recently there has been an increase in the active involvement of publics and patients in healthcare and research, which is extending their roles beyond the passive recipients of medicines. However, there has been noticeably less work engaging them into decision-making for healthcare rationing exercises, priority setting, health technology assessment, and coverage decision-making. This is particularly evident in reimbursement decision-making for ‘orphan drugs’ or drugs for rare diseases. Medicinal products for rare disease offer particular challenges in coverage decision-making because they often lack the ‘evidence of efficacy’ profiles of common drugs that have been trialed on larger populations. Furthermore, many of these drugs are priced in the high range, and with limited health care budgets the prospective opportunity costs of funding them means that those resources cannot be allocated elsewhere. Here we outline why decision-making for drugs for rare diseases could benefit from increased levels of publics and patients involvement, suggest some possible forms that involvement could take, and advocate for empirical experimentation in this area to evaluate the effects of such involvement. Focus is given to the Canadian context in which we are based; however, potentialities and challenges relating to involvement in this area are likely to be similar elsewhere.