Nicholson L, Langham S, Adkins E. The patient perspective in health technology assessment reimbursement decisions for orphan drugs across different countries. Value Health 2019:22(Suppl 3):S860

Abstract

Objectives: There is growing recognition that the patient perspective is fundamental in assessing the value of new treatments in health technology assessment (HTA). This is particularly relevant for orphan drugs (ODs) as there is often limited evidence describing disease burden and unmet need, and the impact of treatment on patients’ day-to-day lives may not be fully captured in clinical trials. This study reviewed how patient input was provided and how it influenced reimbursement decisions in recent HTAs of ODs from countries that commonly include patient input as part of their HTA process.

Methods: We searched HTA agency websites (www.pbs.gov.au/ [Australia], www.cadth.ca/, www.cadth.ca/pcodr [Canada], www.nice.org.uk/ [England], and www.scottishmedicines.org.uk/ [Scotland]) to identify HTAs of the 46 ODs that received European marketing authorisation from 2016 to 2018.

Results: We identified 47 HTAs relating to 22 ODs: 8 in Australia, 8 in Canada, 15 in England, and 16 in Scotland; input from patients/patient advocacy groups was included in 50%, 100%, 87%, and 94% of HTAs, respectively. A high proportion of HTAs included patient input (85%), with 92% of those being positive reimbursement recommendations. However, the type of input presented differed between HTA agencies, from a focus only on unmet need in England to an input on unmet need, clinical efficacy, tolerability, quality of life improvements, and treatment convenience in Scotland. Positive HTA decisions were reported in 33% (1/3) HTAs in Australia that included patient input, 92% (12/13) in Scotland and 100% in Canada and England.

Conclusions: Patient input into HTA decision making varied between countries. Despite a generally positive association between patient input and decision, the impact of patient involvement on final HTA decisions remains unclear. There is a need for HTA agencies to formalise patient participation and publish criteria demonstrating how it is incorporated into the decision-making process, particularly for orphan diseases.