Multistakeholder Panel Weighs In on Which Factors to Consider for Reimbursement of Orphan Drugs

From the decision analysis came an agreement that reimbursement for an orphan drug should be based primarily on its effect on health-related quality of life, its effectiveness, and the availability of other treatment options.

Analyzing various factors related to orphan drugs, a group of stakeholders has determined which and how each criteria should factor into decisions being made for reimbursement of these drugs to treat rare diseases.

From the decision analysis came an agreement that reimbursement for an orphan medicinal product (OMP) should be based primarily on its effect on health-related quality of life, its effectiveness, and the availability of other treatment options.

“These criteria and their relative importance weights can serve as a starting point to guide and inspire health authorities and the rest of decision makers involved to promote the development of a specific framework of multicriteria evaluation and move forward to a structured plan for the evaluation of OMPs,” wrote the researchers.

The group consisted of 28 stakeholders including physicians, hospital pharmacists, health economists, patient representatives, and health authorities.

Thirteen criteria related to the patient population, the disease state, treatment, and economic evaluation were included in the review. Based on the analysis, the group identified 9 criteria that should be considered when making decisions about reimbursement:

  • Health-related quality of life (relative importance, 23.53%)
  • Treatment efficacy (relative importance, 14.64%)
  • Availability of treatment alternatives (relative importance, 13.51%)
  • Disease severity (relative importance, 12.62%)
  • Avoided costs (relative importance, 11.21%)
  • Age of the treatment population (relative importance, 7.75%)
  • Safety (relative importance, 4.72%)
  • Quality of evidence (relative importance, 3.82%)
  • Size of the treatment population (relative importance, 3.12%)

The remaining 4 criteria—economic burden of disease, cost of treatment, cost-effectiveness, and safety—were determined to have a relative importance of less than 3%.

“According to the results obtained, preference was given to a scenario that financed OMPs for diseases with a target population of lower prevalence, of nonpediatric age, and prioritizing the most severe pathologies,” explained the researchers. “It was important that the orphan drug presented nonserious versus serious adverse events and that no other treatment options were available. In terms of the efficacy criteria, treatments that were curative or that significantly increased survival were preferred over other benefits, as were treatments that had high-quality evidence from controlled clinical trials with comparators.”

The group added that treatments that improved health-related quality of life while avoiding higher costs associated with the disease were prioritized.

Notably, the multistakeholder panel did place a higher weight on cost for treatments for nonpediatric patient populations.

Reference

Andrés‐Nogales F, Cruz E, Calleja M, et al. A multi‐stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU‐MCDA study). Orphanet J Rare Dis. Published online August 17, 2021. doi:10.1186/s13045-021-01135-w