Lawrenceville, NJ, USA—December 18, 2024—, the official journal of , announced today the publication of a special themed section of research papers that document challenges in rare disease evidence and economic evaluation and policy as well as ways in which the environment is evolving to address them. Guest editors for the themed section are Brian Rittenhouse, PhD, Massachusetts College of Pharmacy and Health Sciences, Boston, MA, USA, and Elena Nicod, PhD, Dolon, Ltd, Milan, Italy. The series was published in the December 2024 issue of .
In their , Rittenhouse and Nicod note that specific policies, legislation, and process adaptions for orphan drugs have focused on improving the attractiveness of rare disease innovation via different types of regulatory incentives. These policies also adapt pricing and reimbursement frameworks and processes to help manage the challenges of limited evidence available at time of launch and the high cost of these medicines. The Guest Editors point out, however, that few initiatives have focused on new approaches to generating and evaluating clinical or cost-effectiveness evidence. “The motivation for this themed section was that we wanted to revisit the question of whether rarity should be treated differently, focusing on what has changed, what may have been incorrect or more debatable in the past, and what may have not been addressed in previous work,” they said.
The themed section includes 5 research papers:
- “,” by Charles Manski, PhD
- ",” by Alexa Klimchak, MA; Lauren Sedita, MS; Eleanor Perfetto, PhD; Katherine Gooch, PhD; and Daniel Malone, PhD
- “,” by Darius Lakdawalla, PhD; Charles Phelps, PhD; and Hong-Hanh Nguyen, MA
- "," by Katherine Clifford, MPH; A. Alex Levine, MPH; Daniel Enright MS; Peter J. Neumann ScD; and James Chambers PhD, MPharm, MSc
- "," by Lea Wiedmann, MSc; John Cairns, MPhil; and Ellen Nolte, PhD
The evidence from these papers suggests that while ultra-orphan drugs may offer substantially larger health gains than non-orphan drugs, they face significant challenges in demonstrating cost-effectiveness due to their high costs. Traditional health economic evaluation methods using quality-adjusted life-years (QALYs) may disadvantage treatments for rare severe diseases and disabilities. Alternative approaches, such as the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) framework, modified statistical methods, and consideration of societal preferences for horizontal equity, may be needed to appropriately evaluate these treatments—an important consideration as orphan drugs represent a growing share of new drug approvals and healthcare spending.
“Some of the suggested methods in these papers may solve challenges in rare disease evidence and economic evaluation and policy and reduce the need for further initiatives as they may supply solutions to long-perceived difficulties in rare disease economic evaluation and policy,” concluded Rittenhouse and Nicod. “However, challenges will remain due to the inherent difficulties in providing rare disease evidence and because current proposed solutions challenge traditional approaches."
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