Value Frameworks For Rare Diseases: Should They Be Different?

The US health care system is increasingly focusing on value as a basis for reimbursement of pharmacotherapies and devices, and as a result the use of “value frameworks” for measuring and comparing treatment value has grown in recent years. However, the therapies assessed by most frameworks frequently apply to modest-to-large disease populations, rather than the smaller populations affected by rare diseases, where the factors driving value may differ. Rare diseases are different from diseases affecting larger populations in several fundamental ways. In the United States, a rare disease is defined as one that affects fewer than 200,000 people a year, or one that affects more than 200,000 people per year but for which the costs of drug development are expected to exceed revenues for the product. Because rare diseases are frequently characterized by their high complexity, small patient populations that limit the speed of clinical trial enrollment, and limited availability of clinical experts, the development of therapies for rare diseases is particularly challenging. Within a small market of patients for which the per-patient costs of research and development may be sizable, higher per-patient prices are needed to generate sufficient investment returns on successful innovations. The difficulty of justifying much higher prices may result is an “unmet need trap” that impedes innovation and leaves behind a significant number of people who do not have adequate treatments avai...
Source: Health Affairs Blog - Category: Health Management Authors: Tags: Costs and Spending Drugs and Medical Innovation Quality Orphan Drug Act Precision Medicine rare disease treatment treatment value value frameworks Source Type: blogs