Market Exclusivity for Drugs with Multiple Orphan Approvals (1983–2017) and Associated Budget Impact in the US

FDA Orphan Designations, Approvals, and Regulatory Review Time since the Enactment of the Orphan Drug Act (1983)

“…High activity companies with a large number of designations and approvals were more successful than small companies developing new drugs for rare diseases and had higher odds of obtaining orphan approval. Orphan drug market exclusivity further delays market entry to generics and biosimilars and contributes to higher drug prices (23). Also, pharmaceutical companies marketing orphan drugs are more pro table and have a higher market value than those engaged in the development of drugs for common diseases (24), which may explain in part why large multinational companies allocate more resources to orphan drug development.…”

Can the Consideration of Societal Costs Change the Recommendation of Economic Evaluations in the Field of Rare Diseases? An Empirical Analysis

“…However, this impact on budgets could be scarily significant when all RDs are considered together. 52,[56][57][58] So it is not surprising that considerable uncertainty surrounds the decision making about the financing and the price of these treatments, 59,60 keeping in mind the small health benefits that such therapies might provide in the long term compared with its high costs. In this regard, it should be noted that, given the results usually achieved by the economic evaluations of health interventions in rare diseases, an appeal has been made to the use of other methods that complement or even substitute the previous ones, and that help either to expressly include elements that should be present in the decision making about financing and prices, or to introduce innovative reimbursement schemes to reconcile the patients' access to treatments with the requirements of good value for money 16,[61][62][63][64][65] and affordability.…”

Modifying the Criteria for Granting Orphan Drug Market Exclusivity

“…For biologics, US discount rates available in the literature are often drug-specific, and therefore we used the drug-based estimates that provided the widest discount range (10%-38%) to account for potential differences in the price trajectories of drugs impacted by the policies in our study. 23,24 Second, we accounted for the possible overlap between orphan drug exclusivity and other FDA-granted market exclusivity periods by reviewing our calculations assuming that all drugs approved via new drug application (NDA) were granted 5 years of new chemical entity exclusivity 25 and all drugs approved via biological licensing application (BLA) were granted 12 years of reference product exclusivity for biologics 26 at the time of first market approval.…”