Orphan diseases and its management
The web survey focused on the current management of 9 orphan agents selected to achieve a mix of oncology and nononcology agents, orphan and ultra-orphan drugs, various modes of administration, existence of treatment alternatives, and multiple indications (Table).
Among the most significant findings was that no new management approaches have been developed or appear to be in development specifically for managing unique orphan drugs that treat very small populations. Instead, orphan drugs are likely to be captured in the same net of tools, tactics, and benefit designs already in use or planned for controlling costs of biologics, injectables, or other expensive specialty products used for the treatment of diseases far less rare than most orphan conditions.Figure 1
Sensitivity to drug cost ranges considerably among surveyed plans, but for 54%, scrutiny increases when a drug is priced =$50, 000 per patient per year. The remaining respondents cite thresholds =$50, 001 per patient per year, with 2 plans indicating that the drug cost must exceed $250, 000 per patient per year for greater scrutiny (Figure 1).Figure 2
Not surprisingly, clinical data associated with an orphan drug are ranked highest among the factors that drive benefit design or restrictions for orphan drugs in most plans, followed closely by overall cost exposure (Figure 2). Clinical data—FDA-approved indication, trial design and results, formulation, and requirements for administration—provide the basis for prior authorization and continued use. Overall cost exposure—a function of disease prevalence, number of indications, potential for off-label use, and the availability of less expensive treatment options—serves to further raise the visibility of an orphan drug and to guide management.Figure 3
Most of the plans surveyed require prior authorization to ensure that the proposed use matches the FDA-approved indication for each of the orphan drugs (Figure 3). Payers, however, are likely to vary significantly in how they use clinical information and FDA labeling to restrict access to orphan products as illustrated by 2 cases involving eculizumab (Soliris), a product for treatment of paroxysmal nocturnal hemoglobinuria.
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