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P70: Time to Clinical Guidance Inclusion of Orphan Drugs After Expedited Approval

Poster Presenter

      Rachel Brunner

      • Drug Information Pharmacy Resident
      • University of Illinois-Chicago, College of Pharmacy
        United States


The objective of this study is to determine the time between approval and inclusion in clinical practice guidance documents for FDA-approved orphan drugs and biologics (non-oncologic) with expedited program designations.


Eligible products with =1 expedited designations approved since 1992 were reviewed. In June 2020, literature databases were searched for guidances (e.g. treatment guidelines) influencing US clinical practice for each product to determine time to first inclusion, evaluated through survival analyses.


Overall, 135 non-oncology orphan products were reviewed, of which 97.0% (n=131) were designated with priority review, 49.6% (n=67) fast-track, 16.3% (n=22) breakthrough therapy, and 14.1% (n=19) accelerated approval. Sixty percent of products (n=81) received =2 designations. Overall, 74.1% of products (n=100) were included in a guidance document. The median time to guidance inclusion was 2.87 years (95% confidence interval [CI], 2.21 to 4.18) for the entire cohort, and did not significantly differ across priority review, accelerated approval, breakthrough therapy, and fast-track designations (log-rank p=0.24). However, compared with products with 1 program designation, time to guidance inclusion was significantly shorter for products with =2 designations (median, 2.21 vs 4.18 years; Cox proportional hazards ratio [HR], 1.84; 95% CI, 1.21 to 2.79). Of 35 products not included in a guidance document, 54.3% (n=19) were approved in 2018 or later.


According to this study, it takes nearly 3 years from approval for non-oncology orphan drugs with FDA expedited program designations to be included in clinical guidance documents. Products with =2 designations were approximately twice as likely to be included in guidance documents before those with only 1 designation. This suggests that greater recognition of potential therapeutic value via regulatory designations may be associated with more timely guidance inclusion. Nonetheless, while these regulatory designations encourage market availability, they do not ensure patient access, and payer decisions are strongly influenced by clinical practice guidance documents in determining product coverage. Our findings suggest that efforts by professional societies to more rapidly address these products in guidance documents may clarify their place in therapy, facilitate coverage decisions, and promote expanded product access.