. "Appendix B: Innovation and the Orphan Drug Act, 1983-2009: Regulatory and Clinical Characteristics of Approved Orphan Drugs." Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press, 2011.
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Rare Diseases and Orphan Products: Accelerating Research and Development
FIGURE B-1 Annual orphan drug approvals by “newness.” The thick line represents number of orphan drugs approved each year where the molecular structure is completely new. The dotted line represents the number of orphan approvals each year for drugs previously available on the market in the United States or elsewhere. The thin line represents the number of orphan drugs approved each year that were variations or members of the same class of previously approved drugs.
for which the details were not located included 9 clotting factors or immune globulins, 7 already-marketed drugs,31 and 1 other product.32
Of the 30 drugs for which the full medical officer reviews were analyzed, the NDA and IND dates were obtained for 17 of the products. An average of 3.8 years lapsed from the date of the IND to the date of the orphan drug designation, while an average of 5.9 years lapsed from the date of the IND to the date of the NDA. Approximately 0.7 years passed from NDA submission to approval.
The 30 drugs collectively underwent a total of 71 trials evaluating their efficacy. These efficacy trials enrolled a median of 75 participants (inter-
They are raloxifene (Evista) for reduction in the risk of breast cancer in postmenopausal women with osteoporosis, adalimumab (Humira) for the treatment of juvenile rheumatoid arthritis, bevacizumab (Avastin) for renal cell carcinoma and glioblastoma with progressive disease following prior therapy, sorafenib (Nexavar) for treatment of unresectable hepatocellular carcinoma, doxorubicin liposomal injection (Doxil) for use in combination with bortezomib for the treatment of patients with multiple myeloma who have not previously received bortezomib and have at least one prior therapy, thyrotropin alfa (Thyrogen) for use as an adjunctive treatment for radioiodine ablation of thyroid tissue remnants in patients who have undergone thyroidectomy for well-differentiated thyroid cancer and who do not have evidence of metastatic thyroid cancer, and somatropin (Norditropin) for short stature associated with Noonan’s syndrome. This information theoretically could be obtained via a Freedom of Information Act (FOIA) request, although the long duration required for such requests to be filled made the FOIA pathway impractical for this study.
Capsaicin (Qutenza) for management of neuropathic pain associated with postherpetic neuralgia.