. "Appendix C: Medicare Part D Coverage and Reimbursement of 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
(1) Treatment of adults with Philadelphia chromosome-positive acute lymphoblastic leukemia with resistance or intolerance to prior therapy. (2) Treatment of adults with CML with resistance or intolerance to prior therapy including imatinib.
Indicated for patients with Hunter syndrome (mucopolysaccharidosis II, MPS II)
10/6/06
Vorinostat
Zolinza
Treatment of cutaneous manifestations in patients with cutaneous T-cell lymphoma (CTCL) who have progressive, persistent, or recurrent disease on or following two systemic therapies
Treatment of short stature in patients with Noonan’s syndrome
6/15/07
Ambrisentan
Letairis
Treatment of pulmonary arterial hypertension (WHO group I) in patients with WHO class II or III symptoms to improve exercise capacity and delay clinical worsening
Long-term treatment of acromegalic patients who have had an inadequate response to or cannot be treated with surgery and/or radiotherapy
9/13/07
Raloxifene
Evista
Reduction in risk of invasive breast cancer in postmenopausal women with osteoporosis and reduction in risk of invasive breast cancer in postmenopausal women at high risk for invasive breast cancer
For the use for chronic phase (CP) and accelerated phase (AP) Philadelphia chromosome positive chronic myelogenous leukemia (CML) in adult patients resistant to or intolerant to prior therapy that included imatinib
