Table 2 Drug plan listings for 33 unique rare disorder drugs with successful price negotiations
Generic (brand) names | Clinical indication | Date most recent price negotiation completed | Drug plan listingsa | |
|---|---|---|---|---|
No | % | |||
Riociguat (Adempas) | Pulmonary hypertension | 1/2015 | 9 | 81.8 |
Stiripentol (Diacomit) | Dravet syndrome | 5/2015 | 10 | 90.9 |
Icatibant (Firazyr) | Hereditary angioedema | 8/2015 | 11 | 100.0 |
Nintedanib (Ofev) | Idiopathic pulmonary fibrosis | 8/2016 | 11 | 100.0 |
Pirfenidone (Esbriet) | Idiopathic pulmonary fibrosis | 9/2016 | 11 | 100.0 |
Sodium phenylbutyrate (Pheburane) | Urea cycle disorders | 11/2017 | 8 | 72.7 |
Selexipag (Uptravi) | Pulmonary arterial hypertension | 12/2017 | 10 | 90.9 |
Glycerol phenylbutyrate (Ravicti) | Urea cycle disorders | 12/2017 | 10 | 90.9 |
Asfotase alfa (Strensiq) | Hypophosphatasia | 1/2018b | 5 | 45.4 |
Taliglucerase alfa (Elelyso) | Gaucher disease | 5/2018 | 4 | 36.4 |
Obeticholic acid (Ocaliva) | Primary biliary cholangitis | 6/2018 | 11 | 100.0 |
Cysteamine (Procysbi) | Nephropathic cystinosis | 7/2018 | 7 | 63.6 |
Migalastat (Galafold) | Fabry disease | 8/2018 | 5 | 45.4 |
Nitisinone (Orfadin and generics) | Tyrosinemia type 1 | 11/2018 | 6 | 54.5 |
Elosulfase alfa (Vimizim) | Mucopolysaccharidosis IVA | 11/2018c | 3 | 27.3 |
Tocilizumab (Actemra) | Giant cell arteritis | 12/2018 | 8 | 72.7 |
Nusinersen (Spinraza) | Spinal muscular atrophy | 6/2019 | 7 | 63.6 |
Ivacaftor (Kalydeco) | Cystic fibrosis gating mutations | 7/2019b | 8 | 72.7 |
Mercaptamine (Cystadrops) | Cystinosis | 8/2019 | 6 | 54.5 |
Cerliponase alfa (Brineura) | Neuronal ceroid lipofuscinosis type 2 | 1/2020 | 4 | 36.4 |
Sapropterin (Kuvan) | Phenylketonuria | 2/2020 | 5 | 45.4 |
Edaravone (Radicava) | Amyotrophic lateral sclerosis | 4/2020 | 10 | 90.9 |
Inotersen (Tegsedi) | Transthyretin amyloidosis | 4/2020 | 7 | 63.6 |
Teduglutide (Revestive) | Short bowel syndrome | 10/2020 | 7 | 63.6 |
Lanadelumab (Takhzyro) | Hereditary angioedema | 10/2020 | 7 | 63.6 |
Sebelipase alfa (Kanuma) | Lysosomal acid lipase deficiency | 10/2020 | 6 | 54.5 |
Patisiran (Onpattro) | Transthyretin amyloidosis | 11/2020 | 8 | 72.7 |
Macitentan (Opsumit) | Pulmonary arterial hypertension | 12/2020c | 6 | 54.5 |
Tafamidis (Vyndaqel) | Transthyretin amyloidosis | 2/2021 | 8 | 72.7 |
Lumacaftor/ivacaftor (Orkambi) | Cystic fibrosis, F508del mutation | 6/2021d | 6 | 54.5 |
Elexacaftor/tezacaftor/ ivacaftor (Trikafta) | Cystic fibrosis, F508del mutation | 9/2021 | 9 | 81.8 |
Burosumab (Crysvita) | Hypophosphatemia (X-linked) | 9/2021 | 3 | 27.3 |
Onasemnogene abeparvovec (Zolgensma) | Spinal muscular atrophy | 10/2021 | 2 | 18.2 |