Efficacy and costs of spinal muscular atrophy drugs

Sci Transl Med. 2020 Nov 11;12(569):eaay9648. doi: 10.1126/scitranslmed.aay9648.

Jonathan J Darrow¹, Monica Sharma², Mansa Shroff³, Anita K Wagner^{4

5}

¹ Program on Regulation, Therapeutics, and Law, Brigham and Women's Hospital and Harvard Medical School, Boston, MA 02120, USA. jjdarrow@bwh.harvard.edu.
² IP-Dhyaan LLP, Dwarka, New Delhi 110075, India.
³ Harvard T.H. Chan School of Public Health, Boston, MA 02115, USA.
⁴ Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, MA 02215, USA.
⁵ Harvard Pilgrim Health Care Inc., Wellesley, MA 02481, USA.

Abstract

Evaluating the benefits, risks, and costs of two drugs to treat spinal muscular atrophy raises questions about the future of rare disease medicines.