The Regulatory Repercussions of Approving Muscular Dystrophy Medications on the Basis of Limited Evidence-Reference-Cited by-同舟云学术

The Regulatory Repercussions of Approving Muscular Dystrophy Medications on the Basis of Limited Evidence

Published:2023-09 Issue:9 Volume:176 Page:1251-1256
ISSN:0003-4819
Container-title:Annals of Internal Medicine
language:en
Short-container-title:Ann Intern Med

Author:

Bendicksen Liam¹^ORCID,Zuckerman Diana M.²^ORCID,Avorn Jerry³^ORCID,Phillips Sophia²,Kesselheim Aaron S.³^ORCID

Affiliation:

1. Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital, Boston, Massachusetts (L.B.)

2. National Center for Health Research, Washington, DC (D.M.Z., S.P.)

3. Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital, and Harvard Medical School, Boston, Massachusetts (J.A., A.S.K.).

Funder

Arnold Ventures

Publisher

American College of Physicians

Subject

General Medicine,Internal Medicine

Reference13 articles.

1. Approving a Problematic Muscular Dystrophy Drug

2. Regarding “Eteplirsen for the treatment of Duchenne muscular dystrophy”

3. Association of Conflicts of Interest for Public Speakers for the Peripheral and Central Nervous System Drugs Advisory Committee of the US Food and Drug Administration With Their Statements

4. Patient-Centered Drug Approval: The Role of Patient Advocacy in the Drug Approval Process

Cited by 3 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. The FDA and Gene Therapy for Duchenne Muscular Dystrophy;JAMA;2024-05-28

2. Spending on Targeted Therapies for Duchenne Muscular Dystrophy;JAMA;2024-04-02

3. Characteristics of Patients Receiving Novel Muscular Dystrophy Drugs in Trials vs Routine Care;JAMA Network Open;2024-01-24