Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage

The High Cost of Prescription Drugs in the United States

(2016) Aaron S. Kesselheim et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Indication-Specific Pricing for Cancer Drugs

(2014) Peter B. Bach   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Regulators adopt more orphan drugs

(2014) Sara Reardon   NATURE

When Everyone Is an Orphan: Against Adopting a U.S.-Styled Orphan Drug Policy in Canada

(2013) Matthew Herder   Accountability in Research-Policies and Quality Assurance

Prevalence of Off-Label Use and Spending in 2010 Among Patent-Protected Chemotherapies in a Population-Based Cohort of Medical Oncologists

(2013) Rena M. Conti et al.   JOURNAL OF CLINICAL ONCOLOGY

The Prevalence and Cost of Unapproved Uses of Top-Selling Orphan Drugs

(2012) Aaron S. Kesselheim et al.   PLoS One

Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer

(2011) Aaron S. Kesselheim   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Pivotal studies of orphan drugs approved for neurological diseases

(2009) Jun Mitsumoto et al.   ANNALS OF NEUROLOGY

The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?

(2009) Olivier Wellman-Labadie et al.   HEALTH POLICY

Off-Label Promotion, On-Target Sales

(2008) Adriane Fugh-Berman et al.   PLOS MEDICINE