Estimating the clinical cost of drug development for orphan versus non-orphan drugs

Research and Development Spending to Bring a Single Cancer Drug to Market and Revenues After Approval

(2017) Vinay Prasad et al.   JAMA Internal Medicine

Innovation in the pharmaceutical industry: New estimates of R&D costs

(2016) Joseph A. DiMasi et al.   JOURNAL OF HEALTH ECONOMICS

The Evolving Drug Development Landscape: From Blockbusters to Niche Busters in the Orphan Drug Space

(2014) Ashish Kumar Kakkar et al.   DRUG DEVELOPMENT RESEARCH

Clinical development success rates for investigational drugs

(2014) Michael Hay et al.   NATURE BIOTECHNOLOGY

A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov

(2014) Stuart A Bell et al.   Orphanet Journal of Rare Diseases

The Pricing of Breakthrough Drugs: Theory and Policy Implications

(2014) Moshe Levy et al.   PLoS One

Pricing for Orphan Drugs

(2013) Brian P. O’Sullivan et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Orphan drug development: an economically viable strategy for biopharma R&D

(2012) Kiran N. Meekings et al.   DRUG DISCOVERY TODAY

Characteristics of Clinical Trials Registered in ClinicalTrials.gov, 2007-2010

(2012) Robert M. Califf   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Pricing and reimbursement of orphan drugs: the need for more transparency

(2011) Steven Simoens   Orphanet Journal of Rare Diseases

Rare diseases, orphan drugs and their regulation: questions and misconceptions

(2010) Erik Tambuyzer   NATURE REVIEWS DRUG DISCOVERY

Pivotal studies of orphan drugs approved for neurological diseases

(2009) Jun Mitsumoto et al.   ANNALS OF NEUROLOGY

Does orphan drug legislation really answer the needs of patients?

(2008) Marlene E Haffner et al.   LANCET