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Proposed 'grant-and-access' program with price caps could stimulate development of drugs for very rare diseases.

Publication ,  Journal Article
Valverde, AM; Reed, SD; Schulman, KA
Published in: Health Aff (Millwood)
November 2012

The 1983 Orphan Drug Act created incentives for the development of orphan drugs. Despite its successes, including a substantial increase in new drugs, approved orphan drugs still treat fewer than 5 percent of registered rare diseases. In addition, concerns have arisen about the high prices of many of these therapies, which can cost hundreds of thousands of dollars per patient each year. In this article, we propose a new "grant-and-access pathway," in which drug developers could opt to compete for federal grants to subsidize the costs of clinical testing. In return for the grant funding, companies would no longer claim orphan drug tax credits and would agree to price caps for marketed products based on the duration and costs associated with drug development, expected market size, and target rate of return. We identify scenarios in which such a policy could provide a net benefit to society.

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Published In

Health Aff (Millwood)

DOI

EISSN

1544-5208

Publication Date

November 2012

Volume

31

Issue

11

Start / End Page

2528 / 2535

Location

United States

Related Subject Headings

  • United States Food and Drug Administration
  • United States
  • Rare Diseases
  • Program Evaluation
  • Orphan Drug Production
  • Humans
  • Health Resources
  • Health Policy & Services
  • Health Care Rationing
  • Financing, Organized
 

Citation

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Valverde, A. M., Reed, S. D., & Schulman, K. A. (2012). Proposed 'grant-and-access' program with price caps could stimulate development of drugs for very rare diseases. Health Aff (Millwood), 31(11), 2528–2535. https://doi.org/10.1377/hlthaff.2012.0235
Valverde, Ana M., Shelby D. Reed, and Kevin A. Schulman. “Proposed 'grant-and-access' program with price caps could stimulate development of drugs for very rare diseases.Health Aff (Millwood) 31, no. 11 (November 2012): 2528–35. https://doi.org/10.1377/hlthaff.2012.0235.
Valverde AM, Reed SD, Schulman KA. Proposed 'grant-and-access' program with price caps could stimulate development of drugs for very rare diseases. Health Aff (Millwood). 2012 Nov;31(11):2528–35.
Valverde, Ana M., et al. “Proposed 'grant-and-access' program with price caps could stimulate development of drugs for very rare diseases.Health Aff (Millwood), vol. 31, no. 11, Nov. 2012, pp. 2528–35. Pubmed, doi:10.1377/hlthaff.2012.0235.
Valverde AM, Reed SD, Schulman KA. Proposed 'grant-and-access' program with price caps could stimulate development of drugs for very rare diseases. Health Aff (Millwood). 2012 Nov;31(11):2528–2535.

Published In

Health Aff (Millwood)

DOI

EISSN

1544-5208

Publication Date

November 2012

Volume

31

Issue

11

Start / End Page

2528 / 2535

Location

United States

Related Subject Headings

  • United States Food and Drug Administration
  • United States
  • Rare Diseases
  • Program Evaluation
  • Orphan Drug Production
  • Humans
  • Health Resources
  • Health Policy & Services
  • Health Care Rationing
  • Financing, Organized