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 In this section:

                Designating an Orphan Product: Drugs and Biological Products

Designating an Orphan Product: Drugs and Biological Products

                        Orphan Drug Designation Request Form
          Orphan Drug Act - Relevant Excerpts
          Frequently Asked Questions (FAQ) About Designating an Orphan Product
          Orphan Drug Designation: Disease Considerations
          Clinical Superiority Findings
          Instructions for Searchable Designation Database
          Public Identification of Orphan Drug Designation
          Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases; FDA Report to Congress
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          Designating an Orphan Product: Drugs and Biological Products
          Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases; FDA Report to CongressDesignating an Orphan Product: Drugs and Biological Products Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases; FDA Report to Congress
  
  	
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Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases

In Response to: Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act, 2010, Public Law 111-80, Section 740

Department of Health and Human Services,
Food and Drug Administration
March 2011

INTRODUCTION

Section 740 of the fiscal year (FY) 2010 Appropriation Act (Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriation Act, 2010, Public Law 111-80), dated October 21, 2009, required the Commissioner, Food and Drug Administration (FDA), to establish two review groups within FDA to make recommendations to the FDA Commissioner on appropriate preclinical, trial design, and regulatory paradigms and optimal solutions to prevent, diagnosis, and treat (1) rare diseases and (2) neglected diseases of the developing world. Section 740 requires these groups to submit a report to Congress that describes their findings and recommendations.



Section 740 of the Appropriations Act
	(a) The Commissioner of Food and Drugs shall establish within the Food and Drug Administration a review group which shall recommend to the Commissioner of Food and Drugs appropriate preclinical, trial design, and regulatory paradigms and optimal solutions for the prevention, diagnosis, and treatment of rare diseases: Provided, That the Commissioner of Food and Drugs shall appoint individuals employed by the Food and Drug Administration to serve on the review group: Provided further, That members of the review group shall have specific expertise relating to the development of articles for use in the prevention, diagnosis, or treatment of rare diseases, including specific expertise in developing or carrying out clinical trials.

(b) The Commissioner of Food and Drugs shall establish within the Food and Drug Administration a review group which shall recommend to the Commissioner of Food and Drugs appropriate preclinical, trial design, and regulatory paradigms and optimal solutions for the prevention, diagnosis, and treatment of neglected diseases of the developing world: Provided, That the Commissioner of Food and Drugs shall appoint individuals employed by the Food and Drug Administration to serve on the review group: Provided further, That members of the review group shall have specific expertise relating to the development of articles for use in the prevention, diagnosis, or treatment of neglected diseases of the developing world, including specific expertise in developing or carrying out clinical trials: Provided further, That for the purposes of this section the term ‘‘neglected disease of the developing world’’ means a tropical disease, as defined in section 524(a)(3) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360n(a)(3))

(c) The Commissioner of Food and Drugs shall (1) submit, not later than 1 year after the date of the establishment of review groups under subsections (a) and (b), a report to Congress that describes both the findings and recommendations made by the review groups under subsections (a) and (b); (2) issue, not later than 180 days after submission of the report to Congress under paragraph (1), guidance based on such recommendations for articles for use in the prevention, diagnosis, and treatment of rare diseases and for such uses in neglected diseases of the developing world; and (3) develop, not later than 180 days after submission of the report to Congress under paragraph (1), internal review standards based on such recommendations for articles for use in the prevention, diagnosis, and treatment of rare diseases and for such uses in neglected diseases of the developing world.

                                                                                  Content current as of:
      02/26/2019Designating an Orphan Product: Drugs and Biological Products

          
                        Orphan Drug Designation Request Form
          Orphan Drug Act - Relevant Excerpts
          Frequently Asked Questions (FAQ) About Designating an Orphan Product
          Orphan Drug Designation: Disease Considerations
          Clinical Superiority Findings
          Instructions for Searchable Designation Database
          Public Identification of Orphan Drug Designation
          Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases; FDA Report to Congress
          Contact Us
 
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