Senate and House Conferees Agree on Rare and Neglected Diseases Language

By Kurt R. Karst –      

We previously reported on a Senate floor amendment to the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act of 2010 (H.R. 2997) co-sponsored by Senators Sam Brownback (R-KS) and Sherrod Brown (D-OH) that would establish within FDA two new review groups to recommend solutions for the prevention, diagnosis, and treatment of rare diseases and neglected diseases of the developing world.  Earlier this week, after Senate and House conferees hammered out a compromise bill, a Conference Report was released that retains the Brown/Brownback amendment, albeit with some changes.

The amendment, which is reprinted below, and is now included as Section 740 of the bill, requires FDA to establish within the Agency “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,” and another group that is required to do the same with respect to neglected diseases of the developing world.  The previous version of the amendment stated that FDA may establish the groups.  Also, whereas the previous version of the amendment stated that each review group would be composed of 8 FDA employees, the conference version omits any reference to the number of FDA employees in each review group.  It is unclear whether any additional guidance will be given as to the composition of each review group.

SEC. 740. (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.