Senate FY 2011 FDA Appropriations Bill Should be Another Big Step Forward for Rare and Neglected Disease Patients and Advocates

By Kurt R. Karst –   

As the U.S. Senate Committee on Appropriations begins its markup and consideration of appropriations bills for Fiscal Year (“FY”) 2011, and in particular the Agriculture, Rural Development, FDA, and Related Agencies Appropriations bill (S. 3606), rare and neglected disease (i.e., orphan disease) advocates appear to be poised to make big gains (once again!). 

The FY 2011 Senate bill includes a $2 million increase (for a total of  $16,035,000) for FDA’s Orphan Product Development Grant program.  The FY 2010 funding for the program was about $14 million, of which approximately $10 million funded noncompeting continuation awards, and approximately $4 million of which funded 10 to 12 new awards.  The FY 2011 increase is the first since FY 2005.

The FY 2011 Senate bill also includes funding of $1 million for the new Office of the Associate Director for Rare Diseases in the Office of New Drugs in FDA’s Center for Drug Evaluation and Research (“CDER”).  FDA announced the position of Associate Director for Rare Diseases in February 2010.  This orphan drug czar “will serve as CDER’s focal point to the rare disease drug development community and assist stakeholders and developers of drug and biologic products in navigating the complex regulatory requirements for bringing safe and effective treatments to patients in need.”  The $1 million funding will be used to hire additional staff with specific expertise in facilitating the development and review of products to treat rare and neglected diseases. 

Finally, we understand that the manager’s package that will hopefully be adopted by the Appropriations Committee will include the provisions below, which are intended to build on § 740 of the Agriculture, Rural Development, FDA, and Related Agencies Appropriations Act of 2010 (Pub. L. No. 111-80) co-sponsored by Senators Sam Brownback (R-KS) and Sherrod Brown (D-OH).  As we previously reported, the Brownback/Brown amendment requires FDA to convene a committee of expert Agency employees to consider the ways FDA reviews products to treat people with rare and neglected diseases, and consider policy improvements that might help people with rare diseases get better treatments faster.

The proposed language in the manager’s package (tentatively identified as § 741 in Senate Report 111-221 accompanying S. 3606) states:

Sec. __. (a) When implementing the authority provided in paragraphs (2) and (3) of section 740(c) of the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act, 2010 (Public Law 111-80) that requires the Commissioner of Food and Drugs to develop updated guidance documents and review standards for the development of safe and effective products to treat rare diseases and neglected tropical diseases, the Commissioner shall —

(1) maximize the use of accelerated approval where feasible and appropriate;

(2) work with sponsors to facilitate expanded access to investigational therapies;

(3) increase coordination and interaction with the World Health Organization, European Medicines Agency, and other international regulatory agencies;

(4) implement mechanisms for enhanced collaboration between the Food and Drug Administration and National Regulatory Authorities in developing countries;

(5) develop guidance on clinical development programs for rare diseases;

(6) develop guidance on the use of surrogate endpoints that are reasonably likely to predict clinical benefit of drugs and biological products under the regulations under subpart H of part 314 of title 21, Code of Federal Regulations and subpart E of part 601 of title 21, Code of Federal Regulations; and

(7) increase coordination among individual drug, biological product, and device review divisions across Food and Drug Administration centers to support the development of safe and effective medical products for rare and neglected diseases.

(b) The Commissioner of Food and Drugs shall submit a report to the Committee on Appropriations of the Senate and the Committee on Appropriations of the House of Representatives not later than 180 days after the report required in section 740(c)(1) of the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act, 2010 (Public Law 111–80) is submitted: Provided, That the report submitted in response to this section shall describe in detail how the Food and Drug Administration is implementing subsection (a).

The latest legislative push to address rare and neglected disease issues has been spearheaded by the National Organization for Rare Disorders (“NORD”) and Dr. Emil Kakkis’s Kakkis EveryLife Foundation.  NORD has issued a press relase on the FY 2011 appropriations bill.  The NORD Board of Directors is chaired by Hyman, Phelps & McNamara, P.C. Director Frank J. Sasinowski.

There have been several events leading up to the appropriations bill language.  On June 23, 2010, the Senate Appropriations Committee held a hearing to discuss FDA’s review process for products to treat rare and neglected diseases.  That hearing was followed up by a two-day FDA public hearing on the same topic (see our previous post here).  In addition, Representatives Joseph Crowley (D-NY) and Fred Upton (R-MI) recently announced the establishment of the Rare and Neglected Diseases Caucus.