Senate Agriculture/FDA Appropriations Bill Amendment Seeks to Streamline Development and Regulation of Products for Rare and Neglected Diseases

By Kurt R. Karst & Frank J. Sasinowski –      

A Senate floor amendment to the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act of 2010 (H.R. 2997) 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.  The amendment, reportedly backed by recently-confirmed NIH Director Dr. Francis Collins, and co-sponsored by Senators Sam Brownback (R-KS) and Sherrod Brown (D-OH), was first proposed in the Senate in July, and was agreed to on August 3; however, we understand that the amendment could be further changed in Conference Committee. 

The Brown/Brownback Amendment follows recent significant discussions about how to improve the process for developing and approving products for rare and neglected diseases.  In May 2009, the National Organization for Rare Disorders (“NORD”) held a “Partners in Progress Summit” at which thought leaders, including Dr. Collins, several FDAers, and Hyman, Phelps & McNamara, P.C.’s Frank Sasinowski, discussed ways to encourage innovative research and ensure that patients have access to treatments for rare disorders.  In addition, the non-profit Kakkis EveryLife Foundation has advocated for a new Office of Drug Evaluation for Rare Biochemical and Genetic Disorders as part of a larger science driven regulatory policy approach to accelerate treatments for rare diseases.  Dr. Emil Kakkis, President of the Foundation, told FDA Law Blog that “We are pleased with the support of Dr. Collins and Sen. Brownback in advocating for practical changes at the FDA, and with the broad support we have received from others for our initiatives.”  

Under the Brown/Brownback Amendment, which is included as Section 745 in H.R. 2997, the FDA Commissioner may 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.”  The rare disease review group would be composed of 8 FDA employees who must 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.”

With respect to “neglected diseases of the developing world” (i.e., “tropical diseases” as defined in FDC Act § 524(a)(3) concerning priority review vouchers), the FDA Commissioner may 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 neglected diseases of the developing world.”  The neglected diseases review group would also be composed of 8 FDA employees, but who have “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.”

The Brown/Brownback Amendment also requires FDA to submit a report to Congress describing the Agency’s findings and recommendations of the rare and neglected disease review groups, and to issue guidance and develop internal review standards based on their recommendations.

The Brown/Brownback Amendment appears to be a more evolved and specific version of language included in the Senate Report accompanying the version of the Agriculture/FDA Appropriations Bill introduced in the Senate (S. 1406).  The Senate Report accompanying S. 1406 stated:

Neglected Diseases.— The Committee is concerned about the challenge of increasing the number of approved treatments for diseases that, although not necessarily rare, may have few if any therapeutic options.  The Committee recognizes that the definition of a rare disease or condition under the Orphan Drug Act includes many tropical diseases or conditions that affect more than 200,000 persons in the United States.

Because the Orphan Drug Act already embraces therapies to treat many tropical diseases, the Committee urges FDA to take active steps to stimulate orphan status and support their development.  Where appropriate, FDA should engage in partnerships and collaborations to identify compounds that may be suitable to treat this subset of orphan diseases and work in a proactive way to identify compounds to treat such diseases.

Sens. Brown and Brownback have a longstanding interest in backing neglected disease product development.  They are the authors of  FDC Act § 524 – “Priority Review to Encourage Treatments for Tropical Diseases” – that was added to the statute by the 2007 FDA Amendments Act.  FDC Act § 524 provides for a transferable priority review program – the so-called “treat and trade” program – in which applicants for certain new drugs and biologics for “tropical diseases” that have received priority review may receive a priority review voucher entitling the holder to a 6-month priority FDA review of another application that would otherwise be reviewed under FDA’s standard 10-month review clock.  As we previously reported,  FDA granted the first priority review voucher earlier this year when it approved COARTEM (artemether; lumefantrine) for the treatment of acute, uncomplicated malaria infections in adults and children weighing at least five kilograms.