By Kurt R. Karst – FDA Law Blog
Hyman, Phelps & McNamara, P.C.
Yesterday, Senator Sam Brownback (R-KS) (along with Sen. Sherrod Brown (D-OH)) introduced S. 3697, the Creating Hope Act of 2010. The bill would amend FDC Act § 524 to change the transferable Priority Review Voucher (“PRV”) program created by the 2007 FDA Amendments Act (the so-called “treat and trade” program), and in particular with respect to rare pediatric diseases. The introduction of S. 3697 comes on the heels of a July 2010 Senate Health, Education, Labor and Pensions Committee hearing, titled “Treating Rare and Neglected Pediatric Diseases: Promoting the Development of New Treatments and Cures,” at which the idea for the bill was reportedly (according to FDA Week) first floated. Both Sens. Brownback and Brown have shown a particular interest in rare and neglected diseases, having sponsored provisions in the Fiscal Year 2010 FDA appropriations bill and spearheaded efforts to have provisions included in the Fiscal Year 2011 appropriations bill (see our previous post here).
As we previously reported (here and here), under FDC Act § 524, applicants for certain new drugs and biologics for “tropical diseases” that have received priority review may receive a PRV 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 To our knowledge, FDA has granted only a single PRV – in connection with the April 2009 approval of NDA No. 22-268 for COARTEM (artemether; lumefantrine) for the treatment of acute, uncomplicated malaria infections in adults and children weighing at least five kilograms.
Among other things included in the 17-page bill, S. 3697 would amend the PRV program to extend it to applications for a “rare pediatric disease” – that is, a disease “recognized in the medical community as affecting a pediatric population” and that is “a rare disease or condition, within the meaning of section 526” (i.e., the Orphan Drug Act). Such an application must be: (1) a “human drug application” (as defined under PDUFA); (2) “for prevention or treatment of a rare pediatric disease;” (3) “that the Secretary deems eligible for priority review;” (4) “that is for an innovative treatment” (a new term defined in the bill); (5) “that relies on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population;” and (6) “that does not seek approval for an adult indication in the original rare pediatric disease product application.”
S. 3697 would also amend the PRV eligibility requirements for tropical disease applications. Currently, in order for a drug product to be eligible for a PRV, four requirements must be met. Under the bill, in addition to clarifying that only an application for an “innovative treatment” will be PRV-eligible, S. 3697 requires that the application “is for a drug that has not been approved for commercial marketing for any tropical disease indication by a government authority outside of the United States for more than 24 months before the tropical disease product application is submitted.”
With respect to PRV use and transferability, S. 3697 clarifies that “[t]here is no limit on the number of times a priority review voucher may be transferred before such voucher is used.” This is consistent with FDA’s interpretation of the current law. Specifically, FDA clarified in a draft guidance document that although FDC Act § 524 allows for only a single actual transfer of a PRV from the original recipient to another sponsor, “contractual arrangements such as the use of an option or transfer of the right to designate the voucher’s recipient could comply with the terms of the statute.” S. 3697 would also amend the law to add new notification requirements, timelines, and user fee procedures.
Finally, the bill would establish a process by which a sponsor can request designation of its product as one for a “rare pediatric disease” or that is an “innovative treatment.” (Perhaps providing a back-door mechanism for a sponsor to learn whether its product would qualify for 5-year new chemical entity exclusivity under the FDC Act or 12-year reference product exclusivity under the PHS Act.) As mentioned above, the term “innovative treatment” is a defined term in the bill – specifically, as:
(A) a human drug that is the subject of an application submitted under section 505(b)(1), if that drug contains no active ingredient (including any ester or salt of the active ingredient) that has been previously approved in any other application under section 505(b)(1), 505(b)(2), or 505(j) or section 351 of the Public Health Service Act; or
(B) a biological product that is the subject of an application submitted under [PHS Act § 351(a)], if that biological product—
(i) does not have the same structure as a biological product that has been previously licensed in any other application under [PHS Act § 351(a) or (k)] or approved under [FDC Act § 505]; and
(ii) is not biosimilar, within the meaning of section [PHS Act § 351(i), to a biological product that has been previously licensed in any other application under [PHS Act § 351(a) or (k)] or approved under [FDC Act § 505]. [(emphasis added)] The limitation of PRV eligibility to 505(b)(1) NDAs is contrary to how FDA has interpreted the current PRV law, which some might say is not entirely clear on whether 505(b)(2) applications are also PRV-eligible. For example, during a December 2008 public hearing concerning additions to the list of tropical diseases identified at FDC Act § 524, FDA noted that 505(b)(2) applications are eligible for PRVs.