Posted: 23 Mar 2015 06:49 PM PDT
By Alexander J. Varond & Josephine M. Torrente –
On March 17, 2015 FDA approved Cholbam (cholic acid) for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders. In approving Cholbam, FDA also issued the third rare pediatric disease priority review voucher (“Pediatric Voucher”), triggering the Pediatric Voucher program’s 1-year sunset clause in Section 529(b)(5) of the FD&C Act.
Recall that a Pediatric Voucher is a voucher issued to the sponsor of a “rare pediatric disease product application” that entitles the holder of such voucher to priority review (instead of a longer standard review) of a single NDA or BLA after the date of approval of the rare pediatric disease product application. A qualifying rare pediatric disease product application is an NDA or BLA for a drug or biologic intended to prevent or treat a rare pediatric disease. Such drug or biologic may not contain any active ingredient (including any ester or salt of the active ingredient) previously approved in any drug or biologic application. The application must also be deemed eligible for priority review and rely on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population. A sponsor cannot seek an adult indication as part of a rare pediatric disease application.
FDA defines a “rare pediatric disease” similar to that as a “rare disease,” but in pediatric patients. That is, a “rare pediatric disease” is a disease that affects fewer than 200,000 individuals primarily aged from birth to 18 years in the U.S. The definition also extends to diseases that affect more than 200,000 individuals primarily aged from birth to 18 years in the U.S. but for which no drug or biologic treatments are available because a company cannot reasonably expect to recover the costs of developing and marketing such products. We blogged about FDA’s recently released Draft Guidance on the Pediatric Voucher program here.
Specifically, Section 529(b)(5) states:
The triggering of the sunset clause also means that the General Accountability Office will be required to submit a report to Congress on the program within a year. Independent of GAO’s report, the triggering of the sunset clause will certainly attract attention from Congress. It may spur legislation to reauthorize the program, either as stand-alone legislation or as part of the 21st Century Cures legislation currently being discussed. We discussed the 21st Century Cures legislation here, here, here, andhere.
As a recap:
It is at least somewhat ironic that--as the Pediatric Voucher program begins to wind down (unless it is reauthorized)--only 3 months ago, the tropical disease priority review voucher (“Tropical Disease Voucher”) program received a major boost. This boost happened on December 16, 2014 when President Obama signed the Adding Ebola to the FDA Priority Review Voucher Program Act (“Adding Ebola Act”). We previously discussed the Tropical Disease Voucher program here and here.
The Adding Ebola Act added Filoviruses, including Ebola, to the list of tropical diseases eligible for Tropical Disease Vouchers. It also brought several of the Tropical Disease Voucher program’s features in line with the newer Pediatric Voucher program. These changes are listed below:
Perhaps the most important change made in the Adding Ebola Act was that FDA is now able to add diseases to the list of eligible diseases by order, rather than rulemaking. This makes it substantially easier for FDA to respond to emergencies and add diseases that have long been discussed as being good candidates for addition to the list (e.g., Chagas disease).
On March 17, 2015 FDA approved Cholbam (cholic acid) for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders. In approving Cholbam, FDA also issued the third rare pediatric disease priority review voucher (“Pediatric Voucher”), triggering the Pediatric Voucher program’s 1-year sunset clause in Section 529(b)(5) of the FD&C Act.
Recall that a Pediatric Voucher is a voucher issued to the sponsor of a “rare pediatric disease product application” that entitles the holder of such voucher to priority review (instead of a longer standard review) of a single NDA or BLA after the date of approval of the rare pediatric disease product application. A qualifying rare pediatric disease product application is an NDA or BLA for a drug or biologic intended to prevent or treat a rare pediatric disease. Such drug or biologic may not contain any active ingredient (including any ester or salt of the active ingredient) previously approved in any drug or biologic application. The application must also be deemed eligible for priority review and rely on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population. A sponsor cannot seek an adult indication as part of a rare pediatric disease application.
FDA defines a “rare pediatric disease” similar to that as a “rare disease,” but in pediatric patients. That is, a “rare pediatric disease” is a disease that affects fewer than 200,000 individuals primarily aged from birth to 18 years in the U.S. The definition also extends to diseases that affect more than 200,000 individuals primarily aged from birth to 18 years in the U.S. but for which no drug or biologic treatments are available because a company cannot reasonably expect to recover the costs of developing and marketing such products. We blogged about FDA’s recently released Draft Guidance on the Pediatric Voucher program here.
Specifically, Section 529(b)(5) states:
TERMINATION OF AUTHORITY.—The Secretary may not award any priority review vouchers under paragraph (1) after the last day of the 1-year period that begins on the date that the Secretary awards the third rare pediatric disease priority voucher under this section.Because the sunset clause was triggered on March 17, 2015, FDA will not issue Pediatric Vouchers to drugs approved after March 16, 2016. We note that, depending on FDA’s calculations, May 17, 2016 may actually be the applicable date.
The triggering of the sunset clause also means that the General Accountability Office will be required to submit a report to Congress on the program within a year. Independent of GAO’s report, the triggering of the sunset clause will certainly attract attention from Congress. It may spur legislation to reauthorize the program, either as stand-alone legislation or as part of the 21st Century Cures legislation currently being discussed. We discussed the 21st Century Cures legislation here, here, here, andhere.
As a recap:
- BioMarin received the first Pediatric Voucher on February 14, 2014 for Vimizim (elosulfase alfa) for Mucopolysaccharidosis Type IVA (Morquio A syndrome). We blogged about it here.
- United Therapeutics received the second Pediatric Voucher on March 10, 2015 for Unituxin (dinutuximab) for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy. We blogged about it here.
It is at least somewhat ironic that--as the Pediatric Voucher program begins to wind down (unless it is reauthorized)--only 3 months ago, the tropical disease priority review voucher (“Tropical Disease Voucher”) program received a major boost. This boost happened on December 16, 2014 when President Obama signed the Adding Ebola to the FDA Priority Review Voucher Program Act (“Adding Ebola Act”). We previously discussed the Tropical Disease Voucher program here and here.
The Adding Ebola Act added Filoviruses, including Ebola, to the list of tropical diseases eligible for Tropical Disease Vouchers. It also brought several of the Tropical Disease Voucher program’s features in line with the newer Pediatric Voucher program. These changes are listed below:
Feature | Old Tropical Disease Voucher | Pediatric Voucher | New Tropical Disease Voucher |
Limit on # of transfers? | Yes; limited to 1 transfer | No; no limit | No; no limit |
Notice required before use of voucher on subsequent application | 1 year | 90 days | 90 days |
Method of determining whether a disease is eligible for a voucher | Required lengthy rulemaking to add new disease to tropical disease program | Determinations made on a case-by-case basis | FDA can now add diseases to list of eligible disease by order |
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