The Creating Hope Act Overview

THE CREATING HOPE ACT:  THE RARE PEDIATRIC DISEASE PRIORITY REVIEW VOUCHER (PRV) INCENTIVE PROGRAM
21 U.S.C. 360ff

 

Creating Hope Permanent Reauthorization Act of 2019

The Creating Hope Act passed in 2012, was reauthorized several times, and sunsets September 30, 2020. On September 20, 2019, a bill seeking permanent reauthorization of the Creating Hope Act was introduced in the House of Representatives.

The Creating Hope Act pediatric priority review voucher program:

• Creates an incentive for companies to develop drugs for pediatric rare diseases: a pediatric priority review voucher. Vouchers are fully transferable. Exercise of a voucher entitles a company to faster FDA review of any other future drug, including non-pediatric rare disease drugs.

The pediatric priority review voucher program is wildly successful:

• Vouchers are trading at $100 million to $350 million, creating a significant incentive for pediatric rare disease drug development.
• $2 billion in vouchers have traded, with no cost to taxpayers or patients.
• Since the enactment of the Creating Hope Act, 19 new drugs for children with life threatening illnesses have been approved, including 2 for childhood cancers.
• The voucher incentive is funded by the pharmaceutical industry as companies that earn vouchers by developing pediatric rare disease drugs sell vouchers to other companies

It is indefensible that the pediatric priority review voucher program is the ONLY voucher program that is not permanent:

• The voucher programs for drugs developed to treat tropical diseases and medical countermeasures are permanent.
• The Creating Hope Act voucher program for drugs developed to treat children with life threatening illnesses is not permanent.

For more information, please contact:

Caitlin Van Sant, Rep. G.K. Butterfield, Caitlin.VanSant@mail.house.gov, 202-225-3101
Thomas Rice, Rep. Michael McCaul, Thomas.Rice@mail.house.gov, 202- 225-2401
Nancy Goodman, Kids v Cancer, nancygoodman@kidsvcancer.org,  646-361-3590

Background on the Creating Hope Act
In the 20 years before the enactment of the Creating Hope Act, there had been only two drugs developed to expressly treat a pediatric cancer that were initially FDA approved for kids:  Erwinase and Clofarabine.

Under the Creating Hope Act, companies that develop drugs for kids with cancer and other life-threatening diseases receive a voucher from the FDA. The voucher gives rights to faster FDA review of any future drug. And the voucher is transferable.  The  vouchers have sold for up to $350 million; over $1 billion in vouchers have been sold.

By enacting the Creating Hope Act pediatric PRV program – the Rare Pediatric Disease Priority Review Voucher Program, 21 U.S.C. 360ff  — Congress created a market incentive for the development of drugs for rare pediatric diseases through the establishment of a priority review voucher.  Under this program, a sponsor that develops a drug or biologic for a rare pediatric disease and receives FDA approval for that drug or biologic also receives a voucher.  That voucher comes with rights to FDA priority review for any other drug or biologic, including a large-market adult drug or biologic that would otherwise receive standard review.  This would result in the other drug or biologic getting to market many months earlier, creating significant value.  The voucher is fully transferable.

The Creating Hope Act voucher is based upon a neglected tropical disease priority review voucher program that was signed into law as a provision of the Food and Drug Administration Amendments Act of 2007.

The U.S. Senate passed the 21st Century Cures Act and with it, reauthorization of the Creating Hope Act pediatric priority review voucher program on December 7, 2016, voting 94-5. Thank you to our sponsors, Congressmen G.K. Butterfield, Michael McCaul and Chris Van Hollen, and Senators Bob Casey and Johnny Isakson, for their commitment to children with cancer and other rare diseases. Thank you to the FDA for executing this critical program.

Pursuant to the 21st Century Cures, the Creating Hope Act pediatric PRV is reauthorized until September 20, 2020.  Moreover, drugs that receive rare pediatric designations by that date will qualify for vouchers as long as they are approved by September 20, 2022.

FDA Draft Guidance

In July 2019 FDA updated its guidance on the Creating Hope Act Priority Review Vouchers, entitled: Rare Pediatric Disease Priority Review Vouchers, Guidance for Industry, which the agency initially issued in November 2014.

For companies developing pediatric rare disease drugs: 
The criteria for a Rare Pediatric Disease Product Application leading to a pediatric voucher are that a drug or biological product:

• Is for the prevention or treatment of a Rare Pediatric Disease;
• Contains no active ingredient that has been previously approved in any other application;
• Is submitted under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act;
• Itself qualifies for priority review;
• Relies on clinical data derived from studies examining a pediatric population and dosages of the drug intended for a pediatric population.

The term “rare pediatric disease” means a disease that is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years.

The Creating Hope Act provides for a rare pediatric designation process to create greater certainty earlier in the drug development process that a drug or biologic would qualify for a voucher, should it be approved.  The request for a rare pediatric designation can be made at the same time or after a request for an orphan designation is made.

For companies wishing to exercise a voucher: 
A sponsor who wishes to exercise a priority review voucher would likely have a large-market adult drug or biologic that would otherwise be reviewed by the FDA pursuant to a standard review.  By exercising a priority review voucher, the sponsor would then be able to receive a priority review for that drug or biologic.  This would get the drug or biologic to market more quickly, perhaps ahead of a similar competing drug or biologic, thereby generating significant value.

The priority review voucher program does not alter the criteria for a FDA approval.  Drugs subject to priority review must still conform to all the FDA requirements to be approved.

A sponsor of a drug or biologic application that is the subject of a priority review voucher shall pay a user fee.  The amount of the user fee shall be based on the difference between the average cost incurred by the FDA of a priority review and the average cost incurred by the FDA of a standard review.  This amount shall be adjusted annually to reflect current costs. For FY2020 the fee rate is $2,167,116,  which is the lowest amount since FY2011, when the Food and Drug Administration Amendments Act created the fees for redeeming PRVs, and which is significantly less than the $2,457,140 fee in FY2019.

The Creating Hope Act provides that the sponsor must notify FDA of its intent to submit a drug application with a priority review voucher at least 90 days prior to submission.  The user fee shall be due upon the notification.

Pursuant to the Creating Hope Act, the FDA may revoke a priority review voucher awarded if the Rare Pediatric Disease Product Application for which such voucher was awarded is not marketed in the United States within a year.