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The Creating Hope Act Reauthorization Act of 2019 (H.R. 4439)

The Creating Hope Act established the rare pediatric disease priority review voucher program (PRV) which incentivizes the pharmaceutical industry to develop drugs for children with cancer and other life-threatening illnesses. The Creating Hope Act was passed in 2012 and was re-authorized until 2020 as a part of 21st Century Cures.

The Creating Hope Reauthorization of 2019 would permanently reauthorize this life-saving program.

Why the Creating Hope Act is Needed:

  • Pediatric cancer remains the number one disease killer of American children.
  • While survival rates have improved for some types of pediatric cancers, thousands of children are lost to cancer each year and many more encounter life threatening complications related to harsh chemotherapies.
  • Since 1980, the FDA has approved only four new drugs for treatment of childhood cancer, compared to dozens for adults.
  • Despite this significant need, pharmaceutical companies have been reluctant to develop drugs for children with cancer because the markets are so small.

How the Pediatric Rare Disease Works:

  • If a drug sponsor gets a drug targeting a rare pediatric disease approved the FDA can award it a PRV which the drug sponsor can keep or sell (thus earning revenue it can reinvest into additional products) and entitles the PRV redeemer to a 6-month priority review (rather than 10 months standard) for a drug of its choice.
  • Drug sponsors redeeming the PRV also pay a special user fee to cover any of FDA’s additional costs that are incurred because of the priority review.
  • The GAO report notes that FDA confirmed that PRV reviews account for less than 1 percent of all drug application reviews in any given year.
  • If the sponsor receiving the PRV does not bring its pediatric rare disease therapy to market within 1 year of approval the FDA can revoke the PRV.
  • Note: There are also voucher programs for medical countermeasure drugs and neglected tropical disease drugs. The medical countermeasures PRV program will expire on October 1, 2023 and the neglected tropical disease PRV is permanent.

The Pediatric Rare Disease PRV is Working:

  • Of the 31 PRVs awarded by FDA, 19 have been for pediatric rare diseases.
  • Since the creation of the Pediatric Rare Disease PRV, the FDA has awarded vouchers for two pediatric cancer drugs: Unituxin (for neuroblastoma) and Kymriah (for leukemia).
  • $2 billion in vouchers have traded, with no cost to taxpayers or patients. Vouchers are trading around $100 million, constituting a compelling incentive for pediatric rare disease drug development.
  • Drug companies are now prioritizing pediatric cancer drugs and drugs for rare pediatric illnesses.

Endorsers:

  • 94 organizations endorsed the bill including: Alliance for Childhood Cancer, Kids v. Cancer, NORD, and the Coalition for Pediatric Medical Research
  • Creating Hope Act and Pediatric Priority Review Vouchers — Overview
  • Statutory text
  • FDA Guidance on Pediatric Vouchers
  • Pediatric Vouchers Issued and Sold, 2012-present
  • 2020 Testimony in Support of Making the Pediatric Voucher Program Permanent
  • Creating Hope Act News
President Obama before signing Creating Hope Act
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