Advancing Hope Act of 2016 (Public Law 114-229)
What it does
Temporarily renews and modifies the FDA Priority Review Voucher Program for rare pediatric diseases.
The Advancing Hope Act of 2016 amends section 529 of the Food, Drug and Cosmetic Act to temporarily extend and modify the Rare Pediatric Disease Priority Review Voucher Program by:
- Extending the program through December 31st, 2016;
- Modifying the definition of a rare pediatric disease (as defined in Section 529 – (21 USC 360ff) by specifying that the disease must be serious or life-threatening or have serious or life-threatening manifestations (the definition maintains those primarily affected must be under 18 years of age);
- Clarifying that a sponsor may receive only one voucher per drug;
- Mandating that the Comptroller General of the United States, in association with the Government Accountability Office (GAO), create a report studying the impact of the Rare Pediatric Disease Priority Review Voucher Program. The report must:
- Provide indications for each drug awarded a priority review voucher;
- Determine if the program was effective in incentivizing the development of rare disease drugs;
- Analyze the drugs for which the priority review vouchers were redeemed, including indication, number of potential users, value of the voucher if transferred, time between award and redemption of the voucher, and if the drug met an unmet need, including:
- Whether an alternative therapy was previously available, and
- If the drug submitted for this program provided a benefit compared to another available alternative therapy.
- Review the impact of the voucher program on Food and Drug Administration (FDA) resources, the impact on public health for drugs approved through the voucher program, and potential improvements.
- Be completed no later than January 31, 2022, and submitted to the Senate Committee on Health, Education, Labor, and Pensions and the House Energy and Commerce Committee.
<p>The Advancing Hope Act of 2016 is an amendment to the Food and Drug Administration Safety and Innovation Act (<a href="https://www.congress.gov/bill/112th-congress/senate-bill/3187?q=%7B%22se... – Public Law 112-144</a>), which added Section 529 of the Federal Food, Drug and Cosmetic Act (<a href="https://www.gpo.gov/fdsys/pkg/USCODE-2012-title21/html/USCODE-2012-title... USC 360ff</a>). Section 529, enacted in 2012, made regulation that offered priority review vouchers for sponsors of rare pediatric disease product applications. As part of the 2012 law, though, there was a three-voucher cap leading to expiration of the voucher program on March 17, 2016.</p>
<p><a href="http://www.fda.gov/forpatients/approvals/fast/ucm405405.htm">Priority review</a> means expedited review and action by the FDA within 6 months of application instead of the standard 10-month review. If a drug company submits an application for a drug aimed to treat a rare pediatric disease, assuming it meets agency criteria, the FDA will reward a priority review voucher to the sponsor. This voucher can be redeemed to receive FDA priority review for a subsequent application submitted for a different product. The company may also choose to transfer or sell the voucher to another company.</p>
<p>Other legislation to encourage development of drugs for pediatric patients include the <a href="https://www.congress.gov/bill/97th-congress/house-bill/5238">Orphan Drug Act of 1983</a>, which provides incentives for drug companies to make drugs aimed at rare diseases. The <a href="https://www.congress.gov/bill/107th-congress/senate-bill/1789?q=%7B%22se... Pharmaceuticals for Children Act of 2002</a> provides mechanisms for studying on- and off-label drugs in children. The <a href="https://www.congress.gov/bill/108th-congress/senate-bill/650?q=%7B%22sea... Research Equity Act of 2003</a> allows the FDA to require pediatric studies as part of the New Drug Application approval process.</p>
<p>There are multiple entities within the National Institutes of Health (NIH) that work on research in one or more rare diseases, including <a href="https://ncats.nih.gov/rdcrn">Rare Diseases Clinical Research Network (RDCRN)</a>, <a href="https://ncats.nih.gov/trnd">Therapeutics for Rare and Neglected Diseases (TRND)</a>, <a href="https://ncats.nih.gov/grdr">Global Rare Diseases Patient Registry Data Repository (GRDR)</a>, and the <a href="https://ncats.nih.gov/gard">Genetic and Rare Diseases Information Center (GARD)</a>. The Food and Drug Administration (FDA) also has an <a href="http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTob... of Orphan Products Development (OOPD)</a>.</p>
Rare diseases, as defined by the statute, generally affect small patient populations, each typically fewer than 200,000 individuals in the United States (21 USC 360bb). Altogether, more than 25 million Americans are affected. An estimated 80% of rare diseases are thought to be due to mutations in genes or chromosomes, although most do not yet have an identified genetic cause. Genetic causes are often why certain rare diseases are passed along in families, while others mutations are de novo (new) cases. Given both the rarity and pediatric age group of individual diseases, recruitment for studies can be difficult. There are nearly 7,000 rare diseases (most serious or life-threatening), with about half affecting children, according to a March, 2016 a GAO report, Rare Diseases: Too Early to Gauge Effectiveness of FDA’s Pediatric Voucher Program. Few, although not specifically quantified, have “viable treatments.”
According to a 2016 report by the Pharmaceutical Research and Manufacturers of America (PhRMA), the pharmaceutical industry estimates that development of a single drug takes 10-15 years; average cost is $2.6 billion. Less than 12% of drugs that enter clinical trials are approved, leading to a large cost deficit incurred from drug development. Since drug production is costly and time-intensive, and there is a relatively small target audience for any one rare disease, recouping costs and generating profit is challenging. As a result, the incentive for pharmaceutical companies to pursue rare disease treatments is relatively low.
Endorsements & Opposition
- Kids v Cancer, an organization focused on changing the landscape of pediatric cancer research, issued a press release praising the passage of this bill, urging “Congress to pass the Advancing Hope Act on a permanent basis,” as the current legislation is temporary.
- National Organization for Rare Disorders (NORD), in a press release, endorsed the signing of the Act: “For nearly two years, NORD has helped to lead advocacy efforts for the permanent authorization of the program.” The organization is also pushing for permanency of the legislation, stating “with the extension, we will need advocates to join us and fight this battle again in November so that this important program does not go away.”
- The American Academy of Pediatrics (AAP), in principle, supports incentives for pediatric drug development, but qualifies support for this bill’s concept, as evidenced by a review of the 21st Century Cures Act, which also extended the rare pediatric disease priority review voucher program: “While there is a potential for the program to ultimately prove to be a helpful incentive for the development of drugs for children, it is not yet clear whether it will be able to incentivize the development of pediatric drugs that would not have otherwise been developed…AAP is concerned that FDA is not adequately resourced to implement the program.”
- The Food & Drug Administration (FDA) opposes program reauthorization, as documented in the March 2016 GAO report: “FDA officials reported that they have seen no evidence that the program has encouraged increased development of drugs for rare pediatric diseases.” It also “interferes with [the FDA’s] ability to set priorities on the basis of public health needs.” Lastly, according to the FDA, the pediatric voucher program places a “substantial strain on its workload.”