The Advancing Hope Act of 2015 (S 1878, 114th Congress)
What it does
Extends the rare pediatric priority review voucher program and includes sickle cell disease and pediatric cancer as qualifying conditions within that program.
S 1878 amends the Food, Drug, and Cosmetic Act to extend the Rare Pediatric Disease Priority Review Voucher Program by:
- Extending the life of the program beyond the end of September 30, 2016; and
- Extending the inclusion parameters of the voucher program to include (1) any form of sickle cell disease and (2) pediatric cancers.
The Priority Review Voucher program
- This voucher program incentivizes pharmaceutical companies to develop drugs to treat or prevent rare pediatric diseases.
- Vouchers operate by providing pharmaceutical or drug companies (the “sponsor”) that bring the FDA a human drug application aimed at particular diseases an opportunity to obtain priority review for subsequent drug applications of that sponsor.
- Note that sponsors may transfer vouchers as they see fit.
- Priority review entitles the holder to have a future new drug or biological product application acted upon by the FDA within six months.
- The 1992 Prescription Drug User Fee Act (PDUFA) set a 6-month goal for priority review of new drug applications (NDAs) versus a 10-month goal under standard review. In FY 2009 and FY 2010, 78% of priority NDAs and 94% of standard NDAs were reviewed on time, according to the FY 2010 PDUFA Performance Report (p. 10).
Sickle cell disease (SCD) describes an inherited group of red blood cell disorders characterized by the presence of abnormal hemoglobin, a protein in red blood cells that carries oxygen throughout the body.
- Normal red blood cells (RBCs) are disc shaped. Sickled RBCs are shaped like a half-moon, or sickle, due to the misshapen hemoglobin. SCD cells tend to stick to vessel walls and each other, forming clots and preventing oxygen from reaching tissue. Consequences of oxygen deprivation include severe pain and organ damage.
- Sickle cells are more fragile than healthy RBCs and less able to change shape to fit through small vessels; as a result, they have a shorter lifespan than normal red blood cells. The body is continuously creating new red blood cells, but it often cannot keep up the pace with the loss of sickled cells, leading to anemia. Anemia is a condition in which an individual has fewer red blood cells than normal, leading to low oxygen levels in tissues and lack of energy.
- Sickle cell disease is an autosomal recessive disorder. This means that an individual must inherit two abnormal hemoglobin genes, one from each parent, to experience symptoms.
- An individual is a carrier for SCD if he or she inherits one normal hemoglobin gene and one abnormal hemoglobin gene. Carriers do not exhibit SCD traits under normal conditions.
There are many common types of pediatric cancers, such as leukemia and brain and central nervous system tumors, the causes of which are mostly unknown.
- While most pediatric cancers are thought to be caused by genetic mutations, some are inherited from a parent, but most result from random, acquired mutations specific to an individual.
- Genetic mutations which are hypothesized to cause cancer, are often described in three groups - proto-oncogenes, tumor suppressors and DNA repair genes.
- Proto-oncogenes produce proteins which promote cell growth and division or inhibit normal cell death. Proto-oncogenes are referred to as oncogenes when they acquire mutations that promote continuous activity, leading to uncontrolled cell growth and division.
- Tumor suppressor genes code for proteins that stop cell division if damage is present – the damage is either repaired, or, if the damage is irreparable, the cell self-destructs. When tumor suppressor genes are mutated, cell growth cannot be inhibited, and cells continue to grow and divide at an unhealthy rate.
DNA repair genes are intended to prevent mutations which lead to cancers. Thus, a mutated or damaged DNA repair gene can no longer make repair proteins, and cancerous cell growth can occur.
Endorsements & Opposition
- Kids v. Cancer: “The Advancing Hope Act will…continue the program of pediatric priority review vouchers as an incentive for pediatric rare disease drug development.”
- The following groups also support this legislation: Rare Disease Legislative Advocates, Association of Pediatric Hematology/Oncology Nurses, Biotechnology Innovation Organization and Everylife Foundation for Rare Diseases.
- The National Center for Health Research strongly opposed S 1878, stating, “[The bill’s] ability to stimulate innovation is questionable and it does not efficiently use FDA resources to benefit public health. The purported intent of the bill to encourage new treatments for rare pediatric diseases is admirable, but there is no evidence that the program will accomplish this goal...It does not allow the FDA to prioritize matters of the highest public health importance….While the program has not resulted in new treatments for children, it has dramatically increased profits for pharmaceutical companies.”