Cooley’s Anemia Advocacy Forum Update: May 1, 2015

May 1, 2015 – The Cooley’s Anemia Advocacy Forum (CAAF) is a Cooley’s Anemia Foundation project aimed at helping thalassemia patients and families learn how to make their voices heard on federal issues of importance to the thalassemia community. (A brochure about CAAF can be downloaded by clicking here.) Following is a report from Lyle Dennis and Katie Schubert.

CAF Asks Congress to Make Combating Rare Pediatric Disease a Priority

Seal_of_the_United_States_Congress_svgIn an effort to spur the development of novel treatments for children with rare pediatric disease, CAF has strongly urged Congress to make the FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) Program permanent.

With the PRV program set to expire March 2016, CAF joined with 115 organizations strongly supportive of rare disease research calling on Congress to make this a permanent program.

Currently, drug manufacturers face significant obstacles that can hinder the pursuit of rare disease therapies for children, including difficulties associated with conducting clinical trials. First established in 2007, the PRV program gives the FDA the option to award a priority review voucher to the manufacturer of a drug or biologic that targets a neglected or rare disease. The provision applies to New Drug Applications (NDAs) or Biological License Applications (BLAs).

Under current law, the FDA aims to complete and act upon reviews of priority drugs within six months instead of the standard ten-month review period, and shortening by as much as a full year the overall time it takes for the company’s drug to reach the market.

The letter to Congress notes that of the nearly one in ten Americans with a rare disease, approximately two-thirds are children. Of the 350 most “common” rare diseases, 27 percent result in death before the child’s first birthday.

CAF and other stakeholders underscored the fact that the PRV program provides greater hope to children suffering from rare conditions by encouraging manufacturers to invest in the development of novel treatments.

Congress Agrees on Budget Agreement, But Health Spending Limits in Question

Despite some major challenges, a joint House-Senate conference committee has agreed to a so-called “budget resolution” that sets spending and legislative parameters for fiscal year 2016 and lays out a budget vision for the next decade.

But questions remain as to how medical research, prevention and public health programs will fare.

The budget blueprint will set the table for a GOP effort to use a legislative mechanism, called budget reconciliation, to repeal and replace the Affordable Care Act later in the year. If it is adopted, the fiscal blueprint also could lead to negotiations between Congress and the White House over a budget deal to raise limits on spending that were put in place three years ago.

For the time being, however, the House and Senate budgets adhere to those tight spending limits set back in 2012. On that score, House lawmakers recently agreed to cut a collection of health, education and employment training programs $3.7 billion below current funding levels. If approved, this would represent a cut of about 12 percent, or $20 billion, since 2010, adjusted for inflation.

21st Century Cures Initiative Boosts NIH; Rare Disease Incentives Under Consideration

House Energy and Commerce chairman Fred Upton (R-MI) and Diana DeGette (D-CO) recently released a 200-page draft of legislation that would overhaul the government’s system for evaluating medical products and open the way to increased funding for NIH.

The bipartisan proposal, still considered a work-in-progress, would authorize Congress to appropriate an additional $1.5 billion for NIH each year for three years, and includes a separate $10 billion for a new NIH innovation fund, targeted in part for precision medicine research and young emerging scientists. That funding, which would not be part of the annual appropriations process, would be given to NIH in $2.0 billion per year increments for five years.

Still at issue is what, if any, provisions will be added to the measure to incentivize drug makers to take on the costly and time-consuming task of creating new medicines for rare diseases.

CAF Opposes Gene-editing Research Involving Human Embryos; NIH Agrees

NIH_Master_Logo_Vertical_2ColorCAF recently issued a statement that while it supports ethical applications of technology that advance the search for a cure for thalassemia, the Foundation does not condone the use of technology in a clinical setting in a manner which is inappropriate or inconsistent with U.S. regulatory guidelines. In so doing, CAF made it clear that an international consensus on the proper use of such technology is warranted and should be followed.

In a statement issued April 29, NIH Director Francis S. Collins, MD, PhD, concurred, stating that “NIH will not fund any use of gene-editing technologies in human embryos.” Dr. Collins added that altering the human germline in embryos for clinical purposes raises both safety issues and ethical issues.

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