Baby Steps in Congress

One Step_edited-1

Getting things done for childhood cancer in Congress is not easy. For one, even as a community, we have very little political clout. One of the best things going for us is that  we have the Childhood Cancer Caucus in the House of Representatives and that is certainly a huge help. At the present time, almost one fourth of the House are members. The caucus was formed in 2009 with a mission to serve as a clearinghouse for information on pediatric cancer and a forum to aid Members of Congress in working together to address pediatric cancer. One of it’s major accomplishments was to get the Creating Hope Act passed.

The Creating Hope Act offers the incentive of issuing FDA priority review vouchers for those companies that produce a drug for a rare disease. While it costs the government very little to administer the program, the voucher is valuable because it allows a company to speed up FDA approval on another drug of their choice. The vouchers can also be sold to other companies.  So far, in three years, the FDA has approved three drugs for rare diseases. The vouchers earned have produced over $650 million in revenue for the developers. One drug approved this year was for neuroblastoma. It’s the first new drug developed for a childhood cancer in twenty years. That sounds great and it is, but now we have a problem.

The award of the third pediatric voucher for the neuroblastoma drug triggered a little-known sunset clause contained in the 2012 law which will cause the Creating Hope Act to expire on March 17, 2016.  The Creating Hope Act reauthorization was put in an all encompassing 21st Century Act that sailed through the House. Now, this big bill is tied up in the Senate. Unless something changes soon, it appears the 21st Century Cures Act will not pass the Senate in time to save the Creating Hope Act by March, 2016.

AdvancingHopeHeader_edited-1Prior to the passage of the 21st Century Cures Act, Rep. George “G.K.” Butterfield Jr. of North Carolina, introduced the Advancing Hope Act.  His bill expanded the cost-neutral Food and Drug Administration (FDA) priority review voucher (PRV) program, and eliminated the sunset clause altogether. The bill has had some push backs. By making the Act permanent, the FDA feels it will cause problems in that more and more companies will be using it and therefore causing a strain on their normal work flow and ability to approve all drugs for use.

All the drug developers now see the uncertainty in using priority review vouchers and have therefore delayed any work that may earn a voucher until the incentive program is reinstated.

We can not allow this program to have a gap or much worst die after so much success in only three years. One strategy is to ask Congress to approve the Advancing Hope Act (even if the FDA does not like it) and by doing so, show that there is a real need for these vouchers. Another strategy is to quickly re-authorize the present bill now, but we are not seeing an effort to do this since it is in the 21st Century Cures Act. What else has produced three new drugs for rare diseases? The answer is, nothing! Without the incentive of a voucher, it’s just not profitable for companies to invest in rare disease drug development.

Please help by asking your Representative and Senators to co-sponsor the Advancing Hope Act. Click HERE for more information on the Act and how you can help. Maybe we can force Congress and the FDA to come together and develop a real solution to get this effective program up and running for rare disease and childhood cancers!

We don’t have time for baby steps when we need to be running. We need Creating Hope, Advancing Hope or an alternative  to improve drug development for children with cancer.

Author: Joe Baber






This entry was posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized and tagged , , , , , , , . Bookmark the permalink.

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