News Roundup – Rare Disease Legislation is Moving

Today co-sponsors in the House, Representatives Edward Markey (D-MA) and Cliff Stearns (R-FL) introduced the “Improve Access to Clinical Trials Act”.  The legislation would allow patients with rare diseases to participate in clinical drug studies without losing their eligibility for government health care coverage. Current law prevents many people who receive Supplemental Security Income (SSI) from accepting research compensation because it would make them ineligible to continue receiving government medical benefits. This financial penalty prevents significant numbers of people with rare diseases from participating in clinical studies.

Michael Astrue, Commisioner of the Social Security Administration, asked industry to help expand the agency’s “compassionate allowances” program, which expedites eligibility decisions for individuals seeking disability benefits because of serious diseases. The “compassionate allowances” program, launched in October 2008 to “provide benefits quickly to applicants whose medical conditions are so serious that their conditions obviously meet disability standards.” “We need your help” to expand the program to more disease areas where beneficiaries may currently face long delays in receiving Medicare coverage for disabling illnesses, Astrue said during a discussion of orphan drug policy sponsored by Shire Pharmaceuticals, the National Chamber Foundation and the US Chamber of Conference in Washington, DC June 4.

Astrue’s interest in orphan drugs long predates his current role. As an HHS official in the Reagan Administration, he helped craft the implementing regulations for the Orphan Drug Act. Then, as a biotech industry executive, he saw first hand how the exclusivity incentives for orphan products became a vital asset to many biotech firms. Astrue was also on the first short-list of FDA commissioner candidates in the Bush Administration in 2001.


Two competing bills reintroduced in March would create an approval process for generic biopharmaceuticals, but the measure favored by brand-name drugmakers so far has attracted more support from House members.

In recent years, lawmakers have offered legislation to create a single pathway for generic biologics. The goal is to allow faster approval of generic versions of expensive cancer-fighting treatments and other medications and to make them more affordable. But lawmakers have been unable to agree on a few key points, including the length of time manufacturers of an original drug would be protected from generic competition.

The Generic Pharmaceutical Assn. is hoping members of Congress will embrace the Promoting Innovation and Access to Life-Saving Medicine Act, reintroduced March 11 by Rep. Henry Waxman (D, Calif.), chair of the House Energy and Commerce Committee. The bill would give original biologic drugmakers an initial five years to market their drugs without generic competition, and it would allow an extension of up to an additional four years. Kathleen Jaeger, GPhA president and CEO, said the Waxman bill is based on the standards of the 1984 Hatch-Waxman Act, which created an approval process for generic versions of chemical drugs. “For health care to be affordable and sustainable, this bill needs to pass,” she said. Waxman’s bill has seven co-sponsors. The GPhA and several business and labor organizations support the bill.

The Pathway for Biosimilars Act, reintroduced March 17 by Rep. Anna Eshoo (D, Calif.), initially would provide 12 years of exclusive marketing to original biologic drugmakers, which could be extended by up to an additional 2½ years. That measure has 54 co-sponsors and also is supported by the Biotechnology Industry Organization. “This new biosimilars approval pathway will promote innovation, competition and lower prices but also ensures that patients are given safe and effective treatments that have been subjected to thorough scrutiny and testing by the FDA,” Eshoo said.

Sen. Edward Kennedy (D, Mass.) also plans to reintroduce his generic biologics bill, said a spokesperson from the Senate Health, Education, Labor and Pensions Committee, which Kennedy chairs. His measure from the last Congress, which the HELP panel approved in 2007, would have allowed 12 years of exclusive marketing.



~ by marbleroad on June 15, 2009.

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