Meeting the need for speed

WASHINGTON, D.C.—In concert with the Biotechnology IndustryOrganization (BIO), North Carolina Democratic Senator Kay Hagan has crafteddraft legislative language that both political parties hope will streamline andbetter organize the way that potential drugs come to market.

The proposal is known as TREAT, an acronym for Transformingthe Regulatory Environment to Accelerate Access to Treatments.

Tentative plans being crafted by Senator Hagan and BIO wouldpermit manufacturers such as GlaxoSmithKline PLC (GSK), which has a significantpresence in Hagan's home state, to leap customary clinical trial hurdles beforebringing products to market. The draft proposes allowing approval of some drugsafter successfully completing two of the usual three phases of clinical trials.

"GSK appreciates Senator Hagan's ongoing interest infostering patients' access to new and innovative therapies," Melinda Stubbee, aGSK spokeswoman, tells ddn. "We are inthe process of reviewing the draft legislative language."

Specifically mentioned are classes of drugs to address AIDSand cancer. Also identified by Congressional definition are a class of diseasesknown as "orphan diseases," which are classified as rare disorders affectingfewer than 200,000 patients in the United States. With more than 6,000 of theseconditions identified, these afflictions impact millions of Americans. In thepast five years, the U.S. Food and Drug Administration (FDA) has approvedapproximately 85 rare-disease therapies.

In the current regulatory environment, exemptions to thethree-trial rule are occasionally permitted under decades-old rules, and thecriteria used are termed by the industry as "unclear and unpredictable."Accelerated approval allows the shorter trials to prove a measured effect of atrial therapy, in place of a clinical outcome. After the fact, the drug companymust prove that the drug is effective. Physicians may also assist patients ingaining access to medications that they feel will benefit a patient.

The legislation provides that if a drug were approved foruse in other countries, this would carry significant weight in approving drugsquickly in the United States. In addition, the draft legislation addresses apossible overhaul of the fee system pharmaceutical companies use to fund FDAreviews. Discussions between the FDA and the drug industry have leaned toward a6-percent fee increase as part of a plan that extends to 2017.

The legislation would also enable the creation of aninternal management review board at the FDA, which would be intended toincrease the efficiency of the agency. Twenty-two individuals would be appointedto the board, with some members coming from within the agency. The biotech,drug, food and medical device industries would also be represented.

The appointment of a chief innovation officer would alsobecome a reality if TREAT becomes law. It would also set in stone the term ofthe FDA commissioner at six years, but not limit reappointments of thatindividual to additional terms.

Hagan's office and BIO representatives did not return callsfor comment for this story.