As chairman of the House Energy and Commerce subcommittee on health and the environment for 15 years, he engineered enactments of the Orphan Drug Act in 1983 and the 1984 Hatch-Waxman Act, which established the modern generic drug industry. At first, pharma companies were not enthusiastic about the orphan drug bill, he recalls, because it "highlighted an area where they had dropped the ball." To make it more attractive to develop drugs for rare diseases, Waxman and others offered a period of exclusivity when sponsors of such therapies would not face competition, laying the basis for a highly successful program.
The generic drug legislation also reflected a need for tradeoffs between drug development and affordable medicines. Waxman recalls that pharma companies first tried to get Congress to extend patents for the months FDA took to review a market application. After that strategy failed, Waxman helped craft a compromise: extended patents plus delay of generic approval until five years after approval of the brand, in exchange for permitting generics to rely on the brand's safety and effectiveness data, with FDA approval of generics during that five-year waiting period. Now 70 percent of prescriptions are filled with generics at huge savings, Waxman notes. The program is "not perfect," he says, possibly because pharma companies continually seek to extend the period of patent coverage as much as possible.Waxman also supported the landmark drug user fee program, even though he regrets the need to take this route, considering it a federal responsibility to support FDA operations. But he acknowledges that the fee program has served its purpose, and is looking to extend it next year, possibly with additional drug safety requirements.
Drug prices are a long-time interest for Waxman. He hopes to establish rebates on drugs for Medicare dual-eligible beneficiaries, which would eliminate what he considers a big windfall for industry under the Medicare drug benefit program. "I believe there should be much more drug price negotiation than what we've seen in the US," Waxman says. He observes that pharma companies are quick to cite Europe's longer patent exclusivity policy because it serves industry's purpose, but notes strong precedents in Europe for negotiating prices because the government is the major purchaser of drugs. – Jill Wechsler