Any legislation to create a regulatory pathway at the US Food and Drug Administration for follow-on biologics should provide no less than 14 years’ data exclusivity for pioneer products, the Biotechnology Industry Organization has stated.

“Biotechnology companies must have some certainty that they can protect their investment in the development of new breakthrough therapies for a sufficient period of time, in order to secure the necessary resources from venture capitalists and other funding sources,” said BIO’s chief executive, Jim Greenwood. Such certainty can most predictably be provided through lengthy data exclusivity, and anything less than 14 years would be insufficient, he added.

The industry group made its demand just as Congress was being told that the protection of monopolies and the financial bottom line have harmed the industry’s goal of “solving the mysteries of disease.” “Our hope is Congress will allow the FDA to evaluate biogenerics on the basis of scientific facts and not the politics of the bottom line,” Geoffrey Allen, chief executive of biotechnology company Insmed Corp, told a hearing of the House Energy and Commerce Committee’s health subcommittee, held to discuss the approval pathway for biogenerics proposed by Democrat Henry Waxman in his Access to Life-Saving Medicines Act (HR 1038).

Dr Allen, chairman of the new Coalition for Biotechnology Innovation (CBI) which represents small biotechnology companies, called for “a practical, science-driven pathway” which would enable the FDA to determine case-by-case the scientific issues and amount of data required. General guidance documents for biogenerics are expected from the agency at some time, but these are not absolutely necessary, he said, nor would companies wait for guidance to be issued before submitting applications to the FDA. “Close interaction and dialogue with the FDA on a case by case basis would allow a more robust approval process than would result from a broad guidance system,” Dr Allen told the panel.

He forecast that the development of biogenerics would create an “explosion of both investment and innovation in the biologics market,” a view which was shared by Bruce Downey, chief executive of generics manufacturer Barr Pharmaceuticals. The market competition generated by generic biologics would unleash incentives for further innovation of newer medicines, just as Hatch-Waxman did over 20 years ago, and also lower the cost of treatment with existing medicines, Mr Downey told the hearing.

He also emphasised that Congress must enable the FDA to draw on its decades of experience with biological products, by granting it the discretion to require clinical studies only when it determines that they are needed. The regulatory pathway must also be free of artificial barriers and unnecessary roadblocks, and include a mechanism for speedy resolution of patent disputes, he said.

Recipe for disaster

Mr Downey warned that the dual impact of higher prices and greater utilisation of biopharmaceuticals “presents a recipe for disaster which will end in price controls,” but added that the alternative is competition. “Effective generic biologics legislation very well could be the most important piece of consumer legislation enacted this year,” he told the panel.

Industry consultant and former FDA official William Schwieterman urged legislators to “proceed with caution.” A truly workable pathway will bring safe and effective biogenerics to patients in a timely manner, while one filled with needless requirements and hurdles will not accomplish what Congress wants, he said.

Congress must not deprive the biotech industry of incentives to innovate, said Ruth Hoffman, executive director of the Candlelighters Childhood Cancer Foundation, who pointed out that, today, all children with cancer are still being treated solely with highly toxic cancer drugs that were developed 20-30 years ago. “We have reached a plateau with survivorship rates, and we have reached the limit of toxicity for current chemotherapy drugs and radiation treatments,” she said, adding: “the best hope for children with cancer rests in the R&D of new and targeted biologics.”

Testimony from industry strongly echoed BIO’s call for ample data exclusivity. The legislation should not alter existing rules under patent law that protect innovative products, or make changes that favour biosimilars over innovations, said lawyer Richard Kingham, a partner at law firm Covington & Burling.

Biosimilars cannot be treated as generic drugs, said Mr Kingham, and both he and David Schenkein, vice president for clinical hematology/oncology at Genentech (testifying on behalf of BIO) commended to the panel the European Union (EU) system for regulating biogenerics.

The EU system’s key features stem from the recognition of the unique characteristics of biotechnology-derived proteins, said Dr Schenkein, and its pathway strives to achieve follow-on biologics that are truly highly similar to a reference product, while acknowledging that important clinical differences may still exist upon market approval; this makes post-marketing clinical studies and safety surveillance important. By Lynne Taylor

- Meantime S 623, the Senate companion bill to HR 1083, is due for mark-up by the Senate Health, Education, Labor and Pensions Committee on June 13, panel chairman Edward Kennedy has said. However, agreement on its details must first be reached between Senate leaders and the bill’s sponsors, Democrats Hillary Rodham Clinton and Chuck Schumer.