Regulatory Beat: Washington Seeks New Therapies to Combat Bioterrorism, Cancer, Obesity

Despite escalating criticism of exorbitant prices for new drugs and biotech therapies, coupled with demands for access to cheap imports, policymakers continue to support the development of new biomedical products, especially those that combat life-threatening diseases and terrorist threats. The need to maintain incentives for biotech innovation, while addressing such hot-button issues such as follow-on generics and Medicare reimbursement, will challenge Rep. James Greenwood (R-Pa) as he becomes the head of BIO (see "Greenwood to Lead BIO").

Jill Wechsler

Promoting Countermeasures

One main goal of the Bush administration is to encourage private sector development of new vaccines and treatments to protect against biological warfare. After almost two years of disputes and delay, Congress approved Project BioShield in July, and President Bush signed the bill soon after. The legislation provides $5.6 billion over 10 years to stockpile vaccines and other countermeasures to biological and chemical warfare. The bill also permits the National Institutes of Health (NIH) to expedite approval of grants for countermeasure R&D, gives FDA authority to allow emergency use of not-yet-approved treatments, and encourages speedy FDA approval of these products. FDA is urging manufacturers with investigational new drugs (INDs) that may be bioterror countermeasures to come in for "pre-pre-IND" meetings to ensure that researchers conduct necessary analyses in early studies.

While the bill was delayed for months due to a dispute over budget control, officials at the departments of Health and Human Services (HHS) and Homeland Security (DHS) have been negotiating preliminary agreements with manufacturers for new countermeasures. One major project will provide $700 million to purchase 75 million doses of an improved anthrax vaccine made with recombinant technology, high-volume fermentation, and modern protein purification methods. A related initiative seeks to acquire antitoxin treatments for inhalation anthrax disease, including monoclonal anti-PA antibodies, polyclonal anti-anthrax antibodies, and human immunoglobulin. HHS also is seeking antidotes and other products to treat and protect against smallpox, botulism, plague, Ebola virus, and radiation from "dirty bombs."

Missing Protections

Although the legislation offers incentives for manufacturers developing these products, it has a serious shortcoming from industry's perspective: it fails to include liability protections for companies if bioterror vaccines or drugs cause adverse events. And because the federal government is the main customer for countermeasures, manufacturers expressed concerns about regulated prices and lower return on investment in this field. Big pharma companies are generally delaying major investment in bioterror contracts, leaving the field to smaller biotech manufacturers.

Legislators and industry are discussing a BioShield II bill to provide liability protection for manufacturers and additional incentives for product development. Sens. Joseph Lieberman (D-Conn) and Orrin Hatch (R-Utah) expect hearings this fall on new tax and patent provisions, such as a proposal to offer companies that develop countermeasures "wild card" patent extensions — the opportunity to apply a two-year patent extension to an unrelated patent. Lieberman also suggested adding incentives for developing new antibiotics, and other critical treatment categories may be included in the next bill.

Development of counter-terrorism treatments also may become a campaign issue. In June, Democratic presidential nominee Sen. John Kerry (D-Mass) called for building a "medical arsenal of democracy" by speeding the transition from basic discovery to clinical trials for new drugs and vaccines. Kerry has accused the Bush administration of failing to address weaknesses in the nation's bioterrorism defenses, but his proposals for responding to biological threats appear similar to the president's.

Spurring Cancer Treatments

As part of a renewed anti-cancer campaign, last year FDA and the National Cancer Institute (NCI) announced a broad range of collaborative efforts to facilitate development and access to new oncology therapies (see

BioPharm International

, August 2003). One aspect of the initiative involves a joint proteomics and genomics program to develop standards for safety, purity, and potency of tumor vaccines. Another program aims to identify biomarkers to validate diagnosis and early detection of cancer, which was discussed at an NCI/FDA conference in July.

To help speed promising new treatments to market, FDA is streamlining its oversight of oncology therapies, including biotech products. The agency is forming a new Office of Oncology Drug Products (ODP), combining three CDER divisions: the Division of Oncology Drug Products, the Division of Therapeutic Biological Oncology Products, and the Division of Medical Imaging & Radiopharm-aceutical Drug Products. FDA launched a national search for a director of the office, which will house 100 reviewers and staffers.

While the reorganization does not significantly alter FDA operations, it aims to provide more consistent oncology policies and reviews, including decisions on which applications merit accelerated approval status. ODP also will run an FDA Oncology Program to work with other FDA centers and outside organizations on new products, policies, and standards. FDA aims to fully implement this change by April 2005, when much of CDER moves to FDA's new White Oak facility.

Fighting Fat

HHS Secretary Tommy Thompson also announced in July a revision in Medicare policy that could spur R&D for new anti-obesity interventions. HHS eliminated language from its Medicare coverage policy that stated that obesity is not an illness — a change that could allow coverage of effective weight-loss products and treatments for the elderly. This policy shift is expected to influence coverage decisions by private plans and payers, creating a much broader market for weight-control drugs scientifically proven to improve health outcomes.

At the World Obesity Congress in July, FDA Commissioner Lester Crawford outlined FDA efforts to combat obesity through development of new therapies. Crawford described medical problems associated with past weight-control drugs such as amphetamines and dexfenfluramine, acknowleding that doctors have few effective treatments for obesity. An FDA Obesity Working Group has been examining this situation and crafting proposals to facilitate development and marketing of safe and effective weight-control products.

In September, FDA's Endocrinologic and Metabolic Drugs Advisory Committee will review a 1996 FDA draft guidance that tried to set a framework for developing chronic-use weight-loss drugs. Crawford noted that agency experts believe combination therapies and head-to-head comparisons of new and existing treat-ments "are critical for progress."

Greenwood To Lead BIO

After a six-month search, BIO selected Rep. James Greenwood (R-Pa) as its next president. He will replace retiring leader Carl Feldbaum, who headed the trade group since its establishment in 1993 (see BioPharm International, April 2004). Greenwood is completing his current term in Congress and will assume his new position in January.

Greenwood appears an ideal choice for the job. As chairman of the Oversight & Investigations Subcommittee of the House Energy and Commerce Committee, the congressman has been involved in many of the research and reimbursement issues important to biotech companies. He played a lead role in recent revisions to the Prescription Drug User Fee Act and in negotiating the Medicare Modernization Act last year. A liberal Republican, he is also an advocate of stem cell research.

Greenwood said in July that he would not have left Congress for any other trade association but that he sees this as an opportunity to advance the development of biological treatments critical for human health. At the helm of BIO, which represents more than 1,000 large and small companies, Greenwood will face many challenges and controversies, including "follow-on" or generic biologics, intellectual property protections, and pricing and reimbursement, to name a few.