Budget Crunch, Political Battles Shape Policy Agenda for Year - Pressure to approve new user fees opens the door to action on drug shortages, prices, and regulation. - BioPharm International


Budget Crunch, Political Battles Shape Policy Agenda for Year
Pressure to approve new user fees opens the door to action on drug shortages, prices, and regulation.

BioPharm International
Volume 25, Issue 1, pp. 11-15


The loss of patent protection for a wave of blockbuster medicines is driving pharmaceutial companies to search for new models for drug development to fill an admittedly dry drug pipeline. Public and private backers of biomedical research talk more about "game-changing, transformational leaps" in discovery, as opposed to the incremental gains that traditionally lead to important scientific advances. There is growing enthusiasm for developing personalized medicines that provide more effective treatment based on individual genomic and metabolic characteristics. This will require the development of more diagnostics to identify key response factors.

Expanded international research efforts are tapping into public–private partnerships for developing important therapies for malaria, tuberculosis, and other diseases most prevalent in tropical climates. Health authorities are pressing for more research on new antibiotics, along with treatments for rare conditions and killer diseases such as cancer and AIDS. There is growing excitement about new vaccines, which are attracting more industry investment as markets mature around the world.

FDA can help the process, according to Commissioner Margaret Hamburg, who has been promoting the campaign to bolster FDA involvement in regulatory science initiatives to provide new tools and methods to accelerate the R&D process. Several programs are underway to validate biomarkers that can identify potential safety problems early on and improve the efficiency of clinical studies. Other coalitions are looking to streamline the long and costly R&D process by developing research protocols for "adaptive" clinical trials and promoting electronic methods for recruiting patients and collecting research data.

Yet, manufacturers complain that a risk-averse tendency at FDA and demands for more, larger studies keep many promising medicines off the market and raise R&D costs. The recent FDA decision to revoke the metastatic breast cancer indication for Avastin has generated questions about the future of FDA's accelerated approval process and the threshold for bringing new cancer therapies to market.

FDA officials point to last year's jump in approvals for new molecular entities (NMEs) as evidence that the agency is not keeping important new medicines from patients. A number of the approvals involve treatments for rare conditions and serious cancers that carry less risk for patients and lend themselves to speedy FDA evaluation.

The rise in overseas clinical research activity, as pharmaceutical companies seek more efficient drug development operations and data to support global marketing efforts, continues to focus attention on research ethics and policies to ensure compliance with good clinical practices. Several federal agencies are examining past unsafe research practices and weighing changes in policies and standards for clinical studies sponsored by the federal government or regulated by FDA.

Clinical research activities also face more scrutiny at home under transparency requirements that expand disclosure of active clinical trials and study results on the http://clinicaltrials.gov/ website. Health reform "sunshine" provisions require pharma companies to disclose payments to physicians and other health professionals, a process that involves major revisions in corporate policies and information systems. The transparency campaign, moreover, may result in broader FDA disclosure of information on drug safety and effectiveness, possibly even proprietary data that manufacturers might prefer to keep confidential. The assurance that US-supported investigators fully protect research participants and ensure the validity of clinical data is critical to improving public confidence in the pharmaceutical R&D process.

"Patient centeredness" will continue to shape regulatory and research initiatives. FDA is encouraging sponsors to incorporate patient needs and opinions into clinical-trial protocol design, patient recruitment, drug delivery, and safety evaluation. This approach will be supported by research sponsored by the Patient-Centered Outcomes Research Institute (PCORI), which is slated to have a $500 million annual budget by 2014 to study effective treatments for important conditions. PCORI plans to finalize priorities for its research agenda by March 2012, and its Methodologies Committee aims to report in May on research methods and standards for this field.

Jill Wechsler is BioPharm International's Washington editor, Chevy Chase, MD, 301.656.4634,

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