New Year, New Issues - Pharmaceutical Executive


New Year, New Issues

Pharmaceutical Executive

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The search for new drug development models to fill an admittedly dry pipeline will intensify with pharma's loss of revenues from blockbuster medicines. Public and private leaders of the biomedical research community talk more about "game-changing, transformational leaps" in discovery, as opposed to incremental gains. There is growing enthusiasm for developing "personalized" medicines—and relevant diagnostics—that provide more effective treatment based on individual genomic and metabolic characteristics.

Public/private partnerships are supporting more international research on therapies for malaria, tuberculosis, and other diseases most prevalent in tropical climates. Health authorities are pressing for more R&D on new antibiotics, treatments for rare conditions, and therapies for killer diseases such as cancer and AIDS. There is growing excitement about vaccine discovery, which is attracting more industry investment as markets mature around the world.

The growth in overseas clinical research, though, will continue to raise concerns about unethical biomedical research practices. A White House panel has examined past U.S.-backed studies in Guatemala and proposed changes in current policy. That exercise may help shape a broader overhaul of rules and standards for all clinical studies sponsored by the federal government.

The drive for transparency, moreover, will spur more efforts to expand disclosure of active trials and study results on the website. And FDA and the NIH are strengthening financial conflict-of-interest policies to address perennial concerns that economic interests could compromise research results.

Health reform "Sunshine" provisions, which HHS finally unveiled last month, will require pharma marketers to disclose payments to physicians, raising fears that physicians will be discouraged further from participating in clinical research, as well as pharma marketing programs. Despite these concerns, assurance that biomedical research practices fully protect patients and provide valid clinical data is critical to building public confidence in the R&D process.

"Patient centeredness" will shape a range of regulatory and research initiatives. FDA is encouraging sponsors to incorporate patient needs and viewpoints into clinical trial protocol design, patient recruitment, 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, and its Methodologies Committee aims to report in May on research methods and standards for this field.

FDA commissioner Margaret Hamburg will continue to seek support for regulatory science initiatives that can accelerate the R&D process. She points to multiple biomarker validation projects that can identify potential drug safety problems early on and reduce trial failures. Coalitions are looking to streamline clinical studies with "adaptive" clinical trials and electronic methods for recruiting patients and collecting research data.

Despite these initiatives, pharma and biotech companies complain that a risk-averse tendency at FDA keeps promising medicines off the market. Agency officials, though, point to last year's jump in approvals for important new drugs as evidence of its flexibility. FDA promises to do more to assist emerging companies navigate the regulatory process, but it remains to be seen if the recent spike in approvals is a fluke, or a signal of real progress on the innovation front.

Jill Wechsler is Pharmaceutical Executive's Washington correspondent. She can be reached at


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