Filling the pipeline
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.
Finalizing user fees
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
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. But it remains to be seen if the spike
in approvals is a fluke, or a signal of real progress on the innovation front.
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 (GCPs). 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 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
"Patient centeredness" will continue to shape a range of 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 Pharmaceutical Technology's Washington editor, 7715 Rocton Ave., Chevy Chase, MD 20815, tel. 301.656.4634, firstname.lastname@example.org