Slowdown at FDA
Some of the blame for longer, more costly drug development falls on the shoulders of FDA. Stepped-up demand for more safety
and efficacy data, prior to approval as well as after a drug comes to market, can add to development costs, and ultimately
weaken less robust pharmaceutical R&D programs. Moreover, success rates remain notably low for new drugs in clinical development,
despite years of efforts to better inform the clinical-research process to avoid wasting millions of dollars on unsuccessful
studies. The Biotechnology Industry Organization (BIO) reported in February that only one in ten new drugs make it from Phase
I studies to FDA approval, based on an analysis of thousands of drug-development efforts from 2003 through 2010.
Most disappointing is an actual decline in new drug approvals by FDA last year, a troubling shift after two years of slight
increases. The agency cleared only 21 new molecular entities (NMEs) in 2010, down from 25 in 2009. Even more discouraging
are reports that fewer applications for innovative new therapies were filed with FDA last year, squelching optimism about
an upturn in product approvals in the near future. Yet, FDA is caught in a hard place, as patient advocates demand earlier
access to promising therapies, while policymakers and consumer groups insist on more scrutiny of test products to better detect
potential safety problems.
The approval downturn kept several highly touted experimental products from the market. Cardiovascular safety issues prompted
FDA to reject new diabetes therapies and kill several new weight-loss drugs, while also pulling Abbott's Meridia product from
the market. More recently, a promising fast-acting inhaled insulin product (MannKind's Afrezza) was put on hold following
FDA requests for more data on product usage and safety. The agency drew heat for turning down an application from Cell Therapeutics
for pixantrone, a treatment for non-Hodgkin's lymphoma that showed some efficacy, but not enough for FDA approval. Cancer
advocates continue to oppose a move by FDA to narrow its approved indication for Avastin, insisting that the benefits outweigh
new evidence of serious side effects.
There are some bright spots amidst these disappointments. Last month, FDA approved a new treatment for melanoma, Bristol-Myers
Squibb's (BMS) Yervoy (ipilimumab). The drug is the first to prolong lives of patients with this deadly skin cancer and a
vast improvement over existing therapies. Even better for the manufacturer is an expectation that this new monoclonal antibody,
which enlists the body's immune system to attack cancer cells, could lead to similar treatments for other cancers.
FDA also made headlines earlier this year by approving the first new treatment for lupus in more than 50 years—Benlysta (belimumab),
developed by Human Genome Sciences with support from GlaxoSmithKline. The drug's discovery relied on information resulting
from human genome mapping and represents the first real success in this field after multiple failures.
BMS cites its new cancer drug as evidence that an increased focus on pharma R&D can yield big dividends. "R&D pays," stated
CEO Lamberto Andreotti in an interview with the Wall Street Journal, noting that the firm is investing research money very carefully in a range of disease classes and expects to have four more
new therapies approved by FDA in another year. Merck similarly told Wall Street analysts in February that it's not cutting
back on its $8 billion R&D spending plans, even though the decision may result in missing long-term profit forecasts.
Last year's slim drug-approval list included several notable products. Amgen won approval for osteoporosis treatment Prolia
(denosumab), and Roche's Genentech brought out Actemra (tocilzumab), an intravenous drug for rheumatoid arthritis. Boehringer
Ingelheim won the race to bring to market a new blood-thinner Pradaxa (dabigatron), although others may catch up soon. Probably
the most exciting new product was Dendreon's therapeutic prostate cancer vaccine Provenge (sipuleucel-T). Other new vaccines
for meningococcal disease and pneumococcal disease also were approved by the Center for Biologics Evaluation and Research