A Faster, Less Costly Route to New Drugs

Raleigh News & Observer

News_and_ObserverBy Michael Rosenberg of Health Decisions

New data from the Food and Drug Administration and a December report from the Government Accountability Office leave no room for doubt: the pharmaceutical industry is experiencing a productivity meltdown of historic proportions.

The industry’s annual R&D spending reached an all-time high of $40 billion in 2006 while approved truly new drugs bottomed out at 18, the lowest in eight years. With health care costs approaching 16 percent of the economy, the drug industry’s productivity problems threaten the nation’s economic health. The industry’s vital role in the health care system means lives are at stake as well as money.

The productivity shortfall in 2006 was not an anomaly. Since 1986 the FDA has reported an average of well over 10,000 products in active clinical testing. A mere 18 approved new drugs sends an unmistakable signal. Rather than claiming credit for a golden age of drug development, the industry must acknowledge and confront its productivity crisis. Clinical research is one area where we have the tools and understanding to do much better. Clinical trials typically cost $100 million to $200 million and last more than six years. However, leading research organizations and progressive research companies have already proved the effectiveness of a new approach – adaptive research – that cuts the cost and duration of clinical trials by one third.

To take a simple example of the benefits of adaptive methods, suppose early information in a clinical trial shows all the drug dosages tested are ineffective. In today’s typical trial, no one would know. Study sponsors would continue paying for subjects to take a drug that does not help them until all the data has come in. In addition to exposing patients to ineffective treatments, sponsors then face a grim choice: write off their entire investment or fund an entire new trial with different dosages.

By contrast, an adaptive trial would allow designated high-level managers to examine
the data as it trickles in, with safeguards to preserve the “blindness” of front-line investigators. The informed managers would either stop the trial quickly or adjust the dosages, perhaps finding an effective range to continue the trial to a successful conclusion.

Adaptive methods, at long last, permit clinical research to take full advantage of technical advances that have boosted the productivity of other industries for years – modern business basics such as electronic data collection and continuous Web access to up-to-the-minute data on critical processes. Resulting efficiency gains will enable drug companies to cut development timelines and costs, identify failed drugs earlier, optimize the allocation of research funds and recoup investments faster.

Since reduced development costs will make drugs profitable at lower prices and sales volumes, adaptive research also promises to lessen the pharmaceutical industry’s dependence on high-risk “blockbuster” drugs. All too often blockbusters intended to treat vast numbers of patients and generate billions in revenues turn out to be just plain busts – like Pfizer’s torcetrapib, a cholesterol drug abandoned in December after an investment of more than $800 million, dropping Pfizer’s market value $21.3 billion in a single day. There is much less risk for pharmaceutical companies and their shareholders in spreading investments among several drug candidates, each of which can be profitable at lower cost.

The Food and Drug Administration recently announced it will begin providing guidance for the pharmaceutical industry on the use of adaptive research. This is a commendable first step, but both the FDA and the pharmaceutical industry must move with a sense of urgency befitting the productivity crisis in drug development. Adaptive methods have been proven in the field. The potential benefits are huge and far-reaching.

Therefore, the FDA should move swiftly to encourage the adoption of adaptive research, and the drug industry should take prompt action to modernize clinical trials.

Adaptive research is a win for everybody – the drug industry, patients in need of new treatments and every individual, business and agency that strains to pay the high costs of drugs today. For the sake of all concerned, government and industry leaders must insist on a speedy transition to a new era of greater efficiency in clinical research.

(Michael Rosenberg, M.D., is president and CEO of Health Decisions, Inc., a clinical research organization headquartered in Chapel Hill and Oxford, England. Health Decisions specializes in adaptive research and works with the pharmaceutical industry.)