At the risk of redundancy, I will comment on the torcetrapib decision. For those who do not know what torcetrapib is – read this NY Times article – End of Drug Trial Is a Big Loss for Pfizer. Torcetrapib (trying saying that quickly) raised HDL levels. Many investigators think that raising HDL is the next frontier in secondary prevention of coronary artery disease.
Prior to getting FDA approval, drug companies must do clinical trials to demonstrate efficacy and safety. Unfortunately, torcetrapib failed the safety test.
The news came to Pfizer’s chief scientist, Dr. John L. LaMattina, as he was showering at 7 a.m. Saturday: the company’s most promising experimental drug, intended to treat heart disease, actually caused an increase in deaths and heart problems. Eighty-two people had died so far in a clinical trial, versus 51 people in the same trial who had not taken it.
Within hours, Pfizer, the world’s largest drug maker, told more than 100 trial investigators to stop giving patients the drug, called torcetrapib. Shortly after 9 p.m. Saturday, Pfizer announced that it had pulled the plug on the medicine entirely, turning the company’s nearly $1 billion investment in it into a total loss.
The abrupt decision to discontinue torcetrapib was a shocking disappointment for Pfizer and for people who suffer from heart disease. The drug, which has been in development since the early 1990s, raises so-called good cholesterol, and cardiologists had hoped it would reduce the buildup of plaques in blood vessels that can cause heart attacks. Just last Thursday, Pfizer’s chief executive, Jeffrey B. Kindler, said publicly that the drug could be among the most important new developments for heart disease in decades and that the company hoped to get Food and Drug Administration approval for it in 2007.
“I’m terribly disappointed,†said Dr. Steven E. Nissen, chairman of cardiovascular medicine at the Cleveland Clinic and lead investigator of an earlier torcetrapib clinical trial. “This drug, if it worked, would probably have been the largest-selling pharmaceutical in history.â€
For people with heart disease, torcetrapib’s failure means that progress may be slowing after two decades of substantial advances against the disease. Medicines to lower blood pressure and bad cholesterol are already effective and widely used, yet heart disease remains the biggest cause of death in the United States, killing 911,000 people in 2003, according to the American Heart Association.
Heart disease patients do so much better in 2006 then 30 years ago. While a drug to increase HDL might help patients (obviously an unproven hypothesis), we actually have better ways available. Unfortunately, many patients would rather have a magic pill than to engage in the appropriate lifestyle changes.
The number one preventive measure remains smoking cessation. This totally trumps all drug therapy Weight reduction (for the obese) and exercise probably do as much as most drugs.
The problem with lifestyle changes is that they require patients to actively change their lives. Too many patients would rather search for that magic pill rather than confront the correctable lifestyle choices which they make daily.
The Food and Drug Administration said it endorsed Pfizer’s decision to end the trial and believed the company had acted properly. Torcetrapib’s failure is disappointing, but developing new drugs is risky and difficult, said Dr. Robert Meyer, director of the agency’s office of drug evaluation.
“Research is research,†Dr. Meyer said. “If you knew the answer, you wouldn’t be doing it.â€
We should never forget that great theories do not always produce great results. We need randomized controlled trials to test theories. We should never skip that step.
Withdrawing this drug is a victory for patients, although we mourn those who died prematurely participating in this study. These studies are a victory for careful research and independent monitoring boards.
KevinMD has linked to a large number of blogs who have commented on this issue – Kevin MD on The torcetrapib disaster: Blogosphere response