This is an important event that marks the approval of the first cancer vaccine. Unlike other vaccines, Provenge is not meant to prevent prostate cancer. It  boosts the immune system to fight the cancer more effectively.

The treatment is said to cost about $90,000. Not cheap considering today’s cost containment pressures but it is certainly good news for Dendreon, which has spent more than $700 million and 18 years to develop the product.

Only 30 percent of executives representing the health care industry in the United States say their companies are ready for reform and changing economic conditions… 76 percent say the impact of reform on the industry will be significant, and 54 percent say the same about the effects of the current economic crisis.

This report also found that those who feel prepared are the ones  that “drive innovation in a wider range of areas, including product design, customer service, and IT.”

Innovation and controlled experimentation are now necessary ingredients for healthcare organizations if they wish to survive, adapt, and truly prosper.

Prasugrel was tested against Plavix recently in a cleverly-designed and somewhat controversial trial called TRITON-TIMI 38. In this trial, prasugrel was shown to be about 3% better than Plavix in preventing the combined primary endpoint of death from cardiovascular causes, nonfatal myocardial infarction, or nonfatal stroke in patients with a recent heart attack and scheduled catheterization. Unfortunately prasugrel also increased the risk of major and fatal bleeding in patients, especially the elderly and those with a history of stroke. In TRITON-TIMI 38, for every one major fatal heart attack prevented by prasugrel, one person died from a fatal bleeding event.

I’ll have to think very carefully before giving prasugrel to any of my patients and the comfort with which clinicians favor this new drug over Plavix in the management of heart attack patients will determine the scope of it’s initial success. Bleeding complications have always been a concern with Plavix and now even more so with prasugrel.

The premise:

Medical schools and teaching hospitals in the United States are essential producers of basic scientific and clinical knowledge that drives our supply of new medicines, devices, and other health care innovations. Today, the funding for this work is dwindling, rendering the current structure of the biomedical research enterprise unsustainable. Given the economic crisis, the fiscal and operational models of this enterprise must be restructured if the stability of academic institutions is to be maintained and our growing health care needs are to be met.

Financial support for biomedical research in the United States comes from 3 main sources:

• Government (federal and state)
• Pharma/biotech industry
• Nonprofit foundations

The problem:

For the near future, the outlook for research funding from any of these sources is rather bleak. States are expected to reach a combined budget shortfall exceeding $200 billion…  Industry is spending more on research and development, but fewer drugs and medical devices are being approved for the market, and tightened regulation may be hurting profits… Evidence of a downturn in foundation support is already emerging. Since last October, unprecedented declines in the stock market have reduced foundations’ endowments by an average of 30%.

The solution (this one is a lot more difficult to predict and articulate):

If U.S. science is to continue playing a key role in global progress, some major belt tightening will be required. …major reform, with an eye toward long-term sustainability and management of research-enterprise growth, is essential. Although academic institutions face great challenges, our country’s unprecedented hardships may provide them with a long overdue stimulus to make needed changes. A failure to seize this opportunity could have dramatic consequences for the health of the research enterprise.

Merck expects to save about $3.5 billion annually beyond 2011 and double the number of potential medicines in late development to a total of 18 drugs.

Large pharmaceutical companies are combing the market and buying up their smaller counterparts, which sometimes have more promising medicines in the pipeline.  A few months ago, Pfizer bought Wyeth for about $62 billion.

We can expect to see more mergers and acquisitions in the pharmaceutical sector in the near future to fulfill the need for more innovative drugs in the face of increasing development costs and a future that may bring declining revenues for drug makers as payers (insurance companies, the government) tighten their belts.

These are the signs of our times, signaling the need for, and emergence of, novel ways of thinking that can open new windows of opportunity to carry us forward into the next phase of prosperity.

This, my friends, is the time to innovate.

Because of the increased efficacy of prasugrel over Plavix, the FDA granted it priority review back in February of this year, promising to announce its decision within six months. The FDA is now delaying its decision and asking for more data and 3 more months to complete its analysis. The bottom line, however, remains that for every one person that we save from a fatal heart attack, one dies from a fatal bleed with prasugrel, giving it a net effect of zero in saving lives and reducing mortality. Now the question becomes if it’s worth spending millions on prasugrel to get such a margin of net real life mortality benefit.

More info: TRITON, FDA Priority Review, FDA Delay

HDL has long been praised as the “good cholesterol,” as opposed to its evil twin LDL, aka the “bad cholesterol.” Low levels of HDL and high levels of LDL are associated with increased risk of heart disease. High levels of HDL are thought to be protective against heart disease and can offset some of the risk associated with having high LDL levels, or so we think. A recent study published in JAMA by Ruth Frikke-Schmidt and colleagues is challenging the notion that having low HDL levels is harmful. This study, which looked at patients with genetically low levels of HDL due to mutations in a gene called ABCA1, found no increased risk of heart disease after 30 years of follow up.

The results of this study can weaken the arguments about the protective effects of HDL and raise doubts about the success of designing drugs that increase HDL levels. You may remember what happened to Pfizer’s torcetrapib, a drug that was talked up to be the next big thing for the company. Despite significantly increasing HDL levels, torcetrapid actually ended up increasing the risk of death and heart attacks, leading Pfizer to halt its entire program. Some experts noted that the adverse effects of torcetrapib may have been due to the fact that it raised the patients’ blood pressure. Merck currently has a drug like torcetrapib called anacetrapib under development, which reportedly increases HDL levels without raising blood pressure.

With Pfizer’s disappointing experience with torcetrapib and Frikke-Schmidt’s new study, Merck may be on the wrong track. I wonder if we should spend more resources in promoting better-proven ways of reducing the risk of heart disease, such as eating healthy and exercising. Easy to say, I know. I too sometimes wish for a magic pill.

More info: torcetrapib, anacetrapid

Since the controversy over the ENHANCE trial and the drug Vytorin, Schering-Plough’s stock has taken a hit. On April 2nd, the company announced the launch of their productivity and transformation program to confront their new challenges. “We are taking the tough actions that are needed to respond to a tough situation,” said Fred Hassan, Chairman and CEO about the program. A day later, the company announce laying off 10% of their workforce, about 5,500 employees.

Although the media has certainly blown the results of the ENHANCE trial out of proportion, Schering-Plough should have been more transparent in the handling of the results, which were reportedly kept secret even from some of the key investigators involved in the study.

More info: Schering-Plough Press Release

Vtyorin, a cholesterol drug made by Merck and Schering-Plough Pharmaceuticals is a combination of ezetimibe (Zetia) and the generic drug simvastatin. The results of the ENHANCE trial, announced last month, showed no benefit of the combination of these two drugs over simvastatin alone. The ENHANCE trial was dogged with controversy for several months prior to the release of its results. The controversy surrounded allegations on delayed reporting of results and issues about the design of the study.

The congressional committee’s investigation was sparked by anonymous entries in an online forum designed for pharmaceutical representatives called Cafepharma. According to lawmakers, one March 2007 anonymous entry on Cafepharma said “have a buddy at (Schering-Plough Research Institute). He says that the study is a bust. Adding Zetia to already maxed out statin is useless.” Another from June 2007 said “heard it crashed and burned!” These entries, in addition to several others like them, were posted on Cafepharma months before the results of the ENHANCE trial were released. Cafepharma has told investigators that they do not collect information on anonymous users and that none of the comments in question came from registered members.

Regardless of the outcome of the congressional investigation, this story highlights the need for pharmaceutical companies to maintain transparency regarding the results of their trials. Today, it’s become very difficult to control the flow of information. Honesty is, therefore, the best policy.

More information: ENHANCE trial, Cafepharma