My Body, My Choice?

By Daniel R. Matlis

No, it’s not what you think; I’m not going “pro” this or “anti” that on you.

I am referring to the Food and Drug Administration’s (FDA) proposal to make experimental drugs widely and easily available to seriously ill patients.

According to Dr. Andrew C. von Eschenbach, Acting FDA Commissioner “This proposed reform is carefully designed to balance several objectives.” von Eschenbach added “One goal is to enable many more patients who lack satisfactory alternatives to have access to unapproved medicines, while balancing the need for safeguarding the individual patient. Another equally important goal is to ensure the continued integrity of the scientific process that brings safe and effective drugs to the market.”

The proposed rule is a direct response to a 2 to 1 decision by the U.S. Court of Appeals for the District of Columbia Circuit overturning a lower court’s ruling in a case brought by the Abigail Alliance for Better Access to Developmental Drugs.

The court ruled that “barring a terminally ill patient from the use of a potentially life saving treatment impinges on the right of self-preservation.” Further, the court held that once drugs have passed safety trials, they should be made available if they might save someone’s life.

Judge Judith W. Rogers wrote “If there is a protected liberty interest in self-determination that includes a right to refuse life-sustaining treatment, even though this will hasten death, then the same liberty interest must include the complementary right of access to potentially life-sustaining medication, in light of the explicit protection accorded ‘life’.”

Dr. Janet Woodcock, FDA’s Deputy Commissioner for Operations stated “FDA hopes this proposal will increase awareness in the healthcare community of the range of options available for obtaining experimental drugs for seriously ill patients,” she added that “By clarifying and streamlining the processes, FDA also hopes to encourage companies to make such drugs available, and reduce barriers for healthcare practitioners in obtaining them.”

The proposed rules, which are open for comment for 90 days, are described in detail at the following FDA web address: http://www.fda.gov/cder/regulatory/applications/IND_PR.htm

I hope and pray that none of us ever face such a choice, but if we do, it should be our choice to make.


Senate Confirms von Eschenbach as FDA Commissioner

Last week, Dr. Andrew von Eschenbach was confirmed by the Senate to head the U.S. Food and Drug Administration. Dr. von Eschenbach is 65-year-old oncologist, surgeon and cancer survivor. He has been acting commissioner at the FDA since September 2005, and is the former director of the National Cancer Institute.

His appointment will provide the agency with strong leadership as it heads into a potentially tumultuous year in light of legislative changes on Capitol Hill. The nomination, which passed the Senate by a vote of 80-11, will now be sent to the president.

Dr. von Eschenbach is a strong proponent of transforming the FDA to keep pace with changes in healthcare. He commented that the Agency must establish itself “as the bridge that supports new molecular-based interventions as they move across the discovery, development, and delivery continuum.” His vision for the Agency calls for aligning, adapting, and recognizing the importance of FDA’s leadership role and its responsibility in the metamorphosis that will define a new era in Healthcare.

 “This healthcare system of the future will be personalized by developing a sophisticated understanding of not only the disease process, but also of the individual patient. In understanding both the disease and the person at the molecular level, physicians will be able to provide treatment options uniquely suited to a patient’s particular needs. Instead of empirically prescribing a pill and hoping it works, patients will receive treatments designed specifically for them. We will use the new tools of molecular medicine to develop new individualized interventions and in the very process of molecularly monitored delivery to patients our understanding of the biology of disease in individual humans will be elucidated. In this way, delivery will become, in itself, a platform of discovery” commented Dr. von Eschenbach in a recent speech. He went on to say “I expect that this healthcare system will be much more participatory. Increased utilization of information technologies and improved communication strategies will allow patients to assume a substantially more active role in their healthcare.  It will be important for us at FDA to consider how we can work together with doctors and patients to make sure that they have the greatest opportunity to maximize the benefits of new medical products in a way that preserves their ability to make individualized choices free from restrictive burdens, and that the data derived from health care can be used to further enhance the safety and effectiveness of these products.”

It is clear from his comments that he has the right ideas to lead and transform FDA. However, implementing these ideas is not going to be easy. Although Industry and Agency staffers agree that the FDA is in need of increased funding, allocating resources may prove to be difficult under the current budget pressures.


Pharma’s Business Model Doesn’t Seem to Be Working

That’s not just me saying it. Alan Murray, Managing Editor at the Wall Street Journal agrees.

In an Interview on CNBC, Mr. Murray said “The Fact of the matter is the way they (Big Pharma) develop drugs, the way they sell drugs and the way they interact with government regulators and the public all needs to change”. 

His comment on “The era of the blockbuster looks like is probably over” conveyed a similar message as of my last article on the “Beginning of the End for Big Pharma”. 

Click here to see the complete interview. By the way, you can fast forward the commercial to get to the interview.


Are We Witnessing the Beginning of the End for Big Pharma?

by Daniel R. Matlis 

Recently I attended a Pharmaceutical Industry panel and heard a high ranking industry executive illustrated the beginning of the end for the Pharmaceutical Industry.

He described the last 30 years as the golden era for research based Pharmaceutical companies. He lamented that most of the “easy drugs” have been invented and explained that the rate of invention for new pharmaceuticals is slowing.

In addition, 20 years from the invention date, the drug goes off patent (it typically takes 12 to 15 years for a drug to get approved after invention) and a robust generics industry is ready to take the drug off Big Pharma’s hands.

He went on to say that it’s only a matter of time before all pharmaceutical compounds are discovered and developed, at great expense to big pharmaceutical companies. These drugs will then be produced by the generics industry, and sold at a much lower price.  He questioned whether, at that point in time, there will be anybody around to develop new pharmaceuticals.

As I was listening, I thought about the saying “the only constant is change”. I assumed this was a relatively recent refrain, maybe dating back to the industrial revolution. After some research I found, to my surprise, that the phrase was coined nearly 2500 years ago by the Greek philosopher Heraclitus.

It makes sense; after all, human progress has demanded change throughout history. The stone-smith was put out of a job by the bronze-smith and he in turn by the iron-smith and so on. And one day, the Pharma-smith will be put out of a job by the personalized-medicine-smith. This cycle will go on until the end of time, or until we stop progress.

Is Big Pharma doomed?

This reminds me of the movie “Millennium”.  As the film is about to end, Kris Kristofferson and Cheryl Ladd step into a worm hole to the unknown. A voice says: “It is not the end.  It is not the beginning of the end.  It is the end of the beginning”.

So too, in our industry, we are not witnessing the beginning of the end of our fight against disease and human suffering, but the beginning of a new chapter.

As I discussed in “Chemicals to Cell Culture“, the Life-Sciences industry is undergoing a metamorphosis. Historically separate disciplines, like pharmaceuticals, devices, diagnostics, biotech, and nanotech, are converging.

This convergence is enabling the development of personalized treatments in our quest to eradicate disease and end human suffering.

In my opinion, those Life-Science companies that embrace change and seek new technologies will not only survive but thrive.

Those who hope that it will go away, will go the way of the stone-smith and the bronze-smith and the….