Saturday, February 16, 2008

FDA looks to 'broaden drug use'

According to the New York Time (Feb 16th FDA seeks to broaden the use of drugs) the FDA is considering allowing pharma companies to give doctors articles on off-label use of their approved drugs.  Docs have always been able to use their discretion to prescribe the drugs they see most fitting for a disease whether the drug is approved fro that indication or not.  However, the FDA has come down pretty hard on companies that actively promote their drugs for such off-label uses (A few years ago Pfizer had to pay up almost half a billion dollars for promoting an epilepsy drug for non approved indications that they themselves had data that showed it did not work for). Now the FDA is saying companies can give docs articles that show or suggest utility for indications other than currently approved, in the hope that 'life-saving' treatments can be more easily apparent to docs in deciding what to prescribe for their patients.

Off-label prescribing is usually good for pharma but not always.  A drug for macular degeneration, Genetech's Lucentis, suffered from same-company poaching when the same company's cancer drug Avastin was adopted by retina specialists as a cheaper alternative to Lucentis.  In most other cases off-label prescribing leads to more prescriptions written and higher sales for a given medicine.  If it benefits the patient, what is the problem with off-label prescribing and what the FDA is proposing?  Well, for starters, off-label prescribing is inherently risky.  The drugs in question have not been tested for safety issues in any group apart from the approved-for population and their proposed efficacy also does not have rigorous data behind it.  A second issue with allowing pharma companies to give articles to docs is that the companies can be selective about what they give, choosing to distribute only the supportive articles and not the detractors.   Rarely is data cut and dried and there is usually plenty of evidence to support a contrary view to the one that supports an alternate indication.  A third issue is that pharma companies often sponsor trials to show their drugs are useful for additional diseases. None of these points automatically mean it is unethical for anyone to show a doctor an article on anything that is out there in the public domain.  We know for sure that is is unlikely most docs get much time to go through the literature and develop a balanced view on what is out there.

So, where does this leave the issue?  Here are the 'facts':
1) Drugs often have uses beyond their current approved indication.  
2) Doctors are in a unique position to see their patient as a whole, and to prescribe what they feel is best for each individual
3) Pharma companies are often more aware of off-label experiments that most, and even sponsor a number of them
4) Doctors and health professionals do not have time for a regular detailed review of the literature
5) Pharma usually gains from encouraging off-label drug use 
6) Patients should be able to get access to the best drug for their disease, approved or not

The latter point speaks to an earlier argument where patients are not allowed access to truly potentially life-saving options because the drugs are in trial stage and not approved for anything yet.  If the patients do not fit the trial criteria (set to optimize the chance of success of a drug)  then they cannot get access to their experimental medicines. To my mind, this is where the most action by the FDA is required.  For many, these experimental drugs are a genuine last hope and I believe if patients are willing to assume the risk they should be allowed to take the meds.  

So this brings us to patients and what they are prepared to risk.  Since an off-label indication is risky, then shouldn't the patient also have a say?  I don't believe it is enough to 'educate' the physician with the articles as suggested by the FDA. Shouldn't materials also be provide for the patient?  Perhaps a balanced view of what is known and what is not, and a statement about potential risks acknowledging rigorous studies have not been done?  I think balance is the key for both docs and patients.  Give them the info, sure, but be sure all aspects are represented--then the doc and the patient can have the discussion and make the choice.  Seems obvious to me but I'd be interested in what others think.

Friday, February 8, 2008

Some predictions...

  • Targeted medicines for all diseases. Few side effects, few efficacy failures--we get it right first time!
  • We understand how to help the body help itself. We fight off disease and destruction every day without realizing it. In the future we will know how we do it and be able to coax our body to heal itself at will
  • The era of over dosing on prescriptions drugs is coming to an end. We are beginning to know too much to be fooled by simplistic treatment regimes that pile on one med after another. Holistic, gentler approaches will come into vogue in the next 10 years. In the meantime, there will be a public backlash against high priced medicines that we are told we must take. Anti-infectives will be the exception, and the most lucrative area in the near future for pharma and biotech
  • Big Pharma is definitely dying. The model cannot withstand the shift to targeted medicines and increased generic use. As we understand our disease states better, we will be better able to create personal medicine approaches using old and/or new meds. There will be less demand for sexy new meds that cost millions. No more big houses for pharma execs and fancy off-site meetings in the sun...:(
  • Biologics will be seen as a flash in the pan. A red herring. This is a wild suggestion, but it's an intuition. Biologics are fraught with problems and horrendously difficult to make. We thought we understood proteins once before when we were into macromolecules... The future is somewhere else

Thursday, February 7, 2008

Physiologists-come back-all is forgiven

Google this story.....Diabetes Study Partially Halted After Deaths

Published: February 7, 2008 New York Times

...and you will be amazed. Apparently lowering blood sugar in Type II diabetics (90% of the diabetic population have Type II) increases your chance of dying. It was supposed to be the other way around. This is a quick post so I'll get to the point. Perhaps the physiology of the diabetic patient is fundamentally different to that of a non-diabetic? This is not a radical statement, nor a novel one, but the pharmaceutical profession do tend to think of a diseased individual as a person having 'normal' physiology with an 'abnormal' disease superimposed on it. So, we just add drugs, or drug cocktails, to 'fix' all the abnormal bits (for eg the high sugar, or the high cholesterol). Most drugs are assessed by their effects on receptors and organs rather than whole systems as they go through drug development. They are marketing in the same manner. No wonder we tend to pile up the meds to counter all the deviant aspects of a person's physiology rather than stepping back to assess the overall picture. The 'overall picture' is a scary concept. Modern medicine doesn't give us the tools to assess nor treat it.
In the current age of high profile drug cocktail-related deaths such as the recent passing of Heath Ledger, and Ann Nicole Smith, perhaps it is time for those of us in the health professions to think of how we can begin to see the human as an ever evolving system over the years, that becomes fundamentally changed as we reach the tipping point for various disease states (diabetes is only one--dementia, autoimmune disorders, depression, anxiety etc, etc are others). When we do become diabetic, depressed, etc, our baselines have changed. And, because we are an interconnected mess of tissues and cells, these baselines affect every aspect of our being from our response to drugs to our mental view of the world. We can't just keep throwing on more meds until the blood sugar is low enough, or we feel calm and serene enough to get back to work. It's time we spent more time looking at the heart of our diseases and the dynamic physiology that underpins them; that complex backdrop that determines our unique response to everything that interacts with us, natural or imposed.