Critical Path Made Personal

  • by: |
  • 08/29/2006

Here’s a new way to think about whether or not FDA’s Critical Path initiative is important …

… Ask yourself, “What if it was my child?”

And then read this article from today’s edition of the New York Times.

A Conversation With Mary V. Relling
Saving Lives With Tailor-Made Medication

By CLAUDIA DREIFUS

MEMPHIS — In Mary V. Relling龝 office in St. Jude Children’s Research Hospital sits a small ceramic statue of St. Jude Thaddeus, the patron saint of impossible causes.

Dr. Relling, the head of the department of pharmaceutical sciences at St. Jude, has a fondness for impossible causes.

Her own is pharmacogenetics, a clinical discipline in which doctors use high-tech genetic testing to custom-make drugs to patients’ individual needs.

Though pharmacogenetics is controversial and not yet widely done, Dr. Relling, 46, travels the country advocating its use. At St. Jude, patients with leukemia are now routinely given genetic tests to determine their individual response to a medication. “We’ve seen it save lives here,” she said. “That’s made me a believer.”

When hundreds of patients are given a drug, she continued, “some will get no benefit, others will have terrible side effects, and still others will get benefits with tolerable side effects.”

Gene variants may be the cause.

Q. How is this tailoring of drugs different from the way they’re currently ordered?

A. Till now, there’s been a one-size-fits-all approach. In most cases, an average dose of a medication is ordered, and then, if the patient suffers side effects, the dosage is adjusted. With gene testing, we can customize the prescription.

Here at St. Jude, we’ve been gene-testing every child who comes to us with leukemia. I study acute lymphoblastic leukemia — A.L.L., the most common childhood cancer. When a youngster comes in with A.L.L., we get a sample of their DNA. We put it on a special computer chip that scans a half-million different places on the genome. Mostly, we’re looking for unusual variations of the genes and misspellings of the genetic code.

We have a database from earlier patients that helps us predict a patient’s risk of relapse and which misspellings are likely to result in drug sensitivities.

Q. Are there other diseases where the process might be useful?

A. The same medicine we use to treat leukemia is also prescribed for Crohn’s disease and ulcerative colitis. So that same genetic test could be employed to reduce side effects with those conditions.

At the moment, there seems to be a lot of promise for pharmacogenetics in the treatment of arthritis, heart disease, colon cancer and even psychiatric diseases like depression and schizophrenia.

Q. One can almost hear economists everywhere groaning, “Oh, no! Not another test to add to health care costs!”

A. The basic research behind pharmacogenetics — figuring out which genes are important with the various drugs and diseases — is costly. But on the clinical level, you can save money. With leukemia, we’ve seen that testing costs are minor compared to the savings gained by avoiding drug reactions, blood transfusions and additional hospitalizations.

Q. How widespread is genetic testing for prescriptions?

A. It’s very rarely used. Most probably, you can find it at some academic centers in big cities. And, of course, for many medications, the research isn’t in yet about which genes are important and why. But even where tests have been approved, insurers don’t cover all the costs, and that’s hindered this from growing.

Most prescribers don’t understand genetics very well. The fruits of the Human Genome Project have only been out for about five years, and a lot of doctors and pharmacists did their training before that.

I’ve heard people say that medicine won’t change until there are major lawsuits against prescribers who fail to use tests to individualize therapy.

Q. How have the drug companies responded to the promise of pharmacogenetics?

A. Unfortunately, they are not set up for it. The big pharmaceutical companies have a different business model. They make their money from blockbuster drugs that reach millions of people with standardized doses. They don’t want their markets to fragment, which is the obvious effect of pharmacogenetics.

Genomic testing is going to mean that we define smaller and smaller markets for every drug. Instead of one medication for high blood pressure, a manufacturer will have to produce dozens of variants and combinations.

It’s already difficult to get drugs that benefit smaller numbers of patients. “Orphan” drugs are often not manufactured because they help only a small group.

In pediatric cancer, we see what happens to orphan drugs because children’s cancers are rare and, thus, orphan diseases. Of the nine drugs we regularly use for leukemia, seven have been unavailable for varying periods of time during the last decade.

Q. What can you do in that situation?

A. We’re trying to start making it here at St. Jude. We’re not a drug manufacturer, and it isn’t easy for us to do. We’re also working with pharmaceutical companies to see if we can’t help them make it available. But we can’t depend on market forces for our supply.

We’ve seen that the pharmaceutical companies, if they are interested in cancer drugs at all, are mostly concerned with adult cancers — the larger market.

Q. How did you become a pharmacist?

A. I attended the University of Arizona in the late 1970’s, as a French major. In my freshman year, I took the required chemistry course with a brilliant teacher, William Lippincott. He made chemistry come alive. Becoming a pharmacist seemed like a practical route to a chemistry career, with good job possibilities after graduation.

Even today, it’s a great profession for a young person to consider. There is a tremendous demand.


CMPI

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

Blog Roll

Alliance for Patient Access Alternative Health Practice
AHRP
Better Health
BigGovHealth
Biotech Blog
BrandweekNRX
CA Medicine man
Cafe Pharma
Campaign for Modern Medicines
Carlat Psychiatry Blog
Clinical Psychology and Psychiatry: A Closer Look
Conservative's Forum
Club For Growth
CNEhealth.org
Diabetes Mine
Disruptive Women
Doctors For Patient Care
Dr. Gov
Drug Channels
DTC Perspectives
eDrugSearch
Envisioning 2.0
EyeOnFDA
FDA Law Blog
Fierce Pharma
fightingdiseases.org
Fresh Air Fund
Furious Seasons
Gooznews
Gel Health News
Hands Off My Health
Health Business Blog
Health Care BS
Health Care for All
Healthy Skepticism
Hooked: Ethics, Medicine, and Pharma
Hugh Hewitt
IgniteBlog
In the Pipeline
In Vivo
Instapundit
Internet Drug News
Jaz'd Healthcare
Jaz'd Pharmaceutical Industry
Jim Edwards' NRx
Kaus Files
KevinMD
Laffer Health Care Report
Little Green Footballs
Med Buzz
Media Research Center
Medrants
More than Medicine
National Review
Neuroethics & Law
Newsbusters
Nurses For Reform
Nurses For Reform Blog
Opinion Journal
Orange Book
PAL
Peter Rost
Pharm Aid
Pharma Blog Review
Pharma Blogsphere
Pharma Marketing Blog
Pharmablogger
Pharmacology Corner
Pharmagossip
Pharmamotion
Pharmalot
Pharmaceutical Business Review
Piper Report
Polipundit
Powerline
Prescription for a Cure
Public Plan Facts
Quackwatch
Real Clear Politics
Remedyhealthcare
Shark Report
Shearlings Got Plowed
StateHouseCall.org
Taking Back America
Terra Sigillata
The Cycle
The Catalyst
The Lonely Conservative
TortsProf
Town Hall
Washington Monthly
World of DTC Marketing
WSJ Health Blog