Wednesday, August 08, 2007

D.C. Court of Appeals limits access to experimental drugs

In a court case Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach that's been working it's way thru the appeal process, the Washington DC US Court of Appeals ruled 8 to 2 yesterday that terminally ill patients do not have a constitutional right to force the FDA and drug manufacturers to allow access to experimental drugs.

The court wrote, "We conclude there is no fundamental right 'deeply rooted in this nation's history and tradition' of access to experimental drugs for the terminally ill," said Judge Thomas B. Griffith, a Bush appointee, citing a Supreme Court decision that rejected the notion of a constitutional right to die.
"Although terminally ill patients desperately need curative treatments ... their deaths can certainly be hastened by the use of a potentially toxic drug with no proven therapeutic benefit. Thus, we must conclude that, prior to the distribution of a drug outside controlled studies, the Government has a rational basis for ensuring that there is a scientifically and medically acceptable level of knowledge about the risks and benefits of such a drug. We therefore hold that the FDA's policy of limiting access to investigational drugs is rationally related to the legitimate state interest of protecting patients, including the terminally ill, from potentially unsafe drugs with unknown therapeutic effects."

Griffith's opinion was a strong bipartisan decision joined by both conservative and liberal members of the D.C. appeals court.

The plaintiff's argument centered around the contention that the policy deprives dying patients of their right to self-defense and violated the Fifth Amendment clause that people cannot be deprived of life, liberty or property without due process of law. While it may sound heartless at first glance, the rejection of this claim does make sense for several reasons and its supported by organizations like the American Society of Clinical Oncology, the National Organization for Rare Disorders, the Marti Nelson Cancer Foundation, and the National Breast Cancer Coalition.

The suit was spearheaded by Abigail’s Alliance for Better Access to Developmental Drugs, and was founded to honor Abigail Burroughs. Ms Burroughs died in 2001 of head and neck cancer at age 21 while fighting to gain access to drugs not yet FDA approved.

In an article in The New Republic, Medical Oncologist and ethics expert, Dr. Ezekiel Emmanuel argues:
The Abigail Alliance approach would eliminate the kind of careful monitoring on larger groups of patients [that we need] before widespread access becomes available....Instead, unproven drugs would be tested for safety in fewer than 80 people [in phase I trials] and then could be sold to patients. The benefit of a few desperate patients would come at a steep cost for the rest of us.”

Emmanuel and others worry that if people can do end runs around the protocols for testing experimental drugs, the number of patients willing to participate in randomized clinical trials (the gold-standard for drug testing) where half of participants receive the placebo or conventional drug will dry up. If this decision had gone the other way “it would have undermined the entire drug approval process,” said William B. Schultz, a former deputy commissioner of the Food and Drug Administration, who wrote an amicus brief to the court supporting the FDA's position.

Who will be willing to risk receiving a placebo or a conventional therapy instead of the “breakthrough” drug? Not everything new is good in terms of chemotherapy agents. Keep in mind that over 40% of the products that make it to Phase III FDA trials (the step preceding approval) ultimately are abandoned as they prove ineffective and/or prohibitively toxic.

Several other tricky aspects crop up in the scenario of allowing purchase of these drugs if they've been excluded or rejected from clinical trials. It will be VERY expensive in many instances, which is going to exclude the poor. It will also create incentive to "game" the system by having yourself excluded from clinical trials (and avoid the chance of getting the placebo control) in order to pay for the product. Uncontrolled access to the drugs makes it dramatically harder to track adverse events as easily and identify trends between groups.


Anonymous said...

Everything that you says makes sense, and in the abstract I completely agree with you. However, having cancer yourself changes things. I know that were I in the situation where the available well had run dry, I'd do whatever I could to get my hands on a drug that I thought might help.

Imagine how difficult it would be to be an oncologist who has to tell a dying young patient that they can't try a promising new drug, even if you, the doc, believes it might save them.

It was only a few years ago that herceptin was approved by the FDA, and in fact, it's still not approved in Europe. At what point would you think it appropriate to make it available to advanced BC patients? Not everyone that a drug could help is qualified or accepted by trials.

Perhaps there's a middle ground of providing drugs that look especially promising to terminally ill patients. Or, perhaps the trial system should be changed to have a special category for high risk patients, and to make inclusion of these patients automatic and at the expense of something other than the drug companies.

All rational aside, denying hope to the dying just seems cruel.

Dr. Rob Oliver said...

I agree with the dilemma you pose. However, this is going to come down to what's the greatest good of society (ie. better information on the efficacy/safety of treatments)trumping the proposed right of an individual to go "off the grid" for experimental therapy.

There already is a mechanism we have for testing these experimental drugs, the case here involved someone who was not qualified for the trial. While it may seem capricious and patronizing, the rules we have re. this come from centuries of medical experiments with snake-oil and folk-medicine proffered to patients.

Again, you just have to look at the hundred of drugs abandoned late in clinical trials to see how rare some of these medications make dramatic differences in cancer treatment.

It's still kind of a truism that if you can't surgically remove any solid cancer that's contained locally (not luekemia/lymphoma types), you can't cure a cancer. Hopefully some of these immuno therapies and directed treatments will rewrite that book