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Weekly UpdatesWith the U.S. Court of Appeals for the District of Columbia Circuit ruling 8-2 last week that terminally ill patients do not have a constitutional right to obtain access to unapproved prescription drugs, pressure is building on Congress to act to ease the restrictions on patients who have exhausted traditional treatment options.
As a result, lawmakers likely will reintroduce legislation that would require FDA to allow terminally ill patients to purchase experimental drugs with the recommendation of their physician.
Sen. Sam Brownback (R-KS) and Rep. Christopher Shays (R-CT) last session introduced the Access, Compassion, Care and Ethics for Seriously Ill Patients Act, but the bill did not go anywhere at the time. This session the bill likely will be introduced by Brownback and Rep. Diane Watson (D-CA).
Public polls indicate the public is strongly behind allowing terminally ill patients access to experimental drugs. An online poll taken on MSNBC Aug. 16 found that 94% of participants disagreed with the court decision.
The pharmaceutical industry generally sees the matter differently. It has resisted any government mandate to provide experimental treatments to patients. Companies point to ethical issues involving unapproved uses of drugs and concerns over whether patients provide informed consent.
"Our position is that it should be left to the individual companies to make that decision," said Alan Goldhammer, vice president of regulatory affairs for Pharmaceutical Research and Manufacturers of America (PhRMA).
Providing experimental drugs to all comers could hamper clinical trials, he noted.
"Companies must be careful to make sure that after making experimental drugs available to more patients, they are able to find enough volunteer patients willing to abide by the restrictions and rules of a clinical trial," Goldhammer said in a statement.
The recent decision is the latest chapter in a long and controversial saga about the rights of patients and the role of FDA in protecting the public health.
In 2003, the Abigail Alliance for Better Access to Developmental Drugs and the Washington Legal Foundation filed a lawsuit against FDA to obtain access to experimental medications for terminally ill cancer patients.
The lawsuit asked FDA to provide a special initial approval of experimental medications that appear effective and allow their sale and distribution to terminally ill patients who have no other approved treatment options. FDA argued that programs currently exist to provide experimental medications to terminally ill patients and that increased access to such treatments would lead to unacceptable risk.
Frank Burroughs, co-founder of the alliance, believes that the increased media attention of the appeals case will bolster the possibility of legislation being passed. "It was a devastating decision for the Abigail Alliance, but the upside of it is it has generated media coverage and that coverage is definitely positive," Burroughs said.
He continued: "The Abigail Alliance is dumbfounded that most of the justices tragically missed the merits of the case of Abigail Alliance v. FDA. The majority opinions are terribly flawed. The Abigail Alliance feels it is going to be hard for FDA and its supporters to spin the court's decision that "dying patients have no right to pursue life?"
Part of Burroughs' frustration likely is due to the fact that a three-judge panel of the same appeals court ruled last year that the terminally ill did indeed have a constitutional right to experimental treatments. FDA asked the full appeals court to reconsider the decision, which it did, voting 8-2 to side with the government.
Advocates were hoping that the court would allow quicker access to experimental drugs. Today only those who have been accepted into clinical trials have access to experimental therapies. And the three-stage approval process can take up to 10 years. Occasionally, pharmaceutical companies make some unapproved treatments available to terminal patients on an emergency "compassionate use" basis.
FDA stated that it was pleased with the court's decision because it "upholds the constitutionality of the FDA's role," spokeswoman Susan Cruzan said earlier in August.
While FDA facilitates "appropriate treatment access to investigational therapies" it also requires that drugs are "proven to be safe and effective" before being marketed to U.S. consumers, Cruzan said.
FDA proposed in December a regulatory change that would make experimental drugs more widely and easily available to seriously ill patients with no other treatment options. The agency is reviewing comments on the proposal and does not have a specific time for issuing a final rule, Cruzan said.
Christina Marcus, a lawyer with King & Spalding LLP in Washington, noted in an Aug. 16 interview that the citizen's petition promoted by the Abigail Alliance, and the recent decision, may already have affected FDA policy.
"Although FDA did not identify the underlying Abigail Alliance citizen petition as a contributing factor, it may have stimulated or strongly supported FDA's recent (Dec. 14, 2006) publication of a proposed rule to clarify existing regulations and add new types of expanded access (including for individual patients) for treatment use.
"The proposal identifies FDA willingness to allow seriously ill patients early access to some unapproved therapies where there is no comparable and satisfactory alternative therapy available.
"However, a few important differences in approach appear to be that: (i) under the FDA proposed regime, the burden of proving appropriateness of expanded access remains on the patient on a factual, case-by-case basis, and (ii) FDA would retain the right to judge whether the potential benefit to a patient justifies the potential risks and that the risks are not unreasonable in the context of the disease or condition to be treated.
"By contrast, the Alliance petition would create a regime that presumes the right to early access and largely eliminates FDA from the risk/benefit analysis."
Physicians also are concerned about what some opponents to the proposal call a drug "free-for-all."
Allen Lichter, chief executive of the American Society of Clinical Oncologists, worries about the effect looser restrictions would have on clinical trials. There's only so much of any experimental drug to go around, he said, and having a treatment available to some might persuade others to avoid the trials altogether, where they might be treated with placebos.
"We don't believe the solution is to open the floodgates," Lichter said.
Jill Anderson, a prominent attorney with Moses & Singer in New York, noted in an Aug. 16 interview the following pros and cons of the Abigail decision:
Pros
* A decision favorable to Abigail's Alliance could have impeded the progress of research by de-motivating potential patients from participating in clinical trials.
* Researchers will remain able to minimize exposure of patients to deadly side effects.
* A decision favorable to Abigail's Alliance would have made it difficult for FDA to monitor the safety of individuals receiving the drug.
Cons
* Terminally ill patients can still only receive a potentially life-saving drug through participation in a clinical trial; and they still run the risk of receiving placebo.
* Patients arguably have the right to choose whether to take a life-saving medication or die untreated.
At the online physician network www.sermo.com, the response to the decision was mixed. Some physicians wrote comments indicating that they did not think it was a bad idea for FDA "to go slow, wait to have enough data, and thoroughly test for adverse effects."
Another pointed out: "I'm afraid that FDA has been hopelessly burned by late coming adverse event reports, as in the Vioxx case, and that any drugs with the safety profiles that our drugs do will have little chance at accelerated approval unless they raise the dead with no definable side effects."
Several others argued that FDA needs to take into better consideration the severity of the disease and treatments available when approving new drugs--which boils down to FDA simply executing on its existing intent and promise of accelerated approval for patients with terminal illnesses, according to the site.
The case may end up being heard by the U.S. Supreme Court. Anderson noted in the interview that while it is unlikely that the court would alter the decision, the large amount of publicity the case is receiving makes the possibility of legislation more likely.
By Joseph Pickett, Managing Editor
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