USA - Judge Orders Drug Maker to Provide Experimental Treatment to Terminally Ill Teenager

[Metomni]

http://www.nytimes.com/2008/08/21/bu...dystrophy.html

A 16-year-old patient, terminally ill with a rare form of muscular dystrophy, should be allowed to use an experimental drug treatment despite objections from the drug’s developer, a federal judge in Newark ruled on Wednesday.

The case, which touches on major ethical issues, is being closely watched by the pharmaceutical industry.

Under the ruling, Jacob Gunvalson, of Gonvick, Minn., would be able to start taking a drug intended to treat Duchenne muscular dystrophy, a rare and fatal disease that strikes boys and young men. The developer, PTC Therapeutics of South Plainfield, N.J., contends that Jacob does not meet the criteria to be a part of the drug’s clinical trial, and is planning to appeal. Federal regulators must also approve his application to use the drug.

It was not immediately clear what implications the ruling would have for other patients seeking access to clinical trials of experimental treatments. Drug developers tightly enforce parameters on who can participate in these trials, citing concerns about safety and the validity of the research.

Those who do not meet the guidelines are excluded, often because their cases might alter the results, even if the patients consider the trial a final hope in the face of a devastating illness.

In Wednesday’s ruling, Judge William J. Martini, of the United States District Court in Newark, said that Jacob should be allowed to take the experimental drug even though he does not meet the criteria for eligibility. The details of the order were still being hammered out late Wednesday.

“We’re very pleased with what has happened,” said Marc E. Wolin, a lawyer for the Gunvalson family. “This was the relief that we sought. It’s a big step in the right direction.”

But the chief executive of PTC Therapeutics, Stuart W. Peltz, said he was concerned the decision could encourage patients to sue to gain access to clinical trials.

“To have the courts decide who gets the drug, it’s something we should all be concerned about,” Mr. Peltz said. “It’s not the way clinical trials should be thought of, in our opinion.”

The Gunvalsons’ lawsuit was not intended as a challenge to widespread practices at drug companies. Its circumstances were unique, complicated by allegations of deception by Cheri Gunvalson, Jacob’s mother.

In the suit, Ms. Gunvalson said that officials at PTC Therapeutics led her to believe that her son would be allowed to take part in a clinical trial, only to ultimately say his disease had advanced to a point where Jacob no longer met the proper criteria for the study.

She said she helped the company obtain federal grants to research the disease, and that the company discouraged her from enrolling Jacob in an earlier study with the understanding that he would be allowed to take the drug in a later trial.

PTC Therapeutics denied those claims.

“The company made a variety of promises to Cheri about access of the drug,” said Michael A. Hatch, a former attorney general of Minnesota, who represented the Gunvalson family. “We’re here only to try to help Jacob get access to something that was promised to him,” he said.

While drug developers occasionally allow exceptions for patients who do not meet a study’s criteria, some companies fear overuse of this process could discourage patients from volunteering for trials, where many participants receive placebo pills.

“We understand the pressure and the needs of the community, but we have to do it in a way that collects information in a safe, effective and fair way,” said Mr. Peltz.

But Mr. Hatch said that the drug company’s decision to appeal Wednesday’s ruling was misguided. “They’re worried about a floodgate,” he said. “This is not a floodgate case. The situation involving Jacob is extraordinarily unique, concerning the patients and the company.”

[newgrower]

I hope it does become a floodgate. There are companies that do 'compassionate use', where they let people take unapproved drugs once they have shown promise and some general safety. The reason the companies want to limit the people on allow the treatment is because they will still have to report results on these patients to the FDA and don't want to get bad results because the ideal candidates weren't used.

Usually when there is a problem with a newly approved drug, it's found out in post-approval studies where its easier for companies to bury unfavorable results. Things show up in these studies that didn't in the pre-approval studies because you have a larger and more diversified population.

The companies must tell the FDA everything about the study and what analysis they are going to do before they start the study and must report all results for pre-approval studies, even negative results. But if they did more compassionate use studies and had to report these results on a more diversified population before final approval, some of the rarer side effects may come to light earlier.

Of course you do need to balance the safety of these subjects in compassionate use trials with the possible benefits to the patients and society as a whole in this expanded trials. Also you need to balance the added burden for approval with the time to approval of new medication. But I think an expanded use of reported compassionate use trials would be a good addition.

Reputation Comments on this post:

Spot on wrap-up sir, it's sad that they're willing to risk the health of others simply to get favorable results.