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CenterWatch Commentary: Experimental Drug Access Wars Continue

Friday, August 22, 2008

A U.S. District judge Wednesday ordered PTC Therapeutics to supply its experimental drug to 16-year-old Jacob Gunvalson, who has Duchenne muscular dystrophy, a terminal disease for which there is no known cure.

Gunvalson’s parents brought a lawsuit against PTC Therapeutics because they claim that the company had promised them that he could participate in a 96-week clinical trial of PTC 124, even though Jacob was not able to participate in the 28-day preliminary phase of the clinical trial due to exclusion criteria. The company claims no such promise was made.

What seems to have hurt PTC Therapeutics’ case was the company’s particularly close relationship with the family. The Gunvalsons claim that PTC employees made promises to the family and that a senior vice president once hosted them at her house overnight. “They had a special relationship that this court considers more than typical,” the judge said.

Although PTC president and chief executive Stuart Peltz denied there was anything unusual about the company’s relationship with the Gunvalson family, this situation should serve as a lesson to all in the industry.

You cannot form the kind of relationship with a patient where an overnight is normal while still maintaining the line between company and potential clinical trial participant. If nothing else, PTC Therapeutics blurred the line between friendship and a business relationship. And it’s not surprising that, during the many hours the Gunvalsons spent with PTC representatives, the family was led to believe their son could participate in a PTC Therapeutics clinical trial.

In a statement, Peltz said, “In contrast to big pharmaceutical concerns, it is quite natural for our team to form close relationships with patients and other members of the rare disease community.”

Do all small pharma and biotechs studying orphan diseases form these kinds of close relationships? It’s one thing to be invested in a trial’s participants and know them all by name, but it’s another to host sleepovers. PTC crossed a line and opened itself up to misinterpretations and even liability.

What if, for example, a family has reservations about their child’s participation in a clinical trial and only agrees to participate after developing a “close relationship” with the drug company’s representatives? If something goes wrong during that clinical trial, could the family claim that they did not give a truly informed consent? 

Even though I side with PTC in regard to limiting drug access, it’s easy to understand why the Gunvalsons would assume that Jacob would have access. They spent time together; they were hosted by company executives overnight; they were friends.

People who form close relationships with each other have expectations. One is that the other party will help you when you are in need.

This judgment will most certainly be overturned, but in the meantime, small biopharma companies should draw a bright line between their clinical trial participants and friends.

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