Patient Choice in Clinical Trials

Lancet Editorial: Patient Choice in Clinical Trials 6/11/2005

Source: Lancet; 6/11/2005, Vol. 365 Issue 9476, p1984, 1/2p

Document Type: Editorial

Two patients with Parkinson's disease are at the centre of an ongoing court case being held in New York against Amgen, which last year stopped a phase II trial of GDNF (glial-cell-line derived neurotrophic factor) to treat Parkinson's disease because of safety concerns. The patients, who were both participants in the trial, claim they experienced no adverse effects from the therapy and that the treatment reduced their tremors. They want the judge to rule that Amgen cannot withhold treatment. Amgen says it made the decision to halt the trial after reviewing preclinical data, which emerged after the start of the phase II trial, showing that monkeys given an infusion of GDNF developed cerebellar lesions. The company cited additiona l concerns that the treatment might cause production of antibodies that react to endogenous GDNF, thereby worsening the patients' symptoms. But some scientists dispute these findings, saying that no such sideeffects were observed in trial participants and that the monkeys received a very high dose of drug. GDNF is still a candidate treatment in Amgen's pipeline, but the company says more investigations need to be done to understand how GDNF affects the brain. Patient advocacy groups have seized on the controversy surrounding the trial to mount a vocal campaign to make the therapy available for trial participants and to encourage more research.

This case highlights several important clinical issues, but chief among these is patient choice. Amgen reacted responsibly to potential safety risks of a treatment with uncertain benefit by stopping the trial. But in looking for a consistent effect among the population of participants, the company could not exclude the potential benefit of the therapy to some individuals. The fact that the case has ended up in the courts highlights the lack of a suitable mechanism through which evidence from individuals can be taken into account in decision-making about experimental treatments. The case also shows that although patient choice has become a mantra for health services, when it comes to setting the research agenda, patients have barely any involvement at all. _ The Lancet Patient choice in clinical trials