Can Patients Help Evolve Clinical Trials?

Can Patients Help Evolve Clinical Trials?

04.25.11 | By Kate Connors

In today's Wall Street Journal, reporter Amy Dockser Marcus writes about how an online community called PatientsLikeMe helped to inspire a clinical trial of lithium to treat amyotrophic lateral sclerosis (ALS), otherwise known as Lou Gehrig's disease. The trial, published in Sunday's Nature Biotechnology journal, was inconclusive. However, despite this result, Marcus writes that the experience of using an online community to spur a clinical trial may ultimately add an important new element to clinical research. In this way, patients can help to positively affect clinical research.

The research ecosystem in America is becoming increasingly complicated, with R&D costs growing and clinical trial complexity increasing according to the Tufts Center for the Study of Drug Development. And while experts interviewed for the WSJ article stress that social media won't replace randomized, double-blind, placebo-controlled clinical trials - "the gold standard for generating medical evidence" - it could still provide value: "new models are needed," says Lee Hartwell, a Nobel Prize-winning scientist now at Arizona State University, and formerly president of the Fred Hutchinson Cancer Research Center in the article.

For example, "the [inconclusive] result was apparently nine months after the study was launched. Conventional trials typically take more time just to enroll patients.... [Also,] costs for drugs and recruiting patients were avoided."

The trial was also aided by the involvement of the PatientsLikeMe management, the article notes. Although the idea was spurred by patients who use the Web site to discuss the potential of lithium, the company itself took pains to promote scientific validity. For example, they developed a tool to standardize the collection of data, borrowed from conventional ALS self-reporting guidelines, and created an algorithm for comparing patients.

The article clarifies that though network-generated data can offer valuable insights, Merit Cudkowicz, an ALS researcher at Harvard Medical School who was an investigator on a standard lithium clinical trial, "cautioned that the PatientsLikeMe study was not a substitute for more rigorous studies. Two conventional ongoing ALS studies are designed to see if lithium has a very small effect on survival, something the PatientsLikeMe study wouldn't be able to pick up."

It's an interesting story that reminds us not just that science continues to evolve, but the way that we use it does, too.

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