Developing new drugs for the treatment of sarcoidosis isn’t easy. First, the cause of sarcoidosis is unknown. Second, prednisone, a remarkably effective medication for the treatment of sarcoidosis, limited only by its adverse side effect profile, is tough to beat. Third, sarcoidosis is a rare disease, which affects fewer than 200,000 people in the US per year. These challenges notwithstanding, researchers at Mount Sinai will be testing a new drug for the treatment of sarcoidosis. In late 2015, the Division of Pulmonary, Critical Care and Sleep Medicine will be enrolling sarcoidosis patients, who meet prespecified entry criteria, into a clinical trial to evaluate the efficacy and safety of KiactaTM for the treatment of sarcoidosis.

Clinical trials are prospective biomedical or behavioral research studies in humans that generate safety and efficacy data designed to answer specific questions about biomedical or behavioral interventions. These trials may evaluate novel vaccines, drugs, treatments, functional foods, dietary supplements, devices or new ways of using known interventions. They are conducted only after satisfactory information has been gathered that satisfies health authority/ethics committee requirements in the country where approval of the therapy is sought.

Conducting clinical trials, however, is a difficult undertaking. A recent study suggested the following:

• Nearly 80 percent of all clinical trials fail to finish on time and 20 percent of those are delayed 6 months or more.
• 85 percent of clinical trials fail to retain enough patients.
• The average dropout rate among all clinical trials is approximately 30 percent.
• Over two-thirds of trial sites fail to meet original patient enrollment for a given trial.
• Approximately 50 percent of trial sites enroll one or no patients in their clinical studies.

The lackluster recruitment of patients into clinical trials has prompted sarcoidosis researchers in the Division of Pulmonary, Critical Care and Sleep Medicine to collaborate with Transparency Life Sciences (TLS) to innovate the administration of clinical trials. Among other things, TLS utilizes crowdsourcing and mobile health technology to efficiently develop promising drugs for unmet clinical needs, like sarcoidosis. Crowdsourcing enables patients, physicians, researchers and other stakeholders in the clinical trial process to contribute to the design of clinical trials, resulting in protocols that are focused on parameters most relevant to clinical decision-makers and patient needs.

Protocol Builder is TLS’s crowdsourcing survey tool to help develop clinical protocols. Each clinical trial project has its own Protocol Builder that asks specific questions about the study’s design for examining the experimental therapy. In September 2014, sarcoidosis researchers at Mount Sinai and TLS launched separate online patient- and researcher-centered Protocol Builder tools to solicit feedback about the protocol, clinical trial design and utilization of mobile health technology intended for use in the clinical trial evaluating Kiacta in sarcoidosis. The answers to the questions posed in the Protocol Builder survey tools, which are currently being analyzed, will be used to fine tune the design of the protocol for the trial. To date, more than 200 researchers/physicians and patients have participated. Sarcoidosis researchers at Mount Sinai and TLS are confident that the crowdsourcing will provide valuable information, which will improve the design of the trial and enhance patient participation. Stay tuned!

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Adam Morgenthau, MD, is an Assistant Professor in the Pulmonary, Critical Care and Sleep Medicine Division of the Icahn School of Medicine at Mount Sinai. He is a member of the multidisciplinary team of the Mount Sinai – National Jewish Health Respiratory Institute.