Kathy Zheng, MPH, Manager, Project Management and Clinical Innovations, PROMETRIKA, LLC.
Companies that specialize in drugs for the treatment of rare diseases face a number of unique challenges during the development lifecycle of their products. One innovation that these companies have turned to is the use of mobile health (mHealth) tools. While the implementation of an mHealth strategy within clinical trials for all indications comes with challenges, the obstacles are even greater in rare diseases. Despite these hurdles, rare disease companies, in their efforts to improve patient experience, have an even greater appetite for technologies like mHealth. Rare disease researchers face increasing pressure from regulatory agencies, investors, and high profile patient advocacy groups to innovate in an effort to expedite the drug development process. The small number of rare disease patients also magnifies the need for an efficient and accurate data collection process.
Companies embarking on a new technology implementation pathway have additional considerations around motivation and goals when the indication is a rare disease. Some of these considerations only become evident once the implementation process has begun. For example, the idea of strapping a wearable activity tracker that collects mobility information to a patient with a certain rare disease sounds like a great solution to data collection. However, it quickly becomes evident that the cognitive impairment that accompanies that disease also manifests severe tactile sensitivities, thus rendering a wearable device intolerable to the patient. While this effect may have been foreseen in patients with diseases that are better understood, it is more difficult to anticipate these types of issues for rare disease patients because their symptoms and challenges are often poorly characterized.
Other considerations in the implementation of mHealth are more scientific in nature. For example, currently, researchers use most mHealth outcomes to support traditional, validated clinical endpoints. The hope is that, over time, mHealth outcomes will be validated and will become endpoint for the studied disease. While advancement in this area is moving along nicely for indications such as Parkinson’s disease or sleep disorders, the mHealth experience in rare diseases is currently limited. The bar to prove that an mHealth endpoint is valid in a particular rare disease may be set high because there is no precedent endpoint. Without available literature to support whether an mHealth endpoint is valid in the rare disease of interest, researchers can only test a hypothesis and hope that the clinical endpoint reflects the proposed objective endpoint. The hoped-for result is that mHealth solutions can provide companies with a new and expanded repertoire of accepted endpoints in rare diseases.