Machine learning in rare disease: is the future here?

The healthcare industry is increasingly focusing on niche patient populations. Around half of FDA approvals in the past two years were for rare or orphan drugs that serve fewer than 200,000 patients in total in the US and 1 in 2,000 patients in Europe. By 2024, orphan drug sales are expected to capture one-fifth of worldwide prescription sales. However, finding these hard-to-reach patients is difficult and keeping them engaged over time even more so. Could machine learning platforms that deliver personalized experiences for patients and caregivers be part of the answer?