Patient engagement a resource-intense but de-risking exercise, says NDA Group

According to NDA Group scientific director, Markku Toivonen, patient engagement – which covers patient-centric approaches to pharmaceutical R&D and patient-derived outcomes – is attracting increased attention in the drug development landscape.

“Patient engagement is everywhere. It has been adopted by both big pharma and smaller companies,” ​Toivonen told delegates at Nordic Life Science Days last month.

And while implementing patient-centric strategies throughout the development stage can cost time, money and planning, according to Toivonen, it ‘pays off’ in the long-term.

“It is a de-risking exercise, particularly in rare conditions where…clinical data will help you understand which patient-reported outcomes should be taken to confirmatory trials,” ​he said, adding that understanding the clinical relevance of a treatment’s effect can be one of the “main stumbling blocks” ​for new drug applications.

“If you have relevant endpoints measuring…the subject’s benefits, you are much better off.”​

Looking ahead​

According to Toivonen, in the future, patient-reported outcomes – designed to reflect benefits truly perceived by patients – will carry more weight in regulatory reviews and “take a major role in health technology assessment”. ​

However, pharmaceutical companies must take responsibility, and act now, he told delegates. “I want to emphasise: Do not leave patient engagement only to the regulators…or their public advisory boards.”​