For start-up biotech companies, the potential benefits of engaging patients early and often are clear: insights on unmet patient need, guidance on how to value clinical improvement and reduced side effects, and a new way to give investors confidence in the future market for potential medicines. But making PE happen is far from straightforward.

‘Doctors usually serve as a proxy for patient voices,’ says Hanne Damgaard Jensen, CEO of ROS Therapeutics. ‘It would be better to hear from patients but there are many barriers, particularly for small and medium-sized enterprises (SMEs).’

ROS Therapeutics is a Danish biotech start-up developing candidate drugs for inflammatory diseases. Despite viewing patient engagement (PE) positively, young companies face several barriers and few incentives to prioritising PE. In fact, it is possible to bring a drug from the lab bench to the patient – a process that takes several years – without ever having to speak with the patient community about symptoms, side-effects or unmet needs.

‘The current rheumatoid arthritis (RA) treatment has short-comings, namely side-effects that patients find difficult to tolerate,’ says Damgaard Jensen. ‘We’re working on a treatment that would mitigate some of these side effects – and we are testing, in animal models, whether it might also improve clinical outcomes if patients were able to tolerate higher doses.’

This would mean incremental benefits for some patients and a game-changing treatment option for others. Around 25% of patients drop out of treatment on the current mainstream medication, according to the ROS Therapeutics founder.

Doctors know best?

The trouble for smaller companies is that understanding the value patients’ attach to improved symptom control or reduced side effects is challenging. Clinicians work closely with patients but, without living the disease, can they really know?

‘Doctors may not see side effects in the same way patients do. That’s why the patient voice matters,’ says Damgaard Jensen. ‘When we reach early clinical stages, we need to learn more about how patients balance effectiveness and side effects. However, you need resources for this and there are many other pressures on SMEs.’

One problem is that despite the publication of guidelines on drug development, good manufacturing practice, and treatment algorithms on how approved medicines are used, there is very little mention of patient dialogue along the way: ‘If you develop a drug for rheumatoid arthritis you can go right through from pre-clinical research to product launch without talking to a single patient.’

This problem is more acute for drugs that address ‘big’ diseases like arthritis, diabetes and cancer, compared to medicines for rare diseases. For rare conditions, researchers and companies need to find out about the disease from those who have it – because few doctors have experience of diseases they so rarely see. ‘Rare diseases patients are more likely to be invited to help design a trial than a patient with a disease that clinicians believe they understand well.’

Stick and carrot

One solution could be to require drug developers to work with patients along the road to market approval. When patient engagement (or anything else, for that matter) is optional, it is often pushed to the bottom of the ‘to do’ list. Here, regulators could help by asking manufacturers to show they have listened to patients.

However, adding to the burden on SMEs could serve as yet another barrier to biotech entrepreneurs. So, how could smaller players be positively supported in patient engagement? ‘It would be good if there were practical guidelines or toolkits for SMEs on PE,’ says Damgaard Jensen. ‘This would make it easier than feeling as though you are starting from scratch when reaching out to patients.’

Making PE a reflex for drug developers may even help attract investors to back early-stage biotech companies if they feel comprehensive market research has been conducted. It also serves what ROS Therapeutics views as the prime driver for biotech research: ‘Engaging with the patient community is an opportunity to develop medicines that are tailored to their unmet needs – helping patients through science is why we do what we do.’