Franchetti: The patient voice needs to be incorporated into all phases of research and commercialisation, and the earlier the better. Trials in the oncology and immune disorder space are beginning to do this.

In rare diseases it is even more important to speak to the patients and care partners as often there are not many other options in clinical research to choose from, and it’s important we don’t assume their wants, needs and barriers. The rare disease populations are very unique and we need to fully understand the ecosystem/nuances surrounding them.

Consider the example in which a very sick child with a rare disease who is ventilator-dependent and wheelchair-bound has to travel back and forth to the trial site. It would be easy to assume that the parents would welcome a home nurse trial visit, but historically, many parents of children with rare and orphan diseases have given feedback opposed to a home visit because they view their home as the only space where their child feels safe from being poked and prodded and made to feel uncomfortable.

We rely on a variety of means – everything from one-on-one in-depth interviews, in person workshops to video journaling a ‘day in the life of a patient’ to gain a robust understanding of the patient perspective and perception. And, while there’s no substitute for direct patient input, it’s also equally important to also gather insights from caregivers, advocacy groups, and investigators.

As for digital technology to collect patient-reported data, it’s a phenomenal tool, but it also may not be right for every study or every patient. You really need to look at your patient population and consider factors such as patient age, health literacy, and socioeconomics. It is also imperative to consider what data is being collected and the rationale behind it. Patients want to understand why the monitoring is important and how it can drive their clinical outcomes and care.