The world’s first patient-powered registry for Gaucher’s Disease
Dutch not-for-profit organisation, Fair Medicine was founded to shine a light upon – and improve the complicated and often opaque process used to develop new pharmaceutical treatments. A system they believe doesn’t work well for the patient – or drug purchasers.
Since 2015, Fair Medicine has been working with Patients Know Best to develop a more open model for drug evaluation though building a registry for patients with Gaucher’s Disease – a rare genetic condition which severely impacts the independence of those who live with it.
In time, Fair Medicine’s founders Frans de Loos and Prof. Hans Buller hope to replicate the open, patient-led drug evaluation model they create around Gaucher’s with other patient groups and other treatments. As de Loos explains:
“At the moment, it’s often unclear how effective many drugs are and why some cost as much as they do. Many clinicians struggle to objectively recommend one course of treatment over another because there’s no independent analysis – we want to change that. We founded Fair Medicine to add transparency to this system and to encourage the production of effective treatments at a fair price.”
Drugs are normally developed using patient registries – lists of patients with specific conditions which pharmaceutical companies use to consult during the development process. However, these registries are often controlled by individual companies and are not freely accessible to medical professionals which means that their use is limited.
“Companies often don’t evaluate a drug thoroughly enough once it’s on the market – but that’s a vital part of the process missing,” said de Loos. “We looked around the world for an independent system we could use that would enable patients to feedback on the effectiveness of drugs – Patients Know Best offers the best model in the world for us. We simply wouldn’t have been able to do this without it.”
Patients with Gaucher’s Disease will be given a Patients Know Best account and different drugs and treatments will be evaluated through consulting with the patients – and the health professionals treating them. This will ensure that the clinician can understand what the effects (and non-effects) of different treatments are and develop their treatments, recommendations and diagnosis accordingly. Prof Buller said:
“If you listen carefully to patients they often know what is best for them. When you bring patients with the same disease together it’s fascinating to see what they find important – and it’s often not what the doctor thinks is important. As a doctor, Patients Know Best provides an opportunity for patients to tell us what’s important to them – not just the other way around. I truly believe that PKB could revolutionize the way we think about patient platforms.”
Whilst currently being trialed in the Netherlands across small groups of Gaucher’s patients, Fair Medicine plans to use Patients Know Best to roll-out its patient-powered registry to all Gaucher’s patients in the Netherlands and then, across the EU. In two years, Fair Medicine aims to be working within 20 institutions across Europe and to be using the system to both evaluate the effectiveness of drugs and understand how those drugs interact with other courses of treatment. Prof Buller continues:
“Our work with Gaucher’s disease is just the beginning. We know that patients really want an environment where they connect with each other and learn independent information from a range of different sources. Through my work as a pediatrician, I also know how useful it will be for clinicians to understand what impact different treatments are having – so we can know what is and is not working for the patient.”
“There are only around 80 – 100 people in the Netherlands with Gaucher’s and that means there’s only around 30 GPs who have even heard of the condition. People are often terrified of doing things that we all take for granted – like going on holiday – because a flare up of their disease could mean they get treated by an unfamiliar doctor. However, with a patient-controlled approach that person carries their medical history with them wherever they go and that’s life changing – and life saving.”
Dr Mohammad Al-Ubaydli, CEO of Patients Know Best, said: “Fair Medicine’s patient-powered research registry model will spread across Europe. PKB works in 19 different languages and gives the patient access to a single record across all providers, something all patients with rare diseases need.”