A revolution is underway. Rooted in the European EUPATI academy, the Dutch spin-off EUPATI-NL has already trained more than 50 motivated patient representatives. These individuals are ready to improve (and accelerate) therapy development. So researchers, pharmacists and physicians: work with them on core outcome sets, appropriate PROMs, design of clinical trials! This was a key message I delivered at FAST’s first ATMP webinar on March 11.

Participation of patient representatives in FAST
Together with Annemiek van Rensen, board member of FAST and some staff members of patient organizations, we are bringing together experienced patient representatives around the topic of Education. The first spearhead we are working on together with the experts from consulting firm Lysiac is further explanation of the role of the patient organization in reimbursement and availability of (innovative) drugs in the Netherlands. They find this tricky matter, with tailor-made training so far lacking. Special access routes such as DAP, DRUP, ODAP and the Conditional Access Regulation are discussed, as well as very concrete instruments such as the form practice description introduction drug (PIG) for patients. We will use the FAST Forum as an exchange and meeting place.

Engagement of patient representatives at RARE-NL, FAST’s hub for rare diseases
Not surprisingly, given my background, I am particularly focused on therapies for rare diseases. Innovative gene & RNA therapies for inherited rare diseases finally reached patients from the lab in recent years but access is a bumpy road. Let’s start working on diseases for which there is no treatment yet, with a high unmet medical need. The holistic approach to patients, made known early in development, can contribute immensely to adequate treatments.

Genetic therapies for inherited disorders
There are over 7,000 rare diseases, 85% of which are very rare (with at most a few dozen patients in the Netherlands or less). Because these diseases are hereditary, the severe forms often come to light at birth, i.e. in infants and young children. These are special populations for clinical trials. How do we encourage that every patient with a rare disease has a chance of receiving treatment?

I see plenty of opportunities, especially for neurological and eye disorders with the RNA therapies. At the VSOP, we also see that our member organizations want to prepare for the use of these types of therapies. It is good to see that FAST is actively trying to join forces by setting up collaboration hubs such as RARE-NL. That way you create critical mass to address the challenges. At this early stage, let’s be keenly aware that the perspective the patients are concerned with is also well positioned.

With RARE-NL and parties like Epilepsie.nl working on affordable therapies for rare diseases, drug rediscovery and personalized genetic therapies we are looking at how to give patients and patient organizations a good position in therapy development. FAST’s RARE-NL hub is a wonderful initiative that deserves attention and support and can also provide support, including for rare disease patient organizations. This creates a reciprocal collaboration that is also future-proof.

Together strong for Rare – towards a new National Plan Rare Disorders
It is nice to report that at the National Conference Rare Disorders on April 4, RARE-NL will present itself to the mixed audience of patient representatives, physicians, researchers, pharmaceutical industry and policy makers. Together strong for rare and on to a beautiful National Plan Rare Disorders where knowledge and expertise are combined to work effectively on innovative and affordable treatments!

About Mariette Driessens
Mariette Driessens is a policy officer at VSOP – Patient umbrella organization for Rare and Genetic Diseases and the NVHP for anyone with an inherited coagulation disorder. Trained as a medical biologist, she has worked in research and Regulatory Affairs. She has enjoyed working in the patient movement since 2013. As a patient representative on the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), Mariette promotes patient involvement in the development of therapies for people with rare diseases. In the Netherlands, she participates in the Orphan Drug Round Table at the Care Institute and the Orphan Committee with medical specialists, in order to promote access to and appropriate use of orphan drugs in practice. At FAST’s request, she contributes thoughts on meaningful patient participation in the themes FAST is working on.