A visionary overview of the ideal Rare Disease landscape in 2030
Elin Haf Davies, Child health advocate and Founder of Aparito Health
Elin Haf Davies kicked off the symposium. Under the motto “Never underestimate a woman with a mission” she told the audience about her mind-blowing adventures rowing across the world’s oceans to raise money for metabolic diseases. By drawing parallels between this trip and the current development process of orphan drugs, Elin pinpointed present challenges and possible solutions in the diagnosis, monitoring and assessment of orphan diseases. Elin strongly advocated an adaptive approach for a sustainable development of treatments for all currently untreatable orphan diseases.
Setting the Scene
Remco de Vrueh, Lygature
“Yes there are challenges, but you are doing great!”. With this positive note, Remco de Vrueh enlightened the audience on the status of orphan drug development, and the important role of collaboration. After decades of experience, public-private-partnership (PPP) has become mainstream. The number of consortia as well as the number of stakeholders engaged in these consortia is on the rise. As a result, the main question no longer is ‘Why?’ but rather ‘How?’. How to make sure everybody speaks the same language? How to make every voice heard? According to Remco, PPP might be the facilitating platform to get everybody on the same page for orphan drug development, too.
After these inspiring talks, participants had the chance to visit two break-out sessions, each of which was based on challenges in the development of orphan drugs. In each session, one of the tracks focussed on better and faster drug development and the other on access to treatments.
Better and faster drug development: how to cope with benefit-risk
In most industries, innovation is customer-centred. However, in biotech the voice of the patient in determining the risk-benefit ratio for treatments could be stronger. Currently, according to Violeta Stoyanova (CBG-MEB) both the CBG-MEB and EMA are working on appropriate ways to include multiple stakeholders in the assessment of risk-benefit (i.e. via PRIME and adaptive pathways). In addition, there are almost 450 guidelines in place to help you to streamline and optimize your research. Simultaneously, other initiatives have arisen to stimulate earlier access to treatments for patients with an unmet medical need. The concept of myTomorrows, as explained by Ronald Brus (myTomorrows), is to enable access to treatments after proof of concept in a phase II study within (or outside of) the EU. They subsequently gather Real World Data to demonstrate effectiveness of these treatments. Overall, it is clear that multiple initiatives are arising to stimulate safe, yet early access to new treatments.
Better and faster drug development: patients in the driver seat
Patients should be key contributors in drug development, according to Hanka Dekker (VKS). They are the ones who can tell you what symptoms they suffer from most in their daily life and thus which symptoms are important to tackle. Professionalization and collaboration of patient organisations is contributing majorly to an increased awareness for orphan diseases. Ana Mingorance (Dracaena consulting) shares Hanka’s view. We shouldn’t be working ‘for’ patients, but rather be working ‘with’ patients. According to her, the impatient patient will soon take the lead in patient-driven innovation. In conclusion, it will only be a matter of time before patients will actively be involved in all aspects of orphan drug development.
Access to treatments: a sustainable healthcare system
The increasing pressure on affordability of health care, and the considerable price tags of some orphan medicines, the sustainability of our health care system is an important topic to discuss. As such, Martin de Graaff (ZiN) presented his feverish futuristic dream for 2030. His many suggestions ranged from EU-level price setting to adaptive reimbursement of treatments based on pre-defined treatment goals. Joep de Groot (CbusineZ) launched the Sustainable Manufacturing Practice (SMP) as a counterpart to the current GMP practices. His partnership approach aimed to incentivize development under SMP for all stakeholders involved, including faster time to market and a simplified regulatory trajectory. By working under SMP, innovation could be stimulated in a more sustainable fashion. The lively discussion clearly illustrated the many ideas to shape and stimulate the future development of orphan drugs.
Access to treatments: how to improve the actual use of effective treatments
Innovative models can be used to deliver effective treatments to the right patients. Organoids provide an opportunity to create cellular models of human disease, Rob Vries (Hub4Organoids) explained. Organoids can be used to determine whether patients respond to orphan drugs or not. This will have major benefits for effective treatment, not only for rare diseases, but for health care in general. Pete Chan (Raremark) took another approach to stimulate effective treatment. Instead of finding the right drug for a patient, they try to find the right patient for a drug. The goal of Raremark is to accelerate trial recruitment and to bring patient experience into drug development. Initiatives like Raremark and scientific breakthroughs like organoids will boost the effectiveness of treatments for the right patients: an important benefit for rare disease patients.
House of Commons
Based on the contents of the break-out sessions, the moderators formulated a statement. These statements were subsequently discussed during the closing House of Commons debate. Under the watchful eye of Roderik van den Bos (DebatAcademie) all participants engaged in a passionate debate, in which proponents and opponents competed to convince as many participants as possible of their stance. Those who changed their opinion, were welcomed with a warm applause.
Overall, we look back at a day that gave us a lot of inspiration. Inspiration that was translated into a shared ambition. Together we stand stronger in shaping the future for rare disease patients!
Photography: Nils van Houts