In recent years, the landscape of medical research and innovation has been significantly shaped by a growing focus on rare diseases. At Teamit, our journey into this crucial area has been marked by a series of impactful initiatives and collaborations aimed at addressing the unique challenges faced by individuals affected by rare diseases. The REMEDi4ALLproject, which seeks to establish a sustainable European platform for enhancing medicine repurposing or the EU-PEARL project which has delivered an innovative framework for more efficient clinical research are just two examples of initiatives central to Teamit endeavours for better health outcomes through multistakeholder collaboration. But how can we all ensure that more and better medicines and therapies reach those who most need them? Last week we had the opportunity to take part in the 37th EURORDIS Round Table of Companies (ERTC) in Brussels. Discussions underscored the importance of regulatory, Health Technology Assessment (HTA), and reimbursement processes in the final stages of drug development, prompting critical reflections on how to better navigate these pathways for the benefit of rare disease communities.
We had the opportunity to take part in the 37th EURORDIS Round Table of Companies (ERTC) in Brussels last week.
Dialogue, Collaboration and Innovation
Rare diseases are an area where multi-stakeholder collaboration is the only way forward in pursuing break-through solutions and timely access to treatments and quality care to patients. The EU already perceives rare diseases as a crucial playing field where successful pilots could be scale up to other research and innovation fields. For instance, the solutions being developed by REMEDi4ALL to make the repurposing of medicines the new normal could enormously benefit people living with rare and ultra-rare conditions. This strategy, consisting of finding new uses for already known drugs, has enormous potential to reduce time and costs of bringing novel therapeutic options to patients compared to developing a new drug from scratch, a process that can take up to 20 years! Results delivered by drug repurposing will first impact the rare diseases community but can then be replicated to other fields.
Market access and availability, a final complex stretch to bring drugs to patients
The 37th ERTC, discussed the last stretch of the drug development pathway; that is the regulatory, HTA and reimbursement processes. The regulatory approval alone does not guarantee the effective entry of these treatments or solutions into market or accessibility thereof to patients. It is only when these approved treatments pass through a positive evaluation from the pertinent HTA agencies and then health authorities agree on the reimbursement and pricing strategies, that we can assert these solutions are accessible and available to patients.
PRIME and MOCA, instruments already available
The 37th ERTC also illustrated some examples of ongoing initiatives to facilitate multi-stakeholders’ interactions and anticipate regulatory pitfalls in the orphan drug development road map. For instance, since 2016, EMA has aimed to reinforce early and iterative scientific and regulatory advice for priority medicines (PRIME programme), targeting mainly products of major public health interest. Products that have gone through PRIME advice have shown an increase success rate for granted products and ATMPs represents a quarter of the total applications to the programme1. However, one of the limitations of this programme is the lack of involvement of HTA bodies.
A second instrument facilitating early and multi-stakeholder dialogue for regulatory and HTA process is the Mechanism of Coordinated Access to orphan medicinal products (MOCA). This includes not only regulatory bodies, patients’ representatives and pharmaceutical companies, but also national competent authorities for pricing.
As highlighted in the discussions, by leveraging multi-stakeholder dialogues and learning from ongoing initiatives such as the PRIME programme and MOCA, we can already accelerate the pace of progress and ensure that no patient is left behind. Looking to the future, we invite you to join us on this journey of discovery and transformation, embarking on exciting new partnerships and endeavours when possible.
Let’s continue working as a diverse community
Today, let’s celebrate International Rare Disease Day and strive to continue sharing knowledge and experiences with our community and partners, learning and improving our methods and expertise with the common goal of guaranteeing healthier outcomes.
By leveraging multi-stakeholder dialogues and learning from ongoing initiatives such as the PRIME programme and MOCA, we aim to accelerate the pace of progress and ensure that no patient is left behind. Looking to the future, we invite you to join us on this journey of discovery and transformation, embarking on exciting new partnerships and endeavours when possible.
Stay tuned to our social networks for updates on our upcoming opportunities to contribute to this vital cause.
