Author: Brently Berx, Medical Affairs Officer
This month, Kintiga BeNeLux attended the Healixia-EUFEMED joined conference in Belgium: “Mastering increasing complexity in developing innovative therapies”. It was a festive occasion, as Healixia and EUFEMED both celebrated their respective 5th and 10th anniversary!
Ten years ago EUFEMED was founded by 4 national European human pharmacology associations: BAPU in Belgium, Club Phase I in France, AGAH in Germany and AHPPI in the United Kingdom to progress science and methodology in human pharmacology on a European level and to increase awareness for the importance and specific needs of exploratory medicines development.
In addition, 5 years ago Healixia was created, when 4 Belgian associations (BAPU, BRAS, BeAPP and ACRP.be) joined forces in setting-up education and networking sessions for professionals active along the life cycle of medicines, medical devices, in vitro diagnostics and other health related products.
The conference started with an engaging keynote lecture on the recent developments in gene therapies, in rare diseases (eg congenital deafness and ALS). This included a very insightful and gripping testimony from an ALS patient participating in a first-in-human clinical trial for a novel therapy. His testimony highlights the importance of the patient’s perspective in developing innovative therapies.
Broader strategies to optimise clinical development in rare diseases were also discussed, including:
Multidisciplinary and collaborative approach:
- Early involvement of experts in clinical, regulatory, scientific, access and operational fields.
Flexible regulatory strategies:
- Regulators emphasised seeking early advice and firmly recommended engaging in discussions, particularly regarding outcomes and their relevance.
Patient-centric development:
- Including PROs in trial design. This is accompanied by a need to describe the methods used as precise as possible. These methods should be discussed in advance with the regulators.
Parallel advice:
- The Joint Scientific Consultation offers a significant advantage by facilitating discussions with regulators simultaneously and ahead of pivotal clinical trials.
The team gained perspectives from the regulatory agencies and industry on the position of Europe within the global development of innovative therapies. While Belgium is a frontrunner in R&D of innovative therapy, Europe as a whole is lagging behind the rest of the world. However, the overall take home message was that Europe does not need to copy the US or Asia, but can differentiate and innovate to remain competitive in medicine development.
Additionally, there was an introductory session on the new EU HTAR. It seems that this topic needs further strategic clarification and preparation for many companies, as health technology assessment will run in parallel with EMA review, so one year earlier compared to current practice. Given the importance of these new regulations, health technology developers should be well prepared from the earliest stage to ensure a successful registration of their innovative medicines. Kintiga possesses invaluable knowledge and expertise to guide partners through this complex journey.
The conference continued with insights from a regulatory perspective on orphan designation, a framework that is subject to change under the new pharmaceutical legislation. Something we as Kintiga will monitor closely! Finally, there were discussions on real-world data, revealing the large potential, particularly within the scope of EU-HTA.
During the conference, Maud also had the chance to present our Kintiga poster about determinants for Market Access to ATMPs in Western-Europe, which she developed together with Marlies Boeren, Ben Barron-Millar, Malcolm Boyd, Ingo Hantke, Malte Glüsen, Kristof Theys, Priscila Mazzola and Melanie van Riemsdijk.
We’d like to congratulate Healixia and EUFEMED with their joint anniversaries and especially praise our colleague Erik Present as the chair of Healixia for his achievements.
To explore these insights further or learn how Kintiga can support you, contact us at enquiries@kintiga.com.
