Collaboration and innovation drives RealiseD Project to Change the Paradigm for Clinical Trials in Rare and Ultra-Rare Diseases

Andrea_Puletto — Fri, 28 Feb 2025 12:31:57 +0000

Marc Vandemeulebroecke, Biometrics & Data Science

Andrea_Puletto Fri 28/02/2025 - 13:31

Imagine a world where every rare disease, no matter how uncommon, gets its moment in the spotlight of scientific innovation. Where the challenges of small patient populations and varied symptoms don't hinder progress but inspire a new approach to clinical studies. This is the mission at the heart of RealiseD, and ��ֳ� are proud to be part of this project which is funded by the Innovative Health Initiative (IHI) and will run until 2029.

With millions of people worldwide living with a rare disease, and many of these conditions being ultra-rare, the need for new treatments has never been more urgent. Yet, the path to developing these treatments is fraught with obstacles, from enrolling participants in clinical studies to navigating fragmented regulatory frameworks. That's where RealiseD steps in, with the aim of transforming the paradigm of clinical studies for rare and ultra-rare diseases.

But how, you might ask, can such an ambitious goal be achieved? The answer lies in collaboration and innovation. RealiseD is not just another research project; it's a comprehensive, collaborative effort to overhaul how clinical studies are conducted for the rarest of diseases. By bringing together experts from every corner of the healthcare ecosystem - academia, regulatory bodies, clinical research institutes and hospitals, patient organisations, and pharmaceutical companies – RealiseD is working to craft new gold standards for clinical trials in rare and ultra-rare diseases.

So, what can we take away from the ambitious mission of RealiseD? It's a reminder that in the face of daunting challenges, collaboration and innovation can light the way forward. It's a call to action for all of us in the healthcare industry to support and participate in efforts that push the boundaries of what's possible. And most importantly, it's a beacon of hope for the millions of people living with rare diseases, a promise that they are not forgotten and that the future holds the promise of new treatments.

The journey of RealiseD is just beginning, but its vision is crystal clear: a world where every rare disease is recognized, researched, and treated. ��ֳ� is proud to stand together in support of this noble mission, because we think that the impact of RealiseD will resonate for generations to come.

To stay updated on the progress of RealiseD and learn more you can visit their new website by clicking here.

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Comment:

Posted by Andrea Wilkinson , 28 February 2025

So proud to hear of your contribution to this important global initiative to drive changes in EU that we can all learn from! More, More, More!!

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Do advances in clinical trial diversity go far enough for women?

Vanco Vlastimil (��ֳ� S.A.) — Fri, 16 Jun 2023 11:06:38 +0000

Do advances in clinical trial diversity go far enough for women?

Marie Teil, Women of Childbearing Age Mission

Vanco Vlastimi… Fri 16/06/2023 - 13:06

The clinical trial landscape has evolved significantly in the last years – with a focus on more inclusive approaches to ensure medicines get to the right patients.

For example, the Consolidated Appropriations Act of 2023, passed by Congress earlier this year, makes it mandatory in many instances for drug sponsors to submit clinical trial diversity action plans, an action that was previously recommended by FDA guidance.

While diversity has become an increasingly important priority in clinical research, the inclusion of pregnant and breastfeeding women in clinical trials still falls short, leading to few available medications that have been adequately tested and labelled to inform potential use during pregnancy and lactation. Currently, 7 in 10 women may have to take at least one prescription medicine during pregnancy – with physicians and women with chronic diseases forced to face the difficult task of deciding whether to stop or continue a medication during pregnancy and breastfeeding, despite the unknowns.

In partnership with clinical teams, ��ֳ� strives to address knowledge gaps in the care of women of childbearing age through the provision of validated evidence and quality disease management tools. Our pregnancy and lactation trials that are completed or ongoing in four of our products provide evidence to support disease management and help clinicians and women with chronic diseases make more informed decisions around their health and family planning.

We are now working to address further knowledge gaps by systematically evaluating the opportunity to conduct pregnancy and lactation trials in each of our current and future therapeutic areas. We are also collecting follow-up data in infants exposed to our medications during pregnancy and breastfeeding, to identify potential risks and to help support patient decisions.

Additionally, we continue to work in close collaboration with our external partners such as the ConcePTION Consortium and Coalition to Advance Maternal Therapeutics (CAMT) on research specific to pregnant and lactating women, TransCelerate to collaborate on common drug development challenges, and as a member of a working group with the ICH initiative to enable inclusion of pregnant and breastfeeding individuals in clinical studies.

We hope to continue our collaborations with physicians and women around the world by identifying potential educational needs and seeking to offer important information regarding patient care before, during and after pregnancy.

Want to learn more about ��ֳ�’s ambition for women of childbearing age? Click here to learn more. Empowering Decisions for Women of Childbearing Age | ��ֳ�

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Patient Women of Childbearing Age clinical trials diversity

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Collaboration and innovation drives RealiseD Project to Change the Paradigm for Clinical Trials in Rare and Ultra-Rare Diseases

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Do advances in clinical trial diversity go far enough for women?

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