In the highly regulated world of clinical research, an innovative approach is emerging that promises to transform how treatments are developed. One2Treat, a pioneering company, is challenging traditional clinical trial design by putting patients at the heart of the process. https://bit.ly/One2Treat

RARE Revolution poll reveals patient needs are worryingly overlooked in clinical trial design. insights brings you the latest views from your global RARE community. https://rarerevolutionmagazine.com/rare-insider/rare-revolution-poll-reveals-patient-needs-are-worryingly-overlooked-in-clinical-trials

Bob Stevens, CEO of the MPS Society, argues that traditional randomised controlled trials are unethical for rare and ultra-rare diseases, especially when neurological involvement leads to irreversible decline. https://bit.ly/BobRCTs

Discover the vital work of Action for XP, the UK's sole charity supporting individuals and families affected by xeroderma pigmentosum (XP). This rare genetic condition causes extreme UV sensitivity, leading to life-threatening cancers. https://bit.ly/A4XP

In this insightful article, Owen Marks explores the crucial stepping stones to successful rare disease medicine launches in the UK. He highlights the "Rare Disease Gap" in funding, the imperative for early engagement with communities. https://bit.ly/RareSteppingStones

When was the last time you read something that mattered? RARE Revolution insider® brings you straight to the people that matter. Trusted stories. Expert perspectives. No spin. Claim your free October trial: https://my.rarerevolutionmagazine.com/

Discover how the UK's current medicines pricing and access policies are failing those with rare conditions. Henry Burkitt, MD at Oxygen Strategy, calls for a complete rethink. https://rarerevolutionmagazine.com/rare-insider/when-policy-fails-people-reimagining-medicines-access-for-rare-conditions/

We asked our community "If you/your advocacy organisation have participated in any co-design activities with industry were the activity/project outcomes fed back to you?". insights brings you the latest views from your global RARE community. https://bit.ly/CoDesignInsights

Innovative trial designs are reshaping rare disease drug development, offering smarter, faster, and more inclusive approaches. Dr Billy Amzal of Phastar spotlights key tools. https://rarerevolutionmagazine.com/rare-insider/how-innovative-trial-designs-de-risk-drug-development-in-rare-diseases/

Stacy Pike-Langenfeld, co-founder of KrabbeConnect, shares her powerful journey from grief to purpose, championing patient-led research and data-driven advocacy in rare disease. https://rarerevolutionmagazine.com/rare-insider/stacy-pike-langenfeld-of-krabbeconnet/

Time is short. Complexity is high. RARE Revolution insider® brings you straight to the strategies that matter and highlights the people, innovations, and ideas shaping rare disease development. Independent. Intelligent. Accessible. Free trial this October: https://my.rarerevolutionmagazine.com/

Dr Andrés Moreno-Estrada and Carmina Barberena-Jonas from Cinvestav unveil MexVar, a new research platform revealing specific risk variants. Early findings could transform treatment. https://rarerevolutionmagazine.com/rare-insider/mexvar-helping-to-increase-the-scientific-independence-of-mexico/

Don’t just search. Find what matters. RARE Revolution insider® subscribers get full access to insights powered by our librarian Cognito. Every search is across trusted, compliant, approved content. Fast, accurate, independent. Free 1-month trial here: https://my.rarerevolutionmagazine.com/

Discover how Sumair Nizamuddin from Nuffield Bio is tackling mistrust in healthcare by addressing medical racism and advocating for community engagement. Learn more! https://rarerevolutionmagazine.com/rare-insider/breaking-the-cycle-rebuilding-trust-in-medical-research-and-delivery/

Have you participated in co-design with industry? Access insights from our global RARE community: https://rarerevolutionmagazine.com/rare-insider/have-you-or-your-advocacy-organisation-taken-part-in-any-co-design-activities-or-projects-with-industry/

Too much noise. Not enough clarity. RARE Revolution insider® gets straight to what matters. Independent stories, human voices, new ways of doing things in rare disease. Not PR. Not hype. Just clarity. Try your free month in October. https://my.rarerevolutionmagazine.com/

In "The Power of Words," Florence Cornish illustrates how clear science language benefits patients, professionals, and policymakers. Get impactful writing tips! https://rarerevolutionmagazine.com/rare-insider/the-power-of-words-how-should-we-write-about-science #PlainLanguage #MedicalWriting

Join us this week to explore the vital work of Childhood Tumour Trust with CEO Vanessa Martin! Learn about #NF1 and its unmet needs. https://rarerevolutionmagazine.com/rare-insider/putting-you-in-the-heart-of-the-rare-community-childhood-tumour-trust/#NF1#RareDisease #PatientAdvocacy

Revolutionising rare disease pharmacovigilance! AI is transforming safety monitoring for diverse patient populations. https://rarerevolutionmagazine.com/rare-insider/strengthening-rare-disease-pharmacovigilance-with-curated-real-world-data/ #Pharmacovigilance #RealWorldData #RWD #AIinHealthcare

Discover how Prof. Gareth Baynam's 'Lyfe Languages' project bridges healthcare and Indigenous culture by translating medical terms! #IndigenousHealth #HealthcareInnovation #LyfeLanguages #RareDisease rarerevolutionmagazine.com/rare-insider...

We asked our community "Do you understand what is meant by co-design in the context of advocacy and industry partnerships?". insights brings you the latest views from your global RARE community. To access insights click below. rarerevolutionmagazine.com/rare-insider...

Discover the future of rare disease research/treatment as the UK RD Framework enters its final year. A recent Westminster Health Forum Policy Conference highlighted crucial advancements, from closing data gaps to significant reforms in trial regulations. rarerevolutionmagazine.com/rare-insider...

Discover how AI is revolutionising pharmacovigilance in rare diseases. Learn why human oversight remains crucial and how to align AI deployment with evolving regulatory expectations for enhanced patient safety. rarerevolutionmagazine.com/rare-insider...

Delve into the inspiring career of Alice Tuff-Lacey, Service Owner at Genomics England. Discover what motivated her path in science, from early work at Cancer Research UK to leading the Generation Study, screening 100k newborn babies for rare conditions. rarerevolutionmagazine.com/rare-insider...

We asked our community "Is your organisation paid fair market value when engaging with pharma/industry activities such as patient engagement and recruitment collaborations, or insights work?". insights brings you views from your global RARE community. rarerevolutionmagazine.com/rare-insider...

#ICYMI Exciting news in the world of rare diseases! The Undiagnosed Hackathon is revolutionising the way we diagnose rare conditions. rarerevolutionmagazine.com/rare-insider...

Exciting progress is being made in delivering rare therapies! Dr Meriel McEntagart, clinical lead for rare disease technologies at Genomics England, shares her optimism for accelerated progress in the next three to five years. rarerevolutionmagazine.com/rare-insider...

This week, 'In the Thick of It' features Mary Wheatley, CEO, National Scleroderma Foundation. Discover their vital mission to advance medical research, raise disease awareness, and provide essential support and education for those affected by scleroderma. rarerevolutionmagazine.com/rare-insider...

Traditional pharmacovigilance systems face significant challenges in rare diseases due to small, fragmented patient populations and limited natural disease history. Lucy Fulford-Smith at TMC, says new approach is urgently needed Read here: rarerevolutionmagazine.com/rare-insider...

The future of organ transplants is being rewritten! Scientists are pushing the boundaries of bioprinting with the ambitious goal of printing a functional kidney in just 24h. Could this be the breakthrough that transforms healthcare forever? Read more here: rarerevolutionmagazine.com/our-goal-is-...

The European Life Sciences Strategy aims to re-establish Europe as a global leader in life sciences. This strategy focuses on optimising the research ecosystem, enabling rapid market access for innovations, and boosting public trust. rarerevolutionmagazine.com/rare-insider...

Lucy McKay, CEO, and Megan Pullein, research project manager, Medics for RARE Disease (M4RD), discuss their involvement in the RDI Lancet commission and highlight what they see as refreshingly unique about this milestone initiative rarerevolutionmagazine.com/rare-insider...

Discover groundbreaking advancements in ITP treatment with Dr. David Kuter's insights into a novel anti-CD38 therapy showing remarkable promise. This research also holds potential for other rare haematological disorders. rarerevolutionmagazine.com/rare-insider...

Discover Nisha Venugopal, Associate Director at IndoUSrare, in our latest 'Women in RARE' feature. Learn about the opportunities and barriers for women in this vital field, and how remote work is opening new doors. rarerevolutionmagazine.com/rare-insider...

We asked our community "When considering grant funding for your charitable organisation, what type of funding is the most beneficial to your organisation?". insights brings you the latest views from your global RARE community. Access insights here: rarerevolutionmagazine.com/rare-insider...

In our latest interview, we had an insightful conversation with Dr. David Kuter. Watch a sneak peak of his interview below. The interview delves into how a novel therapy is showing promising results for those living with immune thrombocytopenia (ITP). rarerevolutionmagazine.com/rare-insider...

By collaborating with clinical experts, industry partners, and PAGs, the INPDR aims to accelerate progress in care, support, and treatment for those affected by these ultra-rare conditions, ultimately improving the quality of life for patients and families. rarerevolutionmagazine.com/rare-insider...

The European Life Sciences Strategy aims to re-establish Europe as a global leader in life sciences. For our community, this strategy provides a concrete framework to unite behind and keep momentum alive in the face of challenges. rarerevolutionmagazine.com/rare-insider...

As we approach the final year of the UK’s Rare Diseases Framework, significant strides have been made. Dr Kath Bainbridge from the Department of Health and Social Care highlighted key achievements at a recent conference. rarerevolutionmagazine.com/rare-insider...

Trust is the cornerstone of effective Public Patient Participation Inclusive and Engagement (PPIE). Dr Sondra Butterworth, CEO of RareQoL, shares her insights on the importance of trust at personal, system, and organisational levels. rarerevolutionmagazine.com/rare-insider...

A rare disease board trustee reveals the behind-the-scenes efforts to bridge gaps between research, advocacy, and industry. Discover the untold stories of dedication, collaboration, and innovation that drive progress for patients. rarerevolutionmagazine.com/rare-insider...

In our latest interview, we had an insightful conversation with Dr Sondra Butterworth, CEO RareQoL, community psychologist and patient advocate about why building trust is the cornerstone of PPIE . Watch a sneak peak of his interview below. rarerevolutionmagazine.com/rare-insider...

Virginie Bros-Facer, CEO at EURORDS, has dedicated her career to advancing research and advocacy in the rare disease space. Her commitment to patient-centred approaches and policy change is paving the way for a brighter future for the rare community. rarerevolutionmagazine.com/rare-insider...

In our latest interview, we had an insightful conversation with Lucy Mckay and Megan Pullein from Medics for Rare Diseases (M4RD) about their involvement in the Lancet Commission. Watch a sneak peak of his interview below. rarerevolutionmagazine.com/rare-insider...

Discover how Sumair Nizamuddin, a policy officer at Nuffield Council on Bioethics, is addressing the deep-rooted mistrust in healthcare and pharmaceutical industries. rarerevolutionmagazine.com/rare-insider... #MedicalResearch #Healthcare #Trust #Bioethics #DecoloniseHealthcare

This week, we hear from Linda Stone, trustee (former chair) of @SjogrensUK, as she shares the top three unmet needs and main asks of the industry to better serve the rare disease community. rarerevolutionmagazine.com/rare-insider... #RareDisease #PatientAdvocacy #SjögrensUK

The disbanding of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) has sent shockwaves through the rare disease community. Read more about the impact and the path forward in our latest article by Amy Gaviglio. rarerevolutionmagazine.com/rare-insider...

Discover groundbreaking insights from Sumair Nizamuddin on how historical contexts of medical racism and unethical research practices continue to impact trust in healthcare today. Watch a sneak peak of his interview here: rarerevolutionmagazine.com/rare-insider...

Dive into the latest advancements and challenges in the rare disease world. From groundbreaking gene therapies to new reports on healthcare innovation, Nicola Redfern's August article covers it all. #RareDiseases #HealthcareInnovation #GeneTherapy rarerevolutionmagazine.com/reasons-to-b...

Henry Burkitt, Managing Director at Oxygen Strategy, calls for a complete rethink to ensure faster access to life-changing therapies and fair rewards for innovation, prioritising health impact over fiscal accounting! Read more: rarerevolutionmagazine.com/rare-insider...