Vitaccess

Rare diseases affect the whole family. Many rare diseases are genetic and diagnosed in childhood. They are often life-limiting and have no cure. As such, they inevitably have a profound and lasting impact on the wider family, including siblings. This report, "The impact of RARE disease on sibling experience", published by @RARE Revolution Magazine, shares insights from young siblings of people with rare diseases. The report is based on data from 52 young siblings of people with a range of rare diseases from the UK and US. It discusses the impact of rare diseases on siblings' relationships with peers and family, their ability to make plans and experience "ordinary" activities typical of their age, responsibilities, education and employment, mental health, and more. The report stresses that support for siblings is vital, but is currently limited and can be difficult to access. It recommends, among other initiatives, more training for families, particularly around planning for the future, improved school support systems, and awareness-raising activities. This publication is incredibly interesting and insightful - it's definitely worth a read, for both those working in rare diseases and others. https://bit.ly/3QENK7Z

Celebrating the incredible women of Vitaccess this #InternationalWomensDay! 🌟 At Vitaccess, women aren’t just part of the team – they are the team. With women consistently making up over 50% of our staff, their expertise, leadership, and innovation are at the heart of everything we do. From driving research forward to shaping patient-centred insights, their impact is felt every day. Whether it’s through mentoring, supporting flexible working, or amplifying diverse voices in research and real-world evidence, we’re committed to fostering an environment where women thrive. Today, we celebrate our colleagues, our friends, and all the women who inspire us – not just at Vitaccess, but across the world. 💜 [Picture of a few of the team enjoying the sun on the office rooftop terrace...bring on summer 2025] #IWD2025 #WomenInResearch

NICE has added new guidance about qualitative research to its real-world evidence framework. We've summarized the key takeaways for you. ✔️Qualitative data can be used in health technology assessment to understand people's experiences, beliefs, preferences, attitudes, behaviors, interactions, and social context. ✔️In the protocol for a qualitative study, provide a clear rationale for taking this approach, as well as justification for the proposed methods for sampling, data collection, and analysis. ✔️Involve stakeholders in the study design, including members of the target population and subject matter experts. ✔️When reporting, clearly describe the sampling strategy, including the rationale for stopping sampling (data saturation or otherwise), and the methods for data collection, handling, and analysis. ✔️Describe approaches taken to ensure the credibility of data collection and analysis, such as considering researcher reflexivity (how researchers' own values and preferences may influence the study), exploring negative cases that do not align with the majority, independent coding, and triangulation with other data sources. ✔️Clearly state the key findings of the study. Look out for an upcoming blog from Vitaccess on this subject, where you can learn more about how we are leading the way in qualitative research. You can also get in touch with one of our team at info@vitaccess.com. Read the NICE recommendations in full here: https://bit.ly/4ijvP2A

With Spring fast approaching 🌺 we thought it’d be a good occasion to present who we are at Vitaccess! We specialize in patient-centered real-world research. Through our RWE generation, patient-centred outcomes solutions and analytics, we are dedicated to amplifying the patient voice. We support you in gathering relevant, reliable data, draw robust conclusions, and develop compelling evidence - aiming for better outcomes for patients while strengthening the impact of your product. We collaborate with those who are serious about patient-centered outcomes and patient-centricity. From nimble biotechs to global pharma giants, we bring scientific rigor, digital innovation, and a friendly, down-to-earth approach to every partnership. Our team of experts blend industry knowledge with cutting-edge methodologies and tech, delivering tailored research solutions that capture the complexities of real-world patient experiences. As we move forward in 2025, we’re excited to continue empowering healthcare decisions with robust evidence. Let's shape the future of healthcare together! #RealWorldEvidence #PatientCentredOutcomes

💙 Today, on Rare Disease Day, we stand with the 300 million people worldwide living with a rare disease and their families.💜 Rare Disease Day is a globally coordinated movement, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease. At Vitaccess, we're proud to contribute to the rare disease community by generating real-world evidence that can help support the development of life-changing therapies. By amplifying patient voices and providing insights that matter, we aim to help researchers, clinicians, and industry partners bring treatments to those who need them most. https://bit.ly/3CV0XGk Factsheet on rare disease statistics in the UK from Genetic Alliance UK

Today, on National Caregivers Day, we honor the incredible dedication of caregivers - family members, friends, and professionals - who provide essential support to those in need. Their compassion, strength, and advocacy make a profound impact on patients' lives. At Vitaccess, our research has shown that caregivers play a critical role in patient well-being, from managing medications to offering emotional support – yet, their own health and needs are often overlooked. That’s why we’re committed to advancing research that amplifies caregivers’ voices, ensuring they receive the resources and recognition they deserve. Visit our publications page at https://lnkd.in/e3NU28y8 to learn more about the caregiver-focused research we have conducted, including: • Quality of life impact of caregiving for patients with MCT8 deficiency: results from a cross-sectional survey (Akosua Ofori, Mark Larkin PhD) https://bit.ly/4gSH808 • Development of two conceptual models to describe the patient and caregiver reported impacts of living with X-linked myotubular myopathy (XLMTM) (Octavia Borecka, PhD, Jack Lawrence, Sam Llewellyn) https://bit.ly/3D2j656 • Caregiver burden in growth hormone deficiency: a targeted review (Fatemeh Amini, Octavia Borecka, PhD) https://bit.ly/41ds7QO • What role does caregiver burden play in health technology assessment bodies’ appraisals of highly specialized technologies? (Akosua Ofori, Sam Llewellyn) https://bit.ly/419zRUb • Patient and caregiver-reported burden of transfusion-dependent β-thalassemia measured using a digital application (Emma Bagshaw, Amber Kudlac, Mark Larkin PhD) https://bit.ly/41qAaLD This #NationalCaregiversDay, let’s celebrate these unsung heroes. #PatientCentredResearch

Next Friday, 28th February, we will be celebrating Rare Disease Day. Rare Disease Day is a global movement, coordinated by 65+ national alliance patient organization partners, which aims to raise awareness and generate change for the 300 million people worldwide living with a rare disease, their families, and carers. Hear their stories and be part of the progress at: https://hubs.ly/Q030ZV-w0

Exploring EQ-5D in real-world research – see you in London! This Thursday, Jack Lawrence will be heading to the EuroQol UK & Ireland EQ-5D Researchers Meeting in London! 🏛️✨ At Vitaccess, we use EQ-5D across many of our real-world and patient-centered outcomes studies, helping to generate health utility values that support cost-effectiveness modelling, HTA submissions, and economic evaluations. Whether it’s in chronic diseases, oncology, or rare conditions, EQ-5D plays a vital role in capturing patient perspectives in a way that truly informs decision-making. With such a great lineup of talks (including discussions on methodological advancements and applications of EQ-5D across diverse patient populations), we’re looking forward to insights that will help us continue to deliver high-impact research. If you're attending, come say hi to Jack and let’s chat about all things EQ-5D and real-world evidence! #EQ5D #PatientCentredResearch #EuroQol

📢 We're excited to announce that Mark Larkin PhD and Fatemeh Amini will be presenting "A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-Reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry", at the 15th Myasthenia Gravis Foundation of America, Inc. International Conference (May 13-15, the Hague, the Netherlands). Are you planning to attend the conference? Get in touch with our team to set up a meeting or to learn more about the registry. https://bit.ly/4jXcC8a

To move beyond symptom management to disease modification, remission, and cure, a deeper understanding of immunological diseases is necessary. Real-world research provides opportunities to gather a wealth of detailed information, by linking patient data from multiple sources. Genetic and molecular data, clinical data from medical records, and patient-reported data about symptoms and adverse events can all be linked, thereby permitting analysis of, for example, underlying drivers of disease severity or predictors of treatment response. At Vitaccess, we specialize in this type of research, with many of our projects involving the prospective longitudinal collection of both clinical and patient-reported data. Get in touch to find out more. https://hubs.ly/Q030-9hR0