Sheela Upadhyaya | Life Science Advisor in Rare Diseases & Special Advisor
Openflex Ltd
Sheela Upadhyaya is a Life Sciences Consulant and a renowed expert in rare disease with over 25 year experience in the healthcare industry. She has broad international experience spanning, UK including Scotland and the EU5 with a record of delivering leading edge innovation for rare diseases. She is co-chair for Health Technology Assesment international (HTAI) Rare Disease Interest Group and is Chiar for the ISPOR Rare Disease Interest Group key project. She has co-authored several papers and presented at many conferences discusisng the challenge in rare disease innovation development, assessment and implementaition. She commissioned rare disease service provision in the NHS and led NICE's Highly Specialised Technology program, evaluating therapies for rare and ulra rare conditions. She is chair of Togther4 Rare Diseases which is an inititaive looking to encourage and improve collaborative working between European Reference Networks and Industry. She has extensive experience and understands well the issues in rare disease facing teh lide scinces sector across all stakeholders from diagnosis, research, servcie provsion and market access. She has worked with many companies large and small including Alexion, Takeda, Sanofi, Biomarin, Biogen and many others developing optimised market access strategies and successful patient engagment activties. She is deeply committed to partnership working and excels at building concencus among disparate groups. Please get in tocuh if you have a proposal or project that could benefit from my experience.
Appearances:
Pre-Congress Workshops - Tuesday 22nd October @ 10:30
WORKSHOP: Hollistic Approaches to Rare Disease Drug Development: Integrating ESG, Patient Engagement, and Commercial Strategy for Improved EU Access [OFFSITE]
This workshop will be covering:
-Unique challenges in rare disease drug development and the need for a comprehensive approach.
-ESG in Rare Disease: Discuss how environmental, social, and governance factors are becoming increasingly important in the rare disease space.
-Emphasize the social impact of developing treatments for underserved populations
-Discuss ethical considerations in rare disease access to treatment
-Patient Engagement: Stress the critical role of patient involvement throughout the drug development process
-Showcase successful patient engagement strategies
-Discuss how patient input can shape access strategy and clinical trials
-Highlight the impact of patient advocacy on regulatory decisions
-Discuss the importance of early market preparation
Day 1 - Wednesday 23 October @ 14:10
Panel: Acting Now for People Living with Rare Diseases : The Aspire4Rare Example
An EU Action Plan will be a significant milestone for PLWRD, however, while the community continues to advocate for this vital progression there remains urgent work to be done at the national level. Aspire4Rare is an initiative developed by UCB Pharma with the input of experts from the Rare Diseases community. It proposes a Rare focussed health system framework with associated measurement approaches and good practice examples which can serve as a practical tool right now to support action on national policies such as rare disease strategies. In this session we will hear about how the Aspire4Rare framework was developed and how it can be used to support action now.
The Aspire4Rare Global report can be found via the below link:
https://www.ucb.com/sites/default/files/2024-05/UCB_Aspire4rare%20GlobalReport_2024_May27th.pdf
Day 1 - Wednesday 23 October @ 15:10
Challenges of Late onset Rare diseases – recommendations from the COLLABORATE Consensus Paper
Day 1 - Wednesday 23 October @ 16:10
Together for Rare Diseases: Learnings from pilot projects
Day 1 - Wednesday 23 October @ 16:40
Panel: Evidence Assessment in rare. Why do we need a change in Paradigm. The work of HTAi RDIG
Day 2 - Thursday 24 October @ 14:40
Public-Private Partnerships to accelerate development and boost innovation in Rare Diseases
- the evolving landscape of European health policy, this high-level panel brings together key stakeholders to address a critical yet often overlooked issue: the urgent need to enhance collaboration between European Reference Networks (ERNs) and industry in rare disease research. With a new EU leadership, this discussion comes at a pivotal moment to shape the future of rare disease policy and research prioritization.
- Leveraging public-private partnerships to accelerate innovation
- Proposals for a "sandbox" framework to facilitate agile collaborations
- Enhancing patient involvement in shaping research priorities
- Addressing funding challenges through novel mechanisms
As we anticipate the Hungarian Presidency's focus on rare diseases and look ahead to the Polish and Danish Presidencies, this panel will generate actionable insights to inform policy recommendations. Our goal is to catalyze momentum for an EU Rare Disease Action Plan that embraces the power of ERN-industry collaborations.
Despite progress, 95% of rare diseases still lack effective treatments, highlighting the imperative for innovative partnerships. This panel will explore how to overcome regulatory, funding, and capacity barriers that hinder optimal ERN-industry collaborations. By bringing together voices from patient advocacy, clinical research, policy, and industry, we aim to forge a path towards a more collaborative and efficient rare disease research ecosystem in Europe.
Key topics include:
Day 2 - Thursday 24 October @ 16:30
Keynote: How do we reinvent the healthcare system to meet the demand of not only rare diseases, but potentially all patients in future?
-By 2025 we will have many more gene therapies/ATMPs in development/approved. With this future healthcare systems cannot have bespoke solutions for each new ATMP, how can industry work with the healthcare system to build models of care that are effective and sustainable
-How can industry create solutions facilitate patient access to gene therapies?
-How can companies and the healthcare system work together to make it more equitable?
-What role can clinicians and patients play in developing a system that is efficient and delivers the outcomes expected?
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