TO BE PUBLISHED May 2026
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This conference will consider next steps for rare diseases policy in England.
Areas for discussion include the future direction of national strategy, priorities for research and data, access to innovative and individualised therapies, and regulatory and delivery frameworks needed to support these ambitions - including implications of the Government’s recently published National Cancer Plan for England 2026, which places rare and less common cancers as a foundational priority for the next decade.
It will be an opportunity for key stakeholders and policymakers to discuss priorities emerging from the Department of Health and Social Care’s England Rare Diseases Action Plan 2025, alongside expectations for the next annual action plan, anticipated early in 2026, as the current UK Rare Diseases Framework approaches the end of its term. Delegates will discuss what will be needed to translate aims into improved access to therapies and a more consistent patient experience across diagnosis, treatment and ongoing care - with the Cancer Plan’s recently emphasising ambitions including strengthening national leadership, progressing towards three quarters survivorship of cancer by 2035, and reducing variation in outcomes for rare cancers.
Navigating system pressures & policy delivery
Discussion will focus on approaches for delivering expected policy commitments on rare diseases in practice, including priorities for implementation as responsibility increasingly sits across national bodies and integrated care systems. Delegates will consider issues such as accountability for delivery, coordination between specialised services and local systems, and how rare disease priorities can be sustained alongside wider NHS reform and delivery of the Government’s 10 Year Health Plan for England. It comes with the Cancer Plan announcing a new national lead for rare cancers, as well as reformed oversight structures, aiming to shape system coordination and performance.
Care quality, diagnostics & patient experience
With NICE due to publish a quality standard on rare diseases in February 2026, sessions will consider what high-quality care should look like in routine practice, including expectations for timely diagnosis, coordinated multi-specialty pathways and clearer responsibility for care navigation. Discussion will also look at reducing variation in patient experience linked to geography, ethnicity and deprivation, and the implications of diagnostic delays for clinical outcomes, mental wellbeing and family life - including perspectives from patients and families on navigating care, access and support.
Delegates will assess proposals to improve diagnostic pathways and workforce capability, including the practicalities of scaling genomic testing and referral routes across the NHS, population and newborn screening pilots - as well as wider possible approaches to supporting clinicians to recognise rare conditions earlier, and addressing gaps in specialist expertise. Options for supporting earlier referral and more consistent access to specialist input will be assessed, looking at priorities for education, digital tools and service models that align with the Cancer Plan’s emphasis on artificial intelligence‑enabled clinical decision support, structured safety‑netting through Jess’s Rule, and the use of emergency presentation rates to drive earlier diagnosis in hard‑to‑identify cancers.
Access to therapies, research & innovation
Further discussion is expected on access to medicines and individualised therapies, including variation in uptake following regulatory approval, the time taken to reach commissioning decisions, and the challenges of managing high-cost, low-volume treatments within existing funding arrangements. Delegates will consider approaches to improving the generation of evidence following market entry in cases where patient numbers are small, and how this can best inform commissioning, review and reassessment.
Sessions will also assess progress and draw on insights from initiatives such as the Rare Therapies Launch Pad, examining what has been learned so far about developing regulatory, manufacturing and commissioning pathways for highly individualised n-of-1 therapies, and options for facilitating the move beyond isolated cases towards more sustainable approaches.
Regulation, data & governance
The conference will consider proposals set out by the Medicines and Healthcare products Regulatory Agency in Rare therapies and UK regulatory considerations, including options for more flexible licensing and evidence routes for small patient populations. Discussion will focus on how accelerated access can be balanced with safety assurance, equity for underserved groups and international alignment, alongside practical questions around implementation and post-approval uptake.
Further discussion will look at the role of data, registries and real-world evidence in supporting regulatory decisions, reimbursement and post-market monitoring, and how these tools can be used to strengthen care pathways and reduce inequalities. Consideration will also be given to longer-term governance and workforce priorities, including how rare diseases should be positioned within future national frameworks and four-nation coordination beyond 2027.
All delegates will be able to contribute to the output of the conference, which will be shared with parliamentary, ministerial, departmental and regulatory offices, and more widely. This includes the full proceedings and additional articles submitted by delegates. As well as key stakeholders, those already due to attend include officials from the Department of Health and Social Care; Food Standards Agency; Office for Life Sciences; and Medicines and Healthcare products Regulatory Agency; Government Legal Department.
