Westminster Health Forum

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Next steps for rare diseases and specialised commissioning - policy priorities, utilising genomics, patient engagement and co-ordinating care

February 2021


Price: £95 PLUS VAT
Format: DOWNLOADABLE PDF


***Full-scale policy conference taking place online***
This conference will discuss the key policy priorities and next steps for improving outcomes in rare diseases and specialised commissioning in England.


The discussion is bringing together stakeholders with key policy officials who are due to attend from the Department of Health and Social Care; the MHRA; the Office for Life Sciences; and The Scottish Government.


The discussion at glance:


  • UK Strategy for Rare Diseases - assessing progress in implementation in key areas affecting patients and their care, and research, and understanding what still needs to be done
  • the future:
    • policy priorities going forward with the strategy due to come to an end at the close of 2020 and the next steps following the strategy
    • the shape of medicines and medical devices evaluation and regulation following the new Trade and Cooperation Agreement reached with the EU and the impact on rare disease patients
  • Genome UK - realising opportunities presented by the new national genomics healthcare strategy for improving rare disease outcomes
  • COVID-19 - the impact on delivery of care, and progress this year, and ongoing implications for patients and care

The agenda:


  • Assessing progress and policy priorities going forward for improving outcomes in rare diseases
  • Maintaining momentum in improving outcomes, and the implications of COVID-19 for patients
  • Commissioning specialised services through the pandemic and the ongoing drive to improve quality and delivery in care
  • Improving patient access to treatment, developing collaborative approaches, and learning from the experience of COVID-19
  • Opportunities for utilising national genomic policy developments to improve diagnosis and treatment for rare disease patients
  • Key priorities for rare disease research, and the way forward for funding and collaboration
  • Improving patient involvement in disease specific research areas
  • Next steps for adopting innovation and delivering improved health outcomes for rare disease patients - workforce training, effective diagnostics, sharing best practice, and coordinating care

A scan of relevant developments and background:


  • The UK strategy for rare diseases - with commitments for 2013-2020 on prevention, diagnosis and early intervention, coordinated care, patient empowerment, and advancing research
  • UK Rare Disease Framework - building on the UK rare disease strategy
  • Genome UK: the future of healthcare - which aims to enable earlier interventions with more predictive and personalised care
  • Changes we're making to health technology evaluation - consultation by NICE on their approach to guidance
  • Integrating Care - The next steps to building strong and effective integrated care systems across England document - NHS England outlining the operational future for CCGs and clinical commissioning in an updated integrated care system, with emphasis on aligning specialised services with linked care pathways, and an option for this to be based in legislation
  • Mendelian and Modality NHS Partnership rare disease diagnosis programme - aiming to support diagnosis in primary care, matching health records with published diagnostic criteria for rare diseases

Key areas for discussion:


  • the UK Strategy for Rare Diseases:
    • progress - what has been achieved through the Strategy, including putting in place a national infrastructure to take forward improvements in genomics and rare diseases
    • the UK Rare Disease Framework - assessing its potential to create more coordinated care and faster diagnosis, and to improve training of staff and identification of rare diseases more quickly
    • priorities for future policy - development of a framework to build on the work of the current strategy
    • assessing the success of the Strategy - following a Genetic Alliance UK and Alexion Pharma UK survey which found that there are still many unmet needs for rare disease patients across the entire patient pathway, and subsequent calls for a continuation of collaborative action to improve the quality of care
    • key challenges for moving on from the Strategy:
      • COVID-19 - the ongoing impact and future implications of shielding for patients with rare conditions, delays to treatment, access to care, and the outlook for research
      • the new relationship with the EU - the way forward for medicines regulation and research collaboration now that the transition period is over
    • taking forward key aims of the Strategy:
      • collaboration, care pathways and patient involvement - how they can be supported and developed as part of a future framework
      • the potential impact of the Mendelian and Modality NHS Partnership rare disease diagnosis programme
      • maintaining momentum - priorities for the continuing process of improving outcomes for rare diseases and ensuring that future policy is informed by patients
  • commissioning specialised services - in the context of the pandemic, as well as the priorities going forward, including:
    • care coordination, effective transitioning and integrating care with wider services
    • funding
    • achieving and maintaining quality service provision, and reducing variation in local services
    • COVID-19 - assessing the impact on rare disease care of the recent national lockdown
    • knowledge sharing and utilising networks
    • adapting clinical commissioning into an updated integrated care system following NHS England’s updated strategies for integrated care
  • patient access - improvement for innovative treatments:
    • COVID-19 - what lessons can be taken from the response to the pandemic in terms of accelerating processes and working collaboratively
      • future opportunities - including around:
        • managed access and the possibilities for the Innovative Medicines Fund
        • collaborative approaches and supporting early engagement, adaptation and identification of technologies
    • highly specialised technology appraisals - to what extent do methods and processes need to be updated as NICE consults on its approaches to appraisals in this area
    • pricing models - assessing options for improving patient access by varying the price of medicines depending on what disease they are being used to treat
    • access to medicines - the impact on medicine access following the offer of incentives by the MHRA to encourage the development of medicines in rare diseases
  • genomics - its role in delivering improvements:
    • assessing the vision - opportunities for improving rare disease outcomes through using genomics in diagnosis and treatment, and the progress that has already been made
    • NHS Genomic Medicine Service:
      • opportunities for increasing diagnostic services and advancing genomic medicine for rare disease and cancer patients within routine NHS care
      • assessing the new role of the seven Genomic Laboratory Hubs
      • the impact, on patient service and treatment, of the NHS Genomic Medicine Service Alliances which will oversee and coordinate the embedding of genomics into mainstream clinical care
    • the pandemic - what can be learned from the collaborative genomics response to COVID-19, including the impact of £1.2m in government funding to the CLIMB COVID-19 project, enabling a greater capacity to store genomic data, and how this can be transferred across to work in other disease areas
    • next steps:
      • standardising approaches to genomic testing
      • the way forward for introducing whole genome sequencing in routine NHS care
      • how the NHS can continue to harness genomic advancements with the Genomic Medicine Service
      • the priorities for the advancement of AI for the future of genomic sequencing and the impact on effective diagnosis
    • research priorities for rare diseases - including:
      • the investment and collaboration required to achieve the innovation required to bring improvements
      • the role of technology in repurposing drugs for rare diseases
    • adoption - the effective translation of research into clinical care:
      • ensuring that the workforce receive the right training and are prepared for new methods involving genomics
      • encouraging engagement and the sharing of best practice for those working across the systems involved
    • prevention - potential opportunities and early lessons, with Advancing Our Health: Prevention in the 2020s and Genome UK outlining aims for genomics and personalised treatment
    • patient involvement - priorities for key aims in the Strategy around engagement with patients in research, including:
      • making it easier for research feedback to inform patient care
      • reducing diagnosis times
      • improving how patients can engage with specialist clinical trials in their disease area

Policy officials attending:


Our forums are known for attracting strong interest from policymakers and stakeholders. Places have been reserved by officials from the Department of Health and Social Care; the Medicines and Healthcare Products Regulatory Agency; the Office for Life Sciences; and The Scottish Government.


Overall, we expect speakers and attendees to be a senior and informed group including Members of both Houses of Parliament, and national government officials involved in this area of public policy, together with representatives from the NHS, executive agencies including clinical staff, regulators, those involved in specialised commissioning, the independent and third sectors, local authorities, pharmaceutical and diagnostics companies, research groups and networks, universities, patient groups, law firms, healthcare businesses and their advisors, consultancies, the health technology sector and others affected by the issues discussed, as well as academics, think tanks and reporters from the national, local and specialist media.


This is a full-scale conference taking place online***


  • full, four-hour programme including comfort breaks - you’ll also get a full recording and transcript to refer back to
  • information-rich discussion involving key policymakers and stakeholders
  • conference materials provided in advance, including speaker biographies
  • speakers presenting via webcam, accompanied by slides if they wish, using the Cisco WebEx professional online conference platform (easy for delegates - we’ll provide full details)
  • opportunities for live delegate questions and comments with all speakers
  • a recording of the addresses, all slides cleared by speakers, and further materials, is made available to all delegates afterwards as a permanent record of the proceedings
  • delegates are able to add their own written comments and articles following the conference, to be distributed to all attendees and more widely
  • networking too - there will be opportunities for delegates to e-meet and interact - we’ll tell you how!

Full information and guidance on how to take part will be sent to delegates before the conference



This pack includes

  • Dropbox video recording of the conference
  • PDF transcript of the discussion, including all speaker remarks and Q&A
  • PDFs of speakers' slide material (subject to permission)
  • PDFs of the delegate pack, including speaker biographies and attendee list
  • PDFs of delegate articles