Westminster Health Forum

Improving outcomes for rare diseases in England - research and treatment, specialised commissioning and access to medicine

TO BE PUBLISHED January 2020

This seminar will discuss improving outcomes for rare diseases in England - looking at research and treatment, specialised commissioning, and access to medicine.

Delegates will examine the progress of the UK Strategy for Rare Diseases, following the publication of the Department of Health and Social Care’s 2019 update to the implementation plan for England and policy priorities moving forward from 2020, when the strategy is due to end.

The seminar also follows the publication of NHS England’s update to its implementation plan, and we expect discussion on ensuring collaboration between stakeholders to deliver the ambitions of the twin implementation plans.

Attendees will also assess any barriers to progress so far - including the impact of Brexit on the UK’s participation in European Reference Networks, medicines regulation and on collaboration and knowledge-sharing.

Delegates will discuss government initiatives aimed at improving diagnosis and treatment for rare diseases, including:

Planned sessions will consider the priorities for scaling up to meet these ambitions and their impact.

We also expect discussion on what further measures might be necessary to roll out genomic testing - such as ensuring the workforce has the right training and skills, developing digital infrastructure in the UK, and key issues for managing patient data and ensuring public trust.

Priorities for training in genomics for healthcare professionals across all levels, following the extension of funding to 2020 for the Genomics Education Programme will also be assessed - including progress of key aims to develop strategic collaborations to deliver the Genomic Medicine Service for the NHS.

We expect discussion on the next steps for improving the delivery of care for rare diseases and commissioning specialised services - including allocating long-term funding, integrating specialised services with wider pathways of care and reducing variation in the quality of services at a local level.

It follows plans to implement a set of provider criteria for services treating patients with rare diseases - including ensuring that there is a person responsible for co-ordinating the care of patients with rare diseases, providing patients with an ‘alert card’ containing information about their condition, and improving the transition from paediatric to adult services.

Delegates will also consider the key issues for improving access to medicines for rare diseases, with discussion on managing the affordability of medicines for rare diseases and overcoming regulatory challenges for accelerating the uptake of innovative treatments and maintaining access.

The conference will be an opportunity to assess the impact to industry and patient access following the Government’s decision to charge for NICE technology appraisals and highly specialised technologies, which came into effect in April 2019 - as well as the introduction of the voluntary scheme for branded medicines pricing and access in December 2018.

Further sessions will consider the priorities for research into rare diseases - including supporting collaborative working between researchers, service providers and patients, translating research into best practice and looking at the opportunities for innovation to improve outcomes for patients. It follows the creation of the BioResource for Translational Research in Common and Rare Diseases, which is set to improve collaboration in research.


Price: £95 PLUS VAT

Shortly after every Westminster Health Forum seminar, a briefing document is produced. This is distributed to all delegates on the day as well as to our policymaker contacts in government, and to stakeholders more widely.

A seminar publication provides a timely record of proceedings, and acts as a guide to the latest thinking on current policy issues for those unable to be at the event.

This publication includes


Contributions from keynotes and panellists, including accompanying slides*
*Subject to approval

Delegate Pack

Information from the day, including delegate list, biographies and agenda


Transcript of questions and comments posed to speakers from attending delegates


Supplementary articles from speakers
and delegates