The evaluation and access to medicines challenge
Companies are working hard to create the medicines of the future for people with rare diseases. In order to get those medicines to patients, they are evaluated by a number of regulators to make sure they are safe, effective and value for money for the NHS.
When working on a medicine for a rare disease, companies are inevitably working with smaller numbers of patients and therefore uncertainty in the evidence, which means that getting a medicine for a rare disease approved can pose a challenge. When there is a medicine available for a rare disease, we want to make sure that people who need it can get it quickly.
Opinions
Rare disease day 2026
Equity in the age of advanced therapies: insights from haemophilia
Laurence Woollard is Director of On The Pulse Consultancy and co-chair of ATMP Engage, a multi-stakeholder forum focused on patient involvement in advanced therapies. In his latest blog for Rare Disease Day, he reflects on how advanced therapies are being introduced within the NHS, using haemophilia to explore questions of equity and benefit-risk – highlighting the need for a Patient Charter.
Why we need the new Rare Therapies Launch Pad
For Rare Disease Day, the ABPI’s Director of Regulatory and Early Access Policy Dan O’Connor looks forward to contributing to an innovative initiative to help improve patients’ access to personalised treatments for rare diseases.
Moving beyond box-ticking and lip service – why patient involvement matters in a new era of ATMPs for rare diseases
Laurence Woollard is a patient advocate for rare diseases and a Patient and Public Voice (PPV) Partner on NHS England’s ATMP working group. For Rare Disease Day, his message is that meaningful patient involvement is essential to achieve greater access to potentially life-changing therapies for rare diseases.
By working together we can improve the lives of people with rare diseases
Many people are surprised to learn that rare diseases are actually comparatively common. A rare disease is defined as one that affects fewer than one in 2,000 people and it is reckoned that 300 million people worldwide have one. More than 6,000 rare diseases have been identified and about 72% of those have a genetic origin.
Evolving the UK health system to support innovation for the Rare Disease community
Nicola Redfern, General Manager of Bluebird Bio in the UK, describes the commercial challenges for rare disease medicines in the UK and reflects on the opportunities of gene therapies.
Last modified: 26 February 2026
Last reviewed: 26 February 2026