Blood cancers affect the blood and the immune system, often leaving people feeling extremely tired and more vulnerable to infections. This can have a significant impact on the quality of life of patients and their families.

"With few surgical options available, blood cancer patients are dependent on innovation in medicines to improve their outcomes and quality of life" – Blood Cancer Alliance

Despite the clear need for effective blood cancer medicines, data shows that blood cancer medicines face particular challenges in the NICE appraisal process: appraisals are more likely to be terminated than medicines for other cancer types or disease areas. Challenges arise as many blood cancer medicines are given in combination with other medicines ("combination regimens"), which improves patient outcomes however leads to difficulties in assessing cost-effectiveness using NICE's current methods.

"The disproportionate rate of terminated appraisals of blood cancer treatment suggests there are issues within the system that must be urgently addressed to ensure all NHS patients can access the best possible treatment for their disease." – Blood Cancer Alliance

Ref: Blood Cancer Alliance. https://www.bloodcanceralliance.org/access

Generalised myasthenia gravis (gMG) is a rare autoimmune condition that causes muscle weakness, which makes everyday activities such as walking, eating, speaking and breathing difficult. Symptoms can be debilitating, and in severe cases life-threatening.

Until 2026, no new medicine for gMG had been made available in England for over a decade. The NICE approval of a new medicine for gMG in May 2026 was a welcome step forwards, however several other innovative medicines which could help treat gMG are still not available in England. Medicines for rare diseases like gMG face many challenges in the NICE process, particularly those which meaningfully improve quality of life, reduce symptoms or slow progression, but may not significantly affect survival. In particular, when there is no established standard of care, innovative medicines are compared against low-cost symptomatic treatments which makes it challenging to meet NICE's cost-effectiveness thresholds. Additionally, if the medicine is already approved for a different condition, this may make it more difficult to reach NICE's cost-effectiveness requirements as less flexibility is permitted once an earlier indication is already approved.

"For people living with myasthenia gravis, finding the treatment that works for them can be life-changing, particularly when existing options fail or cause significant side effects. The first targeted therapy is a long overdue step forward – but with others already developed and struggling to get through the system, it is clear the process is not working as it should for patients." – Muscular Dystrophy UK