Published: 2026-02-01 00:19

New initiative aims to boost rare cancer patient access to breakthrough trials

A new government-backed initiative is set to enhance access for rare cancer patients across the UK to cutting-edge clinical trials. The programme, announced by the Department of Health and Social Care (DHSC), seeks to address long-standing challenges in connecting patients with rare and less common cancers to potentially life-saving research opportunities.

Addressing the Unique Challenges of Rare Cancers

Rare cancers, defined in Europe as those affecting fewer than 6 in 100,000 people per year, collectively represent a significant proportion of all cancer diagnoses. Despite their individual rarity, there are over 200 distinct types of rare cancers, accounting for approximately 22% of all cancer cases in the UK.

Patients diagnosed with these conditions often face a more complex journey compared to those with common cancers.

Diagnosis can be delayed due to a lack of awareness among healthcare professionals and the non-specific nature of initial symptoms. Furthermore, the small patient populations for each rare cancer type present considerable hurdles for research and drug development.

This can lead to limited treatment options, with many rare cancers lacking approved targeted therapies or established standard-of-care protocols beyond conventional chemotherapy, radiotherapy, or surgery.

Historically, patients with rare cancers have had fewer opportunities to participate in clinical trials. The geographical spread of small patient cohorts, coupled with the highly specialised expertise required for their management, often means that trials are concentrated in a few major centres.

This can create significant barriers to access for patients living outside these areas, impacting equity and timely access to innovative treatments.

Facilitating Access to Innovative Therapies

The new initiative aims to streamline the process by which rare cancer patients can be identified and referred to appropriate clinical trials. While specific operational details are yet to be fully outlined, the programme is expected to leverage national infrastructure and foster greater collaboration between specialist centres, research networks, and the wider NHS.

One likely focus will be on improving data sharing and patient identification. Enhanced genomic profiling and molecular diagnostics are increasingly vital in identifying specific tumour characteristics that might make a patient eligible for a particular trial, especially those investigating targeted therapies or immunotherapies.

By ensuring that these advanced diagnostic capabilities are more widely accessible and that results can be efficiently matched with trial criteria, the initiative could significantly broaden the pool of eligible patients.

Another key component is anticipated to be the establishment or strengthening of national referral pathways. This would enable clinicians, even in non-specialist settings, to easily identify and refer patients who might benefit from a trial.

Such pathways could help overcome geographical barriers and ensure that patients are not disadvantaged based on where they live.

The programme is also expected to support the infrastructure needed to conduct more trials for rare cancers. This could involve funding for research staff, specialist equipment, and the development of adaptive trial designs that are better suited to small patient populations.

These designs, such as basket or umbrella trials, allow for the testing of a single drug across multiple cancer types with a shared molecular alteration, or multiple drugs within a single cancer type.

Understanding Breakthrough Trials

The term “breakthrough trials” typically refers to early-phase clinical studies (Phase I and II) that investigate novel treatments, often those designated as ‘breakthrough therapies’ by regulatory bodies due to preliminary evidence of substantial improvement over existing options. These trials are critical for bringing new drugs and treatment approaches from the laboratory to patients.

For rare cancers, breakthrough trials are particularly vital because they often represent the first opportunity for patients to access highly innovative therapies tailored to specific molecular targets. These might include:

• Targeted therapies: Drugs designed to interfere with specific molecules involved in cancer growth and progression, often identified through genomic sequencing.
• Immunotherapies: Treatments that harness the body’s own immune system to fight cancer cells.
• Advanced cell and gene therapies: Emerging treatments that involve modifying a patient’s cells or genes to combat the disease.

Access to these trials is crucial for rare cancer patients who may have exhausted standard treatment

Source: DHSC Updates