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Caroline Donoghue, access manager at blood cancer charity Myeloma UK, gives the five steps that are needed for people to have access to the most effective drugs and treatments.
On the surface, progress in myeloma treatment is a huge success story. In the past 30 years, the treatment pathway has been completely transformed, moving from no bespoke treatments to a range of different drugs and combinations that can be given across multiple lines. More people are living longer, and many are living better than ever before.
Yet, myeloma remains incurable, and life expectancy still lags behind many other cancers, with only a third of people living for ten years or more following diagnosis. Treatments have also become more intensive and continuous, meaning the burden of treatment is too high for too many people.
The system that brings innovation to patients is also too slow and uneven. People with myeloma still face delays in access, restrictions on treatment choice, and unfair differences in care depending on where they live.
There is so much more that needs to be done to make the hope for a cure a reality for people living with myeloma.
Fragmented policy
Unfortunately, today’s health innovation and access system is not keeping pace with the changes we urgently need. Policy is too fragmented, and ambitions across research, regulation, and assessment are too often disconnected.
As a result, the UK is falling behind other countries in adopting newer, more patient-led treatment approaches and in approving and using myeloma treatments.
The UK needs to be an attractive place in which to research and launch new medicines to secure faster access to innovation. Recent health policy changes, including the increase in NICE thresholds and the government’s commitment to invest in medicines, should help with this. However, our processes for approving and implementing new treatments and taking innovations from clinical trials to clinical practice are fragmented, complex, and require a lot of time and resources. This can lead to delayed or cancelled launches.
In myeloma, CAR-T cell treatments are a good example of this. These treatments could be transformative for patients, delivering deep, durable responses while giving them highly valued time off treatment. They are licensed in the UK and have been available in parts of Europe for nearly five years, yet they are not available on the NHS. We need a more joined-up approach and a clear access-to-medicines strategy to ensure that research and drug access systems work in harmony and deliver better access to treatment innovations.
Getting innovative drugs approved for use on the NHS is only one step towards faster, fairer access. In myeloma, we have had several treatments approved, but around half of these have come with restrictions that limit their use to specific groups and lines of treatment. These strict rules can block the option that best fits the patient and have resulted in a complex, inflexible pathway shaped by process and commercial pressures rather than by patient need or clinical judgement.
Myeloma is a highly individual cancer, and treatment options depend on risk status, past treatment, the risk of side effects and overall health. The complex nature of myeloma means the restrictions on use can leave some groups of patients without options or having to settle for a harsher, less effective treatment. If we want to give all patients the best chance, treatment pathways must become more flexible and more patient-led.
In recent years, we have also seen growing inequalities in access to newly approved treatments. Truly innovative treatments or treatment approaches often come with different side-effect profiles, new testing requirements and administration protocols that need to be rolled out in already stretched and sometimes underfunded clinics. We have already seen this with new bispecific antibody treatments. They can cause serious side effects when patients start them, so some hospitals need specialist training, additional resources and funding for setup before they can offer them.
Myeloma UK launched the Closer to a Cure report to highlight these issues and remove the barriers preventing people from gaining timely access to the most effective drugs and treatment approaches.
Built on three decades of insight, the report sets out the five urgent steps needed by government, industry and policymakers to bring us closer to a cure and ensure everyone can access the treatments they need, when they need them.
Five steps
STEP 1: What’s the problem? The system that approves medicines in the UK is fragmented, long and complex. As a result, the UK is falling behind other countries in approving and adopting new myeloma treatments.
What needs to change? We need research and drug access systems to work in harmony to deliver better access to treatment innovations.
STEP 2: What’s the problem? Many people with myeloma spend years on continuous treatment and live with serious side effects, repeated hospital visits and constant disruption to daily life. Yet, quality of life is often overlooked when new treatments are being developed.
What needs to change? Innovators must focus on improving, as well as extending, people’s lives.
STEP 3: What’s the problem? Myeloma affects each person differently, yet access pathways still rely too often on a one-size-fits-all model. If we want to find a cure for all patients, we cannot accept this lack of differentiation.
What needs to change? Treatments must be tailored to individual patients.
STEP 4: What’s the problem? Strict eligibility rules and outdated restrictions often stop clinicians from using the treatment that best fits the patient’s needs. As a result, too many patients are excluded from or miss out on new treatments and have to settle for substandard treatments with reduced effectiveness and increased side effects.
What needs to change? Treatment pathways must become more flexible and more patient-led.
STEP 5: What’s the problem? The approval of a drug by NICE means little if local barriers delay rollout of a treatment to a particular trust, hospital or region and lead to patients missing out on the most effective treatment.
What needs to change? Approved treatments must reach patients quickly and fairly.
These changes are achievable, but we need to act now. Progress will only come if everyone involved in getting drugs approved works together and prioritises what patients want: longer lives, a better quality of life and more personalised choices.
