Unlocking patient access to new treatments


By Charity Medicines Access Coalition | Tuesday 17 September 2019

Developments in scientific research will change our understanding and management of many diseases in the future.

Innovative treatments can be life-saving or life-enhancing for patients, their families and carers. Research breakthroughs also drive further scientific discovery, leading to the medicines of tomorrow.

Unfortunately, the newest medicines are usually expensive. In addition, their pioneering nature means it can be harder to measure their effectiveness in the way the NHS has traditionally required before it will pay for a medicine.

With NHS finances under increased pressure, decisions about which new medicines to make available are also under greater scrutiny. This creates a challenging environment to ensure patients continue having timely access to promising new medicines.

But doing so is critical to improve patient outcomes. That’s why, as leading health charities and decision-makers within the healthcare system, we’re working together to unlock patient access to new medicines.

A joint effort: CMAC and NICE

The Charity Medicines Access Coalition (CMAC) brings together ten health charities working to ensure scientific advances benefit the people who need them most. Together the charities represent millions of patients, offering care and support while funding leading research.

CMAC aims to help solve the challenge of ensuring patient access to innovative medicines while protecting the NHS’s financial sustainability.

The National Institute for Health and Care Excellence (NICE) plays a pivotal role in achieving this goal. Through its Health Technology Appraisal (HTA) programme, NICE looks at the clinical benefit and value for money offered by new medicines. This helps ensure patients in England, Wales and Northern Ireland have fair access to clinically effective treatments that offer value for money for the NHS.

Over the last year, CMAC and NICE have worked together to identify where changes to NHS practices could benefit patients and carers. This has allowed the charities to make the case for change, while the opportunity to work with charities helps NICE to identify what’s important to people using health and social care services and their carers.

Together, we’ve identified three areas where we want to make a difference:

  • Increasing patient involvement in the assessment of new medicines.
  • Reviewing how NICE judges the value of new medicines.
  • Using evidence from NHS care, as well as clinical trials, in NICE’s decision-making.

Ensuring patients and carers are heard

CMAC believes patients and carers should be at the centre of choices regarding their care. They should also be involved in decisions about access to new medicines, to ensure these are based on what really matters to patients.

That’s why we’re seeking opportunities to help the NHS better understand patient perspectives, including what they most want from treatments.

NICE’s Public Involvement Team have reviewed and improved opportunities for patients to contribute to NICE’s decision-making. As part of this, patients and representatives from CMAC charities have outlined priorities for change. These include new avenues for voicing patient perspectives and greater support to ensure everyone can contribute equally and feels heard.

NICE and patient representatives will now develop recommendations for how to integrate this feedback into practice.

Heidi Livingstone, Senior Public Involvement Adviser at NICE, said, “We are delighted to be working with CMAC because working collaboratively with patient groups is fundamental to improving how we involve service users, patients and carers to ensure they’re able to shape our guidance.”

Reviewing how medicines are evaluated

Each of the conditions and disease areas represented by CMAC have unique challenges in seeing new medicines become available. But there are also common difficulties, including increased uncertainty around the effectiveness of many new medicines emerging from the research pipeline – in part a knock-on effect of efforts to get these treatments to patients earlier.

Last year, NICE committed to review the methods it uses to assess medicines. This review will streamline how NICE develops its recommendations on medicines, as well as on diagnostics and devices. It will also offer a clearer process for how new technologies (including medicines) are selected for, and go through, NICE’s processes.

CMAC believes this review offers the chance to resolve some of the common difficulties seen across disease areas.

Representatives from NICE and CMAC, as well as academia, the pharmaceutical industry, NHS England, and other patient representative organisations will be involved in this review.

Dr Alison Evans, Head of Policy at Alzheimer’s Research UK and co-chair for CMAC, said: “We are excited to be part of this important work and ready to make real changes that will impact the NHS patients of today and tomorrow.”

Using real-world evidence

For many new medicines, NICE now often has less information than in the past about the long-term treatment outcomes of patients taking the medicine when it is deciding whether the NHS should pay for it. This means NICE can’t be as confident a drug will offer enough patient benefit to justify the money the NHS will pay for the treatment.

‘Real-world evidence’ gathered after clinical trials allows us to monitor patient outcomes in NHS care. This means data from sources like NHS datasets and patient registries can be used to learn more about long-term benefit of a treatment.

CMAC wants to support this approach and explore how it could make medicines that NICE may otherwise reject available to patients.

NICE is already working to assess how real-world evidence could help evaluate new medicines’ effectiveness. NICE’s RWE navigator offers guidance on how to collect real-world evidence and helps explain how results could guide the healthcare decision-making process.

Emma Greenwood, Director of Policy and Public Affairs at Cancer Research UK and co-chair for CMAC, said, “NHS data and patient registries provide a rich evidence base that can allow us to better understand new medicines’ impact on patients. We are committed to ensuring patients see the benefits this approach can provide, including earlier access to innovative treatments.”

Further information

You can learn more about the Charity Medicines Access Coalition, and the charity partners involved, by going to https://alzheimersresearchuk.org/about-us/our-influence/policy-work/charity-medicines-access-coalition/

To read more about NICE’s work, go to www.NICE.org.uk

This is a joint blog post between the Charity Medicines Access Coalition and the National Institute for Health and Care Excellence (NICE).


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Charity Medicines Access Coalition