MAP Insights


The NHS, Rare Diseases, the UN and Patient Advocacy

It is midway through February but it’s already been a busy month.

The new NICE programme manual was released and implemented on the 1st of February. The consultation on the Innovative Medicines Fund (IMF) came to a close on the 11th, the MHRA has launched an inquiry into proposals on legislative changes for clinical trials and we’re also expecting the publication of the Rare Diseases Action Plan for England at the end of the month.

It's still early days on the NICE Manual and the ABPI and BIA have offered their views on the impact of these changes to technology appraisal (TA).  At MAP, we’re looking closely at the introduction of the severity modifiers and the new criteria for Highly Specialised Technologies guidance. Some of these methodological and process changes could result in better patient access to innovative treatments on the NHS but we’ll have to wait and see how they are implemented in practice. We are particularly looking forward to some of the modular updates, especially the one on health inequalities as this will help provide clarity on the determinants of health and access to treatment.

The scope of health inequalities goes beyond the definitions of the protected characteristics as laid out in the Equality Act 2010. It takes a wider view, covering factors that impact on an individual’s socio-economic status such as educational attainment, access to good housing, diet and nutrition. In fact, the role of health inequalities will have an important focus in the Rare Diseases Action Plan, so that’s another reason why we should give this the attention it deserves.

These developments should not be seen in isolation and need to be cross-referenced to the proposed IMF (click here to see our blog on the subject).

The evaluation of a technology by NICE, including comprehensive health economic analyses, is followed by the stage which some in industry might consider the most challenging: negotiations with the NHS. In the case of new medicines with a high degree of uncertainty, these include discussions on managed access schemes and commercial arrangements. We are led to believe that price is not the only guiding principle for the NHS, and that “NICE must have regard to the broad balance between the benefits and costs of providing health services or of social care”, the needs of patients and the desire to promote innovation in healthcare. The recent successes of Zolgensma and Libmeldy gives us great hope.

This takes us to Rare Disease Day, an international awareness event that falls on 28 February. MAP will be marking this day so do keep a lookout on our website and social media channels for activity. It is worth noting that within the rare diseases space, there are many conditions; from the mild to the severe, from the well-known to the truly obscure. Sometimes, it is easy to get caught up in the diseases themselves and risk losing sight of the people who are directly affected by them. It is these people – the patients, their families and carers – whose stories need to be told if any real change is to take place.

Which is why we were delighted to read about the UN Resolution on Rare Diseases which was ratified by member states and adopted on 16 December 2021 after several years of committee discussions between different countries, UN agencies and NGOs.

Within the resolution is a recognition that people with rare diseases are likely to be not only vulnerable and socially excluded but also stigmatised and discriminated against. To address this, they need to be empowered through greater equity in their access to care and a widening of their life opportunities. These principles are captured in the Universal Declaration of Human Rights and this resolution shines the light on the very specific needs and rights of people living with rare diseases.

Ultimately, it needs to be stressed that all country signatories have committed to upholding the resolution by improving the lives of those with rare diseases:

‘To progressively cover persons living with a rare disease with quality essential health products, health services and quality, safe, effective, affordable and essential medicines, diagnostics, and health technologies, with a view to covering all persons living with a rare disease by 2030…’

In practical terms, this means policies and programmes are needed to remove social barriers and correct the health inequalities that those with rare diseases face. This shifts the balance of power to the individual patient and patient organisation. With this, patient advocacy is set to have more influence in health and social care decision-making in years to come.

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