Defining a role for policy and public affairs in patient engagement

Defining a role for policy and public affairs in patient engagement

Anyone working in market access will tell you that a well-executed policy and public affairs (PPA) plan can provide crucial support when launching a new medicine or medical device. However, ask them what it means to run a PPA campaign and you may get a few blank stares and the suggestion to speak to their PPA colleagues.

To the uninitiated, policy can seem dry, even a little boring and something that the Government does intermittently when it wants to go down a certain direction with the NHS. On the actual work of public affairs, many are of the erroneous opinion that it only entails having a coffee and chat with an MP in Portcullis House.

There is more to the PPA function of course and this was aptly demonstrated at the recent MAP Patient Access Conference, held at the Royal College of Physicians in London on 22 September 2021.

The key to having a good market access strategy is the production of good quality evidence on the safety, efficacy and cost effectiveness of a medicine. This is also true for a good PPA strategy, as we need the evidence to assess whether a new medicine fits within an existing health policy framework. Complementary to this is the supporting evidence gathered from patients, the users of medicines and services. The patient experience therefore is crucial not only in ensuring that the drugs commissioned by the NHS have a proven clinical benefit but also in the design of patient pathways.

Patients and their carers can provide valuable insight into what works and what doesn’t – whether it is about service provision or access to care. It is often the PPA function (whether in-house or via an agency) that acts as a conduit to enable closer working between companies and patient organisations.

The plenary session at the conference brought together representatives from patient organisations and industry to discuss how these different groups can be brought together for the same purpose – to ensure that the voice of the patient is heard and that timely access to innovative medicines is achieved.

The NHS and industry need to work together so that the best available treatments are developed and, more importantly, made available to patients. This is especially pertinent in rare diseases and orphan medicines where it takes time to develop new treatments and, because the levels of uncertainty and upfront costs are often high, there are concerns that the NHS will not fund them. In the case of promising cell and gene therapies, where the treatment is potentially curative but with only a limited time window for efficacy, the case for early adoption is even more pressing.

Sadly, we heard at the conference about examples where the health technology evaluation process had been particularly slow and of lives lost during the process, that perhaps could have otherwise been saved. Clearly, due process needs to be followed, especially in the consideration of scientific evidence when new data is just emerging and uncertainty is high. It is hoped that current proposals to adapt the NICE Manual so that its methods and processes are fit-for-purpose is a step in the right direction.

The advice from the patient representatives at the conference was that early engagement is essential. Patients can help support and shape services and are also best placed to articulate to health policy bodies why a particular piece of policy will or will not work from the patient and carer’s perspective.

Samantha Barber, Chief Executive of Gene People expressed that those working or involved in patient organisations are by default the experts regardless of the size of the organisation. Indeed, it was felt that there is no correlation between value and cost when deciding on whether a treatment should be made available to a child since all children have value. There needs to be a way to speed up Health Technology appraisals (HTA) to alleviate suffering and improve on children’s life chances.

Natalie Frankish, Policy and Engagement Manager of Genetic Alliance UK said that patient participation presents another viewpoint to the scientific discussions during HTAs. Patients are the experts of their care and their voices need to be heard. Groups like Genetic Alliance are working to get promising medicines to the market quicker so that lives can be saved or quality of life improved, which can take years of campaigning.

Dr Tom Kenny, Chief Executive of Rare Disease Research Partners believes that the current system is not fit-for-purpose because it isn’t patient-centred. He felt that there is a need for HTA authorities to re-evaluate how they work with patient organisations. Dr Kenny reminded us that patient organisations in the very rare diseases space are tiny and vulnerable and don’t necessarily have the resources or experience to understand the complexity of HTA. Relationships between healthcare professionals and these groups should be as transparent as possible to instil confidence that the system works.

Jess Hobart, Trustee of UK Mastocytosis Support Group spoke about how drug companies need to actively engage with patient organisations and charities early on, often before the start of the HTA process. Companies should work more with patient organisations to help support clinical trial design that collects meaningful data that matter to patients and their carers. Patients, in turn, may reveal what they value in a treatment, what good quality of life looks like and how it improves their lives. This data could be used to help reduce the uncertainty highlighted by the current available evidence. This requires a different way of looking at treatment – identifying what is useful to the patient rather than the company or researcher and using patient engagement as a tool to improve evidence gathering.

Charlotte Galvin, General Manager of Amicus Therapeutics provided a perspective from the pharmaceutical industry. She spoke about how the UK is at the crossroads of being a world leader in the life sciences, through the Life Sciences Vision against a backdrop of access and uptake challenges with NICE and NHS England. She highlighted the need for all effort to focus on developing the best quality data even if there is high uncertainty and small populations to draw from. Pharmaceutical companies themselves are often frustrated over the many barriers to getting new medicines to patients and they are keen to collaborate and work together to find solutions. It is also essential to ensure that relationships between industry and patient organisations are open and transparent.

There was much talk about the future landscape of rare diseases and orphan medicines. Commercially, we need to continue to make the case for companies to invest in UK with the patient firmly placed in the centre from the development of clinical trials, through to HTA and NHS implementation. Patient registries were considered as an important step and need to be developed further with support from the health system while remaining independent, removed from the control of any entity with a vested interest other than that of patients.

What’s the conclusion? There is need for better collaboration between the different stakeholders involved but at the heart of the whole process should be the patient.

To view the videos of the conference, please click here      

Gerald Chan

Associate Director, Policy and Public Affairs & Communications
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