Moving beyond box-ticking and lip service – why patient involvement matters in a new era of ATMPs for rare diseases

Laurence Woollard is a patient advocate for rare diseases and a Patient and Public Voice (PPV) Partner on NHS England’s ATMP working group. For Rare Disease Day, his message is that meaningful patient involvement is essential to achieve greater access to potentially life-changing therapies for rare diseases.

In the UK, we are fortunate that all NHS organisations have a statutory duty to engage patients and the public in decision-making about the delivery of healthcare.[1] This is known as ‘Patient and Public Involvement’ or PPI, which is aimed at improving patient safety, experience and health outcomes by partnering on initiatives that are done ‘with’ or ‘by’ service users, rather than ‘to’, ‘about’ or ‘for’ them.[2]

Advanced therapy medicinal products (ATMPs) are those which can repair, replace, regenerate and re-engineer genes, cells and tissues, offering new horizons for people affected by some rare, lifelong conditions.

With growing numbers of ATMPs being developed,[3] NHS England created a working group to ensure that patient priorities are embedded in ATMP research and development, together with raising the public profile of these pioneering treatments and to produce clear patient-facing information. The group is co-chaired by Genetic Alliance UK and the Accelerated Access Collaborative (AAC).

I am one of a number of Patient and Public Voice (PPV) Partners on the group. Some of us live with, or are impacted by, a rare disease, others not; together, we are a diverse team of committed patients, service users, carers and other members of the public willing to share our unique perspectives and experiences to help make health services more responsive to people’s needs. 

In the context of ATMPs, we are holding NHS England and its industry partners to account by challenging their thinking and encouraging them to see aspects through the ‘eyes’ of patients, reinforced by a sense of compassion and respect that underpin patient-centred care.[4]

For example, the Association of the British Pharmaceutical Industry (ABPI) and the AAC recently published their ATMP Roadmap Tool; a first-of-its-kind interactive resource to help companies get their new, innovative products to patients in the UK.

During the development of this tool, as PPV Partners we stressed the importance of keeping patients top of mind throughout the treatment pathway and to engage with patient groups early, giving serious consideration to the diversity of patient populations. This was adopted as one of the four best practice principles of the roadmap and communicated to over 300 ATMP stakeholders. PPV partners also helped make sure that two animated videos from NHS England were simple and effective for lay audiences, while also avoiding the reinforcing of stereotypes about disability and ‘illness’ (e.g., showing a person bed-bound or dependent on others or both). PPV Partners requested for the patient characters to display a sense of meaning and life fulfilment that is possible, even in the face of adversity.

In contrast to conventional medicine, these novel treatments have extremely complex characteristics that are not yet fully understood and present ethical challenges for both provider and patient. There needs to be a greater level of appropriate and tailored patient education, resources and tools made available to ensure that the patient or caregiver has an all-round understanding and is fully aware of the consent they give or deny.[1]   

One of the main criticisms of PPI is the tokenistic nature of participation or ‘box ticking’, limited to a small number of middle-class people on a committee, simply to satisfy a bureaucratic mandate or funding requirements or both (i.e., either not using their input or involving them in a pointless activity to be able to claim that they were ‘involved’).^2,[2] Also, even when participatory methods have been used, power imbalances have been reported, where the experiential knowledge of patients/carers is overshadowed by academics’ research knowledge.[3]

This is wrong - patients and the public can make valuable contributions to any area of healthcare and the greater and more meaningful the level of involvement, the more likely there are to be positive outcomes for all involved.[4] It is most successful when everyone involved embraces partnership working, including easy communication channels, well-defined roles and adequate and respectful remuneration for time and effort.

Our group is being genuinely embraced and playing an important role to help usher in a new era of ATMPs that will hopefully benefit people living with rare diseases and more common ones. I urge NHS England and manufacturers alike to make increasing efforts to adopt PPI norms and values, while reducing unintentional tokenism, through listening to those whom these treatments matter most to: the patient and their family.

End.

Laurence Woollard is Director of On The Pulse Consultancy. Twitter: @TheWoollard

[1] NHS England. Patient and public participation policy. 2017. [Online]. Available at: https://www.england.nhs.uk/wp-content/uploads/2017/04/ppp-policy-edit.pdf [Accessed 3 October  2023]

[2] Ocloo J, Matthews R. From tokenism to empowerment: progressing patient and public involvement in healthcare improvement. BMJ Qual Saf 2016;25:626-32. Doi: 10.1136/bmjqs-2015-004839

[3] Cell and Gene Therapy Catapult. Annual Review 2021. 2021. [Online]. Available at: https://fr.zone-secure.net/64109/.Catapult-Annual-Review-2021/#page=1 [Accessed 30 January 2022]

[4] Pelzang R. Time to learn: understanding patient-centred care. Br J Community Nurs 2010;19(14):912-17. Doi: 10.12968/bjon.2010.19.14.49050

[5] Woollard L, Gorman R, Rosenfelt DJ. Improving patient informed consent for haemophilia gene therapy: the case for change. Ther Adv Rare Dis 2021;2:1-16. Doi: 10.1177/26330040211047244

[6] Gove D, Diaz-Ponce A, Georges J, et al. Alzheimer Europe’s position on involving people with dementia in research through PPI (patient and public involvement). Aging Ment Health 2018;22(6):723-29. Doi: 10.1080/13607863.2017.1317334

[7]Chambers E, Gardiner C, Thompson J, et al. Patient and carer involvement in palliative care research: An integrative qualitative evidence synthesis review. Palliat Med 2019;33(8):969-84. Doi: 10.1177/0269216319858247

[8] Hughes M, Duffy C. Public involvement in health and social sciences research: A concept analysis. Health Expect 2018;21:1183-190. Doi: 10.1111/hex.12825