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Insights on including Patient and Public Involvement (PPI) in Advanced Therapies

6 May 2021

The Cell & Gene Therapy and Regenerative Medicine TINs presented an online event to highlight the importance of Patient and Public Involvement (PPI) in translational research.

In a recent Therapeutic Innovation Networks (TINs) seminar jointly hosted by the Cell & Gene Therapy and Regenerative Medicine TIN, insights were provided on how Patient and Public Involvement (PPI) improves the quality, strength and relevance of such projects, as well as how to get involved.

With a particular focus on advanced therapies, the seminar welcomed the varied perspectives of leading researchers, clinicians and patients in PPI, outlining the importance to research and highlighting the real-world impacts. 

Bringing researchers, patients and public together for mutual learning to support patient-centric research

The first speaker, Linda Von Neree (PPI Lead at NIHR Blood and Transplant Research Unit (BTRU) in Stem Cells and Immunotherapies at UCL) gave an overview of PPI and the benefits, as well as how to approach the PPI section commonly found in translational funding applications. Linda mentions that a common misconception of PPI is simply participation, when rather, PPI is much more than that, and should involve a two-way conversation and a mutually beneficial relationship.  

Linda talk screenshot

A key message throughout the talks is the cruciality of including PPI early on in translational research to maximise research. This is first mentioned by Linda when discussing PPI in funding applications. Including patient and public involvement as early as possible not only demonstrates to funders that you have awareness of needs for a patient-centric strategy and the costs involved, but also allows you to build a relationship with patients to meet these needs by incorporating them into your novel therapeutics to increase chances of clinical uptake. 

For more information about including PPI in your advanced therapies research, please contact Linda Von Neree, Translational Research Office (TRO). 

Are patients willing to have gene therapy?

Professor Matthew Walker (Consultant Neurologist at the National Hospital for Neurology and Neurosurgery) spoke as the first panellist representing an academic researcher perspective, highlighting his involvement of patients in his own gene therapy research on epilepsy. Professor Walker describes how PPI has been important throughout his studies – from initially, determining if patients were willing to have gene therapy as a treatment option for epilepsy, through to providing clarity and reassurance to the public about the new therapies developed. This is especially relevant for cell and gene therapy where viral vectors are injected into the body, which can cause some concern for patients. Therefore, another key area for involving patients, is in drawing up the patient information literature to provide an accurate representation of the therapy in lay language, is an incredibly important factor for successful clinical trial recruitment.

Professor Walker also highlights the type of characteristics to look out for when determining “who” should get involved. His talk runs between 17:34 and 29:02 in the recording link below: 

Watch the PPI event webinar recording

Additionally, if you would like to learn more specifically about Professor Matthew Walker’s work and the portfolio of supporting gene therapy projects that it is a part of, please take a look at our recent TRO 10-year review, which highlights this key work supported by the TRO as a case study on page 21: https://bit.ly/2TRO10 

Gene therapy trials – the patient perspective

As the event agenda turned to the patient perspective, Luke Pembroke showed an impactful video to describe his personal experience as a Haemophilia B gene therapy patient:

YouTube Widget Placeholderhttps://youtu.be/uWjIrFSllrM

Luke’s talk touches on the reality of being a gene therapy patient in a clinical trial, outlining what others are to expect if they are to get involved, including the timelines, struggles (especially during a national lockdown!) and rewarding aspects, such as pushing the science forward to help others. 
 
We additionally heard from Gillian Murphy (Patient & Public Research Panel Member, Blood and Transplant Research Unit at UCL), who brings an interesting hybrid perspective, working in the field of cancer research before being diagnosed with leukaemia. Gillian’s talk highlights her journey from a research career to becoming a patient representative, sharing what was learnt along the way. 
 
Gillian mentions that during her postdoc days, her research was at the very basic, fundamental end of the research spectrum and although it was cancer research, she wasn’t involved with any direct interactions with patients (the closest she got was handling the donated cells). It was when she started working for the NIHR Clinical Research Network facilitating the recruitment of patients, that she was introduced to the importance and value of bringing in patients, carers and members of the public to support research. 
 
When considering her next career step, Gillian unfortunately received a shocking diagnosis of acute lymphoblastic leukaemia, an aggressive form of blood cancer with limited successful treatments for adults. Due to her background and general awareness of the field, one of the first questions Gillian asked upon diagnosis, was “Is there a clinical trial I can join?”. 

Gillian talk screenshot 1
The patients’ trials differ in that where Luke’s was rather experimental, Gillian’s was more large scale, incremental change comparing standard treatment to a new treatment that included additional drugs that could make all the difference. Thankfully, this was the case for Gillian where one of the additional drugs used helped her to reach remission status. 
 
Since then, Gillian has joined many patient groups and describes how she has enjoyed using her experience, working in partnership with researchers, to better research. 

Gillian talk screenshot 2
To listen to the patient perspectives within this Cell & Gene Therapy and Regenerative Medicine TIN seminar, please refer to 29:24 - 1:00:14 in the recording link below:
 

Watch the PPI event webinar recording

Involving PPI in Pre-Clinical Work

Final speaker, Dr Nick Hamilton (NIHR Clinical Lecturer, UCL), brought us back round again to the importance of including PPI early in research, even in pre-clinical stage work. 

As an ENT surgeon, Dr Hamilton’s work involves problems within the throat and looking at the delivery of mesenchymal stem cells to help overcome these. The cells used are taken from the discarded umbilical cord following birth and are grown in the lab before being delivered to the patient.  Something that he thought about when starting this project, was “how will people feel about having these cells taken from that source?”. Dr Hamilton set out to get that early opinion with the guidance of the UCL Translational Research Office. Running a focus group provided the opportunity to adapt and change his therapies before getting locked into a way of doing things that perhaps wasn’t suitable for patients. 

I’m a big believer that we should start to think about PPI early in the translational process, and not wait until it’s too late to engage”.

The presentations were followed by a poll of the audience and an insightful panel discussion with Q&A. To view the full event recording, please complete the form via the link below and join the Cell & Gene Therapy and Regenerative Medicine TINs if you would like to attend future events. 

If you would like to get in touch with any of the speakers and/or explore possible collaborations with regard to the topics discussed, please contact TIN Coordinator Dr Asha Recino (a.recino@ucl.ac.uk).  

Further resources for Patient and Public Involvement (PPI) in research

UCL(H) PPI Leads

Related News

This is a write up of a recent C&G/RegMed TIN Seminar: Patient and Public Involvement (PPI) in Advanced Therapies. The event was recorded and is available to watch in full:

Watch the PPI event webinar recording