Making Research Matter for Families - Dr. David Weinstein
Dr. David Weinstein and his team studied a rare disease in children called Glycogen Storage Disease Type 1a. Children with this disease need corn starch often to manage their condition. Talking to parents, they realized that children with this condition needed a treatment to help them stay healthy throughout the night without needing to wake up every few hours to take their medication. Dr. Weinstein’s team worked with families to find a solution. They created a slow-release treatment that was approved. This made a big difference in the children and families’ lives.
Dr. Weinstein’s team also asked patients and doctors what they thought were the most important things to study about this disease. They worked together and came up with a list of the most important things to research.
Ultimately, Dr. Weinstein and his team listened to families, identified and developed a treatment to improve the quality of life of patients and their families, and asked everyone what should be studied next. This teamwork made things better for the children and their families, and helped researchers know what to focus on.
Read articles:
Research priorities for liver glycogen storage disease: An international priority setting partnership with the James Lind Alliance
Can patients be involved in preclinical research?
Engaging Patients in Ph.D Ovarian Cancer Research - Emma Gerber
As an ovarian cancer research trainee, I know the important statistics and molecular mechanisms related to this disease. However, I lacked a human connection to what I was studying. After learning about patient engagement, I recognized its potential value, but did not know how to begin engaging patient partners in my research. Thanks to support from my university and Ovarian Cancer Canada’s Patient Partners in Research group, I was able to connect with a panel of patient partners with lived experience of ovarian cancer to discuss engagement opportunities in my PhD thesis project. The prospect of engaging with patients in discussions excited me, and the panel’s enthusiasm was contagious. Based on their lived experiences they highlighted the importance of identifying minimally invasive biomarkers to guide ovarian cancer treatment. Importantly, input provided by patient partners enhanced my planned studies. For example, comparing biomarkers of interest at initial diagnosis and in a recurrent setting was suggested, which had not been originally included in my research plan. Embarking on this journey of patient engagement as a graduate student has been immensely rewarding. As someone without a personal connection to ovarian cancer, this experience has transformed my research, infusing it with a sense of grounding and humanity. Amidst the challenges of academic research, this has been a powerful source of motivation. Having had this experience early in my career motivates me to pursue future research collaboratively with those who have lived experience of the conditions being studied.
Sepsis Canada and the National Preclinical Sepsis Platform
The National Preclinical Sepsis Platform (NPSP) provides an example of how patient engagement can shape preclinical laboratory research. The NPSP includes patient partners who are identifying new perspectives and priorities for sepsis (severe infection, which can lead to organ dysfunction) research. Though still in the early stages of their project, through attendance at laboratory meetings, patient partners have identified several clinically relevant outcomes that would be of interest to measure in animal experiments.
To take a thoughtful and tailored approach, the group held an icebreaker/ non-technical session to discuss the NPSP goals, the experimental model and patient partner questions, and worked as a team to co-develop terms of reference.
Additionally, the team has worked together on several grant applications to fund a priority-setting exercise (to identify a patient-important question that could be assessed in a sepsis mouse model), co-conduct of a preclinical systematic review to address this question, and eventually co-development of a laboratory experiment to further study the identified question of interest.
By working with patient partners from the start (e.g., grant/project development) to identify shared goals, the team believes this will have important downstream impact, by ensuring their research is relevant to the patient community.
Public Involvement in Research Funding Processes - Parkinson's UK
Parkinson’s UK is the largest European charitable funder of Parkinson’s research, granting approximately £8m annually in medical research, both clinical and non-clinical. Parkinson’s UK believes that the feedback from people affected by Parkinson’s makes a difference and improves the way the charity does things for people affected by Parkinson’s. Involving people living with Parkinson’s in research funding decisions lets the Parkinson’s community know that they are being listened to and that they have a say in the research being funded by the charity.
Parkinson’s UK has involved lay grant reviewers in its funding decisions for almost 20 years. People affected by Parkinson’s have also been involved in shaping the charity’s research strategy, via the Research Strategy Board. Through reflective practice and evaluation, this work continues to improve, serving the mission of Parkinson’s UK to improve life for everyone affected by Parkinson’s.
Parkinson’s UK believes the value and strength of the lay grant reviewer role is that the personal experience of living with Parkinson’s is brought to the research grants funding process. Through consultation and refinement, Parkinson’s UK has developed approaches and processes to support meaningful involvement in grant funding. This has included training courses, specific lay grant reviewer review forms – tailored to the perspective of people living with Parkinson’s – and more detailed plain English sections of grant applications so lay grant reviewers can fully understand the applications.