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  • Perspective
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Inclusion in neurological research: empowering people living with neurological diseases

Nature Reviews Neurology volume 21, pages 159–170 (2025)Cite this article

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Abstract

The value of involving people living with diseases in the research process is increasingly recognized by professional associations and regulatory agencies alike. Patient contributions range from disease prevention and diagnosis to medication planning, and from advocacy to guideline production and clinical trial design. Thanks to the efforts of activists and advocates, new models of patient inclusion in medical research are being developed to replace outdated non-participative and tokenistic paradigms. New modalities of patient participation in research — for example, the introduction of patient experts, who work closely with researchers and clinicians — have progressively empowered individuals who are living with diseases. In this Perspective, we provide an overview of the current status of patient involvement in medical research, with a specific focus on neurology. We also discuss the existing and future roles of patient experts in neurological research.

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Fig. 1: Timeline of patient involvement in medicine.
Fig. 2: The Wellcome Trust Onion Model of public engagement.

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Acknowledgements

The authors would like to thank Gloria Traina for her expert advice on regulatory and reimbursement processes, and Magdalena Eitenberger and Joann Leeding for useful discussions. The authors also thank the people with lived experience, family members and caregivers who have collaborated with us and inspired our work.

Author information

Authors and Affiliations

  1. Center for Alzheimer Research, Karolinska Institute, Stockholm, Sweden

    Maria Teresa Ferretti

  2. Associazione Italiana Miastenia, Milan, Italy

    Maria Bonaria Uccheddu

  3. European Myasthenia Gravis Association, La Louvière, Belgium

    Maria Bonaria Uccheddu

  4. My Moves Matter, Dublin, Ireland

    Richelle Flanagan

  5. School of Medicine, Trinity College Dublin, Dublin, Ireland

    Iracema Leroi

  6. Global Brain Health Institute, University of California, San Francisco, CA, USA

    Iracema Leroi

  7. Division of Neurology, CHU of Grenoble, Grenoble Alpes University, Grenoble, France

    Elena Moro

  8. Grenoble Institute of Neuroscience, INSERM U1216, Grenoble, France

    Elena Moro

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  1. Maria Teresa Ferretti
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  2. Maria Bonaria Uccheddu
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  3. Richelle Flanagan
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The authors contributed equally to all aspects of the article.

Corresponding author

Correspondence to Maria Teresa Ferretti.

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Competing interests

M.T.F. is the co-founder of the Women’s Brain Project. In the past 2 years she has received consulting and speaking fees from Roche, Angelini Pharma and Prodeco Pharma unrelated to this project. She is currently the Clinical Development Lead of Syntropic Medical. M.U. has received honoraria for consulting services from Alexion, Janssen, Merck, UCB Pharma and UCB S.A., and speaking fees from Alexion and UCB S.A.. She also provided consulting services to Argenx. Travel expenses for congresses were paid by Alexion, UCB Pharma, UCB S.A. and Argenx. R.F. is the co-founder and chief executive officer of My Moves Matter. E.M. has received restricted research grant support from Abbott and France Parkinson. She has also received honoraria from Medtronic for consultation services. I.L. declares advisory boards and honoraria for talks from Roche, NovoNordisk, Eli Lilly and Boehringer.

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Related links

Alzheimer’s Drug Discovery Foundation Diagnostic Accelerator: https://www.alzdiscovery.org/research-and-grants/diagnostics-accelerator

Alzheimer’s Research UK: https://www.alzheimersresearchuk.org/

Chan Zuckerberg Initiative Rare as One project: https://chanzuckerberg.com/science/programs-resources/rare-as-one/

Critical Path Institute: https://c-path.org/about/

European Organisation for Rare Diseases (EURORDIS) — Rare Diseases Europe: https://www.eurordis.org/

European Patients Academy on Therapeutic Innovation (EUPATI): https://eupati.eu/

My Moves Matter: https://www.mymovesmatter.com/

Patient-Centered Outcomes Research Institute: https://www.pcori.org/about/about-pcori

Patient Expert Training Programme: https://learning.eupati.eu/mod/page/view.php?id=1075&forceview=1

Patient-Led Research Collaborative: https://patientresearchcovid19.com/

PatientsLikeMe: https://www.patientslikeme.com/

The Michael J. Fox Foundation: https://www.michaeljfox.org/

Women’s Parkinson’s Project: https://www.womensparkinsonsproject.com/

Workgroup of European Cancer Patient Advocacy Networks: https://wecanadvocate.eu/

Glossary

Citizen science

Any activity that involves the public in scientific research and generates genuine scientific outcomes, from the design of the research question, through data collection and volunteer mapping, data interpretation and analysis, to publication and dissemination of results.

Embedded patient researchers

An embedded researcher works inside a host organization as a member of staff while also maintaining an affiliation with an academic institution. Embedded patient researchers are people with lived experience who carry out research in partnership with health-care teams.

Evidence-based advocacy

The application of scientific principles and evidence to health advocacy.

Health advocacy

Activities aimed at creating positive system change by increasing access to care, navigating the health-care system, mobilizing resources and funding, addressing health inequities and influencing health policy in general.

Patient advocates

People with lived experience as patients or as partners, family members or informal caregivers of a person with a health condition. Patient advocates work for themselves and their patient community to increase awareness, access to resources and help advance research and education.

Patient engagement

Patient engagement encompasses activities such as providing information or disseminating ideas or results to the public and patient communities, which are aimed at increasing awareness of scientific processes. Patient engagement represents a lower level of inclusion in research than patient involvement.

Patient-led PROs and PROMs

Patient-reported outcomes (PROs) and patient-reported outcome measures (PROMs) in which patients have provided input to inform the methods and processes used.

Patient-reported outcome measures

(PROMs). Tools or instruments that are used to measure patient-reported outcomes.

Patient-reported outcomes

The FDA defines a patient-reported outcome (PRO) as “a report that comes directly from the patient (i.e. study subject) about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.”

Preference research

Preference research aims at understanding the preferences, needs and expectations of key stakeholders in the health-care system, including patients, caregivers and health-care professionals.

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Ferretti, M.T., Uccheddu, M.B., Flanagan, R. et al. Inclusion in neurological research: empowering people living with neurological diseases. Nat Rev Neurol 21, 159–170 (2025). https://doi.org/10.1038/s41582-024-01047-6

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