Research and Publications
Patient-reported outcome measures add value as clinical trial endpoints
Norah L. Crossnohere, Anne L. R. Schuster, Jeffrey Bruckel, Ronald C. Chen, Amy M. Cizik, Samantha Cruz Rivera, Christopher Muth, Linda Nelsen, Derek Kyte, Albert W. Wu, Elissa Thorner, Claire Snyder & Michael Brundage
Nature Medicine
Abstract
Asking patients about their symptoms, functioning and well-being during clinical trials can influence trial outcomes, regulatory decisions and clinical practice. Randomized clinical trials (RCTs) are the gold standard for assessing novel interventions in health. Traditionally, RCTs have used endpoints derived from physiological and laboratory measures, clinician-reported outcomes, and performance outcomes to assess the effect of interventions.
News
From patient preferences to patient experience
How patient perspectives are helping shape research, regulation and care in multiple sclerosis
The importance of incorporating the perspectives of people living with multiple sclerosis into research, treatment development and healthcare decision-making continues to gain recognition across the international MS community.
Building on the principles outlined by the U.S. Food and Drug Administration (FDA) regarding Patient Preference Information and patient experience data, recent initiatives have further highlighted the value of capturing the real-world priorities, challenges and unmet needs of people affected by MS.
Among them, the recently released Voice of the Patient Report from the Shaping Tomorrow Together: The MS Externally Led Patient-Focused Drug Development Initiative provides important insights into the experiences and expectations of the MS community, reinforcing the growing role of patient perspectives in shaping research, regulatory discussions and future care pathways.
Artificial intelligence and science of patient input: a perspective from people with multiple sclerosis
Anne Helme, Dipak Kalra, Giampaolo Brichetto, Guy Peryer, Patrick Vermersch, Helga Weiland, Angela White and Paola Zaratin
Published: 17 February 2025
Abstract
Artificial intelligence (AI) can play a vital role in achieving a shift towards predictive, preventive, and personalized medicine, provided we are guided by the science with and of patient input. Patient-reported outcome measures (PROMs) represent a unique opportunity to capture experiential knowledge from people living with health conditions and make it scientifically relevant for all other stakeholders.
News
Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle
Guidance for Industry, Food and Drug Administration Staff, and Other Interested Parties
The U.S. Food and Drug Administration (FDA or the Agency) values the experience and perspectives of patients. FDA understands that people who live with a disease or condition and utilize devices in their care (hereafter “patients”) may develop their own insights into and perspectives on the benefits and risks of devices.1 FDA believes that patients can and should bring their own experiences to bear in helping FDA evaluate the benefit-risk profile of certain devices. This kind of input can be important to consider during FDA’s decision-making for these devices.
Patients provide valuable input to FDA in a variety of forms. Section 569C(c)(2) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as amended (including by section 3001(3) of the 21st Century Cures Act, Pub. L. No. 114-255), states that, for purposes of section 569C, “the term ‘patient experience data’ includes data” that are “intended to provide information about patients’ experiences with a disease or condition.” FDA encourages industry to consider patient experience data in device development and evaluation. This includes data relating to patient preferences for outcomes and treatments. This guidance focuses on “patient preference information” (PPI) as one specific type of patient experience data.
Patient‑Generated Data as Indicators of Impact of Multidisciplinary and Multi‑stakeholder Health Care Systems, in the Age of Personalised Medicine
Paola Zaratin, Per Soelberg Sørensen, Óscar Fernández and Giancarlo Comi
Published: 12 February 2025
Abstract
Using the case study of the Multiple Sclerosis Care Unit Initiative launched by the European Charcot Foundation, we discuss the need to evaluate the impact of multidisciplinary and multi-stakeholder healthcare systems on patient-reported experience and outcomes for the pro- gramming and monitoring of brain and neuro-degenerative diseases in Europe and beyond.
New article
Digital patient experience tools in multiple sclerosis: a landscape analysis of the global Patient-Reported Outcomes in Multiple Sclerosis (PROMS) initiative
Vasilis-Spyridon Tseriotis, Liesbet M. Peeters, Lotte Geys, Marcus D’Souza, Johan van Beek, Ludwig Kappos, Valerie Block, Patrick Vermersch, Paola Zaratin, Robert Hyde, and Letizia Leocani, on behalf of the eHealth working group of the Patient-Reported Outcomes in Multiple Sclerosis initiative
[Background] Electronic health (eHealth) applications can increase the uptake of patient-reported outcomes (PROs) in people with multiple sclerosis. We aim to present the findings of the eHealth tools landscaping exercise conducted by the global Patient-Reported Outcomes for Multiple Sclerosis (PROMS) initiative.
[Methods] A structured survey was disseminated among identified eHealth tool developers. Metadata (up to 10 October 2024) were hosted on the Multiple Sclerosis Data Alliance catalogue, an open-access platform focussing on digital health tools in multiple sclerosis. eHealth tools’ clinical domains were compared using a Kruskal–Wallis nonparametric one-way analysis of variance, based on domain-specific scores (each domain received one point for every test in which it was evaluated), followed by Dunn’s post-hoc tests with Holm adjustment for multiplicity.
We need to engage representative patients’ community rather than ‘expert patients’ to increase impact of research in the era of personalized medicine – Commentary
Paola Zaratin
Published: 11 February 2025
Abstract
As patient-advocacy representative, with experience in neuroscience research in different sectors (academia, pharma, and a patient organization), I have had the privilege to face the challenge of maximizing the impact of patients’ engagement in brain health research from different perspectives.
A digital version of the nine-hole peg test: Speed may be a more reliable measure of upper-limb disability than completion time in patients with multiple sclerosis
Xiaotong Jiang, Marisa McGinley, Joshua Johnston, Jay Alberts, Robert Bermel, Daniel Ontaneda, Robert T Naismith, Robert Hyde, Nick Levitt, Johan van Beek, Zhaonan Sun, Nolan Campbell and Christian Barro
Published: 12 October 2024
Abstract
Background: A digital adaptation of the nine-hole peg test (9HPT) was developed with the potential to provide novel disability features for patients with multiple sclerosis (PwMS).
Objectives: The objectives were to evaluate the 9HPT features based on reliability, prognosis, and discrimination between treatment groups.
The global patient-reported outcomes for multiple sclerosis initiative: bridging the gap between clinical research and care – updates at the 2023 plenary event
Paola Zaratin, Sara Samadzadeh, Meral Seferoğlu, Vito Ricigliano, Jonadab dos Santos Silva, Abdulkadir Tunc, Giampaolo Brichetto, Timothy Coetzee, Anne Helme, Usman Khan, Robert McBurney, Guy Peryer, Helga Weiland, Peer Baneke, Mario Alberto Battaglia, Valerie Block, Luca Capezzuto, Loïc Carment, Paolo Angelo Cortesi, Gary Cutter, Letizia Leocani, Hans-Peter Hartung, Jan Hiller, Jeremy Hobart, Kaisa Immonen, Paul Kamudoni, Rod Middleton, Patricia Moghames, Xavier Montalban, Liesbet Peeters, Maria Pia Sormani, Susanna van Tonder, Angela White, Giancarlo Comi and Patrick Vermersch on behalf of the PROMS Initiative Scientific Committee and Working Groups
Published: 20 June 2024
Abstract
Significant advancements have been achieved in delineating the progress of the Global PROMS (PROMS) Initiative. The PROMS Initiative, a collaborative endeavor by the European Charcot Foundation and the Multiple Sclerosis International Federation, strives to amplify the influence of patient input on MS care and establish a cohesive perspective on Patient-Reported Outcomes (PROs) for diverse stakeholders.