Introducing PALADIN

The past decade has seen a proliferation in the number of investigational therapies and the number of companies sponsoring active clinical trials. At the same time, an unprecedented number of collaborations between patient advocacy groups and biopharmaceutical companies have formed to not only reach and recruit patients into clinical trials but also to shape clinical development plans, to ensure outcomes relevant to patient communities are reflected in drug development activity, and to support access to clinical trials for historically underserved communities.

Patient advocacy groups are often considered the trusted partner in a patient’s health journey, supporting patients and their caregivers. And advocacy groups are interested and may invest in clinical research activity but, for many, their primary mission is not to conduct clinical trials. Biopharmaceutical companies, on the other hand, are focused on expediting and improving the clinical trial process and in bringing new therapies to market.

In recent years, through forums like FDA Patient-Focused Drug Development meetings and social media, patient communities and their families are calling upon their advocacy organizations, health care providers, drug developers and the clinical research enterprise to communicate and collaborate more effectively in order to accelerate the development of new therapies, with the ultimate goal of increasing access to life-saving treatments.

While well-intentioned, with few exceptions, collaborations between patient advocacy groups and industry have been limited in their effectiveness in making clinical research faster and more efficient. This is largely due to wide variation and inconsistency in collaboration expectations, processes, and practices. A major opportunity exists to standardize and structure these R&D-driven collaborations to optimize their impact and reduce the risk of repeating past mistakes. Specific optimization opportunities include:

• improving understanding of respective operating processes; objectives; restrictions; needs; priorities; and timelines.
• establishing standards for contractual arrangements and fair market value language.
• increasing transparency around effective organizational and governance structures, training, operational support, monitoring and evaluation/assessment.
• improving understanding of funding request and delivery cycles.
• improving early communication to anticipate short- and long-term mutual interests and promote effective planning of resources and timelines.
• developing mechanisms to apply lessons learned and measure value and impact to improve future collaborations

Addressing an unmet need

The good news is that all of the underlying conditions and opportunities to optimize collaborative efficiency and effectiveness are addressable. Biopharmaceutical companies and patient advocacy groups have devoted significant attention, effort and financial investment to establishing partnerships. However, there has been limited attention and investment devoted to determining and communicating ‘how’ industry and patient advocacy groups can best collaborate and to establishing an accessible repository of resources, standardized practices and lessons learned.

Recognizing this opportunity, in the spring of 2023, Sanofi provided thought-leadership and funding to launch a pre-competitive, disease-agnostic Consortium whose mission is to optimize advocacy group-industry collaboration to ultimately accelerate the pace of medicines development. Over 20 charter groups launched the Consortium to collectively develop:

• common foundations that improve industry and advocacy R&D-focused collaborations;
• guidance and trainings for advocacy and industry representatives on how to implement patient- and caregiver-informed R&D approaches;
• measures to improve diversity in clinical trials through best-practice sharing across industry;
• awareness programming aimed at educating and connecting patients to clinical trials; and
• knowledge-sharing across therapeutic areas to reduce silos and maximize learnings.

As of May 2023, current members of the PALADIN Consortium include:

• Accelerated Cure Project
• Aimed Alliance
• Allergy & Asthma Network
• American Kidney Fund
• Astellas
• Astra Zeneca
• Black Health Matters
• Bristol Myers Squibb
• COPD Foundation
• CSL
• Merck, known as MSD outside of the United States and Canada
• Michael J. Fox Foundation for Parkinson's Research
• National Alliance for Caregiving
• National Hemophilia Foundation
• Novo Nordisk
• Opus Genetics
• PeDRA
• Pfizer
• Sanofi
• Susan G Komen®
• Takeda
• Tigerlily Foundation

The Tufts Center for the Study of Drug Development (Tufts CSDD), part of Tufts University School of Medicine, is providing project management support for the Consortium including assistance with governance and operating activity; planning and execution of work streams; meeting facilitation; development and production of work products; and a communication strategy to ensure broad dissemination of the work of the Consortium.

Consortium members have collectively created a charter and chosen PALADIN — Patient Advocacy Leaders And Drug (Development) Industry Network — for the Consortium’s name with the mission of Inclusive and Optimal Collaboration Accelerating Drug Development.

Year one – four primary work streams

In its first year, PALADIN will focus on four primary work streams. These areas have been identified as essential and fundamental to collaborative success:

• Common Foundations, Processes and Approaches
• Collaboration Skills and Training
• Guidelines for Optimizing Patient Engagement and Clinical Trial Participation
• Guidelines for Transparent Solutions that Work

Each work stream will meet approximately once per month to develop their respective work products. To ensure that insights, recommendations, and resources are relevant and useful, each work stream will be comprised of, and co-chaired by, representatives from patient advocacy groups and industry. During this launch period, the Consortium’s governance will include one member of each participating patient advocacy group and biopharmaceutical company. All Consortium, work stream activity and deliverables will be consensus-driven.

The key to PALADIN’s success in the first year will be establishing the highest level of engagement, collaboration and consensus among member organizations and creating best-in-class resources (i.e., standard practices, guidelines and skills training). Success also depends on Consortium resources being recognized, applied and adopted by the broader community of patient advocacy and drug development organizations.To this end, PALADIN plans to publish updates and articles on its progress and to provide easy access to finished work products.

Concluding thoughts

The launch of PALADIN is an exciting and important step to establish more effective relationships between patient advocacy groups and industry. We look forward to working together to develop consensus-based guidelines, training, resources and competencies that will optimize patient advocacy group- biopharmaceutical company collaborations. Ultimately, we believe PALADIN’s impact will enable more consistent, effective and efficient engagementsthataccelerate the development of new medical therapies and better align medicines development with patient needs.

It is our hope that PALADIN will continue to innovate and support advocacy-industry collaborations well beyond its launch year. While the focus for year one is on the US ecosystem, the Consortia members believe there will be a future opportunity to leverage learnings to other countries and adjust for local regulations and laws.

If your organization would like to learn more about PALADIN, please contact Trish Davidson at Patricia.davidson@tufts.edu

The authors acknowledge suggestions and insights from Silvia Cerolini, Roslyn Young-Daniels, Mary Elmer, De De Gardner, Sara Loud, Colin Rock, Michael Spigler, Wendy Vargas, Len Valentino, Anthony Yanni

Author Affiliations:

Victoria DiBiaso, Sanofi

Ken Getz, Tufts University

Bruce Miller, COPD Foundation

Swathi Ramesh, AstraZeneca

Lisa LaLuna, Patient Advocate

Patricia Roselle, Sanofi

Tara Hastings, Sanofi

Michael J. George, Sanofi

Ellyn Getz, CSL

Patricia Davidson, Tufts University*

Emily Botto, Tufts University

Abigail Dirks, Tufts University

*Corresponding author to whom inquiries should be directed: Patricia.davidson@tufts.edu

Disclosures

PALADIN is funded by Sanofi US and authors Victoria A. DiBiaso,¹ Patricia Roselle,² Tara Hastings,² Michael J. George² are employees of Sanofi

Institutional Affiliation:

¹Sanofi, 1 Avenue Pierre Brossolette, Chilly Mazarin, France

²Sanofi, 55 Corporate Dr, Bridgewater, NJ, USA