Day-Zero Collaboration:
Patients and Scientists Advancing Breakthrough Therapies Together
By Marcella Debidda, PhD, Ryan Golley | June 16, 2025
1. Why Timing & Method Matter
The first academic article to invite a “patient’s perspective” was published 32 years ago. Yet decades later, many people living with rare or progressive conditions still feel like spectators in the very research designed to help them. To understand how—and when—they want their voices heard, Bionews asked members of its Spinal Muscular Atrophy (SMA), Huntington’s Disease (HD), Sickle Cell Disease (SCD), and Alzheimer’s Disease (AD) communities three questions during one week in April 2025:
- When is the best time to involve patients in the development of new therapies?
- How would you like researchers to work with patients to share their thoughts on a project?
- Do you have any other comments regarding patient involvement in research?
454 individuals responded. Their answers, shared at the second multi-stakeholder summit in Cleveland (26-27 April) with consortium institutions that included the Mayo Clinic, the University of Miami, Case Western Reserve, the University of Toronto, and ETH Zurich, send an unmistakable message: co-creation with patients must begin as early as possible—often even before formal diagnosis—and remain a continuous, bidirectional conversation from first concept to post-publication.
2. Key Insights & Data Points
Start at Day Zero.
86% (n = 388)
chose “at the very start, when deciding which health improvements matter most.”
Multiple open-ended comments stressed involvement “even at the first signs of disease, before a formal diagnosis exists.”
“Patients should be involved at every step—before trials even start.” – SMA participant
Engagement must span the full drug development life-cycle.
Respondents highlighted individualized timing around cognitive or diagnostic milestones.
Flexible, frequent communication outperforms one-off surveys.
Transparency & result-sharing remain pain points.
“We’ve never heard the results and I think that would be helpful.” – HD caregiver
Caregivers need equal footing.
Participants called for recognition of caregiver time, expertise, and emotional load:
“Please also give the patient’s family a voice in decision-making.”
Inclusive, empathetic collaboration is non-negotiable.
“Husband chose to lie/deny after diagnosis—painful!”
3. Recommendations – The “Early-and-Often” Engagement Framework
1. Start With a Patient-Defined End in Mind
Convene co-creation workshops before protocol design to prioritize efficacy and quality-of-life outcomes that matter most to patients and caregivers.
2. Build a Multi-Modal Communication Ladder
-
- Monthly virtual advisory boards for deep-dive discussions.
- Quarterly pulse surveys (≤ 10 questions) to track evolving trends.
- Always-on digital portals where participants can log feedback, view dashboards, and monitor next steps.
3. Compensate Fairly & Recognize Expertise
Align honoraria with clinical-site visit rates; pay caregivers separately and offer travel stipends; publish a transparent compensation grid.
4. Close the Loop—Publicly
- Share lay summaries within 90 days of each milestone.
- Host livestream Q&A debriefs with investigators and patient advisors.
- Archive recordings, infographics, and data in an open repository.
5. Embed Caregiver Perspectives
- Establish parallel caregiver panels; offer mental-health resources and flexible scheduling; include dedicated caregiver breakout sessions at every scientific meeting.
4. Conclusion & Call-to-Action
At the April Cleveland summit we discussed what our communities have long felt: meaningful patient partnership is neither a checkbox nor a courtesy — it is a prerequisite for credible, adoptable science. By inviting patients and caregivers from Day Zero, and by maintaining continuous, two-way communication sponsors can accelerate recruitment, improve protocol relevance and adherence, and ultimately deliver therapies that resonate in the real world.
*Enriching the doctor-patient relationship by inviting the patient’s perspective- Ann Intern Med. 1992 Mar 1;116(5):414-8. doi: 10.7326/0003-4819-116-5-414.