Last Updated: October 20, 2025, 4 pm UTC

Parkinson’s disease (PD) affects an estimated 10 million people worldwide, including more than 1.1 million in the United States. With prevalence rising as the population ages, experts warn of a growing public health crisis and an economic burden that exceeds $50 billion annually in the U.S. alone¹.

Disease heterogeneity, as well as other culprits, have been a barrier in finding a true disease-modifying breakthrough since the introduction of levodopa in the 1960s. Today, a robust pipeline of more than 130 active PD trials is underway, targeting diverse mechanisms from alpha-synuclein to GLP-1 agonists. Still, too many programs fail to advance beyond Phase 2, often for reasons unrelated to science — particularly when recruitment, retention, or patient burden are underestimated^2,4,5.

In this blog, we explore practical steps sponsors can take to design more patient-centric Parkinson’s disease trials—addressing common barriers, partnering effectively with advocacy groups, and applying strategies that protect both participants and data integrity.

Barriers That Keep Patients Out of Trials

For people living with PD, clinical trial participation is often a daunting prospect. Common barriers include:

Stigma and Distrust. Some patients—especially from underrepresented communities—harbor generational skepticism about research, which can deter participation when an investigational therapy feels risky².
Invasive Procedures. Lumbar punctures, frequent imaging, or repeated blood draws can be burdensome and reduce willingness to enroll.
Logistical Challenges. Long-distance travel, multiple site visits, and limited caregiver support make participation difficult.

If these barriers aren’t addressed up front, recruitment stalls and attrition rises—threatening timelines and data integrity.

Building Trust Through Early Engagement

Patient-centric design benefits from engaging patients, caregivers, and advocacy groups early in planning:

Co-Design What Matters. Listening sessions help confirm symptoms and outcomes that are meaningful (e.g., sleep, fatigue), guiding secondary/exploratory endpoints that complement MDS-UPDRS^3–5. At later stages, these insights can also help ensure endpoints align with FDA expectations for registrational trials, balancing patient relevance with regulatory requirements.
Reduce Burden Together. “Walking” the visit schedule with patients and caregivers often reveals opportunities to shorten visits, add breaks, or adjust timing.
Communicate Clearly. Co-develop plain-language FAQs, informed consent explanations, and caregiver guides—ideally with multilingual options for equity.
Keep the Loop Open. Maintain a feedback channel during enrollment so teams can adjust quickly when pain points surface.

Partnering with Patient Advocacy Groups—From Intent to Impact

Early, ongoing collaboration with patient advocacy groups (PAGs) anchors a study in what matters to patients and strengthens outreach and credibility. Four pillars can help guide effective engagement:

Timing of Engagement. Begin before protocol finalization and continue through site start-up and enrollment. Early input informs endpoints, visit cadence, caregiver touchpoints, and patient-facing materials.
Partner Selection. Match organizational strengths to your program’s needs—national foundations can bring registry and endpoint expertise, while regional groups often excel at community engagement.
Collaboration Approach. Define roles, shared goals, and cadence (e.g., monthly check-ins; rapid reviews of outreach content). Preserve trust by inviting PAGs to communicate directly and independently with their communities.
Contributions to Development. PAGs add value by refining secondary/exploratory endpoints and PROs^3-5, supporting recruitment through registries and outreach, providing education on trial logistics, and advocating with regulators for outcomes that matter most to patients.

Collaboration works best when it’s reciprocal: share plain-language updates, lessons learned, and—where possible—aggregate results back to the community.

Simple PAG Engagement Plan (T-12 Weeks to First Patient In (FPI))

Timeline	Key Actions	Example Deliverables / Metrics
T-12 to T-8 weeks	Conduct listening sessions with patients/caregivers; draft FAQs and caregiver guide; align registry and outreach plan.	Draft patient FAQs; caregiver logistics guide; outreach strategy outline
T-8 to T-4 weeks	Review patient-facing materials with PAGs; schedule community webinar; confirm diversity goals and tracking.	Approved FAQs; webinar invite; diversity dashboard
T-4 to FPI	Launch outreach (registries, email, social posts); train sites on FAQs and caregiver scripts; open feedback channel.	Live outreach campaign; site training completion; active feedback loop

Practical Solutions That Put Patients First

There isn’t a one-size-fits-all model, but several strategies consistently make PD trials more accessible and sustainable:

Remote Visits and Wearables.

Building remote visits into the protocol from the outset reduces travel burden. Paired with sponsor-provided wearable devices (plus training/support), teams can capture tremor, gait, or sleep data at home with greater consistency and less exclusion than bring-your-own approaches⁶.

Caregiver Accommodations.

Caregivers often make participation possible. Concierge travel, meal and lodging coverage, and flexible scheduling help families manage complex visits. Clear, proactive communication with caregivers reduces uncertainty.

Open-Label Extensions (OLE).

When families weigh competing trials, OLE access beyond the blinded phase is often a deciding factor—especially in progressive diseases like PD².

Clear and Continuous Communication.

Regular updates, plain-language materials, and multilingual resources signal respect for patients’ time and comfort and contribute to more complete datasets.

A Patient-Centric Path Forward

Scientific hurdles in PD are formidable, but operational hurdles should not be the reason promising therapies fail to reach patients. By thoughtfully addressing barriers, reducing burdens, and engaging advocacy groups from the earliest stages, sponsors can create studies that are both rigorous and realistic. Patient-centric design strengthens recruitment and retention, improves diversity, and protects data integrity, while fostering the trust needed for long-term collaboration.

The next breakthrough in Parkinson’s research may emerge not only from advances in the lab but also from how well the clinical research community listens to—and supports—the patients at its center.

At Premier Research, we bring extensive experience in designing and executing Parkinson’s disease trials, helping sponsors anticipate challenges, protect data integrity, and move programs forward with confidence. Contact us to learn how our neuroscience experts can support your next PD study.

ABOUT PREMIER RESEARCH:

Premier Research, LLC (Premier) is a global leader in clinical research and consulting services with expertise in driving an efficient and effective path to market for the life sciences industry. 

Premier is built with the needs of biotech in mind, turning breakthrough science into life-changing drugs, devices, and diagnostics by addressing trial complexity, overcoming development hurdles, and demonstrating product value. 

Leveraging deep therapeutic expertise, innovative technology, and product development operational proficiency—from preclinical planning to clinical trial execution and commercialization—our integrated approach offers personalized, end-to-end solutions to identify the pertinent data and insight necessary to make informed decisions earlier and deliver accelerated development timelines for a smarter, faster path to approval. To learn more visit premier-research.com. 

References

American Parkinson Disease Association. (n.d.). What is Parkinson’s? American Parkinson Disease Association. https://www.apdaparkinson.org/what-is-parkinsons/

Poewe, W., & Chaudhuri, K. R. (2025). Parkinson’s disease drug development pipeline: Active trials and emerging mechanisms. Journal of Clinical Neuroscience, 96, 123–135.

LeWitt, P. A., et al. (2015). Efficacy endpoints in advanced Parkinson’s disease: Off time, dyskinesia, and quality-of-life measures. Parkinsonism & Related Disorders, 21(5), 462–468.

Pagano, G., et al. (2022). Trial of cinpanemab in early Parkinson’s disease. New England Journal of Medicine, 386(26), 2451–2461.

Pagan, F., et al. (2022). A Phase II study to evaluate the safety and efficacy of prasinezumab in early Parkinson’s disease (PASADENA): Rationale, design and baseline data. medRxiv.

Wang, Y., et al. (2024). Modeling of Parkinson’s disease progression and implications for detection of disease modification in treatment trials. Journal of Clinical Neuroscience, 100, 123–130.