Most discussions around patient involvement in clinical trial design focus narrowly on the participant experience. As a result, input from patient advocates often centers on treatment burden, visit frequency, and potential side effects.

Specifically, concerns of actual participants are as following:

• Will this experimental treatment harm me?
• What are my chances of receiving the active treatment?
• Why am I excluded from participating at all?

What’s often missing from the conversation is a broader perspective:
How does trial design benefit all patients, not just those enrolled in clinical trials?

The reality is that most patients will never take part in a clinical trial, yet they’re still deeply affected by how those trials are designed and conducted. While it’s critical to protect participants, we also need to ensure that studies are built to efficiently deliver better, safer treatments to the broader patient population.

Patients waiting for new therapies care about:

• How quickly treatments reach the market
• Whether new therapies are safer or more effective than existing options
• How many new treatments become available
• Whether those treatments will be affordable and accessible

Efficient and innovative trial designs are key to meeting these needs. This includes approaches that improve dose and treatment selection, reduce time to approval, enable studies in rare diseases, and lower development costs—thereby improving access and outcomes for all.

This broader patient-focused perspective has gained some attention in recent literature (Antonijevic et al., 2021; Beckman et al., 2022; McMillan et al., 2022; Huml et al., 2023), but it’s still far from mainstream.

If we want to create truly patient-centered research, we need to expand the conversation. Patient advocates and study teams must collaborate not only on reducing burden for trial participants—but also on shaping designs that drive progress for all patients awaiting better care.

Table 1. How Innovative Trial Designs Benefit Patients

The table below, adapted from Antonijevic et al. (2021), summarizes the many ways that innovative designs can directly benefit patients—both within and outside of trials. These include faster access to treatments, more appropriate therapies, improved options for rare diseases, and better affordability.

Source: Antonijevic et al. (2021)

A Smarter Path for Sponsors

While designing trials to benefit all patients is clearly in the public’s interest, it also offers tangible advantages for sponsors.

Sponsors who adopt an “all-patient-focused” design approach benefit in several key ways:

• Faster, more predictable recruitment
  Broadening eligibility criteria and designing studies that reflect real-world patient needs can improve recruitment timelines and reduce dropout.
• More meaningful data for regulators and payers
  Inclusive trial designs generate evidence that better supports market access and long-term reimbursement success.
• Better decision-making
  Adaptive and master protocols provide earlier insights and ability to make decisions in a broader context, enabling faster “go/no-go” calls and smarter resource allocation.
• Lower development costs
  Innovative designs reduce trial size, enable shared control arms, and eliminate redundant steps—driving down overall development spend.
• Accelerated time to market
  Streamlined trials that reflect patient reality shorten the path to approval, maximizing both impact and return on investment.

When sponsors design with the entire patient population in mind, they’re not just advancing patient care—they’re also unlocking more efficient, scalable, and competitive development strategies.

“Patient-focused trial design isn’t just the right thing to do—it’s the smart thing to do.”

Conclusion: Designing for Impact

By expanding our focus beyond trial participants and toward all patients awaiting treatment, we create trial designs that are more ethical, more efficient, and more impactful.

Innovative clinical trial designs represent a unique opportunity for alignment between patient needs and sponsor success. When study teams and patient advocates work together to shape trials that serve everyone, the result is a research ecosystem that moves faster, costs less, and delivers more meaningful therapies to those who need them most.

Resources

Antonijevic Z, Liu Y, Tang R, Huml J, Beckman RA, Mayer C, McMillan G. Patient Benefits from Innovative Designs in Rare Diseases. Rare Disease Drug Development: Clinical, Scientific, Patient, and Caregiver Perspectives. RA. Huml. ed., Springer Nature, Cham Switzerland; 2021 pp 147-160.

Beckman RA, Antonijevic Z, Ghadessi M, Xu H, Chen C, Liu Y, Tang R. Innovations in Clinical Development in Pediatric Rare Diseases: Small Patients and/or Small Populations. Peadiatric Drugs. 2022 Nov; 24(6):657-669. doi: 10.1007/s40272-022-00538-7

Huml RA, Collyar D, Antonijevic Z, Beckman RA, Quek R, and Ye J. Aiding the adoption of master protocols by optimizing patient engagement. Therapeutic Innovation and Regulatory Science, 2023 57: 1136-1147.

McMillan G, Mayer C, T R, Lu Y, LaVange L, Antonijevic Z, and Beckman, RA. Planning for the next pandemic: ethics and innovation today for improved clinical trials tomorrow. Statistics in Biopharmaceutical Research, 2022 14: 22-27.