From Rare Revolution Magazine- The co-design revolution: how empowering patients is the future of clinical trials

Defining co-design: beyond the feedback form

One of the most persistent misconceptions discussed was the difference between simply gathering patient feedback and genuine co-design. Wes Michael argued that the distinction is fundamental to the philosophical approach of drug development. Co-design is not “consultative”, where industry listens to opinions and then proceeds with its existing plan. Instead, it is rooted in a powerful concept:

 “People support what they help create…It’s about collaboration. It’s about a two-way street. It’s about engaging patients, not just getting their opinions.”

-Wes

The core principle is taking patients and caregivers seriously and treating their lived experience not as anecdotal input but as core expertise for creating a workable trial. Wes highlighted the risk of industry viewing patient input as a “checkbox”, an administrative task rather than an integrated part of the design. This superficial approach fails to build the necessary buy-in and trust required for trials to succeed.

Siloed advocacy and low awareness

The current relationship between patients and trial designers, particularly in rare disease, is characterised by significant gaps. Mindy Cameron cited startling statistics from a Rare Patient Voice survey: of over 2,000 respondents, 69% were unaware that co-design even exists, and only 4% had participated in any co-design activity. This points to a massive deficit in industry outreach and communication.

Mindy identified a key internal barrier within sponsor companies: the “siloing of the patient advocacy function”. Too often, patient advocacy teams are relegated to patient programmes, events and “free giveaways at conferences”, rather than being strategically integrated into the core clinical development process.

 “Stop siloing your patient advocacy teams and get them involved, because they are the ones that are talking to your patients…and they are learning what the patient experience is like, and that is foundational to clinical trial design.”

– Mindy

The misconceptions held by researchers and sponsors often centre on viewing patient expertise as “singular”. Mindy countered that in the digital age, a patient is an expert whose input reflects the collective experience of their entire community. This patient input is highly “nuanced”, depending on disease progression, geography, access to healthcare and socioeconomic factors. Critically, Mindy emphasised that there is “nobody more motivated than a patient or a caregiver to help you with your clinical trial design. No one.” They are quick learners in a complex ecosystem, driven by the highest possible stake.

Meaningful involvement

What does meaningful co-design look like in practice? Both experts agreed: start early. Wes stressed that drug developers are “never too early to start working with patients”. Early engagement ensures that a trial’s design is not just scientifically sound, but humanly feasible.

The discussion highlighted a critical finding from Rare Patient Voice’s survey on what patients prioritise for influence in trial design. Surprisingly, it was not the scientific endpoints or inclusion criteria, but the practical, everyday matters:

 “The most important thing is support services for the participant. An everyday, practical thing. How am I going to get there? Who’s going to take care of my kids while I’m there? You’ve got to be practical. How is it changing their life, and how can we make it work for them?”

– Wes

This insight underscores a central theme: clinical trial participants are “whole people” with lives to live, not just patients with a disease. Failing to account for logistical and financial burdens is the fastest way to undermine recruitment and retention, regardless of a trial’s scientific merit.

Mindy corroborated this, detailing her own family’s experiences with Duchenne muscular dystrophy. She recounted instances where trial sites were physically incompatible with her son’s level of disability. Furthermore, she targeted patient-reported outcome (PRO) questionnaires, which are often required to be filled out in the clinic, documenting decline in an “upsetting, very emotional” setting. This practice, often rooted in “this is the way we’ve always done it”, is an unnecessary burden that co-design could easily alleviate by allowing home completion. She also pointed to the invasiveness of mandatory procedures like biopsies, which deter participation if not kept to a minimum.

Navigating the “hope” narrative

A common industry pushback against early engagement is the fear of providing “false hope” to a community, especially when a therapy is still investigational. Wes called this a justification for inaction at any stage, noting that drugs can fail even in Phase 3.

Mindy offered a deep, patient-centered reframing of the concept of hope:

“Hope is a commodity…if it weren’t for hope, a lot of patients with these devastating conditions wouldn’t be able to go on, and it is something that is central to our lives, hope.”

– Mindy

She stressed that even failed studies provide comfort, provided the participation “moved the field forward or contributed to the body of scientific knowledge”. The key, according to Rebecca, is communication—being transparent and using plain language to convey that the trial is early and investigational. The presence of research itself, Wes added, sends a positive message: “you want to know that people are paying attention to it, no matter how rare it might be.”

Dismantling invisible barriers to participation

To achieve truly inclusive and successful co-design, several “invisible barriers” must be dismantled. RPV’s survey pointed to three critical factors needed to encourage participation:

explanation of the process: ensuring all materials are in plain language for “any and all patients and their caregivers”.

flexible scheduling: accommodating the practical reality of people’s lives through options like remote participation and flexible time frames.

compensation for their time: compensating participants, which is standard in market research, challenges the flawed notion that payment would “bias the result” in clinical settings. The compensation is seen as fair recognition for the time and disruption involved in participating.

Mindy added two further critical points for inclusivity:

language and diversity: ensuring information is available in patients’ first languages is “hugely important”. Failing to look at the broad range of the patient population—including race, ethnicity and income—means missing “very important pieces that may come back later…and cause big problems”.

income and financial incentives: Mindy highlights the complication of financial incentives for patients on disability who are bound by strict income limits. Companies must be flexible and find “another way for them to participate, or another way to compensate them” that respects these regulations.

The discussion also addressed the challenge of “hard to reach” patients, who are often not connected to the largest advocacy groups. Wes asserted that his company has spent 12 years successfully finding these people by “going to where they are”—attending local walks, conferences and utilising patient-to-patient referral programmes. Mindy noted that “nothing will bring out patients like an approved therapy”, which subsequently expands the pool of patients interested in research.

The blueprint for co-design success

To conclude the ideal co-design partnership, Mindy offered a concise three-point blueprint for industry:

Start early: engaging patients from the inception of an idea.

Pragmatic design: structuring a trial to alleviate patient burden, ideally by mirroring standard clinical care requirements.

Data return: providing patients with their data and explaining the results of the trial (how their participation mattered) once it is over. Mindy views this as essential for patients to make informed decisions about future care. This is her “soapbox”, emphasising the need for better “long term follow up and result sharing”.

“The number one reason patients don’t participate in clinical trials is they’re hard and they take a lot of time. So if we can arrive at a consensus on how we can share enough data during a clinical trial that alleviates the burden on the care and the trial side, that would be a huge measure of success.”

– Mindy

The conversation crystallised that the true value of co-design lies not in what is decided (the scientific endpoints), but in how the decisions are made—the process. As Wes stated, “it gets to the right way of going about doing it, and trust that that process will lead to a better result.” The area “crying out for insight” is not the endpoint itself, but the protocol—the everyday logistics that determine whether a patient can participate and remain engaged. This is crucial for improving notoriously slow trial enrollment. Mindy further highlighted the growing need for patient involvement in long-term follow-up studies to ensure patient engagement for therapies with multi-year outcomes.

To fully realise the promise of patient-centric drug development and address the challenges of slow enrollment, high dropout rates and misalignment with patient needs, the industry must take immediate, actionable steps to transition from consultation to true co-design.

Integrate patient advocacy at inception: sponsors must move the patient advocacy function out of its silo. Patient engagement teams are not merely marketing or event planners; they are intelligence officers. Their expertise must be integrated into research and development from the moment a therapeutic target is first considered. The initial responsibility for patient engagement rests firmly with the sponsor.

Prioritise pragmatic trial design: shift the focus of protocol design from purely scientific metrics to real-world feasibility. Every procedural step (site visit, blood draw, PRO questionnaire, invasive procedure) must be vetted with a diverse patient group. Invest resources into flexible scheduling, remote options, data sharing with care teams to avoid redundant testing and, most importantly, provide fair compensation for time and effort.

Embrace transparency and data stewardship: commit to early, plain-language communication regarding the investigational nature of a therapy to manage the hope narrative responsibly. Furthermore, industry must implement robust systems for data return and results-sharing post-trial. Giving patients their data back and demonstrating how their contribution advanced scientific knowledge is a non-negotiable step to building long-term trust, participation and a sustainable research ecosystem.

Seek out new avenues to connect to patients: actively overcome “hard to reach” barriers. This requires investment in outreach beyond major conferences and national advocacy groups. Utilise diverse channels—regional events, social media networks and grassroots referrals—to ensure co-design reflects the broad spectrum of the rare disease community, including all geographic, socioeconomic and linguistic groups.

Co-design is not an optional ethical veneer; it is a critical business strategy. By collaborating with patients to create trials that are easier, more practical and less burdensome, industry will unlock faster enrollment, higher retention and, ultimately, a better chance of developing a transformative therapy that truly improves the lives of rare disease patients.

About Rare Patient Voice

Rare Patient Voice, LLC provides patients and family caregivers an opportunity to participate in all types of research including market research, health economics outcomes and real-world evidence, user experience/human factors studies and clinical trials. The RPV community includes over 185,000 patients and family caregivers across more than 1,500 diseases, both rare and non-rare, in nine countries. Visit us at www.rarepatientvoice.com.