Myth #1: “I’ll Just Get a Placebo”

One of the biggest misconceptions patients have is the fear of getting a placebo, which many equate to receiving “no treatment at all.” Tina shared that the fear of placebo is a huge barrier for patients considering trials.

“Patients think it’s potluck: Do I get the treatment, or do I get a placebo? They don’t realize it’s not just about one or the other. Trials are usually structured to ensure they’re still getting the standard of care if they’re not receiving the investigational treatment.”

In reality, most trials are designed to include either the experimental treatment or the best available standard of care—rarely just a sugar pill. Patients aren’t left without options, and this misunderstanding often keeps them from accessing the potential benefits of cutting-edge care.

Myth #2: Randomization Is Unfair

For patients, the word “randomization” can feel unsettling. It’s easy to see why: it implies a lack of control in an already vulnerable situation. Tina’s response to this was direct and insightful:

“Patients don’t always understand the randomization process. Some even think their worsening symptoms are tied to whether they got the placebo or not. But in reality, many trials are designed to allow patients access to the investigational treatment if it’s effective, even after the placebo phase.”

As professionals, we must clarify that randomization is a scientific tool designed to eliminate bias, not an arbitrary gamble. It ensures the reliability of trial results, which in turn benefits patients long-term.

Myth #3: Trials Offer Little Care

A common misconception is that clinical trials prioritize data collection over patient care. However, the opposite is often true. Tina shared her experience of receiving exceptional care during her trial participation:

“I was being seen weekly. Granted, it was a lot on me to have my mother drive me two hours each way into New York City, but there was solace in the fact that I had such robust care,” she explained.

For patients with limited access to healthcare or specialists in their disease, the care provided during a clinical trial can be a welcome and much-needed resource. Trials often offer access to tests, monitoring, and resources that help patients better manage their disease. This level of proactive monitoring goes far beyond what most patients receive in routine care. Clinical trials are meticulously designed to safeguard participants’ health, ensuring frequent check-ins and faster response times. This robust care model is a strong counterpoint to fears that trials are impersonal or risky.

Myth #4: Trials Are Only for Severe Cases

Many patients believe that clinical trials are exclusively for those with advanced or severe disease, reinforcing the misconception that trials are a 'last resort.' However, trials often include participants with mild-to-moderate conditions to evaluate early intervention strategies. Tina highlighted the importance of engaging these patients earlier:

"No provider ever told me early on that considering a clinical trial could make a huge difference before my disease became more complex. We need to educate patients about these options much earlier in their journey."

This 'last resort' mindset keeps patients from exploring options that could prevent their disease from progressing to more severe stages. By informing patients and healthcare providers that trials aren’t just for the most critical cases, we can expand access to innovative treatments and improve outcomes.

Myth #5: Clinical Trials Are Inaccessible

Another common misconception is that clinical trials are geographically or financially out of reach. However, decentralization and virtual models are changing the game. Tina shared her perspective:

"Decentralized trials gained traction during the pandemic, and they’re a game changer. If I could have done telehealth visits or had a nurse deliver the medication to my home, it would have been a completely different experience."

Decentralized trials and virtual options reduce logistical barriers, but access gaps still remain. Local trial sites and community-based recruitment can further ensure trials are within reach for diverse populations.

Education Is the Missing Piece

The real issue isn’t the placebo or randomization itself—it’s the lack of education about how these elements work. These myths often have roots in cultural narratives, media portrayals, and historical missteps by researchers and pharmaceutical companies. Distrust stemming from unethical studies in the past, sensationalized media stories, and a lack of transparency in trial processes has compounded the fear and misunderstanding patients feel today. Tina emphasized the importance of involving clinical research coordinators and patient advocates to rebuild trust and break things down for patients:

“Patients need to understand how the trial works, what their rights are, and what guarantees they have. Without that, fear and mistrust will continue to hold them back.”

We can’t assume patients will just get it. Clear communication and resources co-created with patient advocates can make a world of difference in how trials are perceived.

Take Action: Patient-Centric Communication

If there’s one thing I’ve learned from Tina’s advocacy, it’s this: clinical trials must be about more than just advancing science. They must advance trust. For those of us in the pharmaceutical and life sciences fields, this means creating trial designs and communication strategies that prioritize patient understanding and empowerment.

When we demystify the process—placebos, randomization, and all—we unlock the potential to truly partner with patients in advancing medicine. And isn’t that why we’re here in the first place?

If you found this discussion insightful, don’t miss my full conversation with Tina on the Merakoi Voices podcast. It’s packed with even more powerful stories and actionable ideas to transform how we approach clinical trials.

About Tina Aswani Omprakash

Tina Aswani is a passionate patient advocate, founder of the South Asian IBD Alliance, and a globally recognized voice in the IBD community. With years of experience navigating chronic illness, she works tirelessly to bridge the gap between patients and clinical research, advocating for diversity, equity, and patient-centered care in trials.

About Merakoi

At Merakoi, we're passionate about harnessing the power of mini-communities to bridge the information gap and empower patients to take control of their health journeys. By fostering ongoing collaboration between patients, healthcare providers, and pharma companies, we're creating a future where every patient has access to the knowledge and support they need to thrive.

Together, we can build a world where no patient is left in the dark, searching for answers. If this sounds like the kind of healthcare innovation you want to participate in, let's chat!

Episode Overview

Join us for an insightful conversation with Ella Balasa, a patient expert and advocate with extensive experience in clinical trial co-design. This episode explores how early patient involvement can transform clinical research and development, building on Merakoi's commitment to patient-centered healthcare innovation.

Guest Bio

Ella Balasa - Patient expert and advocate specializing in clinical trial co-design, with a unique background combining microbiology lab experience and personal patient journey with cystic fibrosis.

Key Topics

• The evolution from patient advocate to healthcare consultant
• Building trust in underrepresented communities
• The importance of grassroots engagement in clinical trials
• Transforming the patient role from subject to partner
• Strategies for early patient involvement in trial design

 "It's not that patients are just in the center... we are sitting around the table with everybody else. We're a piece of this ecosystem."

Main Takeaways

1. The critical importance of human-to-human relationships in building trust
2. Why early patient engagement matters in trial design
3. How empathy drives better healthcare solutions

Related Resources

"From Patient to Partner: Transforming Clinical Trial Experiences Through Co-Design"

"The Patient Left in the Dark: My Story of Unanswered Questions" 

About Merakoi
At Merakoi, we're passionate about harnessing the power of mini-communities to bridge the information gap and empower patients to take control of their health journeys. By fostering ongoing collaboration between patients, healthcare providers, and pharma companies, we're creating a future where every patient has access to the knowledge and support they need to thrive.

Together, we can build a world where no patient is left in the dark, searching for answers. If this sounds like the kind of healthcare innovation you want to participate in, let's chat!

Ashley's journey through a clinical trial for atopic dermatitis was far from ideal. As a patient living with this condition, she bravely decided to participate, hoping to contribute to the development of new treatments and improve the lives of others like her. However, her experience fell short of expectations. Feeling unprepared and unsupported, Ashley's clinical trial left her feeling like just another number, rather than a valued partner in the process.

In her own words, Ashley shared, "I remember just through my clinical trial experience, it ended very abruptly, and I didn't know what my next steps were or how I was going to continue going on this medication. And so that left me, you know, feeling very isolated and alone and like, wow, I really did just feel like a number in this clinical trial."

This disappointing reality is one that many first-time clinical study participants face, leaving them feeling disconnected and unappreciated. Likewise, many study teams are nervous about the implications of treating patients like just another data point. They ask important questions about the risks this brings in failing to recruit patients for their study, losing patients to dropoff in the middle of the trial, or not winning consent for important substudies and data sharing that are essential parts of today's complex studies. How does treating patients like numbers cloud our views of how they experience trials, who should be in control, and how we should we relate to them?

Reimagining Ashley's Clinical Trial Journey

In an ideal world, Ashley's clinical trial experience would have been a far cry from the disappointment and isolation she felt. Instead of feeling like just another number, she would have been welcomed as a valued partner in the research process from the very beginning.

1. Upon expressing interest in participating, Ashley would have received a warm invitation to join the trial, along with easy to understand and comprehensive information about what to expect. The study team would have taken the time to understand her unique needs, concerns, and preferences, ensuring that the trial design was tailored to her specific circumstances.
2. As Ashley embarked on her trial journey, she would have been treated as a true superhero – a courageous individual willing to contribute to the advancement of medical research for the benefit of herself and countless others living with atopic dermatitis. This recognition would have been woven throughout every interaction, from the personalized welcome pack she received to the genuine appreciation expressed by the study staff.
3. Throughout the trial, Ashley would have felt supported, informed, and empowered. Regular check-ins with the study team would have provided opportunities for her to ask questions, share her experiences, and receive updates on the trial's progress. These interactions would have been characterized by transparency and a genuine commitment to her well-being.
4. The trial site itself would have been designed with patient comfort and convenience in mind. From the moment Ashley stepped through the doors, she would have been greeted by a warm and inviting atmosphere, with comfortable waiting areas, complimentary refreshments, and a range of accessible amenities. Transportation assistance would have been offered to ensure that participating in the trial was as seamless and stress-free as possible.
5. Throughout her journey, Ashley would have had access to a wealth of educational resources and support services. From informative brochures and videos to peer support groups and one-on-one counseling, she would have been empowered to take an active role in her own health journey. These resources would have not only enhanced her understanding of the trial process but also fostered a sense of connection and community with other participants.
6. As the trial progressed, Ashley's contributions would have been continuously acknowledged and celebrated. Small gestures of appreciation, such as personalized thank-you notes and milestone celebrations, would have reinforced the value of her participation and the importance of her role as a patient superhero. These moments of recognition would have served as a powerful motivator, encouraging Ashley to stay engaged and committed to the trial.
7. Upon completion of the trial, Ashley would have been given the choice to receive the study findings in an easily comprehensible way, which she could then share with her community if she desired. Not only would this help disseminate the study findings, but it would also encourage conversation about the trial and position the sponsor in a positive light due to the overall positive experience.

For Ashley, this reimagined clinical trial experience would have been a world apart from the abrupt and isolating reality she faced. Instead of feeling abandoned and uncertain about her future, she would have emerged from the trial feeling valued, supported, and empowered. Her journey would have been an example of what is possible when patients are placed at the heart of clinical research, and when their needs and experiences are given the attention and respect they deserve.

Patients as Partners: The Key to Successful Clinical Trials

Ashley's story highlights the stark contrast between a traditional, patient-agnostic trial experience and the potential of a patient-centric approach. By reimagining her journey, we can see the potential of empowering patients as partners in the clinical trial process.

But how can we make this vision a reality? The answer lies in embracing co-design and patient mini-communities. By bringing together diverse groups of patients who are a good fit for a specific trial, researchers can gain valuable insights at every stage of the design process. Mini-communities allow for open and honest conversations about the challenges and concerns patients face, enabling study teams to create trials that are more engaging from enrolment through to completion.

Imagine how different Ashley's experience could have been if she had been part of a mini-community for her atopic dermatitis trial. As sorts of permutations are possible in co-design, but a simple two cohort approach would suffice. As a first-time trial participant, Ashley would have been invited to a cohort of trial inexperienced patients. By sharing her insights and collaborating with other patients, she could help shape the trial design to better meet the needs and expectations of participants and, in particular, addressing barriers to enrollment. Meanwhile, a second cohort would have consisted of AD patients with previous clinical trial experience. This cohort enables the study team to build on the real-life learnings of previous studies, reducing the risk of repeating others' mistakes and integrating opportunities to improve the study.

Through this co-design approach, the mini-community would enable a more comprehensive understanding of patient needs, ultimately resulting in a trial that is more engaging, inclusive, and effective. Ashley and her fellow participants would feel valued and heard throughout the process, knowing that their insights are making a real difference in the trial design. I know these are real benefits, and to be clear, when I say patients should be "partners," "co-designers," "collaborators," or "experts," I'm speaking metaphorically. Most patients don't have the scientific knowledge, regulatory understanding, or clinical expertise of researchers. So why take the risk? Because as imperfect as the analogy is, working with patients is easiest if you truly think of them as equal partners rather than passive participants or validators of your opinions. 

As a patient experience strategist, I have seen firsthand how pharmaceutical teams benefit from involving patients as partners in various aspects of their work. By collaborating with patients, teams can gain valuable insights into the patient journey, identify unmet needs, and develop solutions that truly resonate with the patient community. Even teams with previous experience working with patients gain value with every round of patient collaboration, as patient needs are continually evolving in tandem with our own understanding of diseases and the treatment landscape.

Embrace Mini-Communities for Your Next Study

To all the teams running clinical trials: it's time to take patient engagement to the next level. Don't just involve patients; embrace the power of mini-communities and co-design. By partnering with Merakoi and their innovative mini-community approach, you can unlock the full potential of patient insights and create trials that truly prioritize the needs and experiences of those who matter most.

To learn more about patient co-design in clinical trials, I recommend reading Dani Benson's insightful blog post, Unleashing Patient Power: Co-Design for Better Trials . Her guide explores the various aspects of patient involvement in clinical trial design and provides valuable insights for teams looking to adopt a more collaborative approach.

Contact us to discuss how to incorporate the voice of patients into your clinical trials.

About Merakoi
Merakoi partners with health and life sciences companies to build mini-communities that guide product development through continuous user insights. Our network of patients/advocates and proprietary community platform enable engaging, longitudinal co-creation between users and developers. The result is human-centered solutions that resonate powerfully in the real world.

Related reading: What Is Patient Co-Design?

Imagine a world where clinical trials prioritize the needs of patients, not solely focus on testing new treatments. Co-designing with patients should be the starting point for clinical trial design, but sometimes their voices get lost in the shuffle. As the people who stand to benefit the most from new therapies, patients have a lot to say about what works and what doesn't. That's why it's so important to involve them from the very beginning.

When we invite patients to be active co-designers in clinical trials, we're doing more than just checking a box. We're recognizing that their experiences, opinions, and concerns are valuable and can shape the way a trial is run. By having open and honest conversations with patients, we can tackle issues that might make it tough for them to join or stay in a study. This could be anything from transportation problems to worries about the trial procedures or unknown results. By understanding each patient's perspective, we can make the trial experience better for everyone. When patients feel good about participating, it can lead to faster enrollment and better retention throughout the study.

But patient engagement is about more than just making trials run smoothly. It's also about building trust and creating a shared goal of improving healthcare. When patients feel like their voices are heard and valued, they're more likely to become champions for clinical research in their communities. This can greatly impact how the public sees clinical trials, breaking down misconceptions and encouraging more people to get involved.

Redefining Insights: Embracing Next-Generation Methodologies

Traditional market research and advisory board methods have long been the foundation of insight gathering for clinical trials. However, as technology and data rapidly evolve, it's crucial to recognize the limitations of these approaches and explore next-generation methodologies.

Market Research:

• Expensive and time-consuming
• One-off, snapshot views of patient experiences
• Limited ability to iterate and adapt based on ongoing patient feedback

Advisory Boards:

• Stale and commoditized audiences
• Lack of genuine diversity and representation of trial-specific target populations
• Tendency for the same voices to be heard repeatedly

While these methods have served us well, they may not be leveraging the full potential of advances in software, big data, and AI. It's time to embrace new approaches that address these limitations and unlock the power of continuous patient insights.

Imagine a solution that offers:

• Cost-effective, iterative processes
• Ongoing engagement with diverse patient segments
• The ability to shape and validate designs for specific patient populations

By adopting next-generation methodologies, we can revolutionize the way we conduct clinical research, leading to more effective, efficient, and engaging trials. 

Mini-Communities: Your Direct Line to Patient Insights

So, how can we make patient engagement easier and more effective? Enter the idea of mini-communities for your clinical programme. At Merakoi, our mini-communities bring together diverse patients who are the perfect fit for a specific trial, giving researchers a direct line to the people who matter most.

Let's say a research team is planning a trial for a new treatment for multiple sclerosis (MS). They could create a mini-community of MS patients with different experiences and backgrounds. This could include patients who have participated in trials before, those who are new to the process, and even patients from different geographic regions or with different types of MS.

By engaging with this mini-community, the research team can get valuable insights at every stage of the trial design process. They can ask patients for feedback on the trial protocol, making sure it's feasible and patient-friendly. They can test different recruitment materials and strategies to see what resonates best with patients. And they can even involve patients in designing the trial itself, getting their input on everything from the number of visits required to the types of assessments used.

Cohorts: Unlocking the Power of Segmentation

One of the most powerful features of mini-communities is the ability to create cohorts, or subgroups, within the larger community. In our MS trial example, the research team could create cohorts based on patients' previous trial experience. They could compare insights from trial-savvy patients to those who are new to the process, identifying potential barriers and concerns for each group. This information can help the team tailor their approach to meet the needs of different patient populations.

Cohorts can also be used to gather insights on specific aspects of the trial experience. For example, the research team could create a cohort of patients who have used wearable devices in previous trials. By learning about these patients' experiences and preferences, the team can make informed decisions about incorporating wearables into their own trial design.

The value of cohorts in patient engagement cannot be overstated. By segmenting patients based on key characteristics or experiences, study teams can gain a more nuanced understanding of patient needs and preferences. This, in turn, allows for more targeted and effective trial design, ultimately leading to better recruitment, retention, and overall trial success.

Case Study: Enhancing a Skin Disease Trial through Patient Mini-Communities

Recently, I had the opportunity to work with a global clinical study team on enhancing a skin disease trial. As someone who has seen firsthand the challenges of patient recruitment and retention, I was excited to put the power of mini-communities and cohorts to the test.

We began by creating a mini-community of patients with the specific skin disease. Within this community, we established three main cohorts: patients who had previously participated in clinical trials and those who were new to the process, as well as a panel of patient advocates and expert. This allowed us to compare insights from trial-experienced patients with those who were trial-naive, giving us a more comprehensive understanding of patient perspectives.

One of the key issues we uncovered through our interviews with these cohorts was the impact of certain invasive procedures, such as skin biopsies, in the trial protocol. Trial-experienced patients were able to share their past experiences and concerns, while trial-naive patients expressed apprehension about these procedures. Patient advocates, though not necessarily the target population for the study, provided vital information on ensuring informed consent. By listening to these groups, we were able to work with the study team to refine the protocol, making it more patient-friendly without compromising scientific integrity.

Another crucial aspect of our mini-community was its ethnic and geographic diversity. We made sure to include patients of different skin colors and from various national healthcare systems. This diversity proved invaluable, as we discovered that patients with darker skin tones often faced delays in diagnosis due to the difficulty in visually identifying symptoms. Patients from countries with more robust healthcare systems reported having better access to information and support networks, while those from regions with less developed infrastructure struggled to find the help they needed. This knowledge allowed the study team to tailor our recruitment materials and patient support programs to better meet the needs of patients from different backgrounds.

For patients in the mini-community around this clinical study, the experience is extraordinarily rewarding:

"You have to make the process comfortable for participants. They're spending their time doing this not only to help themselves but really to advocate for others because there's a lot of people that have this condition that don't have a voice. And what we are trying to do is help them come out of that. They don't have to be an advocate themselves, but they should advocate for themselves at least. And that's so important. You want the process to be smooth sailing and change from the way it has been, and it can change."

Ashley Wall, Merakoi Patient Expert

At the end of the day, a successful clinical trial isn't just about the scientific results. It's also about making a real difference in patients' lives. By putting patients at the center of the process and working together to design better trials, we can unlock the full potential of clinical research. This means faster development of life-changing treatments and a brighter future for healthcare, one trial at a time.

For a comprehensive guide on patient engagement in clinical trial co-design, check out this valuable resource from PFMD: Patient Engagement in Clinical Trial Protocol Design (Merakoi is a contributor)

Ready to change the way we do clinical research? Contact us for more information about mini-communities.

About Merakoi
Merakoi partners with health and life sciences companies to build mini-communities that guide product development through continuous user insights. Our network of patients/advocates and proprietary community platform enable engaging, longitudinal co-creation between users and developers. The result is human-centered solutions that resonate powerfully in the real world.

Related reading: what patient co-design really means

Today, May 20th, we celebrate International Clinical Trials Day, a commemoration of the day that James Lind laid the foundation for modern clinical research in May 1747.

At a time when scurvy was thought to have killed more British seamen and sailors than French and Spanish armies combined, surgeon’s mate James Lind conducted what many refer to as the first clinical trial. Lind selected 12 scurvy-stricken men from the ship. They were divided into six pairs, with each group receiving a different addition to their basic diet.  Some were given cider, others seawater, or vinegar. And the last group, oranges, and lemons,  Those fed citrus fruits experienced, in the  words of Lind “the most sudden and good visible effects.”

Fast forward to today, and the conduct of a clinical trial is a rigorous process involving multiple phases, from preclinical to real-world evidence  after the intervention is approved by regulators.  Clinical trials and their various phases constitute a vital part of clinical research. In this way, new drugs and treatments can be evaluated for safety and effectiveness before they can be approved for general use.

In early phase development, understanding the patient target population requires a coordinated effort to design a trial that will lay the foundation for later-stage success. Timely recruitment of patients is widely recognised as an essential component of successful clinical trials.  However, the clinical research industry faces a significant problem with delays in patient recruitment. Current estimates show that 80% of clinical trials miss their patient enrollment deadlines. In turn, this impacts how quickly new medicines can reach the patients who need them.

In order to better understand the knowledge and attitudes of patients towards early phase clinical trials, we conducted a survey among our patient experts. 

Q1: Are you aware of / do you understand the different phases a drug must go through before it is approved for use?

The majority of survey respondents (76%) understand the different phases a drug must go through before it is approved for use.

Q2: Have you considered or been offered the opportunity to take part in an early phase trial?

Just 18% of respondents have been offered the opportunity to take part in an early phase trial. When asked where they had heard about the trial, respondents mentioned the following sources:

• E-mail
• My consultant (for chronic condition)
• We contacted researchers regarding our ideas
• At the hospital before treatment
• I am an advocate on a Phase I Trial for a preventive breast cancer vaccine. I have supported this research for twelve years and it is finally in process.
• The hospital I attend

Q3: What motivated you to consider/take part in an early phase clinical trial?

Respondents provided the following reasons for taking part: 

• To serve the public
• To help others/better healthcare for me/interesting/trust in the team/potentially better health for longer (degenerative condition - might delay decline)/the 'right thing to do' for people with my condition
• To investigate real needs
• I believe in preventive cancer vaccines - primary prevention of cancer
• It was an 'add on' drug, and it was felt I needed further treatment to prevent my condition deteriorating.

Q4: Are you aware that participation in early phase trials is not just about chemotherapy?

A majority of respondents (79%) knew that participation in early phase trials can give access to interventions beyond chemotherapy.

Q5: What are some of your main concerns about participating in early phase clinical trials?

When asked to elaborate on concerns regarding their participation in early phase clinical trials, respondents gave the following reasons:

• Too many exams
• I might not hear about all of the relevant ones I might be eligible for - and so only being offered a selection (I don't want to 'waste' my data).
• If not designed with patients in mind, then how do I trust the team to know the side effects/outcomes/protocol bottlenecks that are relevant to me? 
• Potentially future damage or worsening condition. 
• Potentially, intervention is not useful or damaging. 
• Not being told of the outcome
• That you’re taking a gamble against treatments proven to deliver good outcomes.
• Placebos
• That there is not enough data and we don't know the long term effects of the drug. The potential long term effects could be significant.

 Q6: How might your concerns be addressed?

Finally, we asked respondents how these concerns could be addressed to allay patient fears.

• Inclusion of patients from the very beginning
• Hearing from others, stats on clinical trial outcomes.
• Making the real drug available once proven effective to all those in the trial
• Only with time.

“I have taken part in several Phase I early Phase II trials - but only if I can be a 'member of the team' and kept fully up to date. I WILL NOT take part if I am just considered a human-shaped lab rat. So! Teams! Get your act together, involve patients EARLY and work out how to be clear about your trial, the side effects, why it is important (Is it important, or just a PhD/vanity project??) and keep us up to date at all times. If you don't make it easy to be part of, why should I jump through hoops to get on board?” - - - anonymous survey respondent

Optimising early clinical trials involves distinct phases focused on understanding patients, the target populations, their needs, and how to reach them, including better education and  ongoing and pertinent communication.  Sometimes programnes that understand these fundamentals are needed for patient behavioural change if retention or recruitment issues already exist.

Conclusion

Critical factors to consider:

1. Inclusion and exclusion criteria 

Generally, clients work with KOLs and investigators to finalize the target patient population and trial objectives for early trials in order to move the trials quickly to the next phase—the "fail and fail fast’ attitude. Budgetary allocations rarely cover patient input. This can be a costly mistake in delaying the overall clinical development program. Direct validation with patient experts and understanding the emotional and practical aspects of considering or participating in a trial—such as choices, attitudes toward participation, risks, and fears—is another aspect that clients will not gain unless they also hear directly from patient experts and their diverse patient networks. 

2. Supporting the clinical trial sites with their patient outreach 

We must consider ways to increase awareness of early phase clinical trials - where do patients go to find out about all the ongoing trials, and what are the pain points (barriers to entry) for them?

3. Getting the messaging right

To maximise recruitment for an event like this, all trial touchpoints need to be validated and thought through, including how to describe trial materials and patient participation materials to ensure maximum uptake and retention. It will help patients and caregivers recognise that this is for them or someone close to them.

So if you are embarking on a  early phase clinical trial and you want to hear more about involving patient experts to ensure success,  get in touch with merakoi through Helen Hey.

Equally, a patient’s journey can involve a lot more than just medical diagnosis and standard interventions.   An early decision point on the patient journey, particularly when receiving a serious diagnosis, is whether to participate in a clinical trial.  The bridge between patients and a clinical trial is the Informed Consent Form (ICF).  The ICF provides potential clinical trial participants with the information they need to decide on whether to volunteer for a research study.

 As the name suggests, there are two parts of the ICF, Informed and Consent.

According to Webster’s Dictionary. 

• Informed is having or showing knowledge of a particular situation; a decision or judgment based on an understanding of the facts of the situation

• Consent is giving permission for something to happen or an agreement to do something

Simply put, the ICF is meant to ‘inform’ the participant about the clinical trial and the patient provides his or her ‘consent’ to participate in the trial as described by signing the ICF.

Essentials Elements of the Informed Consent Form

There are three major elements of an ICF – disclosure of information (inform), competency of the participant or surrogate to make a decision (consent) and the voluntary nature of the decision (consent).  But let’s take a closer view through the patient lens.

It is critical for patients to understand the proposed clinical trial.  The ICF typically provides the potential participant with a description of the clinical investigation.  It outlines potential risks and benefits of the treatment and highlights alternative treatments that may be available beyond the proposed trial.  It covers the schedule for treatments and additional testing or scan requirements.  Usually, the primary investigator or other medical staff review the contents of the ICF and ask the patient if they have any questions.  But is this review enough?

I have reviewed many ICFs in my role serving on internal patient councils with pharmaceutical companies.  The documents are often overly technical scientifically and written with a heavy legal tone.  It is difficult for most patients and caregivers to understand.  After all, few patients have received medical training and do not understand the nuance of what they are reviewing. 

It’s like visiting a foreign country, where not only do you not understand the language, but you don’t even recognize the letters.  

While the medical staff does a very effective job explaining the contents of the Informed Consent Form, the person receiving the information may not be able to understand it in the form and language in which it is delivered.  The layout is confusing.  The flow is often disjointed.  It is not specifically written with a patient as its prime audience.  The goal must be for patients to ‘understand what they are signing up for.’

A few suggestions for study sponsors that should make the ICF easier to digest for patients:

1. Make it more readable for the patient.  ICFs are very dense documents – a lot of technical terminology, detailed tables and appendices, too many acronyms, inclusion and exclusion criteria that might be confusing and difficult to follow.  Informed Consent Form authors need to keep their audience in mind.  Use terms that are better understood by potential patients.  I appreciate that there may be a regulatory distinction between a side effect, adverse event or serious adverse event, but to patients, it’s just a side effect that must be addressed.  Help the patient understand what they are reading.  The MRCT Center at Brigham and Women’s Hospital and Harvard has developed a patient friendly Clinical Research Glossary that provides plain language definitions and examples of commonly used terms.  Additionally, more schematics and symbols should be used and fewer words.  As an oncology patient, I always think in terms of cycles – how long is a cycle, how many site visits are required, what is the sequence of events during that cycle -e.g. – treatment, bloodwork, scans, doctor appointment/telemedicine, providing patient reported outcomes via questionnaires.  It’s a better way to communicate - I’m more likely to remember the picture then a detailed appendix;
   

2. Highlight the benefits and risks so patients can understand these.  Patients do not want clinical trial sponsors to ‘over-promise or over-sell’ the potential benefits of participating in a trial. Patients are always wearing a WIIFM hat (What’s In It For Me). Too often in the spirit of being overly cautious, the potential benefits are under-emphasized, or they are positioned in an altruistic frame – by the patient participating in the trial, we will learn a lot more about the disease for the benefit of future patients.  Don’t misinterpret, altruism is a key driver for many clinical trial participants, but hopefully there could be some benefit to the participant.  Why not present the potential benefits and risks in a more balanced, understandable way?  After all, there is true patient benefit associated with being more closely monitored as part of the study – this should not be minimized.  ICFs are very thorough, including all the possible risks.  Another improvement that could help patients is to provide more details on the potential side effects.  Often these potential risks are presented without any communication of the likelihood of occurrence. Potential participants may be overwhelmed by all the possible risks and cause them to wonder why they would ever consider participating in a trial.  This is required to better inform patients;
   

3. Clearly articulate the patient burden (and associated burden for a caregiver) and take steps where possible to minimize this burden.  Participation in a trial often involves more frequent monitoring leading to more site visits for lab work, doctor appointments, or scans.  Can any of these be done more conveniently for the patient, be it at a local office or testing facility?  Or perhaps some appointments could be via telemedicine?  But let’s make it easy.  Provide the patient with a debit card to capture any covered expenses.  If possible, provide logistical support to help patients and caregivers make reservations and travel plans, be it ride sharing, flights or accommodations.  However, remember that every patient is different and his/her needs and preferences are different.  Ideally, trials should be designed to be flexible and allow patients to select the option that best meets their needs.
   

4. What costs are the patient responsible for?  This needs to be very clear, both for participation in the actual trial activities and beyond those activities should there be any additional cost implications.  For example, after the trial is finished, are there longer-term testing requirements for the patient?  Or might patients be faced with changes over the long term in their ability to secure insurance?  Patients must be informed.

Reaching an Informed Decision

Patients like me need more and should expect more.  They want to understand the trial – what is expected of trial participants?  Potential participants want to be confident that steps are being taken to minimize their burden; they want clarity of the potential risks and benefits.  This information is essential for the participant and caregiver to make an INFORMED DECISION - that is being a truly informed patient.  Potential participants should consider the tradeoffs, both those specific to the trial and those beyond the trial.  Equally important is the understanding of alternative treatments. Are better treatment options available?  Is there another trial that might be a better fit?  All these inputs will influence making an informed decision and be weighed by the personal circumstances and priorities of the patient. These priorities might be psycho-social, logistical, financial or other.  Patients have different circumstances, priorities and preferences which will influence their decision, but to make a truly Informed Decision, the patient must incorporate all considerations, trial specific and beyond.

Regarding consent, a participant’s competency to decide as an informed volunteer is key.  Consider the dynamics of the situation.  A patient has just received a potentially life changing diagnosis.  The patient is trying to quickly learn about the disease and digest information in a ‘foreign’ language.  In many circumstances, time is of the essence, adding additional pressure for requiring and making an informed decision.   The patient tries to determine if there are any trials available that meet his/her needs. The patient can be overwhelmed with information - like drinking from a firehose.

Searching online can be challenging for people without a scientific or medical background.  New tools, like Medifind.com or CenterWatch iConnect help patients find specialist doctors and clinical trials, but it is still a challenge. 

Is the patient ‘competent’ to make an informed decision at such a stressful time? 

 Many patients rely on their doctors to help them navigate to the best course of action, but non-research-oriented general physicians might not be aware of the relevant, most current clinical trials.  Many doctors are most familiar with clinical trials at their institution.  Physicians should be educated on how advocacy groups can support their efforts. Helping patients and families to become broadly informed, beyond just the details of one study, is so important.  The patient needs to trust the depth and breadth of the trial information and be confident they are making the best personal decision.  Patients should seek input and advice from beyond their general practitioners and the clinical investigators and engage disease specialists for their opinions.  Yes, the patient is competent to make a decision but perhaps not always confident that they are making the right decision.  

Finally, the voluntary aspect of consent is well covered in the ICF.  Patients should clearly understand that their participation is voluntary.  They always have the option to leave a clinical trial at any time for any reason.  There should be no pressure to participate in a clinical trial and patients should expect to receive the current standard of care available.

Our Call To Action 

Specifically, on behalf of my fellow patients, here is our call to action to pharmaceutical sponsors. 

1. Incorporate the voice of patient experts in co-creation.  Experts should provide ‘the patient voice and insight’ into the development of patient facing materials and  explore opportunities to reduce patient burden.  Our experience and insight is invaluable throughout the development of new treatments and companies should seek to partner with patient experts. 
2. Train physicians to communicate with patients about treatment options, including clinical trials.  Help physicians and patients find ‘the best’ clinical trials for the individual patient – trials that are medically relevant, inclusive and accessible.   
3. Invest in infrastructure to better leverage technology driven opportunities to support decentralized clinical trials and improve the patient journey.  
4. Update the  informed consent process.  The goal should be to provide the information and context to allow patients to make a truly informed decision.  Develop patient-facing materials that use language that is easily understandable, better graphics and visuals, and a balanced articulation of benefits and risks. Including non-medical risks. 

There are clearly opportunities to further improve the patient journey, to be even more patient-forward in designing and delivering clinical trials.  For many patients, clinical trials provide a ray of hope that otherwise might not be available.  The ICF serves as that bridge to the possible.  

It’s time not just to be informed, but for patients to be empowered to make an Informed Decision.