Why It Pays to Invest in the Patient Experience

Scott Gray - Co-Founder and CEO, Clincierge

The pharmaceutical industry's understanding of the role of patients in clinical trials is evolving. Patients have always been at the center of clinical trials, but this has not always been reflected in the attitudes toward the design and execution of the trials themselves.

Individuals choose to enroll in clinical trials for various reasons. Most often, it is because the treatment available represents the best chance of effective therapy or to aid the drug development progress. Both factors have traditionally played an equally important role in the decision to join a trial. On the industry’s side, a clinical trial aims to develop effective treatments for patients with the potential for commercialization, with efficiency and safety always top priorities.

A trend of patient centricity is emerging within clinical trials, and we are watching its shift from an industry buzzword to a fundamental part of clinical trial design. The main principle of this style of trial design is always keeping the patients’ needs as the top priority. What makes this idea revolutionary? In theory, it is because patient centricity serves to unite the aims of clinical trial stakeholders. Patients benefit from measures designed to respect their views and keep them engaged, ensuring higher trial completion rates. The pharmaceutical industry benefits from an improved recruitment process and increased retention rates through trial completion.

The transition to patient-centric clinical trials makes even greater sense when placed into the context of the pharmaceutical industry’s overall shift in the treatment development process. New medications are being developed for smaller patient populations, increasing the practice of creating customized medicines, often in the orphan drug space. For reference, the number of personalized therapies on the market doubled between 2016 and 2020,¹ while orphan drugs made up over half of the FDA's approvals in 2021.²

When delivering new treatments to smaller patient groups, it is crucial first to fully understand the participant experience to shape the clinical trial. For studies with a smaller number of participants, ensuring the patients feel listened to and valued will help to keep them engaged for the entirety of the trial, leading to a lower dropout rate. This patient-centric approach significantly affects enrollment and retention rates when the existing patient pool for a particular condition is already very low.

Valuing Patient Participation

One key challenge facing the clinical trial process is recruiting enough patients and retaining enough participants once the group is assembled to ensure successful trial completion. Research has shown dropout rates in late-stage trials often exceed 30%.³ This level of dropout in clinical trials can have a significant impact. When a certain percentage of patients decide to leave, there is the possibility the trial will have to be abandoned due to being under-enrolled. The resulting loss of time, money, and trial data is enormous.

With the cost of clinical trials running into the millions of dollars and the average investment deployed in developing a new drug estimated at $2 billion to $3 billion,⁴ stakeholders want to ensure trials do not end due to patient attrition. The cost of trials often overrides other factors in determining the success of the research. One study found that 22% of Phase III studies failed not due to the efficacy of the treatment but instead due to a lack of funding.⁵

The issues facing the successful completion of a clinical trial can become a self-defeating cycle. The recruitment process is one of the most expensive elements of setting up a trial and is only exacerbated by delays created by recruitment difficulties and patient dropouts along the way. Each delay or pause to the trial incurs additional expenses, which increase the risk the trial will have to be discontinued.

Recognizing Return on Investment

Innovative clinical trial stakeholders first seek to understand why these issues arise by learning about the patient experience before, during, and after the clinical trial process. The high value of this patient-centric approach has been supported by research. Studies found drugs developed using a patient-centric design were 19% more likely to be approved, a statistic consistent across different therapeutic areas.⁶

Despite the success of drugs developed utilizing patient-centric methods, some companies are still reluctant to engage patients more deeply in the clinical trial process. One of the reasons behind this hesitancy is the potential financial costs of employing patient-centric measures, which require additional investment dollars. However, research has found that for $100,000 of investment in patient engagement, it was possible to return this sum 500-fold or more.⁷

The reasons for this increase in return on investment are as varied as individual patients' reactions to participating in clinical trials. Focusing on patient input makes it possible to identify eligibility criteria allowing for a more significant number of patients to enroll. Employing measures to ensure continued patient engagement makes the study more attractive to these eligible patients, resulting in improved overall participant satisfaction with their trial experience.

The critical challenges of patient recruitment and retention can be addressed by implementing measures to reduce the inconvenience factor attending clinical trials has on everyday life. This support includes managing the logistics of patient travel to a clinical trial site by providing a patient care coordinator, an approach with numerous advantages.⁸ When patients are offered additional support through patient education or appointment reminder tools, engagement and retention rates increase. When combined, these services accelerate drug development pathways. A more attractive study results in a shorter recruitment process, with a reduced chance of delays due to patient retention issues.

Another central talking point across the industry is diversity in clinical trials.⁹

Studies incorporating patient centricity in their protocols have the added benefit of increased diversity. Historically, the majority of clinical trial participants have been white males, as they were more likely to be able to afford the time away from both work and family, as well as pay for the travel expenses required for participation. By contrast, minority populations were less likely to be referred to a clinical trial and had a reduced likelihood of participating once they learned of the opportunity due to challenges like lost wages and travel logistics.

Increasingly, the pharmaceutical industry is working to rectify this disparity in clinical trial participation, and patient centricity in trial design is one measure likely to improve diversity statistics. Earlier this year, the United States Food and Drug Association (FDA) drafted industry guidance to enroll more participants from traditionally underrepresented racial and ethnic populations in the US.¹⁰ Moving forward, trial regulation will see further emphasis placed on inclusion, so taking steps before such action becomes mandatory will serve drug developers in the long term.

Fostering Regulatory Support

The FDA's push on diversity mirrors similar actions by industry regulators on patient centricity. Greater efforts are being made to ensure clinical trials offer an improved and patient-focused experience for those participating. This change has involved new publishing guidance and materials aimed at promoting the adoption of patient-centric measures.

The FDA recently provided guidelines on patient-focused drug development, highlighting how stakeholders can collect and submit patient experience data alongside other relevant information from patients and caregivers. Through its Oncology Center of Excellence, the agency also announced the launch of 'Project Optimus,' seeking to change how the industry approaches dose optimization for oncology treatments. The project aims to encourage the selection of a dose(s), maximizing the efficacy of a drug and its safety and tolerability. In this manner, patients are no longer subjected to the highest amount of a drug possible (with the associated level of potential toxicity) but a range of doses tested in advance of administration to determine efficacy and safety at different levels.

This action is not limited to the FDA, with European authorities also including 'generation of patient experience data' and 'patient engagement during regulatory assessment' as a vital feature of the agency's strategy through 2025.¹¹

Learning from Patients

The action of regulators is currently focused on several key strategies: increasing the level of patient experience data and encouraging efforts to engage more actively with patients. The emphasis on gathering information and data is crucial for future applications of patient centricity, which is still a developing and evolving trend. Simply understanding how patient-centricity benefits trial stakeholders, whether financially, ethically, or in terms of drug development, is not enough. The key is to learn the most significant factors for patients and what areas they are looking for improvements. Suppose drug developers can understand the reasons why patients are likely to drop out of trials. In that case, action can be taken to address the challenges and provide the participants with an overall better experience. In the long term, this will result in more patients having increased access to better treatment options.

In order to learn what patients found to be the most challenging aspects of clinical trial participation, Clincierge commissioned independent research to understand the most common reasons participants decided to exit clinical trials.¹² The study found that 95% of respondents said travel was the primary reason for the decision to drop out of a trial. Additionally, 62% of patients said travel concerns prevented them from joining a trial in the first place.

The burden of travel to a clinical trial participant will be felt differently based on several factors. The common issues faced by most individuals considering a clinical trial are economic in nature. Participating in a trial will likely disrupt work commitments, leading to loss of income and potentially the loss of their job due to prolonged time off. Travel expenses can deter a patient, with many worrying about the possible need to employ childcare services. Also necessary to consider is the amount of time spent away from the family due to travel to the clinical trial site and the pressures placed on the patient's primary caregiver.

These challenges are only exacerbated when long-distance travel is necessary. When dealing with smaller patient groups, such as in rare diseases or personalized medicine, travel to a clinical trial often involves flights to the site and extended periods away from family. The complexity of arranging travel to participate in a clinical trial increases exponentially if the participant has mobility or cognitive disabilities, making travel more difficult.

The result of these challenges is increased stress on potential clinical trial participants and reduced likelihood of being interested in or able to participate. It is no surprise the researchers found that 100% of respondents said having a dedicated individual who could help them manage trial logistics was essential to them. Providing patient support services is crucial in alleviating the many burdens related to clinical trial travel arrangements. A patient care coordinator who will manage logistics, including travel and reimbursement, allows patients and their caregivers to focus on the primary goal of their clinical trial participation – receiving much-needed treatment while providing the critical data needed to reach trial endpoints.

The foundation of the pharmaceutical industry is built upon improving the well-being of patients through increasing viable treatment methods. A patient-centric clinical trial protocol reflecting this goal will enhance both the patient's experience and the trial's overall outcome. As an industry, we cannot ignore the many benefits this patient-centric model provides to patients. This trend must continue to grow, bolstered by continued research and positive results.

References

1. Personalized Medicine Coalition, The Personalized Medicine Report, December 2020. Accessed August 15, 2022. https://www.personalizedmedicinecoalition.org/Userfiles/ PMC-Corporate/file/PMC_The_Personalized_Medicine_Report_Opportunity_Challenges_ and_the_Future.pdf
2. Evaluate, Orphan Drug Report 2022. April 27, 2022. Accessed August 15, 2022. https:// www.evaluate.com/thought-leadership/pharma/orphan-drug-2022-report
3. Alexander W. The Uphill Path to Successful Clinical Trials. National Library of Medicine. 2013 Apr; 38(4): 225–227
4. John Hopkins Bloomberg School of Public Health, Cost of Clinical Trials for New Drug FDA Approval Are Fraction of Total Tab. September 24, 2018. Accessed August 15, 2022. https:// publichealth.jhu.edu/2018/cost-of-clinical-trials-for-new-drug-FDA-approval-are-fraction[1]of-total-tab
5. Fogel, D. B. Factors associated with clinical trials that fail and opportunities for improving the likelihood of success: A review, Contemporary Clinical Trials Communications, 2018 Sept, 11, 156-164
6. The Economist, Patient-centric trials – Summary. May 2019. Accessed August 15, 2022. https://druginnovation.eiu.com/patient-centric-trials/
7. Levitan B. et al, Accessing the financial value of patient engagement: a quantitative approach from CTTI’s patient groups and clinical trials project. National Library of Medicine. 2018 Mar; 52(2): 220–229
8. Scott Gray, Pharma Outsourcing. Advancing clinical trials with patient support services. September 8, 2021. Accessed August 15, 2022. https://www.pharmoutsourcing.com/ Featured-Articles/579102-Advancing-Clinical-Trials-With-Patient-Support-Services/
9. Scott Gray, Pharma Outsourcing. Achieving clinical trial diversity through actionable steps. June 2, 2021. Accessed August 15, 2022. https://www.pharmoutsourcing.com/Featured[1]Articles/576443-Achieving-Clinical-Trial-Diversity-Through-Actionable-Steps/
10. US Food and Drug Administration. FDA takes important steps to increase racial and ethnic diversity in clinical trials. April 13, 2022. Accessed August 15, 2022. https://www.fda.gov/ news-events/press-announcements/fda-takes-important-steps-increase-racial-and-ethnic[1]diversity-clinical-trials
11. European Medicines Agency. Progressing the concept of patient-centred development in practice. June 4, 2021. Accessed August 15, 2022. https://www.ema.europa.eu/ en/documents/presentation/presentation-progressing-concept-patient-centred[1]development-practice-n-bere-ema_en.pdf
12. Clincierge. Amplifying patient voices in rare disease clinical trials. July 24, 2022. Accessed August 15, 2022. https://info.clincierge.com/vop 

Scott Gray is the co-founder and CEO of Clincierge, a provider of patient support services for clinical trials. Since 2015, Clincierge patient care coordinators have managed logistics and reimbursements in more than 300 clinical trials worldwide. For more information, visit www.clincierge.com.

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