Commitment to Patient Centricity and Diversity: Key ingredients for a successful trial

As a technology led global CRO with specialist therapeutic expertise in oncology, cell & gene therapy, and rare disease, we have been taking a patient centric approach to trial design for over 20 years. This article outlines recent changes in industry requirements and notes some of the important patient centric techniques and strategies we continuously leverage that are gaining visibility across the industry and improving clinical trials for both patients and Sponsors.

What is patient centricity and patient diversity?
Generally, “Patient centricity” in the terms of clinical research means designing a treatment, clinical trial, or other health solution centered around the patient. This approach requires feedback from patients, their loved ones, plus caregivers, as well as making decisions based on that feedback.

Clinical trials rely on volunteers, and age, gender, weight, race, ethnicity, and other factors can trigger different reactions in people taking the same treatment. Ensuring diversity among clinical trial participants allow clinical trials to assess if the treatments are safe and effective for people across different communities

Changes in Industry
Until recently, patients’ input into clinical studies had been minimal to non-existent. New regulations ^1,2,3 ICH GCP E8(R1), place emphasis on including patient perspectives in study design and require studies to take a patient-focused approach to study conduct at all stages of clinical development. Including patient and health care providers’ perspectives help to position potential treatments in the “context of ethical issues, culture, region, demographics, and other characteristics of subgroups within a targeted patient population.” ⁴ Sponsors can benefit by partnering with a CRO who is well versed in understanding patients’ needs and having the expertise to implement a successful patient-centred trial execution strategy.

Patient centricity is not just a ‘nice to have’, it offers clear benefits to Sponsors
A patient centric approach results in trial participants who are more likely to both consent for and complete a clinical study. In a patient survey ⁵ from Boston Consulting Group (BCG) in 2019, respondents said they were more willing to engage and work with Sponsors that they perceived as taking a more patient centric approach to research. In addition, more than 50% of physicians said that with everything else being equal, they were more likely to prescribe medication from a Sponsor they considered be more patient centric. Further, the Future of Drug Development study⁶ reviewed approximately 4,000 Phase II and Phase III trials in oncology, neurology, and rare diseases and concluded that studies that took a patient focused approach reduced their enrolment timeline by more than 50% compared to those studies that didn’t (from 7 months to 4 months on average). In an ever-growing competitive landscape, this benefit to Sponsors and their patients is clear and our vast knowledge and experience aligns with these results, which are: a patient centric approach achieves accelerated recruitment, improves retention, and facilitates faster pathways to launch successful treatments into the marketplace.

Example applications of patient centricity and patient diversity
Treatments with a ‘one size fits all’ approach to drug development no longer meet patient and healthcare provider expectations. Patient and population diversity must be considered and is of the utmost importance to develop drugs that meet patients’ specific needs.

Data from the American Cancer Society ^7,8 show there are differences in disease prevalence, responses to treatment and survival across different populations, therefore studies need to build in strategies which include approaches to identify these differences, when not previously known.

Precision medicine (a patient centric approach which considers individual variability in genes, environment, and lifestyle) continues to advance and is one method to address this need. Our teams are able to assist sponsors in the identification or application of disease or patient specific markers in trial design or completion. An example of this is initial tumor markers or changes in markers during a trial.

It is imperative to utilize the data already available. Below is an example of applying readily available patient demographic information in the trial site selection strategy:

During the initial feasibility outreach stage for an antihypertensive project, we focused feasibility work on sites within African American communities because of the high disease prevalence and knowledge of patient difficulties with travelling for healthcare. Because of this insight, we placed the majority of the study centers in local neighborhood institutions rather than the larger academic-oriented institutions. As a result, the study was able to recruit quicker, provide greater community knowledge of research, and add to available treatment options for this population.

Aixial Group places high importance on partnerships with Patient Advocacy Groups (PAG) and local disease support groups. This can aid in educating the desired patient population while empowering local investigators and research personnel to act as study advocates in their local communities. This link (investigators as study advocates) has shown to be a powerful tool for helping patients feel comfortable with participating in research. This can also serve as a medium for building relationships between study investigators and local primary care physicians for clinical trial education.

Aixial Group is specialized in this and is continuously building and delivering strategies that factor in the social determinants of health that create or participate to the level of these differences on behalf of our Sponsors.

A Pragmatic Approach and Building Trust Leads to a Successful Trial
We continue to implement various methods to remain patient centric, which leads to an increase in enrollment, study participation, and more robust data for the Sponsor’s study. Here are a few ways we make a difference:

• Providing decentralized options such as eConsent, ePROs, home nursing or telehealth, local lab draws to accommodate patient needs in different ways
• Flexible trial visit or assessment schedules
• Patient and/or caregiver concierge support
• Developing companion information for families and caregivers
• Involving patient advocates in recruitment material and informed consent development and review
• Translating trial information and recruitment materials into different languages and/or lay summaries to reach broader audiences
• Ensuring patients can easily understand the informed consent by making it available in their native language or in a validated lay language
• Developing surveys to gather feedback on protocol design and patient-friendly operational logistics. This can lead to an improved, more accepted protocol by the patients and therefore an increased enrollment
• Applying genetic, environmental, and lifestyle information to the trials design including selection or recommendations on investigational treatments, selection of trial sites, assay development, etc.

In Conclusion
Involving patients early in the process has shown to have an increase in their trust in the study, a boost in recruitment, and overall protocol adherence. On the study side, incorporating patient perspectives can also help us understand the lived experience of a condition which contributes to results that are more meaningful to patients and that significantly support the creation of new treatments that are better suited to their needs.

Our approach to patient centricity is pragmatic, inclusive, and proven to be a great benefit to Sponsors. We pride ourselves on our commitment to understanding patient populations and will continue our focus of looking after and caring about the right partners: patients, research sites, PAGs, and Sponsors. With their trust in hand, our work in clinical trials will continue to be successful.

Reach out to us today to discuss an approach that fits your needs. Our services have a global reach with trials operating in over 30 countries across more than 10 therapeutic areas focusing on oncology, cell & gene therapy, and rare diseases in all phases of clinical development.

Email us at: info@aixial.com

References
¹Medicines and Healthcare products Regulatory Agency (MHRA), Opportunities for patients and the public to be involved in the work of the MHRA, Guidance, 2020. https://www.gov.uk/guidance/opportunities-for-patients-and-the-public-to-be-involved-in-the-work-of-the-mhra
²Food and Drug Administration, FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making. Consulted on 21 July 2022. https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical
³European Medicine Agency, Patients and Consumers. Consulted on 21 July 2022. https://www.ema.europa.eu/en/partners-networks/patients-consumers
⁴International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use. General Considerations for Clinical Studies (ICH E8 (R1), 2021. https://database.ich.org/sites/default/files/E8-R1_Guideline_Step4_2022_0204%20%281%29.pdf
⁵Lubkeman, M Lee, G Barrios, What Do Patients Want, and Is Pharma Delivering?, 2020. https://www.bcg.com/publications/2020/what-patients-want-is-pharma-delivering
⁶The Economist Intelligence Unit, The Innovation Imperative: The Future of Drug Development Part I: Research Methods and Findings, 2019. https://druginnovation.eiu.com/wp-content/uploads/2019/05/Parexel-innovations-in-drug-development-part-1_V14.pdf
⁷American Cancer Society. Cancer Facts & Figures for African American/Black People 2022-2024. Atlanta: American Cancer Society, 2022.
⁸American Cancer Society. Cancer Facts & Figures for Hispanic/Latino People 2021-2023. Atlanta: American Cancer Society, Inc. 2021.