By Rashida Challenger and John Ferraro, Halloran Consulting
The process of developing novel cutting-edge therapies within a diverse patient population has always been a challenge for the biotech/pharma industry. The industry is weighed down by the increased cost of bringing new therapeutics to market. The additional pressure to conduct clinical trials that mirror our diverse population presents further challenges that the industry hasn’t quite found the answers to.
Unfortunately, disparities do exist when testing new drugs, therapies, and devices. Lack of diversity in clinical trials applies to race, age, gender, socioeconomic status, and even disease state. For instance, the elderly population is notoriously excluded from clinical trials, which is rather perplexing given that they are a large and fast-growing portion of the worldwide population, making up the biggest share of patients for particular health conditions. For decades, studies were historically enrolled with men, not allowing women to participate. And people who suffer from comorbidities—say, someone who is HIV positive and also diagnosed with cancer — typically aren’t eligible to participate.
Even when a certain disease is more prevalent in a particular minority population, it can still be difficult for trial investigators to reach the right individuals. As an industry — as a group of researchers dedicating our lives to improve the health of others, regardless of their race, age, gender, or disease — how can we do better?
The lack of representation in clinical trials stems partially from oversight. Biotech and pharmaceutical companies design their clinical trials based on what will get their drugs approved and on the market as quickly, safely, and cost effectively as possible. In widening the eligibility of participants — opening the door to potential risk, adverse events, and murkier data — drug developers sacrifice not only time and money but the potential success of their drug.
It was good to see that the FDA recognizes this issue as it released a draft guidance in June 2019, Enhancing the Diversity of Clinical Trial Populations – Eligibility Criteria, Enrollment Practices, and Trial Designs. This draft guidance is a step in the right direction and has some interesting thoughts and potential solutions for the industry to implement. However, does it go far enough?
We do a cursory job of evaluating and ensuring a study population that would, upon extrapolation to the population as a whole, be representative. Why is being representative such a crucial part of bringing a new therapy to market? For one, certain minority patients may very well benefit from early access to therapies, potentially prolonging or saving their lives. For another, we are all biologically and genetically different from one another, making it crucial to test new therapies in diverse populations to firmly conclude whether a drug is safe and works. So, what are some tangible actions we, as an industry, can take to increase diversity in clinical trials?
1. Widen eligibility criteria.
We must map out wider eligibility criteria earlier in the drug development process. We understand this is not an easy ask for smaller companies, which have less resources and capital on hand. However, if we can start making small adjustments to how we recruit patients for clinical trials, we are better off predicting disease onset and treating patients along the continuum of public health with practical data. Achieving proof of concept and securing investor interest tend to be top of mind for companies in early stages of development, making it difficult for them to allocate time, resources, and funds toward inclusive clinical trial design. But if we’re going to move the needle on diversity in study participants, drug developers must be thinking about this in Phase 1, not Phase 3.
2. Tap into community-based medical centers.
Clinical trials often take place in large research and academic facilities located in major hubs like Boston, New York, and Atlanta due mainly to their academic credentials and experience, yet many patients needing care live in rural areas. The financial burden and time commitment necessary to travel regularly to doctors’ visits in these cities often prove to be too expensive and time consuming for many patients. This is particularly burdensome for elderly and low-income individuals. Some clinical trials require participants to visit the doctor’s office weekly or mandate a number of assessments in order for the patient to be considered compliant with the protocol. Plus, with each doctor’s visit and assessment being accompanied by an insurance copay that is not covered by the clinical research company, the cost to participate already diminishes a great deal of the population pool.
Therefore, tapping into the community-based medical centers and clinics can surely help. They may not have “the” big name affiliation or “the” most published studies, but what they do have is accessibility and a keen understanding of their local community. They care about their patients and can often be your trial’s biggest recruiter and cheerleader.
3. Use technology.
A recent buzzword in our industry is “patient-centricity.” Everyone in our industry wants to be more patient-centric. Here is your chance — implement aspects of virtual clinical trials using technology and other home-based and telemedicine technologies to enable the expansion of your trial into underrepresented areas and also reduce the obstacles of distance, costs, and availability.
4. Connect with patient advocacy groups.
Even when certain diseases are much more prevalent in minority or underserved populations, it can still be difficult for companies to reach the right participants. This is where patient advocacy groups can come into play — they serve as a direct plug-in to your patient population. Get to know the organization early on in your drug development process. If you work in the rare disease space, this is a nonnegotiable component of your trial’s success. Take the time to educate the advocacy group on your compound and trial, sponsor their disease awareness campaigns and write content for their newsletters. Meaningful relationships with patient advocacy groups take time to foster, but once trust has been established, the partnership will pay out all through the development process and into commercialization.
5. Support diversity-focused public policy.
In certain countries, maintaining diversity in clinical trials is written into the law. And while this isn’t mandated in the United States, it is certainly a measure for consideration. Of course, we don’t want to needlessly stall innovation. Overregulation in an already highly regulated industry doesn’t benefit anyone. But a compromise may be in the cards to make diverse representation in drug development a higher priority for our industry. A potential solution to enable this would be for the FDA and other regulatory authorities to institute a diversity program similar to the pediatric and/or orphan programs that already exist to assist these niche populations. This would provide monetary benefits that incentivize trial sponsors to include diverse populations in their clinical trials.
6. Make diversity an internal mandate.
At the end of the day, true change in clinical practices won’t happen unless diversity is prioritized within an organization — and from the top down. A company’s executives and clinical leads must be vocal and action-oriented about their commitment to ensure diverse representation at every phase of development. It needs to be a frequent topic of discussion. If that’s not the case, then don’t be afraid to speak up and challenge the processes that are set in place.
What’s the point of developing therapies for patients if we exclude groups of individuals who may benefit from them and provide more robust data for all patient populations? Why do we worry more about the statistical difference between two arms in a clinical trial than whether the trial as a whole represents the community we serve? We must be better about servicing all populations, not just those that are easiest to access. We must be better about achieving biopharma’s ultimate goal: improving the health of all people.
A great place to learn more about increasing diversity in clinical trials is the FDA’s Office of Minority Health and Health Equity.
About The Authors:
Rashida Challenger, lead consultant in clinical operations at Halloran Consulting Group, has more than 13 years of experience in clinical operations with an emphasis in oncology and rare disease. She has held many positions ranging from clinical research associate to clinical project manager to associate director in the pharmaceutical/biotechnology space and with CROs. At Halloran, Challenger is responsible for the management of the overall study operational plan and CRO oversight, which includes project timelines, trial budgets, and quality of deliverables. Prior to joining Halloran, she was associate director of clinical operations at Neximmune, where she was responsible for the oversight and execution of cellular therapy clinical programs.
John Ferraro, principle consultant at Halloran Consulting Group, has more than 28 years of experience in clinical operations/research within the biotechnology and pharmaceutical industry. During this time, he has been successful in all sizes and types of organizations as well as in a myriad of therapeutic areas, most recently oncology, hematology, immuno-oncology, and rare disease. Ferraro is a clinical operations leader who excels at operational strategic planning, oversight, and delivery of clinical development programs. Prior to joining Halloran, he led his own consulting firm, JnC Clinical, where he was engaged with various organization types as VP of clinical operations or clinical advisor.