Let’s run our trials for the real patient populations

There is a significant gap between what our population looks like and what the population of people  participating in clinical trials looks like.

Prostate cancer, for example, is highly over-indexed in African American males. Yet historically, the type of patients included in prostrate trials are white men. For breast cancer, while the mortality burden is significantly higher for African American women, they are often not represented at the same level in clinical research as they are in the population.

This is a growing problem, notably in the eyes of regulatory bodies, but also when it comes to delivering the best treatment for the actual patient population.

There is a significant gap between what our population looks like and the population participating in clinical trials.

Keri McDonough, Leader for Advocacy and Patient Relations at Syneos Health, points to traditional barriers limiting patient diversity. She references the example of finding the “perfect patient” for a clinical trial. As McDonough explains, in the quest for a perfect patient, you can end up excluding your real patients. An example is setting a very limited BMI criterion, which may alienate significant patient populations.

McDonough pointed out that “if you don’t have the data that is reflective of the population you intend to provide medication to, then the FDA is saying loud and clear that you’re not going to be able to proceed.”

“If you don’t have data reflective of the population you intend to provide medication to, then the FDA is saying loud and clear that you will not be able to proceed.”

In its June 2019 guidance, the FDA laid out a roadmap for the industry for how they can increase patient diversity within their clinical trial populations. The core focus areas were eligibility criteria, enrolment practices and trial design.

Keri believes that each of these fields is interlinked. It then becomes a domino effect; if one is out of place, the next part of the process will be impacted. McDonough explains that “if you have too stringent eligibility criteria, then your trial will not be able to recruit patients, so your enrolment practices won’t matter much. Or if you have a trial that is recruitable but you don’t have the right people on the ground in the recruitment process, you’ll run into another problem.” To address these areas, there needs to be an understanding for how each piece fits into the mix.

A fundamental starting point for each focus area is to increase education and awareness. McDonough points out that “if certain patient communities aren’t aware that they can participate and that trials are available, then there’s no hope of recruiting them.”

Effort here should be focused on clinical physicians. As a trusted source of information, they are one of the best routes to reach patients directly. Clinicians may be operating at the moment from a place of personal bias. McDonough points out that “if a physician has their own set of assumptions or barriers in place, then that patient you want to reach is not going to be aware of your trial,” as their physician is not raising the opportunity to them. You can proactively address this by communicating the importance of patient diversity with physicians and creating materials to reach underserved communities. For example, translating clinical trial information into Spanish to reach the Hispanic community. McDonough believes it is crucial that your trial materials connect with the patient communities you want to reach.

“If certain patient communities aren’t aware that they can participate and that trials are available then there’s no hope of recruiting them.”

Trials should also be designed with inclusivity in mind. This may include increasing the number of investigators that represent the communities not traditionally involved in clinical trials. Or ensuring that your clinic sites are suitable for the patient community you want to reach. For example, if you are running a trial for obese patients, have you ensured they are accommodated in the clinic with appropriately sized seating?

Simple and small changes can have a drastic impact on how welcome a patient feels within a trial, which will improve their retention and compliance and directly impact the quality of patient data.

To ensure patient diversity, inclusion must be addressed from the start. McDonough suggests that when you have your first sense of where you want the trial to go, before you develop a full protocol, you should bring in the advocacy community for a closed-door discussion. McDonough believes that “this is the time to discuss your enrolment approach and identify any red flags early on.” She continues that “no one wants to be involved in a last minute scramble to get more representation, when every moment and every second along the path to submission matters.”

“No one wants to be involved in a last minute scramble to get more representation, when every moment and every second along the path to submission matters.”

It is also important to create patient advisory boards that are separate from the leadership and advocacy groups. In particular, you want to get the perspective of patients who are not over-educated on the topic. McDonough notes that while seasoned advocates are an immensely valuable resource who bring so much to the process, they do often have a highly sophisticated line of sight. She highlights the importance to “pause and get some feedback and perspective from normal, everyday people who don’t live and breathe this stuff.” These are the patients you will want to recruit. Where appropriate, run your materials and plans by them and use the discussion as an opportunity to make sure what you create actually resonates.

Taking the time to think with inclusivity in mind and design practices for a diverse patient population will lead to dividends in the long run. It is time to ensure that the trials being run are truly reflective of the patient population they aim to serve. As McDonough points outs, there is a chance to forge meaningful connection between scientific innovators and the full patient population. It is through this relationship that real innovation and effectiveness will be shown.