Clinical Research: Phase 1 - Phase 4

Addressing Research for Women from Every Angle

When sponsors develop drugs and devices specifically for women, they need to create trials that are uniquely female-focused. From protocols and endpoints, to recruiting strategies and messaging, these trials have to take into account the unique needs, expectations and regulatory requirements of a totally female population.

Having spent more than 20 years in this space, I’ve learned a lot about the adaptations sponsors and CROs can make to better meet the needs of regulators, sites, and the women they want to recruit. Many of these changes are subtle, but can have a big impact on trial outcomes.

Regulatory expectations

The first step is honing specific research priorities, which will power the design of the trial. Based on research goals, we can determine how many subjects are needed and from what specific populations, specify inclusion/exclusion criteria, and settle on primary endpoints.

While most study sponsors have a good idea of these goals when they engage a CRO, we help review these objectives from the lens of women’s health research. Having significant past experience working to satisfy FDA and other regulatory requirements, we tailor trial design to improve the odds of study success.

Understanding regulatory expectations and having recent firsthand experience with industry guidance adds efficiency to the review process. Getting this knowledge ahead of time prevents sponsors from having their initial protocol design declined. It also helps in building a more realistic project timeline and budget, making long-term planning easier.

A woman’s perspective

While accounting for regulatory requirements, we must also acknowledge the participant burdens that may interfere with recruiting and seek opportunities to ease these challenges wherever we can. We can’t help a sponsor gain regulatory approval and bring a product to market without effectively engaging women in a study. Ensuring a positive patient experience is always central to what we do.

It’s no surprise that patients’ motivations and expectations when participating in clinical research differ from that of the sponsors and investigators involved. Part of our role is to advocate for what women need, while ensuring data can be collected efficiently at the site level and delivered to the sponsor for regulatory submission.

For example, in early contraceptive studies, participants are required to use back-up contraception to avoid unwanted pregnancy. Without this, we might experience greater enrollment challenges. However, this means sponsors cannot measure effectiveness by lack of pregnancy. Instead, the follicle size of ovaries is measured for visible growth, which indicates ovulation.

But to do this, a woman has to come into the clinic two-or-three times a week for blood draws and transvaginal ultrasounds. This creates a significant time commitment and is fairly invasive. So, these requirements must be balanced with appropriate patient incentives. Where possible, we streamline data collection to reduce participant burden and build in strategies like fair patient compensation. We deliver strategic solutions that will work for everyone.

Inclusive and representative

A close review of inclusion/exclusion criteria through a female-focused lens is also important before a trial protocol is finalized. In some cases, regulatory guidelines will dictate limitations. For instance, the age range for female contraceptive studies is 18-35, which regulators consider the “fertile window.” Women older than 35 can be recruited, but they won’t count towards the efficacy of the contraception because fertility is known to drop starting at 35.

Another growing point of discussion in women’s health studies is attention to the Body Mass Index (BMI) of participants, which can have an impact on dosing, safety, and effectiveness. In the example of contraception, women with higher BMI have a higher risk of blood clots, and may see less effectiveness with some forms of birth control. For this reason, a high BMI often leads to exclusion from trial participation.

These issues must be factored into trial design to ensure patient safety and quality results. But we also have to balance the interest and need for data that shows how an asset will work in a population that is truly representative of real-world users – regardless of age, BMI, or other factors. There are considerations for capturing data and segmenting a trial population to gather everything without risking regulatory success or spending budget unnecessarily. For example, running two concurrent trials.

Sponsor-CRO discussions about end-use and long-term marketing plans early in the planning process support a more comprehensive approach to project design and wiser spending.

Patient testimonials

Keeping in mind that the end goal of any trial is to go to market successfully, building in opportunities to glean insights from participants about the perceived benefits of the product can help sponsors shape marketing strategies and develop product testimonials.

Trial participants are a captive audience. Using face-to-face interviews and electronic surveys, we gather thoughts on intent to buy, likes/dislikes, suggested cost, and whether they would recommend the asset to a friend.

This data is also a great tool for future trial planning, as we can learn more about what participants did and did not like about their study experience. Beyond trial design, these insights can inform recruiting strategies and be used in campaigns intended to drive study enrollment.

The Bottom Line

Trial success is dependent on a number of factors, but, most importantly, we have to have a clear understanding of long-term goals and expectations of all stakeholders – sponsor, end-user, investigator, regulator – from the outset. Working backwards from the end goal with all parties in mind will deliver the greatest reward.