Contributed by SME: Jean Cunningham, PharmD, BCPS, Senior Director of Medical Affairs, Empower Pharmacy

In healthcare, randomized controlled trials remain the gold standard for evaluating safety and efficacy. Pharmacists and other healthcare professionals should continue to advocate for high-quality evidence whenever possible.

However, those who work in compounding pharmacy face an often-overlooked reality. The evidence that licensed healthcare providers need does not always arrive in time, and sometimes it does not arrive at all.

Clinical trials take years to complete and require significant financial investment. While this model works well for commercially manufactured medications with large patient populations and pharmaceutical sponsors, it does not always align with the world of compounded therapies. Many compounded medications serve niche patient populations, address individualized clinical needs, or represent emerging areas of practice where large-scale clinical trials may never be financially feasible.

At the same time, healthcare providers and patients cannot always wait.

Every day, healthcare providers face patients who have exhausted standard options, require customized dosing, need alternative formulations, or seek therapies that are still evolving within clinical practice. These providers must make treatment decisions based on the best information available today, not the evidence that may become available years from now.

The question is not whether evidence matters; it does. The real question is how healthcare providers responsibly navigate uncertainty when the highest level of evidence is unavailable. This is where Medical Affairs plays an increasingly important role.

Historically, Medical Affairs has been associated with pharmaceutical manufacturers, serving as a scientific bridge between research, healthcare providers, and commercial organizations. Yet the need for this function may be even greater within compounding pharmacy.

Medical Affairs professionals help healthcare providers navigate the space between what is known and what is still being learned. This includes evaluating published literature, identifying relevant case reports, reviewing observational data, analyzing treatment patterns, and providing balanced scientific context around emerging therapies.

These insights do not replace clinical trials, nor should they. Medical Affairs helps ensure that clinical conversations are grounded in the best available information, with appropriate transparency about what is known, what is uncertain, and where evidence gaps remain. 

That connection matters. 

Compounding pharmacies often interact with thousands of prescribers across multiple specialties. Through medical information inquiries, educational programs, conference participation, and ongoing provider engagement, Medical Affairs teams gain unique visibility into how therapies are being discussed and utilized in real-world practice.

These insights can help identify emerging treatment patterns, common prescribing approaches, practical implementation challenges, and areas where additional research may be warranted. In this way, Medical Affairs becomes more than a support function. It becomes the connective tissue between published evidence and clinical reality.

As personalized medicine continues to expand, compounding pharmacies have an opportunity to move beyond simply dispensing medications. By investing in robust Medical Affairs functions, organizations can provide healthcare providers with evidence-informed support, facilitate scientific exchange, and navigate uncertainty by bringing scientific rigor and clinical context to the decision-making process.

The future of compounding will depend on stronger evidence generation, more research, better data collection, and continued scientific evaluation. Until then, healthcare providers deserve trusted partners who can help them interpret the evidence that exists today while remaining transparent about its limitations.

Medical Affairs was built to fulfill this role. In a field defined by individualized care, Medical Affairs’ engagement is essential.

References

Angus, D. C., et al. (2024). The integration of clinical trials with the practice of medicine: Repairing a house divided. JAMA.

Frieden, T. R. (2017). Evidence for health decision making — beyond randomized, controlled trials. New England Journal of Medicine.

Reig, S., et al. (2026). Reimagining medical affairs through strategic leadership in patient partnering healthcare. Drug Discovery Today.