Bringing the Voice of Clinical Practice and Patients into Drug Development

Historically, medical affairs was viewed primarily as a post-approval function responsible for scientific communication, publications, and support of marketed products. However, over the past two decades, the function has expanded significantly to include scientific exchange through field medical teams, engagement with external experts and investigators, support of evidence generation, and pre-launch scientific education of the medical community.

Clinical development generated the evidence required for regulatory approval, while medical affairs helped communicate that evidence to healthcare professionals after launch.¹ Today, the increasing complexity of new therapies and the broader healthcare environment are challenging this traditional division of responsibilities.

As therapies become increasingly specialized and healthcare systems place greater emphasis on patient-centered care and shared decision-making, organizations are recognizing the value of incorporating clinical and patient perspectives much earlier in development. Questions related to unmet medical need, treatment burden, patient experience, endpoint relevance, and future evidence requirements could, and arguably should, influence development decisions years before a product reaches the market.

Increasingly, these considerations influence not only the success of development programs, but also the relevance of the evidence ultimately generated for patients, physicians, payers, and healthcare systems.^2-4 This evolution is changing the role of medical affairs.

Increasingly, medical affairs are not simply communicating evidence around and after approval but also helping bring the realities of clinical practice and patient experience into the development process itself. Having worked across commercial, medical affairs, and clinical development organizations throughout my career, I have observed how the questions physicians and patients ask about a therapy often differ from the questions regulators require for approval.

Both are important, but they are not always identical. Understanding these perspectives earlier can help organizations design development programs that are not only scientifically rigorous and regulatorily sound, but also more relevant to the people who are ultimately expected to use and benefit from new therapies.

Beyond Investigator Input: Why the Patient Perspective Matters

Clinical development has always relied heavily on scientific expertise and investigator input. Investigators play a critical role in helping sponsors understand disease biology, clinical practice, study feasibility, and evolving treatment paradigms; however, investigators and patients do not always view clinical trials through the same lens.

Physicians may focus on scientific rationale, unmet medical need, and potential therapeutic benefit. Patients, while equally motivated by the possibility of improved treatment options, often evaluate participation through a different set of considerations.

Concerns about treatment burden, perceived risks, quality of life, family responsibilities, travel requirements, and prior experiences with therapies can all influence willingness to participate in clinical trials or acceptance of a therapy when it is ultimately prescribed in clinical practice following approval.^2-5

As the industry increasingly embraces patient-centricity and patient engagement, there is growing recognition that patient perspectives may provide valuable insights throughout the development process. Understanding how patients perceive disease burden, treatment trade-offs, and participation in clinical research may help development teams identify considerations that are not always evident through scientific, operational, or investigator perspectives alone.

This creates an important opportunity for medical affairs to help bring those voices into development earlier and more systematically.^2-6

Medical Affairs as a Bridge Between Development and the Real World

Medical affairs occupy a unique position within pharmaceutical organizations. Through scientific exchange with healthcare professionals, collaboration with investigators, engagement with patient advocacy groups, and ongoing assessment of disease landscapes, medical affairs develop a broad understanding of how diseases are managed in routine practice and how patients experience those diseases in everyday life.^1,7

As a result, medical affairs are often well positioned to identify gaps between how studies are designed and how therapies may ultimately be experienced by patients and physicians. This role extends beyond traditional scientific communication.

Increasingly, medical affairs contribute insights that can inform evidence-generation strategies, highlight unmet needs, identify barriers to participation, and ensure that development programs remain connected to the realities of patient care. Perhaps most importantly, medical affairs can help ensure that patient perspectives are heard alongside scientific, regulatory, and operational considerations.

Medical affairs can serve as a bridge between external stakeholders and development teams by integrating insights from patients, caregivers, physicians, investigators, and advocacy groups into evidence-generation planning.

When the Missing Insight Came From Patients

The importance of incorporating patient perspectives became particularly clear to me while working on a Phase IIb development program for a promising therapy in an area of significant unmet medical need.

The study was designed not only to evaluate the investigational therapy against placebo but also to include an active comparator arm. This approach was scientifically important.

In addition to supporting development decisions, it would provide an earlier understanding of how the therapy might perform relative to the existing standard of care and help inform future Phase III study design. Before finalizing the protocol, the team sought input from experienced investigators and selected highly regarded sites with deep expertise in the disease area.

The study design was considered robust and thoughtfully constructed. Despite this preparation, recruitment quickly became a major challenge. After approximately six months, 11 of the 12 participating sites had enrolled few or no patients.

As is often the case, attention initially turned toward protocol modifications. Clinical operations teams proposed broadening eligibility criteria, including expanding age ranges and disease duration requirements, in hopes of increasing the pool of eligible participants.

However, there were concerns that doing so could compromise the scientific objectives of the study. Patients with more advanced disease might be unlikely to respond to treatment, potentially making it more difficult to accurately evaluate the therapy's true potential.

At the same time, discussions with investigators yielded little clarity. Each site offered a different explanation for the recruitment challenges. While these perspectives were valuable, no consistent pattern emerged.

At that point, the discussion shifted from understanding investigator perceptions of the recruitment challenges to understanding how potential participants themselves viewed the study. While extensive investigator input had informed the study design, the perspectives of patients who would ultimately be asked to participate had not yet been explored directly.

Working with a market research partner, a survey was developed targeting patients who closely matched the study's inclusion and exclusion criteria. Participants were asked to review key aspects of the protocol and provide feedback regarding their willingness to participate.

The findings were revealing. The primary barrier was not visit frequency, eligibility criteria, or concerns about receiving placebo.

Instead, patients consistently identified concerns regarding the active comparator arm. Within the patient community, a particular adverse effect (AE) associated with the comparator therapy was widely recognized and strongly feared.

Interestingly, this concern had not emerged consistently through discussions with investigators, despite extensive engagement with experienced sites. The patient survey revealed a common theme that had remained largely invisible through traditional channels of feedback.

The organization now faced a dilemma. Removing the comparator would have simplified recruitment, but it would also have reduced the study's ability to generate information critical for future development decisions and understanding the therapy's potential place in the treatment landscape.

Instead, the team chose a different path. The comparator arm was retained, preserving the scientific integrity of the study.

At the same time, the team developed patient education materials and site training resources explaining the actual incidence of the AE, its reversibility, and the procedures that would be followed if symptoms occurred. Additional site training helped ensure that these discussions were conducted consistently and accurately.

The impact was substantial, as enrollment accelerated and the study completed recruitment within approximately three months.

Had the team relied solely on traditional recruitment metrics and investigator feedback, the protocol may have been amended in ways that could have altered the study population and potentially affected interpretation of the results. Patient insights revealed that the true barrier was not eligibility criteria but perception of risk.

This experience reinforced an important lesson: understanding patient perspectives can reveal barriers that may not be apparent through investigator feedback alone and can identify solutions that preserve both scientific rigor and operational feasibility.

Bringing Patient-Centricity into Development Earlier

Patient-centricity has become a common aspiration across the industry; however, achieving it requires more than patient-friendly language or recruitment materials. It requires incorporating patient perspectives into decision-making earlier and more systematically.^2-5

Patients and caregivers can help development teams understand which outcomes matter most in daily life, how disease burden affects decision-making, what risks are considered acceptable, and which aspects of treatment have the greatest impact on quality of life. Importantly, these perspectives influence far more than recruitment.

They can help inform endpoint selection, patient-reported outcome strategies, study feasibility assessments, evidence-generation priorities, and post-approval research plans. They can also help ensure that the evidence generated during development remains relevant to the decisions that patients and physicians face in everyday clinical practice.^2-6

As expectations for patient-centered development continue to grow, organizations that integrate these perspectives earlier may be better positioned to generate evidence that is both scientifically meaningful and relevant to real-world practice.

Looking Ahead

The future of drug development will require closer integration of scientific, clinical, operational, and patient perspectives. Regulatory approval will remain the primary objective of development programs.

However, achieving approval is only one step toward improving patient outcomes. Ensuring that development programs generate evidence that is meaningful to physicians, patients, and healthcare systems requires a broader understanding of clinical practice and patient experience.

Medical affairs have an increasingly important role to play in this process. By helping bring the voices of physicians and patients into development earlier, medical affairs can serve as a bridge between scientific innovation and the realities of patient care.

The goal is not to replace scientific or regulatory decision-making, but to enrich it with perspectives that are often underrepresented during development. As therapies become increasingly complex and patient expectations continue to evolve, organizations that successfully integrate these voices may be better positioned to generate evidence that is not only approvable, but meaningful, usable, and ultimately capable of improving patient outcomes.

About the Author

Natalia Borinshteyn, MD, PhD, is Founder and President of Life Science Excellence Inc., a boutique strategic consulting firm supporting pharmaceutical, biotech, and medical device companies. She brings more than 20 years of experience across clinical development, medical affairs, and commercial strategy, and has contributed to the development and launch of multiple innovative therapies across dermatology, immunology, gastroenterology, diabetes, and HCV.Previously, Dr. Borinshteyn served as Vice President of the HCV Launch Team at AbbVie, where she led Global and US Medical Affairs and played a key role in launch readiness and execution. Earlier in her career, she held leadership roles at Sanofi across clinical development, portfolio strategy, and commercial operations.Dr. Borinshteyn holds an MD and PhD in immunology and completed executive education at The Wharton School and Harvard Business School.

References

1. Medical Affairs Professional Society (MAPS). The evolving role of Medical Affairs across the product lifecycle.
2. U.S. Food and Drug Administration. Patient-Focused Drug Development: Collecting Comprehensive and Representative Input. Guidance for Industry. 2020.
3. U.S. Food and Drug Administration. Patient-Focused Drug Development Guidance Series. Available at: www.fda.gov.
4. Clinical Trials Transformation Initiative (CTTI). Effective Engagement with Patient Groups Around Clinical Trials.
5. Perfetto EM, Oehrlein EM, Boutin M, et al. Value to whom? The patient voice in the value discussion. Value in Health. 2017;20(2):286-291.
6. Levitan B, Getz KA, Eisenstein EL, et al. Assessing the financial value of patient engagement. Therapeutic Innovation & Regulatory Science. 2018;52(2):220-229.
7. IQVIA Institute. Medical Affairs 2030: Transforming Scientific Engagement and Evidence Generation.