Evidence generation in medical affairs represents a systematic approach to producing data that demonstrates the value of products or interventions in real-world settings. Unlike traditional clinical trials and randomized controlled trials (RCTs), which operate under controlled environments with stringent protocols, evidence generation incorporates a broader array of methodologies, including observational studies, real-world evidence (RWE), and qualitative research. This approach evaluates treatment performance in everyday clinical practice, addressing patient outcomes and healthcare needs. For instance, in the therapeutic area of diabetes, traditional clinical trials have shown sulfonylureas' efficacy in controlling HbA1c levels under strict conditions. However, RWE highlights a contrasting reality where patients often intentionally skip doses due to concerns about hypoglycemia. Insights such as these showcase the importance of evidence generation as a complementary tool to traditional trials, enabling treating physicians to make more informed and relevant clinical decisions while enhancing patient care in real-world scenarios.

Medical affairs teams are at the forefront of this transformation. They are responsible for ensuring that the value of a product is understood and appreciated by a diverse range of stakeholders, including healthcare providers, payers, regulators, and patients. To achieve this, they must generate robust evidence that is relevant, reliable, and actionable.

The Role of Evidence Generation

A well-executed evidence-generation strategy can:

1. Support regulatory submissions and approvals by providing the necessary data to secure approvals and market access.

2. Equip HCPs with the knowledge and tools to make informed treatment decisions.

3. Influence reimbursement decisions by demonstrating the cost-effectiveness and value of a product to payers.

4. Improve patient outcomes by identifying real-world challenges and opportunities to optimize patient care.

Medical affairs has a significant responsibility to not only ensure the scientific rigor of generated evidence, but also its relevance for diverse stakeholders including providers, payers, regulators, and patients. This priority is highlighted in McKinsey's Vision for 2030, which calls for MA teams to shift to new evidence-generation strategies that are more coherent, adaptable, and efficient.

Many leaders make evidence generation in silos rather than for the needs of the wider organization and stakeholders. McKinsey highlights that only 20% of leading pharmaceutical companies develop an integrated evidence generation plan across the life cycle of a product.  This lack of a holistic approach hinders the ability to generate meaningful, actionable evidence that supports decision-making, ensures regulatory compliance, and meets the needs of end-users.

This article will explore how medical affairs teams can drive change by coordinating the evidence generation process to best meet stakeholder needs by leveraging the latest approaches, strategic thinking, and technology.

Building an Integrated Evidence-Generation Strategy

Stakeholders in the healthcare ecosystem have varying priorities and require evidence that is relevant to their specific roles and responsibilities. For example, healthcare providers prioritize data that improves clinical decision-making, while payers and regulators focus on the cost-effectiveness and safety of treatments. Patients, on the other hand, value accessibility, transparency, and outcomes that directly improve their quality of life. When developing a strategy for evidence generation, all perspectives must be taken into account. 

To meet these diverse perspectives, medical affairs teams must:

• Develop tailored evidence generation plans aligned with stakeholder-specific needs

• Anticipate and address stakeholder questions and concerns

• Present evidence in formats that are accessible and actionable for different audiences

This coordinated approach ensures MA delivers robust data that supports informed decision-making across the healthcare ecosystem.

Key Steps for Integration:

1. Unifying Internal Leaders: Bring together teams from R&D, commercial, and regulatory functions to develop a cohesive evidence-generation plan.

2. Highlight the Importance of RWE by Upskilling Teams: Empower these cross-functional teams with the knowledge and skills to effectively leverage RWE in evidence-generation planning and execution.

3. Mapping Stakeholder Priorities: Identify the evidence needs of all stakeholders, from healthcare providers to patients, and ensure these are addressed in the plan.

4. Sequencing Evidence Delivery: Create a timeline that ensures a steady flow of evidence to stakeholders throughout the product lifecycle.

ACMA supports medical affairs professionals with the skills and knowledge of building and integrating evidence generation strategies in the Board Certified Medical Affairs Specialist (BCMAS) program, which includes a dedicated RWE module developed with insights from industry leaders. This equips MA professionals with the skills to create targeted evidence meeting the needs of providers, payers, and patients - aligning with McKinsey's call and vision for Medical Affairs by 2030 for cross-functional, lifecycle-based evidence planning.

Innovative Approaches for Faster Evidence Generation

In today’s fast-paced healthcare environment, the ability to generate evidence quickly is essential. Advances in AI and analytics are enabling MA teams to accelerate this process while maintaining scientific rigor. For example, AI can be used to scan databases for early signals, generate hypotheses, and design trials. These methodologies allow MA teams to produce high-quality evidence more efficiently, even in complex therapeutic areas. In the future, pharma companies are expected to leverage innovative approaches and these tools more effectively.

Examples of Innovative Approaches:

• AI-Driven Evidence Analysis: Tools like medaffairsAI use machine learning algorithms to analyze large datasets, identify trends, and generate actionable insights.

• Real-Time Data Integration: Platforms that aggregate data from multiple sources, including RWE and clinical trials, enable MA teams to deliver timely evidence.

• Scalable Methodologies: Techniques that can be applied across therapeutic areas ensure consistency and efficiency in evidence generation.

MA teams can respond more effectively to emerging challenges and opportunities, ensuring that their evidence-generation efforts actually remain relevant and impactful.

How to Tailor Evidence Presentation for Stakeholders

Generating high-quality evidence is only part of the equation; presenting it in a way that resonates with stakeholders is equally important. McKinsey highlights the need for tailored evidence packages that meet the specific needs of different audiences. For instance:

• Healthcare Providers: Use interactive dashboards and visualizations to make clinical data more accessible.

• Payers and Regulators: Provide detailed reports with robust economic models and long-term safety data.

• Patients: Simplify complex information into clear, actionable insights that address their concerns.

Making evidence more accessible allows stakeholders, including clinicians and payers, to use data effectively in their decision-making processes, enhancing the overall impact of MA-generated insights.

Addressing Challenges in Evidence Generation

Despite the huge potential of evidence generation,  evidence generation presents unique challenges due to regional and systemic factors. Within the healthcare ecosystem, a significant obstacle lies in the lack of interest and time among healthcare providers (HCPs) to actively participate in evidence generation. Although many HCPs acknowledge the value of generating real-world data, they often fail to perceive its long-term benefits in improving patient care. Furthermore, within the realm of medical affairs, the lack of standardized expertise remains a critical barrier. While medical affairs teams are generating data, the absence of a systematic approach to evaluate its quality or impact often results in a trial-and-error process. This issue is compounded by regional complexities, regulatory requirements, and the need for compliance with international standards, which can be both time-consuming and demanding. Cultural stigmas, such as those surrounding rare genetic diseases, further hinder data collection and patient participation in such studies. Some of the major concerns that the MA teams have to deal with include data quality, confidentiality, and compliance with the set regulations. Also, the increasing complexity of the therapeutic areas and the increasing expectations of the stakeholders make evidence generation a complex process.

ACMA assists MA professionals in tackling these challenges by offering training and educational programs such as the BCMAS certification which provides knowledge on data integrity, compliance, and advanced evidence-generation strategies to help professionals overcome these challenges.

Conclusion: A Path Forward for Evidence Generation

In today's rapidly evolving healthcare landscape, evidence generation has emerged as a critical tool for medical affairs teams. It's no longer enough to rely solely on traditional clinical trials; real-world data and patient insights are essential to inform decision-making and improve patient outcomes.

Through continuous learning and the application of digital AI tools, ACMA equips MA professionals with the knowledge and resources to excel in evidence generation. As the healthcare landscape continues to evolve, the ability to generate and deliver stakeholder-focused evidence will define the success of medical affairs in shaping the future of patient care.

References:  McKinsey & Company. A vision for medical affairs 2030: Five priorities for patient impact. Oct 2023. https://www.mckinsey.com/industries/life-sciences/our-insights/a-vision-for-medical-affairs-2030-five-priorities-for-patient-impact 

Richard Chandler. Clermont Group. How the healthcare industry can harness artificial intelligence. https://www.clermont.com/download/Chairman's%20Letter%202024-06_v14.pdf

McKinsey & Company. A vision for medical affairs in 2025. Apr 2019. https://www.mckinsey.com/

Setting New Benchmarks: Trends & Standards in Medical Affairs & MSL Organizations.  Sept 2023. https://medicalaffairsspecialist.org/resources/webinars/new-trends-and-standards-in-medical-affairs

Medical Affairs, Evidence Generation and Bridging the Gap: Leading the Way in the Middle East & Gulf Region. ACMA Webinar. Nov 2024.https://medicalaffairsspecialist.org/resources/webinars/evidence-generation-in-medical-affairs