Blog - medidata

Real World Evidence generation at the heart of Medical Affairs

MEDAFFAIRS Leaders Forum Spring
Key topics summary

This 9th edition of MedAffairs Leaders Forum, held in London at the end of February, featured high-level speakers, particularly inspired by the underlying theme of how to enhance the value of their products in order to bring the maximum benefit to "real-world" patients. Several presentations highlighted the incredible growth of the collection and analysis of real-world data, the unique method to take a full picture of a drug’s impact on the life of patients. 
Medineos was glad to attend the event and thus we want to share three main essentials topics from this forum

New trends in Real World Evidence generation

What is undoubtedly clear after listening to the presentations, is that lifescience companies are undergoing a real ‘evidence revolution’. Real-world evidence is breaking out from the confines of market access, as it was previously limited to, and is infiltrating every part of the life of medicine, from discovery to the very late stage.
This revolution has, in part, been driven by technology – the availability of electronic patient level data and the ability to analyze this data by means of artificial intelligence algorithms – and, in part, by the market, as different stakeholders ask for more reliable proof of the value of new medicine as Jacek Nowak, Executive Medical Director, Amgen reminded us.
As a matter of fact, regulators and payers want more RWE for different reasons. RWE analysis will complete conventional RCT data with additional insights from patients who reflects real-world practice, resulting in better understanding of safe and effective use of therapeutic innovations. Christoff Koenen Head of Cardiovascular Development, BMS, said that the use and role of RWE is becoming more and more important due to the 21st Century Cures Act signed by Obama. This act requires the FDA to develop a guidance for real-world evidence to support approvals of new indications for previously approved drugs, and to support or fulfill post-approval study requirements. To complete this ambitious plan, the FDA is developing a framework for a RWE program in cooperation with the drug industry within 2 years. The expected outcome, within 5 years, is a draft guidance on when RWE can be used, as well as standards and methodologies for collecting and analyzing RWE. These are the first signals of future improvements in methodologies and regulatory sciences related to RWD use. Even though real-world evidence based on real-world data is neither a solution nor a replacement for clinical trials, it is expected to bring significant changes to the healthcare industry.

Medical Affairs as the new key player in Real World Evidence

That change, already underway, requires a shift by Pharma Companies from just being competent on the product and disease to becoming masters in effectively communicating clinical evidences through the interpretation of scientific data. The Medical Affairs Dept seems to play a central role in taking charge of interpreting and communicating the proper value of their products. These departments can link scientific and clinical results to patients outcomes and simultaneously gathering feedback from the "real world" generating useful insights for future development plans. It is of furthermore importance to break the "silos rules" in order to leverage partnerships among departments throughout the whole product lifecycle. As shown by Seema Heider, Senior Director Group Lead PHI HEOR, Pfizer, of particular importance is a close cooperation between HEOR and MA teams to merge together methodological knowledge, clinical awareness, and proper multi-channel, multi-stakeholders tailored tools to effectively communicate the evidence generated throughout the product life-cycle . In this context, Medical Affairs teams are the ideal first point of contact in discovering a patient’s unmet needs, as they are in the privileged position of sending internal advice about how to incorporate the patient’s voice in company decisions with greater frequency. Erik Briers, Patient Advocate, EMA CAT, believes a more patient-centric view is still needed. Companies should use "clever" patients as a resource in their clinical development, creating a sort of "Patients’ Advisory Group" by analogy with the more consolidate "Physicians’ Advisory Group".  Companies should increase their awareness of all relevant stakeholder’s needs. This will help companies focus more on clinical outcomes not only perceived by the physician, but also in a broader audience (patients, payers, regulators, caregivers etc.).

The workflow approach to Real World Data projects design

To succeed in this value-driven environment, companies should adopt a multidisciplinary approach to  generate clinical evidence of their products throughout the whole lifecycle.
Using the Project Management Institute Guidelines, companies should begin a conceptual shift from a pure clinical and methodological approach, to a technique which encompasses different steps during the RWD project design. As a first step, companies engaged in generating clinical evidence should be forced to take into account the strategy and business needs leading the entire project. Medineos relies on a step by step approach tested over the years on many different RWD projects.
The main lesson learned is that the design of an observational study is not only about writing a Study Protocol. This is the conclusion, and the easiest step of the whole process. Identifying a "finer" study question and prioritizing expectations ("reasons why"), in full compliance with the diverse regulations and definitions of "Non Interventional Studies-NIS", requires knowledge, experience, focus, and discipline. The 4-step project design work-flow (Listen, Shape, Focus and Define), as stated by Giovanni Fiori, Medineos Managing & Scientific Director, is one of the methods that can support complex multi-disciplinary teams to consider the best trade-off among desirability, viability and feasibility of RWD projects.
To conclude, the promise of Real World Evidence is to enable companies to respond to the new and ever more sophisticated demands from HTA, payers, and regulators as a consequence of the increase in drug prices due to both the clinical development costs, and the even more personalized treatments requirements.  In order to be able to surf this wave of innovation, companies are required to go "beyond the pill" in communicating the value of their products. The challenge is to increase the structure flexibility within pharmaceutical companies in order to adopt cross-functional teams. This change will allow companies to more accurately disseminate the value of their products, thereby improving the channels of communication to their stakeholders.


If you wish to further discuss any of these topics or deepen your knowledge of observational research, please contact us at