Harnessing RWE in rare diseases

Ahead of eyeforpharma Philadelphia 2019, Sonal Bhatia, Pfizer’s vice president, North America medical lead, rare disease talked to us about the importance of collaboration and RWE to her field.

What are your career highlights and tell us what attracted you to this latest role?

I have been within Medical Affairs for the last 14 years, driving medical strategies for medicines across the enterprise. My focus started in diabetes and cardiovascular.

In the last few years, I have shifted to rare diseases, where my passion is to help find and bring transformational and innovative medicines to patients who have rare diseases, working with physicians, patient advocacy groups, payers and regulators to bring the best medicine to our patients.

“There may be misconceptions in the medical literature about what affects patients at different stages of the disease. This could lead to assumed clinical facts about that disease that may be wrong”

As your role involves working in rare diseases, could you tell us about how important it is for medical, commercial and outcomes research teams to work together?

In rare diseases, the registrational trials tend to be much smaller in size than a typical Phase 3 CV trial, primary because the population is much smaller. As such, it is critical for medical to work in close partnership with commercial and outcomes research to understand what are key data gaps and help generate meaningful data through real world evidence (RWE) that will allow our payers , regulators and physicians to build confidence around their decisions for treatments for patients with rare diseases.

What are the main challenges towards gathering RWE in rare diseases?

Often the patient journey is different for individual patients with the same disease, coupled with a small sample size that could lead to a sample size bias. It is also critical to get longitudinal data to truly understand the patient journey, which often requires longer prospective trials.

What are the benefits to gathering this evidence to the rare disease drug development process?

RWE in the early part of the drug development process is critical to garner information on the rare disease. The medical literature typically has minimal information, with only few studies having been conducted (outside of clinical surveys) and there may be misconceptions in the medical literature about what affects patients at different stages of the disease. This could lead to assumed clinical facts about that disease that may be wrong. It is critical to break through misconceptions and increase the level of data/knowledge around a patient/disease journey.

What uses does RWE have when it comes to interaction with regulators and payers?

RWE is playing a more critical role in today’s world, where regulators and payers are looking for additional data to help support registrational studies and show the evidence of how the medicine is working from an effectiveness and safety lens in the real world settings. In a post launch era, RWE helps to address cost effectiveness of the medicine in the real world setting that can be of enhanced value to the payers.

Do you think the need for RWE could lead to the end of the “silo” structure that is so common in pharma?

I think collectively it is our responsibility to showcase the importance of RWE so that we don’t get caught up in these silos, and lean on such critical data to help our physicians have the data to make informed decisions for their patients.

What kind of skills are important to develop the medical co-leadership required to generate and effectively use RWE?

The key skill sets are a collaborative and “end customer” mindset. This will allow the teams to truly understand what the customer needs, and work backwards with a cross functional team to develop the right data for the right customer.

About the interviewee

Sonal Bhatia is the vice president, medical affairs, and North America lead for rare disease at Pfizer. Sonal is an active speaker nationally and internationally, where she has shared her thoughts around how patient centricity and real world evidence can effectively usher in a new era for medical affairs. Prior to her current role, Sonal was a senior medical director, team lead at Pfizer.