Trained as an emergency medicine physician, Yin Ho, M.D., M.B.A., stepped out of the emergency room in 1998 and into the emerging eHealth industry, impelled by the promise of health care innovation driven by new access to data and information.
Today, with 20+ years of experience working in health information technology and providing technology solutions to biopharma in clinical development, market access, reimbursement, and regulatory submissions, she is a recognized leader in data and analytics-driven decision-making, inclusive of real-world evidence (RWE).
After many years of experience in internet startups, pharma, and software, Dr. Ho founded and led Context Matters, Inc., a data analytics software company whose flagship product (Market Access Platform) was used by global health economics and market access teams to support their understanding of what data and analyses both HTA (reimbursement) and regulatory agencies used to make their cost-effectiveness and comparative effectiveness value determinations. And it was in that context that she noticed an increasing interest in RWE inquiries from biopharma.
After a successful exit sale of Context Matters, she joined Aetion and now serves as Chief Product Officer. Her time working in and with biopharma has afforded her unique insights into how real-world data (RWD) can be used to generate decision-grade RWE, and the context of how this data will be utilized and accepted. She shares her insights below.
Responses have been edited for clarity and length.
Q: How have you seen adoption of RWE in biopharma evolve over your career?
A: RWE as a concept has been around for a long time, and pharma has been flirting with RWD for quite some time. While RWD was initially narrowly defined as electronic medical records, the explosion of real-world data sources over the last ten years—smartphones, wearables, patient registries, and genomics—drove an expansion in the definition of RWD.
Pharma is familiar with various RWD sources, as companies often use them to track what happens to a drug after it is introduced into the market. Traditionally, however, the sources used were mainly to support late-phase, post-marketing studies.
There has always been concern that secondary uses of data come fraught with challenges. The primary concern is whether the conclusions drawn from a data set collected for one purpose are valid for another use case.
Additionally, the concept of proving causation has always been a bit controversial. The general thinking is that it is hard enough to prove association, never mind correlation, and rarely for causation. When I first came to Aetion, I winced whenever I heard the word “causation.” I had been trained for so long—as anyone who has worked in or around pharma has—to be extremely careful when using the word “causation.” The conventional wisdom is that causation is something you rarely can reach—at best you could get to correlation. Reputable public health schools do not all agree on whether causation is something that truly can be proven, even if you remove as many confounders as you can.
This is where Aetion is breaking new ground and developing scientific methodologies that address this controversy. The platform is specifically built to enable and run causal analyses, defined by cutting edge methods to address confounders. And pharma’s comfort levels with RWE have also increased, particularly as they see more observational data become available to support the drug development process.
Q: What opportunities do you see for biopharma as we prepare for guidance on RWE from the FDA and other regulatory bodies?
A: Empowerment and the comfort of greater certainty. Biopharma has an opportunity to make use of tools that will provide a better understanding of what their drug might be up against in the market, how it may perform in the real world, and what opportunities and challenges may lie ahead. Basically, if I know that there’s a lot of data being collected about my drug in various places, I would want to use it to my advantage.
Whether it’s a decision to accelerate a clinical trial submission and get to an answer faster, or the difficult determination to shelve a product quicker, judicious use of RWE is preparing biopharma with the data, information, and knowledge to optimize the development process.
Q: What are the main challenges you’ve seen or heard from teams trying to implement an RWE program?
A: It’s critical to think through where RWE sits within an organization, as it gives you a clue as to the value placed on RWE within that organization. It also lets you understand the receptivity of the overall organization to the kind of decisions RWE can render or be used to support.
I have seen RWE sit in a “center of excellence” model at the organizational level, and I have also seen RWE as a branch of a smaller group—within analytics, health economics and outcomes research, or market access. If RWE is just a branch group, there's only so much the team is going to be able to achieve, and their role will be to constantly educate other stakeholders.
Q: Are there any strategies you’ve seen work better than others for ensuring RWE gains traction?
A: It’s important for teams to first evaluate the barriers and challenges for RWE within their organization: What is RWE’s relative influence internally? Which teams and therapeutic areas are best suited to use RWE? Which drugs in the pipeline will most benefit from RWE in assessments? Which potential roadblocks could slow progress?
Additionally, it’s worthwhile to identify which groups at an organization have the more promising portfolio and pipeline, who is the most open to RWE adoption, and which drugs could most benefit from gaining a competitive advantage or being the first to market.
Adopting innovative technology within the walls of pharma can be challenging. If you use a top-down approach, you need to have enough people within the organization who agree with you and are motivated to drive the initiative forward as a key imperative. This enables them to be more open to experimentation. High visibility discussions about RWE can help, too. When done thoughtfully and carefully, a top-down approach can build support for RWE as it moves through an organization, but making RWD and RWE easy to work with at the user level and at the team level will determine the long-term success.
As for qualities of the RWE teams themselves, effective RWE teams pilot new ideas and technology. They are staffed with colleagues who have thought through their goals for RWE, what they want to learn, and championed the path to get there. Importantly, a successful RWE team has gone through the extra step of defining the specific, tangible benefits accrued to each department within an organization to generate true buy-in.
It’s also important that an RWE team has a straightforward path to gaining cross-collaborative alignment. Not surprisingly, it can be difficult for those who are reliant on buy-in from multiple stakeholders to be effective; the involved process of achieving buy-in from many can slow momentum.
A good pilot project for any team involves clear metrics and a clear path to ensure that what has to be done can be accomplished quickly. And there must be some level of agreement (or even an idea) on what will follow a successful pilot.
Q: Where are the opportunities and challenges for RWE adoption across the enterprise?
A: We’re still educating people about RWE, and we need to be honest about its real potential and any real pitfalls. What are the problems that we cannot solve today, but which we believe we can solve with new sources of data and RWE? When we have that honest dialogue, it becomes a bit easier to assess where RWE can amplify biopharma’s processes.
At Aetion, we look to determine where and when RWE is fit to answer a given research question, and which types of RWD are best suited for an analysis. Sometimes, RWE is insufficient to answer a particular question, but the analyses can, for example, complement an RCT to paint a fuller picture of a drug’s overall impact. It’s important to communicate that distinction.
It is simply a matter of time until we have more established pathways to use RWD and RWE to support biopharma and other stakeholders’ decisions. The industry is already moving in the right direction.