Importance of real world evidence in post market surveillance and launch success

In an increasingly competitive disease, drug and technology landscape, winning payer and provider choice can be difficult. The following marketplace conditions can make gaining this choice even more challenging:

• The ever-evolving regulations for medical device manufacturers make staying current with regulations a full-time endeavor.
• Gaining access to a health system or obtaining coverage can be the key to actualizing a new technology’s benefit before the window of competitive advantage closes.  
• Healthcare’s shifting focus to a more patient-centered approach acknowledges the variety of factors that can influence how a drug or medical device interacts with a patient’s life.
• By shining the spotlight on the patient story using real world evidence (RWE) and real world data (RWD), manufacturers have an opportunity to gain an edge in the market.

The importance of RWE and RWD

As healthcare evolves, manufacturers should consider placing greater emphasis on the growing importance of RWE and RWD in crafting a value story. RWE and RWD, as defined by the U.S. Food and Drug Administration (FDA), is “Healthcare information derived from multiple sources outside of typical clinical research settings, including electronic medical records (EMRs), claims and billing data, product and disease registries and data gathered by personal devices and health applications.”1 The FDA can then use this data to monitor the safety and efficacy of products and make regulatory decisions.

The limitations of traditional clinical trials are becoming more apparent and supplementing trial data with RWE can allow manufacturers to collect data that are more representative from a larger, more inclusive patient population. Planning for how this data will be collected and what role it will play in the pre/post approval process can accelerate adoption of the technology by payers or as part of a health system’s preferred offering.

It’s not only the FDA that is interested in using RWE to understand the value of a new device or technology. Payers also look to RWE to support the efficacy claims of a new therapy or device. Having demonstrative evidence of the cost saving or therapeutic benefit of a product can significantly affect its commercialization and coverage success.

Payers are increasingly stressing the importance of outcomes and value in their review and assessment for coverage of pharmaceuticals, medical devices and diagnostics. Medical devices have always been subject to RWE documentation from the FDA, and more than ever, payers and provider organizations are requiring RWE through the use of product experience programs or patient registries that supplement evidence obtained through clinical trial.  

In particular, medical device regulations have grown increasingly stringent in their requirements for RWE and post-market safety data. With the advancement of manufacturing technology, medical devices have embedded themselves in the treatment or management of almost every disease area. Consider the role technology is playing in extending clinical data collection into the patients’ home. With wearable technology and smartphone applications, data is being collected continuously and adherence information is more reliably gathered.

Why use RWD?

RWD can be deeply impactful in successfully negotiating access, securing value based contracting with payers and driving downstream sales. Payers and physicians alike are stressing the importance of data from varied sources, citing that clinical trials alone are insufficient to understand the impact of a drug or medical device.

In a controlled environment such as clinical trials, sponsors can enforce adherence and offer a more structured environment for drug testing. In the real world, external factors such as lack of adherence, environmental factors and potential impact on quality of life play a more significant role in how the drug or technology affects outcomes and disease progression. Sometimes, despite efficacy, there may be a cost story which cannot be derived from a clinical trial alone that needs to be fleshed out in order to gain payer coverage.

The increase in demand for RWE/RWD is apparent when looking at recent movements in the industry. Consider the following:

• A survey from ConvergeHealth found that 90 percent of global pharma companies have implemented or are implementing RWE/RWD analytics.
• The highest priority for attendees at the American Society of Clinical Oncology (ASCO) 2019 Conference was developing robust strategies to obtain RWE2.
• Pfizer’s cancer drug Ibrance gaining additional FDA approval owing to RWE3 and Roche’s acquisition of Flatiron Health4 are a result of RWE/RWD planning.  
• An increasing number of pharma and device manufacturers with commercially available brands are considering implementation of an early experience and/or patient registry program and patient chart reviews to establish RWE that produces clinical and economic benefit for stakeholders.

Channels to collect data

Recent modernization of electronic health records (EHRs) has given the opportunity to use claims as a form of RWD and to analyze patient costs and outcomes. Claims records can reflect the impact of a new therapy by analyzing utilization and costs before and after the implementation of the therapy. This analysis can supplement and sometimes even supersede clinical trial evidence, particularly when the need is to demonstrate cost savings. A claims analysis allows access to a real world story about the implementation of the drug or device, rather than the controlled environment of a trial.

Another novel pathway for collecting such data is conducting a pilot program with self-insured employers. A partnership with an employer allows for the identification of a patient population for the sponsor and allows the employer to control costs of care for their employees through measurement of standards and metrics that align to their business objectives. This mutually beneficial partnership allows for the collection of data on a therapy or device in a population that is easier to identify and enroll. It also gives employers the platform to be innovative and seek out novel therapies, while providing a forum for the sponsor to collect real world evidence.

Baker Tilly advises on various avenues for collecting real world evidence such as claims analysis, patient registries and pilot programs with self-insured employers. We help companies use RWE to leverage the power of their technology and utilize RWD to support their differentiation story.

For more information on this topic, or to learn how Baker Tilly specialists can help, contact our team.