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Covid-19: The RWE tipping point we’ve been waiting for?

Despite investment in RWE, many life sciences leaders question how and when RWE influences medical value assessments.

Consequently, we find a bifurcation across the industry around how much effort and investment in real-world evidence is warranted. For some organizations, the 21st Century Cures Act signaled that investment in RWE would yield positive ROI—if FDA accepts RWE, non-regulatory decision-makers would follow suit. But other manufacturers are in a wait-and-see period, waiting for clear guidelines and consistent customer commitment to accepting RWE studies. With so much uncertainty about how payers, providers, clinical societies, and health technology assessment (HTA) organizations use RWE, we have yet to reach a tipping point on RWE acceptance.

We think Covid-19 could be the catalyst to change that.

The pressure to diagnose, track, and treat—and ideally vaccinate—the population in order to end the pandemic has caused all stakeholders soften their stance around historic concerns about privacy, data sharing, and evidence generated outside of an RCT. FDA is ready to make concessions about the strength and amount of evidence required for approval—evident through FDA’s authorization for emergency use of chloroquine and hydroxychloroquine for Covid-19, despite limited, vetted evidence.

Frontline HCPs are relying on early-stage clinical data and anecdotal evidence from their counterparts around the world to inform challenging treatment decisions and decide which patients to enroll in clinical trials. Progressive payers and providers (such as Oscar Health and Massachusetts General) are creating symptom trackers and diagnostic tools to collect RWE about their populations, to understand symptom progression, and to detect at-risk individuals and direct them to testing sites. Patient groups and research organizations are also creating disease-specific registries to understand Covid-19’s impact on patients with different conditions.

So while FDA’s guidance on RWE has an important impact on the industry’s perception and use of RWE in decision-making, the real tipping point might have nothing to do with regulation. As leaders across the industry harness real-world, real-time data to address the Covid-19 crisis, they are witnessing firsthand that imperfect data can still be impactful—even crucial—for answering important questions about the best way to care for patients.

If RWE proves successful in mitigating the consequences of the Covid-19 pandemic (in many ways, it already has), and if that success is recognized, we could see a lasting shift in individuals’ perceptions about the value of RWE writ large.

Covid-19 as a test case for new uses of RWE

Traditionally, life science manufacturers use RWE for epidemiology studies, and to satisfy regulatory requirements for surveillance and safety. In the past few years, RWE has been touted for its potential to provide insight into five novel use cases: accelerated approval, label expansion, clinical trial recruitment, appropriate use, and innovative payment models. Covid-19 is putting each of these to the test right now.

  • Accelerated approvals: FDA is using a variety of existing programs to speed time-to-market for promising Covid-19 treatments. But beyond traditional or newly created accelerated approval mechanisms, former FDA Commissioner Scott Gottlieb suggested that FDA should conduct “real-time reviews” of potential treatments. He believes FDA should “evaluate data as it is read out from clinical trials, instead of waiting until the trial concludes.” This type of accelerated development not only means approval could occur without a completed RCT, but signals that FDA might be open to working with evidence outside the traditional gold standard RCT.  In a post-Covid-19 world, FDA could expand the mechanisms it uses to accelerate development for treatments with high unmet need, even in the absence of crisis-level urgency.
  • Label expansions: Researchers are testing whether existing drugs can treat Covid-19. While much of that testing will rely on controlled trials, several stakeholders are tapping into a variety of real-world data sources for answers. For example, Columbia University and ODHSI (The Observational Health Data Sciences and Informatics) recently launched a virtual study-a-thon to see if electronic health records and claims data from 600 million patients can help “examine the safety of proposed drugs and the effects of various treatments on historical viral diseases as well as predict outcomes for patients with viral symptoms or complications.” Of course, the concept of using RWE to support label expansions is not new, but in the case of Covid-19, researchers are looking to re-purpose existing drugs for entirely new indications. This isn’t unprecedented – Pfizer’s Viagra (sildenafil) was initially tested for cardiovascular disease before being used for erectile dysfunction – but it’s certainly rare.
  • Clinical trial recruitment: In an effort to accelerate the development of a Covid-19 vaccine or treatment, several organizations are partnering to swiftly identify and recruit patients for clinical trials. For example, TriNetX is amassing Covid-19 real-world data from its global network of health care organizations to connect pharmaceutical companies to sites that are open to receiving Covid-19 clinical trials and observational studies. Karyopharm Therapeutics is partnering with Komodo Health to accelerate their Covid-19 trial recruitment and site selection, using Komodo Health’s RWE database to identify physicians treating large populations of Covid-19 patients. Although an infectious disease is by nature more dynamic and harder to track than non-infectious diseases, these new data partnerships to support Covid-19 trial recruitment are creating the muscle memory with which to accelerate these efforts in the future, well beyond the current pandemic.
  • Appropriate use: Matching right patient to right treatment is paramount in any health care situation. But the stakes are different—and in some ways higher—in the case of a global pandemic. Frontline HCPs, supply chain leaders, payers, and others are forced to make difficult trade-offs to maximize very limited resources. Understanding exactly what the benefits and risks of a given situation are is crucial for swift and decisive action, and RWE can help. For example, stakeholders must understand how Covid-19 treatments might work differently across patient sub-segments in order to prioritize. In fact, several antiviral experts “think [Remdesivir] should primarily be used for patients with more severe symptoms and those who are hospitalized—some 15% to 20% of cases," Andrew Joseph reported for STAT News. In addition to prioritizing access to a given drug-based treatment, providers must also think about the relative efficacy of different-in-kind treatment options: Who would benefit from a drug vs a ventilator? Who should quarantine at home without treatment? There’s also the question of prioritizing access based on indication, if the treatment for Covid-19 turns out to be an existing drug (indicated for another condition). In that scenario, payers may need to create additional access controls to ensure patients who need the drug for its original indication don’t face problems filling their prescriptions because of a shortage.
  • Payment models: Implementing value-based contracts or other innovative payment models is challenging for many reasons—from misaligned incentives to poor data infrastructure to a lack of bandwidth, to even variability in how different stakeholders define value. But Covid-19 is creating a sense of urgency to bring products to market, even if the details of contracting and payment are not ironed out. For example, Peter Bach, director of the Drug Pricing Lab at Memorial Sloan Kettering Cancer Center, and Richard Hatchett, a pandemic expert in Norway proposed that the government should purchase Covid-19 vaccines before they are approved. And while many manufacturers have made public commitments to price approved Covid-19 products affordably, health care leaders are already proposing outcomes-based payment models to ensure purchasers pay only for effective products.

Four questions will determine the success of RWE moving forward

Despite the potential that RWE holds, whether or not Covid-19 is the tipping point will depend on four questions:

  1. Will interoperability and data sharing continue once the Covid-19 dust settles?

    Our take: Despite the abundance of health care data that exists today, most of it lives in silos, which creates challenges for stakeholders trying to generate longitudinal, insightful RWE. However, as a result of Covid-19, there’s been a noticeable uptick in the number of cross-industry stakeholders willing to break silos and share data. For example, Datavant is creating a registry of Covid-19 patients by pooling medical records from hospitals, payers, and pharmacies across the country. Additionally, federal regulators are relaxing rules that let hospitals and vendors share patient medical records with public health officials – further encouraging cross-industry data sharing. However, the extent to which these relaxed regulations will continue in a post-Covid world remains unknown.
  2. How will RWE aggregators, vendors, and researchers protect data privacy – today and moving forward?

    Our take: In addition to sharing existing data with one another, stakeholders are also turning to a variety of new sources of data—such as wearable devices, app-based questionnaires, and web search histories—to better understand Covid-19. But the degree to which this new data remains secure is unknown.

    The federal government is also increasingly opening the door for tech companies to share sensitive user information that can help track Covid-19’s spread. For example, Google is using location data from Google Maps to create “mobility reports” that see if different communities are adhering to social distancing measures. Facebook and IBM are each taking a similar approach. All hope to help public health officials track the spread of Covid-19. However, while patient privacy is always of paramount concern, experts are speculating about the future of these privacy rules and wondering what will happen to patient data privacy when the crisis ends. If relaxed privacy rules fuel innovation and uncover new clinical insight during Covid-19, long-term regulations on data security and privacy may change.
  3. Are early-stage AI and algorithms up to the challenge of accurately assessing limited data?

    Our take: Researchers increasingly rely on AI and algorithms to identify and predict cases of Covid-19. These algorithms often help assemble disparate data sources and synthesize RWE. However, with limited time to test and refine emerging algorithms, potential for bias in study designs, and incomplete or quickly changing data sets, there is a risk of incorrectly analyzing data or diagnosing cases, which may lead to potential patient harm. This is true not only for Covid-19, but for AI and machine learning across the industry.
  4. Will stakeholders’ discomfort about data source, methodology, and bias remain?

    Our take: Today, decision-makers have various concerns about RWE, including discomfort around how the underlying data was assembled and analyzed. There are also concerns about study design and potential biases. But as stakeholders battle the current pandemic, they’re accepting a broader range of data and evidence. While traditional concerns about RWE may be put aside in a time of crisis, it’s likely they will re-emerge after Covid-19.

Covid-19 could be the tipping point that boosts the reputation of RWE and accelerates industry acceptance. But ultimately, whether RWE is incorporated in any given decision is about an individual accepting RWE. Medical leaders must be prepared to understand, empathize with, and appropriately address customers’ concerns about RWE to demonstrate value – both in this time of crisis and in the future.

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