The Rapidly Changing Healthcare Ecosystem
More than ever before, RWD is filtering into healthcare and affecting every aspect of the sector, from drug development to physician-patient interaction. To harness its transformative potential, it’s vital that we fully understand how data is collected and used.
RWD is defined as “longitudinal patient-level data captured in the routine management of patients, which can be repurposed to study the impact of healthcare interventions.”
RWD is generated from a variety of clinic and payer sources, such as electronic health records, disease and product registries, claims and billing databases, and increasingly from information collected by digital therapeutic solutions via daily ePROs.
RWE and Its Link to Digital Devices
The real value of RWD emerges only when it is transformed into RWE, which the FDA defines as "clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data." Unlike clinical trial evidence, the development of RWE involves validating and combining often disparate sources of routinely collected data.
Data analytics have enabled RWE to play a much bigger role in the health sector, but the value of RWE ultimately depends on the quality and type of RWD.
Notably, medical care determines a mere 10% to 20% of health outcomes, and thus traditional, clinic-based sources of RWD restrict the scope of RWE and its potential impact on patient outcomes. Increasing use of remote digital devices in patient care may provide additional out-of-clinic data, enhancing the quality of RWE.
RWE: A Catalyst of Innovative Evidence-Based Medicine
Evidence from high-quality RCTs is still the gold-standard. This evidence is used to support regulatory approval of new drugs and medical devices. But, the need for additional data following RCT-based therapy approval has long been recognized as post-market surveillance using clinical data registries is a routine component of the regulatory process. RWE can bridge this gap by offering a longitudinal view of a patient's well-being beyond the trial setting.
Indeed, the FDA recently published draft guidance on the use of RWE in study design and data quality validation to help address key bottlenecks in evidence generation for licensure. Despite its mundane appearance, experts believe that the guidance document symbolizes a critical step towards fundamentally repositioning RWE in regulatory approval processes and healthcare in general.
Notably, this draft guidance sets quality standards for RWD, paving the way for a much broader use of data from non-traditional sources, including from DTx solutions.
RWE to Optimize Healthcare Value
The recent FDA draft guidance may encourage higher standards of RWE. These higher standards may prove to be beneficial to payers concerned with value and cost. This could be seen as good news, as specialty drug spending has increased by over 20% in the last decade (2012 to 2022), contributing to an already unsustainable level of healthcare expenditure, estimated at 17.5% of the US GDP.
The rising cost of healthcare is a prominent social and health concern affecting patients, physicians, and payers alike.
To maximize value and minimize costs, payers are turning to RWE to gain a better understanding of the real-world risks and benefits of therapies.
Furthermore, an increasingly higher bar has been set for reimbursement, based on real-world performance, including reductions in hospitalization costs, observed adherence, or comparative value with other competing agents. RWE is also being used more and more to inform drug formulary decisions, and, in some cases, to determine payment based on real-world effectiveness.
However, despite its potential to promote healthcare value, outcomes-based reimbursement must be applied carefully to avoid undermining patient health.
A recent study of a Medicare pay-for-performance program found that the use of quality scores disincentivized physicians from treating the sickest patients, resulting in poorer outcomes and higher costs. Conversely, in real-world practice, more expensive novel therapies may be channeled to the sickest patients, resulting in lower effectiveness compared with RCT estimates.
Payers are therefore struggling with how to best use RWE to guide reimbursement. As explained in a 2021 publication, “Payers acknowledge the need for change…but downstream innovation for coverage, reimbursement and patient access to novel treatments is only now, and haltingly, occurring.”
Industry experts have suggested that a more comprehensive approach to RWE is needed in order to avoid potential problems with outcomes-based reimbursement, including greater use of long-term data on patient and clinician-reported outcomes.
RWE: Critical Remedy for Pharma Innovation
The healthcare ecosystem is constantly changing, and pharma companies are feeling the effects. They are under more pressure to show that their therapies are not only more effective than their competitors, but also have better real-world outcomes.
These new demands complicate long-standing challenges for pharma, such as a fragile patent estate and a downward trend in drug approvals, evident in the increasing failure rates of clinical trials and the lengthening of discovery-to-product-launch time scales.
Pharma is facing an acute pressure to innovate. In response, industry leaders have engaged in new approaches to boost efficiencies in drug development, including pre-competitive consortia and partnerships. Unfortunately, these approaches are unlikely to adequately address the new and long-standing challenges to pharma revenue.
However, while RWE intensifies existing pressures on pharma to innovate, it may also enable key transformations in drug development and approval processes that could substantially boost productivity.
In line with the growing prominence of RWE in healthcare, experts suggest that pharma fully embrace RWE in order to enhance efficiencies throughout the drug development process. A new regulatory scheme is the key to the fully integrated RCT/RWE approach, in which approval is first sought for a small patient population based on phase 2 trial results and then expanded to include more indications and patient groups based on RWE.
This adaptive licensing approach, based on generating real-world patient data rather than conducting additional late-stage clinical efficacy trials, aligns with the recent FDA draft guidance delineating what US regulators want to see in RWE.
The RCT/RWE model could benefit a variety of healthcare professionals, dramatically reducing drug manufacturing timelines and R&D investment, and provide evidence of clinical value for payers and patients.