Are Real-World Evidence (RWE) Studies The Answer To More Rapid Regulatory Approval?

Over the last decade, nearly all stakeholders in healthcare have recognised how Real-World Evidence (RWE) and Real-World Data (RWD) complement traditional clinical research methods. More recently, Real-World Evidence (RWE) and Real-World Data (RWD) are driving galvanic disruption in the research field. The insights gleaned from both datasets are fuelling comparative studies on therapeutic procedures and end-point studies on products.

In April 2019, we saw the first FDA approval of a drug based purely on real-world data. The drug in question is Pfizer’s Ibrance (palbociclib), a treatment for for male breast cancer. [1] Elsevier, collaborated with Pfizer to coordinate part of its clinical development programme for Ibrance. Chief Medical Officer Dr Ian Chuang says “The amount of data gathered in such a short time to support the new licensed use of Ibrance exceeds what a clinical trial could have gathered in the same amount of time.”

In a time where patients are reluctant to enter hospitals to take part in clinical trials, data gathered via new technologies in RWE and RWD studies could revolutionise the way we run research. Furthermore, if regulators are open to approving some drugs and therapies based on RWD study data alone, this will propel cutting-edge therapies to market faster.

What is Real-World Evidence (RWE) and Real-World Data (RWD)?

The FDA defines [2] Real-World Data (RWD) and Real-World Evidence (RWE) as follows:

“Real-World Data (RWD) are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.”

“Real-World Evidence (RWE) is the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD.”

To be clear, Real-World Evidence (RWE) is generated when appropriate analytic techniques are applied to Real-World Data (RWD) to indicate what people and patients will actually experience, during and from their care.

Real-World Evidence (RWE) clinical trials may use a whole host of Real-World Data (RWD). This may include data derived from electronic health records (EHRs); medical claims and billing data; data from product and disease registries; patient-generated data, data from inhome- use settings; and data gathered from other sources that can inform on health status, such as mobile devices.

In the same document [2] the FDA states “RWD sources can be used for data collection and, in certain cases, to develop analysis infrastructure to study designs to develop RWE, including, but not limited to, randomized trials (e.g., large simple trials, pragmatic clinical trials) and observational studies (prospective or retrospective).”

The Market for Real-World Evidence (RWE) Clinical Trials Is Growing Fast And Tech Is The Driving Force

This industry forecast by Meticulous Data, released in June 2020, states “The Real-World Evidence (RWE) solutions market is expected to grow at a CAGR of 15% from 2019 to 2024 to reach $1.64 billion by 2024. Factors such as rising geriatric population and burden of chronic diseases, shift towards value-based care, growing focus on personalized health care, delays in drug development and the subsequent increase in development costs, and expanding use of RWE for regulatory decision making supports the growth of RWE solutions market.” [3]

The use of computers, mobile devices, wearables and other biosensors to collect, store and analyse vast amounts of Real-World Data (RWD) is driving this progress. In fact, careful analysis of this data in Randomised Controlled Trials (RCTs) affords improved trial design and better patient outcomes, [4] and the provision of better patient care as novel treatments get to market cheaper and faster.

In June 2020 Fareed Melhem from Medidata [5] stated in Deloitte’s Life Sciences Connect Podcast [6] “We know patients in clinical trials don’t look like patients in the real world in many ways.” Real-World Evidence (RWE) studies and RWD study data can provide the missing piece of the 360 degree view we need to design the most effective studies and interventions.

Why Are Real-World Evidence (RWE) Clinical Trials So Important?

Real-World Evidence (RWE) trials generate convincing observational data outside of randomised controlled trials (RCT). In essence, RWD study data can offer wider perspective on the risks and benefits associated with specific therapies. Subsequently, this data creates more awareness on diseases, products, and patient populations. In simple terms, RWD study data can answer questions unaddressed by RCTs, resulting in the ability to assess a drug in a Real-World context. That real-life assessment delivers better health outcomes and more cost-effective therapies and drugs in the market.

Real-World Data is particular useful when you’re looking for special populations in patient recruitment. IgniteData faced this challenged when working in partnership with Virtus Respiratory Research, who needed to recruit patients with a substantial smoking history, and without a diagnosis of COPD [7].

Stepping Outside Of Sites To A Patient-Centric Approach

As researchers struggle to identify the right patients and enrol them in studies, new approaches are called for. Accordingly, increased awareness of the importance of better engagement and education amounts to larger patient populations. The idea being that if sponsors and researchers forge a stronger connection with the community and take a more patient-centric approach, we all benefit.

This article from September 2020 [8] explains how we could think about increasing the network for trials by reaching out to people in the community and educating them further. “Sponsors looking to increase diversity in clinical trials should, among other things, “meet patients where they are,”. Although this article relates specifically to minorities, it’s a worthy consideration for the wider population. LaTasha Lee vice president for social and clinical research and development for the National Minority Quality Forum (NMQF) says, “It’s important for early and meaningful engagement of those populations, not just when you’re ready to launch a clinical trial, but as you are designing your clinical trials,”

This Real-World Evidence approach to patient recruitment [9] is a good way to build trust in patients populations. Moreover, this mindset could aid the faster search for eligible subjects, in tandem with pre-selection and pre-screening of existing data available in primary and secondary care.

What Are The Challenges In Using Real-World Data (RWD) In Clinical Trials?

There are several challenges which are important to overcome in the world of Real-World Data (RWD) and Real-World Evidence (RWE).

Firstly, we must consider the patient perspective. Hence, when we approach patients we must ensure we treat them like human beings rather than data sources. From the clinical research perspective we’re always data hungry, however, to build trusted relationships with patients we must remember to consider their projected outcome, in conjunction with ours.

Secondly, we want to consider data quality. As we all know, data always follows the “trash in, trash out” principle. Accordingly, if there’s no standard way for a clinician to enter RWD study data, we’re going to find ourselves in trouble. Therefore, a rigorous quality control process is needed to ensure that the data are accurate, timely, and useful. Moreover, Electronic Health Record (EHR) data wasn’t originally created for research purposes and where it may contain some ‘gold’, it may also contain gaps or errors. In the worst case scenario, missing or incorrect data can cause medical harm.

Limitation on the availability of research-grade data is reducing as strategic partnerships are created to ensure access to valid Real-World Data (RWD).

Regulators Are Keen To Support Real-World Evidence (RWE) and Real-World Data (RWD)

In June 2020, Deloitte released details of their latest pharmaceutical industry benchmarking study [10]. They discovered that “More than 40% of this year’s respondents say recent FDA guidance and statements encouraging RWE use in R&D are driving an increased strategic focus.”

Needless to say, the industry is working in alignment with regulators. Deloitte’s study reports. “Since our last RWE benchmarking survey, we have seen numerous statements from senior FDA officials as well as draft guidance issued by the FDA on how RWD and RWE can be incorporated into regulatory decision-making. The European Medicines Agency (EMA) released the EMA regulatory science to 2025 reflection paper which included promoting the use of high-quality RWD in decision-making as one of its strategic goals.”

Conclusion – Are RWE Studies The Answer To More Rapid Regulatory Approval?

Even though the signs are positive, we shouldn’t jump the gun. We want to ensure the core objective remains clear to all and rigorous data quality checks are implemented. The more robust and quality data evidenced, the easier it is for regulators to approve therapies. More importantly, RWE has many applications beyond regulatory approval. Real-World Evidence (RWE) and Real-World Data (Data) enhances many stages of a clinical trial.

Even though there are challenges and barriers still to overcome in the use of Real-World Evidence (RWE) data, stakeholders know that properly designed and conducted studies can discover previously unseen aspects of disease. In turn, this information can guide strategic decisions, better patients outcomes, and in the future it could also drive beneficial changes in clinical practice. To realise the numerous advantages of the data, all stakeholders must work closely with one another.

Technology supports us in finding solutions to some of the issues in Real-World Evidence (RWE) clinical trials, however, it’s important to keep the study objectives of all stakeholders in mind, and continue to build longterm trusting relationships with patients. Actually, it would be reasonable to suggest that as patients understand more about the future power of RWE studies to improve both clinical practice and patient outcomes, the more open and participatory they will become.

IgniteData Can Enhance Your Real-World Evidence (RWE) Clinical Trials

IgniteData supports you in designing and delivering powerful Real-World Evidence (RWE), through the effective analysis of consistently collected and statistically valid consented patient data. Our effective management and analysis of RWD study data will aid you driving budget efficiency, minimising site burden, improving patient recruitment and regulatory adherence.

Get in touch at contact@ignitedata.com to speak with an IgniteData expert today.

References

[1] https://www.pharmaceutical-technology.com/features/real-world-data-improvingoutcomes/

[2] https://www.fda.gov/media/120060/download

[3] https://www.globenewswire.com/news-release/2020/06/23/2052249/0/en/Real-World-Evidence-RWE-Solutions-Market-Worth-1-64-Billion-by-2024-Growing-at-a-CAGR-of-15-from-2019-Market-Opportunity-Analysis-and-Industry-Forecasts-by-Meticulous-Research.html

[4] https://www.ncbi.nlm.nih.gov/books/NBK540105/

[5] https://www.medidata.com/en/

[6] https://www2.deloitte.com/uk/en/insights/life-sciences-health-care.html?icid=top_life-sciences-health-care

[7] https://www.ignitedata.com/case-study/bespoke-patient-data-targeting-patientrecruitment/

[8] https://www.centerwatch.com/articles/25007-researchers-urged-to-meet-patientswhere-they-are-to-build-more-diverse-clinical-trials

[9] https://www.ignitedata.com/solutions/patient-recruitment-company/

[10] https://www2.deloitte.com/uk/en/insights/life-sciences-health-care.html?icid=top_life-sciences-health-care