Filling the black hole: Can Real-World Evidence data meet demand?

Adam Collier examines the emerging need for Real-World Evidence data to meet the increasing scrutiny and demands of healthcare decision makers.

The increasing demand from payer bodies, such as NICE, the SMC, NHS England and local CCGs, to demonstrate product efficacy, not only in clinical trials but also in a real-world setting, is widely known within the pharmaceutical industry. With a payer base increasingly driving healthcare decision-making, there is a growing need to produce more evidence around real-world clinical effectiveness and the real-life safety profile of products than randomised clinical trials (RCTs) – the current “gold standard” – can offer. Real World Evidence (RWE) is fast becoming a significant currency of healthcare. Clinical trials, whilst a valued and needed resource, are recognised for their limitations within the real-world setting due to their “controlled” nature; therefore prompting decision makers to demand more robust evidence around the true effectiveness of medicines before approval.

Moving out of the closed RCT environment and into treatment initiated in daily practice sees a more diverse reality; such as patients with multiple comorbidities, on multiple medications and with compliance issues. As a result, there is a need for data around medications to demonstrate real-world effectiveness in the full patient treatment pathway – both for primary and secondary care-initiated interventions. Until recently, it has not been possible to track patients across the primary and secondary care “divide” in many European countries, with the notable exception of Sweden.

Sir Alasdair Breckenridge, former Chairman of MHRA, emphasised that “the way in which medicines are now being regulated will be changing; the conventional way of regulation is now seen to be too arduous, expensive and not successful enough. Therefore, there is an increasing interest to be able to conditionally licence medicines earlier in the regulatory process; however, this requires there to be very good post-marketing data collection, in which real-world efficacy and safety can be judged outside of the clinical trial setting. The converging needs of both payers and regulators increases the requirement for real-world data evidence within a healthcare environment which supports the principles of Value-Based Pricing.” Breckenridge poses the question of where to get this real-world evidence data; concluding that “observational studies, i.e. large databases, may provide a much needed solution for this treatment pathway ‘black hole’ for any particular product.

“Until recently, it has not been possible to track patients across the primary and secondary care “divide” in many European countries, with the notable exception of Sweden.”

Essentially, healthcare decisions could be better informed based on large patient experiences in the real world—supplementing RCTs that typically involve a few thousand patients in a controlled setting. Using RWE, patients, providers of care and payers can better assess the value of treatments and services based on actual health outcomes and the total cost of care. Against this background, real-world data is gaining recognition as a core element of the clinical and health economic research agenda; an important tool in understanding performance throughout a product’s lifecycle and the patient treatment pathway.

Generating RWE via observational studies and the use of patient registries, is one of the fastest growing areas of pharmaceutical research and development. Such post-marketing studies are increasing in importance as regulatory bodies demand more long-term data demonstrating efficacy, safety and quality. Realistically, key drivers for this breadth of information are the demands of health technology assessors and payers, and their need for evidence-based health economic data, based on local conditions.

When a pharmaceutical company submits a product or device for approval, it is required to supplement its submission with thorough evidence and figures for the cost of treatment with that particular product, including unexpected side effects of the medicine. Many vital medicines receive a negative funding approval outcome due to cost, particularly in comparison to older competitor products established within the market. However, often, this does not account for the costs of treating any side effects that the older treatment options may cause, which the healthcare system must pay for. A database that validates a product with RWE and exposes the burden of existing treatment options will result in patients, payers and healthcare providers being granted access to medicines through a more accurate, representative view of a medicine within an uncontrolled and typical setting.

“Essentially, real-world data can fill in the pieces of the puzzle that RCTs cannot do…”

The UK currently holds leading observation research datasets such as the CPRD, HES, Disease Analyzer, THIN and Hospital Treatment Insights. Further development is expected with Care.Data once privacy issues have been resolved. However, data is still for the most part in separate silos, not covering the full patient pathway from primary into secondary care and often not covering Wales and Scotland. Historically, databases have demonstrated the patient pathway within the primary care setting; but missed a lot of detail around treatment interventions in secondary care.

The pharmaceutical industry is placing greater importance upon hospital-initiated and hospital-treated medicines, so a valid and practical need is emerging for further exposure within the full treatment pathway. Despite the fact that the implementation of Value-Based Pricing is currently on hold, approval or not by NICE has a significant impact on the uptake of newly-launched medicines; and this is based upon demonstration of a product’s value and necessity within a real world setting. Professor David Haslam, Chairman of the National Institute for Heath and Care Excellence (NICE) stated in a recent PMLive interview on Value-Based Pricing that, “our response hasn’t changed, which is that we absolutely support the general principle that the NHS should pay a price that reflects the additional therapeutic benefit of drugs.

Essentially, real-world data can fill in the pieces of the puzzle that RCTs cannot do – how the individual patients are responding and how individual systems fare when you overlay the information on the treatment pathways within an individual market.

Across the healthcare setting, everybody wants information that will help them to do their jobs better. Payers are looking for cost-effectiveness and value for money; physicians are looking for the best way to manage patients; individual payers are looking at the data that they themselves accumulate. The more information that’s available, the more there is the ability to articulate the value of products not just as a price point but as a larger—and ultimately more meaningful—intervention within the healthcare setting.

About the author:

Adam is the Principal of the Patients Insights practice at IMS Health with responsibility for consulting and data related to IMS’ patient data assets in the UK. He has 18 years of commercial experience in the UK and European healthcare industry, including pharmaceuticals, healthcare management consulting and in healthcare provision.

He can be contacted at

Closing thought: Can Real-World Evidence data meet the demands of healthcare decision makers?