Outcomes-based medicine demands real-world evidence

Real-world evidence (RWE) must become a fundamental accompaniment to traditional RCT data in proving value, but uptake has been relatively slow and steady to date.

As the drive towards data-driven decision making in healthcare continues unabated, the value of the ability to measure real-life patient outcomes increases. For pharmaceutical companies, randomised controlled trials (RCT) are the founding fathers of the traditional route to medicines approval and market access.

But in today’s complex ecosystem – where the life science industry must plan for, and address, the transformational shift towards value-based pricing and deliver against enhanced regulatory demands for proof of efficacy and effectiveness – real-world evidence (RWE) must become a fundamental accompaniment to traditional RCT data.

In short, it’s less a question of if RWE will become an expected component of all market access efforts, and more a question of when and how. RWE is data collected in the field to demonstrate not only the efficacy and effectiveness of a particular treatment, but its value in improving patient outcomes.

The use of RWE in the future of health technology appraisal (HTA) was the topic of discussion in a QuintilesIMS webinar earlier this year, featuring Rita Faria, Research Fellow at the Centre for Health Economics, University of York and co-author of NICE’s technical support document for using observational evidence in technology appraisals, alongside QuintilesIMS’s Adam Lloyd, Senior Principal, Health Economics and Outcomes Research, and Eric Yu, Principal, Real-World Evidence Solutions.

Moving towards faster adoption of RWE

With market forces and advances in both personalised medicine and orphan drugs adding new layers of complexity to obtaining and compiling traditional RCT datasets, RWE will need to play a critical role in the coming years if new treatments are to successfully navigate their way to market.

Alongside this, swathes of evidence-based data, driven by climatic changes in digitisation and transparency, can already be leveraged through a variety of private and publicly available sources, including biobanks, demographic data, social media, pharmacy data and electronic medical records.

Yet despite the clear role that RWE has in underpinning an outcomes-based reimbursement system, the pace of adoption is relatively slow and steady. This, revealed Faria, could, in part, be due to continuing uncertainty surrounding RWE methodology and expectations. “We are on moving ground and there’s a lack of precedent in health technology assessment about what’s needed – both for those companies submitting the evidence and for academic review groups in terms of what we’re supposed to be looking for.”

Despite this uncertainty, steps are being taken to clarify regulators’ stances on RWE and the best practice and skills required for data gathering. The aforementioned NICE technical support document is one significant step towards clear guidelines, and several cases of successful RWE use already exist.

Meanwhile, there are pioneering experts among academics and providers who are already experienced in RWE pilot delivery. According to the panel, these pilots have highlighted both successful strategies and potential pitfalls in data collection and methodology that must be considered as the industry works towards the gold standard in RWE.

  • Be cognisant of biases – when utilising data from observational studies companies must make efforts to identify, quantify and adjust for potential biases. For example, selection bias is a genuine risk and one which needs to be monitored.
  • Consider the best selection method – in RWE studies, individuals are not randomly assigned but have had a specific treatment recommended to them for a set of specific reasons that could impact the outcome. Thus, the selection method for groups needs to be thoroughly considered and adhered to rigorously. Selection techniques are covered in detail in the technical support document.

The road ahead

Inevitably, RWE will become the preferred approach for regulators, life science companies and even patients, Yu pointed out, adding, “Intrinsically it is about being patient-centred and employing an augmented approach for evidence collation and evaluation.”

But learning how to lay the foundations for solid RWE means taking a hands-on approach, he went on. “We can always wait for the world’s most perfect solution, but doing is learning.”

Certainly, as value for money becomes the dominating factor in global healthcare, the emphasis on RWE will increase. “We want to give the best possible treatment out of our constrained budget,” Faria noted. “We need to know the effect of the treatment in our patient population over the short and the long term, relative to other treatments available.”

RWE is anything but a replacement for RCTs, Lloyd observed. Instead, he noted, supplementing traditional RCT data with RWE presents a stronger case to health technology assessors that cannot be underestimated.

It is this continued march towards outcomes-based medicine, combined with industry’s focus on personalised medicine and orphan drugs, that has led to the expanding role of RWE in addressing the question of value; value for regulators, value for healthcare systems and value for society.

The inexorable rise of outcomes-based medicine cannot be halted. Is it time life sciences caught up?

Find out more about RWE in QuintilesIMS’s on-demand webinar, or visit their website.


About the author:

Claire Bowie is Head of Publishing at pharmaphorum. She has extensive experience in healthcare communications and publishing, supported by a background in biological sciences.

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