Real World Data (RWD) and Real World Evidence (RWE) are gaining traction across the industry. The front runners are already exploring new ways to collect and use data within their market access strategies and, for some, this is proving an effective way to ‘tip the balance’ from a no to a yes with target agencies. How well is pharma leveraging the data advantage? And how should companies benchmark their own RWD activities against the best of the rest?
At the heart of the Real World Data revolution is the issue of control. Pharma companies used to be in the driving seat when it came to the data produced from trials and studies of their drugs. But now the floodgates have opened and everyone from payers to patients to your competitors can all see and comment on real world drug performance. And they are, in droves. This new data-rich environment presents significant opportunities but also threats. The key to success is finding a strategy that is sustainable, meets commercial objectives, and produces insight that is ultimately trusted enough to influence opinion.
Defining Real World Data and Real World Evidence
Real World Data (RWD) are data used for decision making that are not collected in conventional randomised controlled trials (RCTs). Real World Evidence (RWE) is the evidence generated from RWD. A linked concept is ‘Big Data,’ which is generated from combing multiple sources of RWD. At the heart of RWD and RWE is patient-centricity.
RWD can be clinical, economic and humanistic and come from diverse sources; from patient records to social media and nearly everything in between. However, this proliferation of data also poses challenges for methods and analysis. Further, companies need to keep tabs on RWD and RWE in their fields as, even if they are not generating this data, others might release findings that necessitate a company response.
Real World Data and Real World Evidence for Market Access
Interest in RWD and RWE is increasing, with various stakeholders – including companies, regulators, Health Technology Assessment (HTA) agencies, payers, providers and patients – leveraging the information to inform their decisions. This data is proving particularly relevant to some HTA agencies and payers as they focus on therapies that provide value for money.
RWD and RWE are increasingly being seen as an enabler for market access, with case studies emerging on how RWD and RWE can ‘tip the balance’ from a no to a yes with HTA agencies, including the Scottish Medicines Consortium (SMC), the National Institute for Health and Care Excellence (NICE) and the French Authority for Health (HAS). However, RWD and RWE may need to be coupled with changes in price to secure those positive recommendations. Acceptance of RWD and RWE across agencies is not universal and challenges to using RWD and RWE with payers need to be overcome to increase their influence.
For the companies themselves, meanwhile, RWD and RWE are core to outcomes-based contracts, which currently remain limited, but are expected to grow in the future. Further, RWD and RWE are helpful beyond initial market access and can be used to inform development plans and future business decisions. RWD and RWE can also help differentiate products from those of competitors.
Companies leading on Real World Data and Bench-Marking
The pharmaceutical industry as a whole recognizes the importance of RWD and RWE, although there is significant diversity in terms of company approach. Pfizer is particularly busy – running one of the highest number of real world studies as a sole sponsor, based on analysis of data from ClinicalTrials.gov. Other big pharma companies are also gaining significant ground. For example, Astellas won the first inaugural UK Prix Galien award for RWE in 2016 based on their work on Dificlir (fidaxomin) for colostrum dificile infection in the English National Health Service (NHS). GlaxoSmithKline has been breaking new ground with the first pragmatic Phase III real world trial: the Salford Lung Study (SLS). The pay-off for this multi-million pound study in terms of market access aren’t yet known.
Those companies who are seen as leaders in RWD and RWE share several common features, including:
- Centralising RWD and RWE in the organisation, whilst at the same time striving to integrate this information across the business and work cross-functionally
- Implementing RWD and RWE early in the clinical development process
- Investing in the infrastructure for RWD and RWE
- Gathering data from a diverse set of data sources and performing varied analysis to address the unique needs of various stakeholders
- Collaborating with others so as to provide access to RWD and expertise, as well as to add credibility to a company’s own RWE
Real World Data and Collaboration
Companies need to collaborate with a variety of stakeholders to optimise RWD and RWE. Doing so will not only increase the insights from RWD and RWE, but also increase the acceptance and credibility of RWD and RWE by external stakeholders. Companies have a wide choice of collaborators, including Patient Powered Research Networks (PPRNs), providing a way to work with patients and access patient data.
The future for Real World Data and Real World Evidence
RWD and RWE are going to grow in importance, reflecting the increase in volume of data available, improvements in its quality and the ability to link disparate data sources. RWD and RWE will be in greater demand too,
- Because of the trend for coverage and reimbursement decisions to be based on this information
- As well as being used in outcomes-based contracts that are expected to increase in the future.
Further, the types of data captured and the methods in which outcomes can be generated are also expected to evolve, with patient reported outcomes (PROs) expected to grow in importance. Beyond the pharmaceutical industry, a number of other stakeholders are expected to shape the future of RWD and RWE, with technology companies expected to emerge as a key influencer given their efforts to seek a greater presence in healthcare.