For years, randomised controlled trials (RCTs) were considered the gold standard for generating clinical data on efficacy and safety to support product registration and subsequent prescribing. Recently, analysts and academics have discussed the promise of real-world data (RWD), signalling its potential to contribute to improved health outcomes. Data’s role in normal clinical practices, or in settings that reflect the reality of healthcare delivery, is likely to become increasingly important in ensuring that medicines are accepted by national policymakers and are adopted into practice.
RWD is useful in several phases of product development; it helps reflect priorities to ensure a well-rounded clinical development and market access plan that includes not only RCTs but also more pragmatic research in real clinical practice. These aspects are well recognised by the pharmaceutical industry, reflecting the need for a change in focus toward demonstrating the value of a new medicine.
In Europe, evidence requirements from key stakeholders have moved in a direction where RWD is becoming crucial to decision-making when used in conjunction with clinical trials. Therefore, it seems to have progressively more room to adopt non-interventional studies (NIS) as the key method in gathering this type of evidence. As NIS generally take place in a heterogeneous population and are conducted over a long period, they are considered naturalistic, whether prospective or retrospective, and allow for an unbiased view of real-world outcomes.
European authorities have undertaken massive work to optimise RWD access and use, and new strategies are required to align interests of the various stakeholders typically involved in healthcare. The past decade has seen global commercial clinical trial activity decline significantly due to slow start-up times, low patient recruitment, and high and variable costs.
Increasingly RWD can ensure continuing research activities; the need for data in addition to RCT is becoming more widely accepted. This need is evident in both the pre- and post-approval settings for new medicines. Pre-launch, RWD can enhance the effectiveness of clinical trials through the identification of patients from specific subpopulations, leading to potentially shorter and more effective trials. RWD can also be used to evaluate unmet clinical needs, describe pathways of care, and collect resource use, for example in preparing for a health technology assessment (HTA) as part of the reimbursement process. During the post-launch phase, RWD is used to demonstrate the safety of a drug in the marketplace aligned with governmental priorities, patient needs and national agendas. In some aspects, RWD collection may be essential to satisfy regulators’ requirements where interim conditional approval has been granted to a new medicine.
RWD is becoming crucial to decision making when used in conjunction with clincial trials
Big Data and the effective use of RWD are about to transform R&D. The collection and analysis of RWD can help identify potential new candidates and promote their development into effective, approved and reimbursable products. The average R&D cost for a newly approved medicine is estimated to be around £1bn (including the cost of projects that fail). Industry attrition rates continue to increase despite significant technological advances and adoption of new approaches over the last decade. Driving more rapid progress in adopting the concept of disease stratification and personalised medicine could make a significant improvement to the cost effectiveness and accuracy of the R&D process, consequently improving its competitiveness. Perhaps a light at the end of the tunnel is beginning to emerge with the creation of a strong and coherent alliance among industry, health authorities, biomedical sciences and regulatory bodies, mainly in oncology. Without such an alliance the growth of R&D will be further inhibited and the development of the small diagnostic sector placed in jeopardy.
Although RWD has progressively become central to decision making, the regulations and guidelines for NIS are rapidly changing and are far from being harmonised in Europe. While considerable efforts have been made to demonstrate the accuracy and completeness of the information obtained, researchers and others should always check with local authorities or ethics committees before starting any NIS in Europe. Governmental decision making will be based on evidence of value in the commissioning of healthcare, payment for services, and payment for future new medicines.
Even more challenging, these changes are occurring against a financial crisis and recession in Europe, with tight financial management and most likely no real increases in health funding. Therefore, it is important that the pharma industry, governments, physicians and patients work together to demonstrate the value of new medicines, to ensure data are used appropriately to facilitate access to innovative medicines for patients, and not to delay this process.
