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Dr. Chitra Lele is Chief Scientific Officer, Sciformix Corporation.
Stakeholders around the world can benefit from RWD as it can improve patient outcomes and speed at which products come to market.
As the life sciences market continues to evolve where payers and patients are playing a more integral role in the landscape, organizations are placing greater focus on how they plan for commercialization much earlier in the development process. Companies must provide evidence and demonstrate the value of products to multiple stakeholders. Real World Evidence (RWE) and Market Access (MA) are inherent to reach this goal and increasingly crucial to ensure patient access and commercial success.
Real World Data (RWD) is fundamental to capturing the benefit and risk after a product has received regulatory approval. The FDA defines RWD as data collected from sources outside of traditional clinical trials and Real World Evidence (RWE) as the evidence derived from the aggregation and analysis of RWD elements. RWE links RWD with economic outcomes, demonstrates a product’s value proposition and is essential for market access (MA).
Real World Studies
RWE is being widely used to gain an understanding of disease epidemiology, patient journey, real-world use and effectiveness of treatment options, unmet patient needs and the value that products offer in sub-populations. Real world studies allow inferences to be made on the product’s long term safety and effectiveness, comparative effectiveness and product differentiation.
RWD can assist both innovators and patients in very impactful ways. For the former it can inform marketing strategy decisions and identify target audiences. From the patients’ perspective, it helps identify where quality of care can be improved and how tools for patient education can be developed.
A key requirement for market access is evidence of clinical effectiveness and cost effectiveness of the product. Market access is no longer about simply gaining marketing authorization for the product. Its scope includes pricing and reimbursement, Health Technology assessment (HTA), and formulary inclusions.
Today payers expect that real life evidence match the projected performance at launch, as well as new evidence become available with change in the competitive landscape and the environment. Most importantly, they will assess the value for money of the product. RWE can provide support to back up pricing after a product launch which is illustrated in the WellPoint guidelines.1
HTA is a systematic approach used to evaluate properties, effects and impacts of a wide range of health technologies and interventions. It considers a range of factors including safety, clinical effectiveness, budget impact analysis, organizational impact, equity and ethical issues.
RWE in the Public Domain
The 2015 World Health Organization’s (WHO) global survey on HTA by national authorities found that RWD is not currently being utilized due to a global lack of qualified human resources and an absence of HTA training programs in most countries.2
Although the FDA has released multiple pieces of draft guidance on the use of RWD and RWE, a report from the bipartisan policy center calls for further clarity, stating RWD has been rarely used beyond post-marketing surveillance. The broader use, such as, in label expansions or supporting new drug applications is not yet routinely used by the FDA. It goes on to ask the FDA for formal guidance including levels of use, circumstances and methods under which RWD is acceptable.3In Practice
RWD can benefit multiple stakeholders to support commercialization strategies and provide insightful analysis for differentiation, to assist with regulatory submissions and to influence the market access strategies.
Although the opportunities from RWD are outstanding, its vastness poses challenges as it can become obsolete if not analyzed in real-time with effective technology. It also requires a range of experts to effectively interpret and use the data, and the WHO survey reveals a lack of qualified professionals – investment in this could be highly beneficial across the world.
Finally, the FDA has concerns over the relevance and reliability of RWE as it evolves over time. Yet, using sound statistical principles to collect and analyze data, it can still be used to drive market access decisions.
Stakeholders around the world can benefit from RWD as it can improve patient outcomes and speed at which products come to market. To gain a competitive advantage organizations should make effective use of both technology advances and human resources available.
Chitra Lele is Chief Scientific Officer at Sciformix Corporation.