What is real world data and how does it relate to patient safety and pharmaceutical market access?
Over the recent years, all the stakeholders in the pharma industry have been seriously contemplating the benefits of real world data.
Real world data was primarily used for the safety profiling of specific drug classes or for disease types. This practice further led to the planning of post-marketing surveillance. Similarly, the need for evidence was looked at during the development stages of products to help the decisions of regulators for marketing approval. Given that real world data and real world evidence are playing an ever-increasing role with regard to health care decisions, we at phamax thought it was a good time to look at how and why it is increasingly accepted among stakeholders.
Real world evidence (RWE) rose to fame when evidence generation and data building were streamlined. These capabilities provided aid and supported pharma companies and the relevant stakeholders when it came to understanding the valuation of products and their accessibility. These combined fundamentals described Health Economic and Outcomes Research (HEOR).
Real world evidence studies also complement clinical trials. Clinical studies are set up within a defined design and methodology to generate and analyze specific outcomes. So, the collected information is confined and governed by restricted parameters. Real world evidence studies explore the reality and present all forms of data. They provide the link of relevance in the real setting which clinical trials would not examine.
For example, in the rare diseases domain, substantial evidence cannot be acquired due to the low prevalence and because the treatment for rare medical conditions is sparse. For such rare indications, real world studies can support our medical understanding of the various patterns of treatment measures. It can also help advance our knowledge on the disease burden, which can bring hope to the orphan drug market.
Similarly, real world evidence plays a pivotal role in patient care, particularly in the budding field of patient-customized treatments. The data from these settings support in building the personalized outcomes of patients with the same disease at different stages, and the detection of adverse effects and safety issues for a broader population. Also, evidence studies can provide answers in the development of targeted and genomic therapies where real world data can act as a biomarker for the research.
Real world data can be accepted globally as drugs and medical devices are approved and accepted in global markets.
Due to the growing importance of real world data and evidence-based studies, pharmaceutical companies and relevant stakeholders are eagerly awaiting guidelines or representative formats on how to use real world data from the regulatory authorities. To date, there have been noticeable efforts taken by regulatory and eminent authorities to create a unified platform for acceptance of the data.
Similarly, the United States Food and Drug Administration (US FDA) recently released a draft guidance document for medical devices. The draft guidance elaborates upon the utilization and the process of designing the collection of data, other than the set paradigms of clinical trials. The document also details various data management tools and data sources like Electronic Health Records (EHR), registries, case reports, and the validation process utilized in the collection process.
Accepting real world evidence data has always been a concern among stakeholders, and the initiatives from the WHO and the US FDA will certainly operationalize the attempts made by stakeholders globally to increase market reach.
There is a widespread understanding and acceptance on the applications of real world data. There are limitations that are gradually being addressed, yet the future is promising.
When we talk of evidence, we talk of data, and data is always scrutinized when it comes to its source and quality. Hence, pharmaceutical companies, research organizations, and medical centers should take advantage of the e-clinical and analysis tools to streamline the data and emphasize better quality. As a result, the confidence levels for the data submitted will be higher when reviewed by regulators, experts, and key decision makers.
There is a need is to devise a collaborative approach or a universal model which integrates and synergizes the scientific and commercial teams, thereby increasing the use of real world data generated from RWE. Clinical trials often do not allow for all parameters in a single protocol or design, and could probably cause a steep increase in the constricted budget. Conversely, real world evidence has the potential to gather real-time data across countries within stipulated budgets.
There are various debates concerning the credibility and acceptability of real world data. However, a collaborative approach from stakeholders has cleared the skepticism. The way forward is to collaborate, contribute, and continue to build the confidence to develop effective tools which constantly improve scientific, commercial, and clinical outcomes with the use of RWE and real word data, ultimately driving effective patient-specific healthcare forward.