The healthcare industry is changing its view on how drugs should be assessed and valued. Although randomized controlled trials have long been the gold standard in testing drug safety and efficacy, there are now increasing calls for additional insights into how drugs perform in the real world. This Real-World Data, largely generated by patients’ real life treatment journeys, is fast becoming the new currency in healthcare, with its integration into Real-World Evidence informing key healthcare decisions. But what is patient generated Real-World Data and what advantages does it bring?
The Role of RWD in Healthcare
Until recently, approval of a new drug and its release on to the market entirely depended on assessments of its efficacy and safety in randomized controlled trials (RCTs). But now, the tide has turned. Healthcare decision-makers, including regulators, physicians, payers and patients themselves, are realizing that drugs shouldn’t just work in idealized RCT settings, but that they should also work while patients live their normal, everyday lives.
This changing attitude is driving demand for programs that test whether drugs work in the real world. These programs generate so-called Real-World Data (RWD) reported from a variety of sources, such as patient registries and electronic health records. In turn, these multiple RWD sources are integrated into a wider Real-World Evidence (RWE) base, including insurance claims data and clinical data from electronic health records.
It’s becoming clear that RWE can inform a range of healthcare decisions across all stages of the product lifecycle, from drug development, to launch, and finally to the in-market stage. As such, companies that invest in RWD programs are starting to enjoy significant commercial benefits from the RWE that they generate. One company has estimated that RWE could produce up to $1 billion for a top-10 pharmaco product when it’s applied collectively in key areas:
- Demonstrating product safety and value
- Enabling productivity and cost-savings
- Facilitating initial product pricing and market access
Importance of the Patient Voice
Much of the RWD collected comes from programs in which thousands, or even tens of thousands, of patients take the drugs while living their normal lives and regularly report back about their personal treatment experiences. This rich source of ‘patient voice’ can be used to not only better monitor patients during RWD programs, including patients’ physical and emotional wellbeing, but to also collect valuable information about their treatment journeys that would not otherwise be available.
Such insights can provide a richer understanding of a drug’s effectiveness and its added value compared to those gained from traditional RCTs alone. A precise understanding about what patients need from a drug for their individual disease and situation can only be obtained by listening to the patients themselves. As such, the patient voice and the RWD it produces is now considered one of the most important factors in evaluating the effectiveness and market value of a new drug.
The Benefits of Patient Generated RWD
Patients participating in RWD programs provide healthcare decision-makers with specific types of data that RCTs typically do not provide. Examples of this information include:
- Outcomes that are important to patients
- Reasons why patients make certain decisions, such as adherence to treatments and treatment satisfaction
- Patients’ experiences of illness and care that physicians and program coordinators would not otherwise hear about
- Practical input into future study design and successful study execution
Companies can use patient generated RWD as tangible evidence to persuade healthcare decision-makers such as payers and regulators of a product’s added value. In turn, decisionmakers can use RWD alongside RCT data to make holistic assessments about a drug’s value and efficacy. Ultimately, patient generated RWD promises to ensure that new drugs effectively treat disease while allowing patients to continue living their normal, everyday lives.
A Look Ahead
RWE is increasingly influencing a variety of important healthcare decisions. Pharma companies are starting to respond by harnessing the value RWE can generate. As companies start using RWE strategically, rather than opportunistically to inform all stages of the product lifecycle, patient generated RWD will become even more important in ensuring the commercial success of new drugs, and in informing national reimbursement and regulatory decisions.
It’s becoming clearer that the more we listen to the patient’s voice, the more we can successfully tailor new treatments and programs to their real-life personal needs. This promises to facilitate a personalized approach to disease treatment that could significantly improve patient outcomes as well as contribute to the success of RWD programs in future drug development.
Tim Davis is Vice President, Digital Patient at ERT
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