Patient-centered real-world data is an increasingly important part of the drug development and market access story, providing evidence of the value of the drug as used in clinical practice. This kind of data cannot be attained under the idealized conditions of the clinical trial process but rather needs to be collected during routine clinical care.

Cars and clinical trials: The challenge of ideal data

When companies test cars for their fuel consumption, this is done under ideal conditions. The cars are tested in labs, not on the open road. They can use an 'eco' mode, and the lights, heating, heated windows and air conditioning are all turned off. The cars are unloaded, and the roof rails and even the passenger door mirror can be removed. And the tires can be inflated to higher than the recommended pressure. However, in the real world, cars are driven with the radio on, fully loaded with shopping and kids in the back; with less than ideal tire pressures; often in standard default mode rather than 'eco' mode; and with the lights, windscreen wipers and heaters on because it's cold, dark and wet outside. Therefore, the results are often quite different from those reported under ideal circumstances.

When companies test drugs in clinical trials, this is done under ideal conditions as well. The patients have few if any co-morbidities, are often on no other drugs, and their age and weight is within a specified range. They visit the clinics for regular check-ups. They are reminded to take their drugs, and their adherence is monitored. In the real world, patients may have a range of other conditions. They may be old or young, underweight or overweight. They may take a variety of different drugs, and may forget one or two doses over the space of a week.

The role of the enriched real-world study

Similar to the car company example above, consumers of healthcare along with clinicians, regulatory agencies, and payers want to know how well the drug is working for patients in the real world. Enriched studies are an effective and efficient study design approach to measure how the medicine is being used as well as measuring important effectiveness and safety outcomes. Indeed, enriched studies combine information from existing records such as electronic medical records and healthcare claims data that are routinely captured in practice with protocol-defined primary data collection from patients, clinicians, and care providers. They generally use a minimally interventional approach, and may be observational or randomized.

Because a percentage of the data is patient-reported, or collected through questionnaires and calls, and other clinical endpoints may be collected directly from their medical records, these studies can reduce the burden of data collection on clinical sites, therefore making site recruitment and administration more efficient and cost-effective. Patient recruitment is also simpler, as there are fewer inclusion and exclusion criteria to ensure that the cohort is closer to the real-world population.

The inclusion of patient-centered measures means that the studies are evaluating real-world effectiveness rather than clinical trial-based efficacy. These styles of studies provide a better understanding of the patient, by including patient-centric data such as adherence patterns or how often a patient is seeking additional care. They also assess the impact of treatment on their quality of life, or patients’ preference of one type of injection over another, or improvement of symptoms (such as severe nausea to a new medicine) over time. It is these clinically meaningful and patient centered questions that will drive the use of medicines in the real world.

Demonstrating the Value

The collection of biological, clinical, social, behavioral and cost data from both primary and secondary data sources included in an enriched prospective study allow for a more comprehensive evaluation of the clinical utility of treatments as well as a patient-oriented approach. From these studies, clinicians and patients can begin to determine which medicine is right for which patient at the appropriate time. There are also many other questions that can be evaluated, including the impact of adherence on effectiveness. If a patient takes the medicine only half of the time prescribed, what impact does that have on controlling their condition?  As an example of this, within a controlled clinical trial, an oral daily asthma drug was found to be less efficacious relative to an inhaled corticosteroid because subjects were on average taking their medicines as prescribed. However, in the real world, the adherence was far greater for patients taking the oral drug rather than the inhaled corticosteroid, and not surprisingly, when evaluated, the oral medicine was found to be just as effective as the inhaled steroid in clinical practice. Increasingly, patients and clinicians and other stakeholders demand clinically meaningful evidence, such as enriched real world studies, to understand how well medicines work under real world conditions in addition to their evaluations under ideal settings.