Data from real-world use is important for patients and payers to understand how drugs work in uncontrolled environments.
Patients, insurers, and policymakers are now demanding to know more information about new medications that goes beyond the scope of clinical trials. These individuals want to know information such as which patients the drug will work the best in, how it compares to similar drugs, and which are the most cost effective, according to analysis conducted by AbbVie.
Real-world data could provide information about how the drug will work in environments other than in clinical trials, which is why pharmaceutical companies and academic institutes are analyzing data to understand how drugs are utilized, according to AbbVie. Although stakeholders are inquiring about new information, clinical trials are still necessary to determine the safety and efficacy of new drugs.
The biggest challenge between the translation from clinical trials to real-world use is the patients themselves, the report noted. Patients included in clinical trials are typically very similar, while the general population varies greatly.
Patients could need to take other medications that were not tested with the new drug in clinical trials, or they could potentially have more than 1 disease. Patients may also experience different adverse events, and medication has the potential to work differently in each patient, AbbVie reported.
Such variation can cause challenges for insurers, who have to decide whether or not to include a new drug on their formularies.
“In the end you really need both kinds of studies,” said Glen Schumock, PharmD, PhD. “We will continue to have randomized clinical trials as the gold standard for drug development, but we also need to have these Phase 4 or comparative effectiveness or real-world evidence studies.”
According to the analysis, real-world data can be categorized as clinical data that helps evaluate safety and efficacy, economic data that evaluates value, or humanistic data that evaluates quality-of-life.
To gather this information, researchers buy data from insurance companies, healthcare systems, or insurers that can have significantly more data available compared with clinical trials. However, real-world data can be inaccurate for all patients, especially if the data is collected from only 1 area, and the data typically does not include humanistic data, according to AbbVie.
Researchers are currently looking for supplementary information from sources such as patient networks that detail patient-reported data. PatientsLikeMe includes patient-reported data about aspects of the medication that are important to them.
“We hope that there is patient-generated evidence that can be journal worthy and even find its way into regulatory submissions for new products,” concluded Jeremy Gilbert, vice president of PatientsLikeMe. "If we can get that trifecta of patient-generated data, outcomes data and medical records all in the mix, we'll have a really amazing real-world evidence base to use for all kinds of decisions."