Biopharma embracing real-world evidence, report says
- Results from Deloitte's second annual real-world evidence benchmarking survey, out late last week, show that the variety of sources of real-world data are expanding, including electronic medical records, health insurance records, genomics, social media, and wearables; and the volume of data is growing.
- Between January and April 2018, Deloitte surveyed 20 professionals from real-world evidence, IT, scientific, medical, and business teams at global life sciences companies. Biopharma companies are investing more in real-world talent and technology, and increasingly building in-house capabilities though they face challenges of overcoming barriers to adoption and value.
- The Food and Drug Administration has created guidelines for the use of real-world evidence, and the European Medicines Agency recognizes real-world data for certain approval types.
Real-world evidence is based on data from patients outside of controlled clinical trials, better reflecting how patients take drugs and respond to them in their everyday lives. This includes the kinds of people who wouldn't be included in clinical trials, such as the elderly, or those with a number of different conditions.
The FDA, under the provisions of the 21st Century Cures Act and the Prescription Drug User Fee Act (PDUFA VI), has created timetables for the use of real-world evidence in a regulatory context. The EMA is also involving real-world evidence in its regulatory pathways. Its PRIME (PRIority MEdicines) program uses patient registries, health records, and other sources of data to identify unmet medical needs, providing a route to accelerated assessment and expedited approval for companies that can meet these needs.
At the same time, biopharma company staff are increasingly looking to real-world evidence. This is not only to back up the value story for payers and create the basis of value-based contracts but also to understand the market, support drug development and improve the prospect of drugs making it through regulatory approval.
Responders to the study suggested a number of important current and future roles for real-world evidence. Examples included analyzing treatment impact across subpopulations to improve treatment and management of side effects; better understanding disease, to advance drug development; providing synthetic control arms for quicker market access; and quantifying treatment outcomes for value-based contracting.
There are many different ways to collect real-world data, but one of the most accessible for patients, especially younger patients, is via a smartphone app, which can collect sleep, activity and other datasets, and can also remind people to input other data.
The amount of data available is rising so fast that analysis can create a bottleneck. Artificial intelligence approaches, using blockchain technology and machine learning, can securely access and analyze personal data.
Most responders to the Deloitte survey are still working out how to track the return on real-world data spending. Others, however, are working on different financial and non-financial metrics to measure the short- and long-term impacts. These include numbers of studies published, use of real-world evidence to support health technology assessments, spending with external vendors, and effect on development productivity and product sales.
To make real-world evidence work, end-to-end evidence management will be vital. This ensures that data generated at all stages of drug development, from drug discovery to commercialization and post-marketing studies can be accessed and used to generate and optimize product value.
"This could not only enhance research productivity and assessment of drug effectiveness but also support pricing, customer engagement, and marketing," wrote the reports authors.
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