What clinical real-world data can deliver

This article was first published in STAT

Thanks to new regulatory frameworks like the Food and Drug Administration’s Breakthrough Therapy designation, drugs are coming to market faster than ever. A 2024 analysis of drugs included in this program showed they had 23% shorter clinical development timelines than those that were not part of the program, with no negative effects on their safety profiles. This regulatory innovation is great for patients, pharmaceutical companies, health care providers, and the full health care landscape, getting new medicines to the people who need them in ever-shorter time.

Using real-world data to monitor drug safety and effectiveness adds an important capability to identify ways to improve patient and clinician experiences with a drug, based on side effects, adverse events, prescribing patterns, and patients’ subsequent care needs following their use of a drug.

PA Consulting, the company I work for, recently conducted a review of clinical data from various sources to identify the number of people seeking care in emergency departments who reported using a GLP-1 weight loss or diabetes drug and who had a complaint related to the known and reported side effects of these products, including nausea, vomiting, diarrhea, stomach pains, and constipation.

The analysis identified some patients who presented to emergency departments reporting side effects from GLP-1 weight loss or diabetes drugs.

Although clinicians report in interviews and journal articles that they are seeing patients with these side effects, the prevailing perception among clinicians is that the clinical and therapeutic value of GLP-1 medications outweigh the severity of side effects.

The fact that patients are going to emergency departments and reporting side effects from GLP-1 drugs is interesting not because these side effects exist: the companies that produce GLP-1 drugs are aware of them and openly caution patients and health care providers that they are not uncommon. But the real-world data on a medication’s side effects can offer pharmaceutical companies valuable insights. Real-world data can point to opportunities for innovation and care improvement, and support additive or novel research and development.

Registries and other sources of clinical data can surface safety issues like adverse events or side effects. But they can also deliver new insights that go beyond triggered regulatory reporting and offer useful and actionable insights. In our work with pharmaceutical companies leveraging real-world data to understand the incidence of side effects, we have seen several interesting examples of how the data have been used — not only by those focused on safety and pharmacovigilance, but also by medical affairs, patient advocacy, and clinical operations team members.

When a drug comes to market quickly, there is less time to educate and train clinicians on how it is best used and prescribed, and in what settings. It is the purview of a company’s medical affairs team to relay clinical knowledge gained from trials to health care professionals. But if the trials are significantly shorter, and the overall time to market is reduced, that crucial activity is compressed. As clinical and patient awareness of a new therapy grows, clinicians may prescribe it without having had the benefit of comprehensive education from a medical science liaison or other educator.

Medical affairs personnel can leverage novel data from real-world settings to support ongoing provider education about prescribing the therapy to the most appropriate patients with the most relevant health needs. They can also leverage real-world data to support clinicians on what site of care is most appropriate for a clinician to prescribe a new medicine or use a new device, such as a hospital or outpatient facility. Medical affairs teams can also educate clinicians about how best to support patients who experience a side effect or adverse event, the optimal patient profile and health history for a particular therapy, and how clinicians can collaborate with the pharmaceutical company’s medical affairs team.

Pharmaceutical data experts can also collaborate with patient advocacy colleagues to identify side effects and the best practices for mitigating them. The anecdotal, patient-level experiences of patient advocates can be augmented by real-world data that tells a more comprehensive story at a larger scale, bringing together quantitative and qualitative insights.

The objective of having patient advocates in a pharmaceutical company is to understand and apply patients’ voices and experiences in drug development and commercialization. Real-world clinical data can provide this insight because it offers a detailed record of the treatment patients are given, helping pharmaceutical organizations understand patients’ disease histories, their current states of treatment, and what future care might be useful. With real-world clinical data from health records, patient advocates can access new data and information about clinical treatments from the pharmaceutical company and then provide that information to patients and their health care providers. In this way, patient advocates can support patients on their journeys to effective use of therapeutics and management of their condition because they have better and more complete information about the care and treatment a patient experienced.

Using real-world data, clinical operations personnel can design and implement new or additive studies to support novel claims and/or an improved understanding of a drug’s safety profile. This approach can be done using existing data sets or new ones, leveraging the insights that a real-world dataset can provide to clinical researchers to generate new ideas for study.

Real-world data provide new insights into the care clinicians deliver and patients receive. Clinical notes and medical records can help understand clinician and patient perspectives and identify unmet needs that can be translated into new research to meet those requirements. For example, analyses of real-world clinical data have in some cases identified safety signals of therapeutics and diagnostics on the market that suggested a need for additional research, or additional monitoring requirements or expectations.

Real-world data are now being used in a variety of clinical studies to support label extension and to better understand the safety profile of drugs or diagnostic tools. A study of the quadrivalent human papillomavirus vaccine, for example, assessed long-term effectiveness, immunogenicity, and safety for up to 14 years after the start of the vaccination by leveraging registry data.

Various roles in pharmaceutical organizations can support patients and providers with tools, information, and education to prevent or manage the potential side effects of a treatment. By providing insight into how patients may experience a drug’s side effects or how clinicians can deliver effective guidance to patients when prescribing a therapy, leveraging clinical real-world data can bolster a pharmaceutical company’s goodwill and brand equity with patients and health care providers.

For products in competitive categories, like GLP-1 weight loss or diabetes drugs, leveraging real-world data to improve patient and provider experience can yield a competitive advantage, delivering insight into patients’ and clinicians’ experiences. If a product in a competitive category comes to be known for side effects that result in users seeking emergency care or discontinuing the therapy while its competitor is known for their safety, effectiveness, and a supportive education and medical affairs team, the company that delivers the better experience will win in the market even if the two products have the same side effects.

Leveraging real-world clinical data in novel ways can result in important changes to pharmaceutical companies’ ways of working to deliver a competitive advantage to products that are coming to market more quickly, and safely, than ever before.