Convergence: Regulators, experts weigh dedicated patient experience section in drug labels

PITTSBURGH — Regulators and industry experts considered the pros and cons of creating a dedicated section for patient experience data (PED) in drug labeling on 9 October at RAPS Convergence 2025.

Patient experience data is collected by any persons – including patients, caregivers, advocacy groups and manufacturers – with the intention of providing information about patients’ experiences with a disease or condition. (RELATED: Convergence: FDA, industry experts lead way on patient experience data, Regulatory Focus 15 October 2025)

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) both strongly encourage the collection of patient experience data and patient-focused drug development (PFDD) more broadly.

“Why would we want patient experience data in labeling? FDA states that labeling for prescription medicines is FDA’s primary tool for communicating drug information to healthcare professionals, patients and their caregivers,” said Allison Martin, director of regulatory science and policy in North America at Sanofi.

European regulatory bodies agree that having patient experience data is a key priority in evidence generation and is looked at on par with other clinical data, said Sabine Haubenreisser, PhD, principal scientific administrator, stakeholders and communications, at the EMA.

“We encourage the collection of patient experience data using reliable and validated methodologies as this can contribute to the benefit-risk evaluation, and we see this as complementing primary or secondary endpoints,” Haubenreisser explained.

A high bar for labeling

But getting patient reported outcomes (PRO) data included in labeling can be a challenge as the bar is high. FDA’s 2021 assessment of activity between 2017 and 2017, showed that of 36 novel drug approvals where reviews included patient experience data, 30% of PED was ultimately included in labeling, most commonly patient reported outcomes, but also including information from natural history, observational and qualitative studies. (RELATED: FDA releases first Cures Act report on patient experience data in regulatory decision-making, Regulatory Focus 30 June 2021)

References to PED were most commonly included in labels for drugs treating congenital, familial or genetic conditions (17%), followed by nervous system disorders (14%) and skin or subcutaneous tissue disorders (14%).

“So we are seeing some uptake, but there is room for improvement here,” Martin said.

A Lancet study of novel oncology drug approvals in the US from 2017 to 2022 found that of 45 applications that included PRO data, the vast majority of results (43/45) were not included in the original label, with reviewers citing deficiencies in trial design and data.

In a study of novel oncology products authorized in Europe between 2017 and 2020, out of 128 indications (for 76 products) PRO data were included in 100 (78%) confirmatory trials. However, while PRO data were included for 76 indications (59.4%) were included in European public assessment reports, they were only included for 22 indications (17.8%) in the summary of product characteristics (SmPC).

When considering inclusion in labeling, EMA needs to determine how the data was generated, its clinical relevance, whether the data are scientifically sound, complete and free of bias, Haubenreisser said. In an effort to be more transparent about the generation and use of patient experience data, an expert team assembled and published findings on the topic at the end of September. (RELATED: EMA tells sponsors to incorporate patient experience across product lifecycle, Regulatory Focus 29 September 2025)

“You have four months to provide your input, and we would very much welcome that. And then we will obviously publish the final version,” Haubenreisser said.

Pros and cons

There is some pushback on the idea of a special section for PED in labeling, with some saying the label is for physicians, not patients. In a perfect world, physicians would not only read the prescribing label but also search the literature for relevant information on patient experience, but that does not always happen, Martin said. Including patient experience data in the label could expand access to healthcare professionals (HCPs), patients and caregivers, while helping companies avoid legal risks, she added.

Patients are not typically reading labels, but that’s not necessarily the purpose of including PED in labeling, said Aviva Rosenberg , co-founder and co-president of the Gaucher Community Alliance.

“Once it's in there it can be used for HCP education, and then the HCPs can make appropriate decisions for the families,” Rosenberg said. “It's the prescribers that really know the families and when they have this information about how patients respond, or what patients’ experiences were with a particular product, they're better equipped on the frontlines to be able to say [whether a] treatment might work.”

The healthcare provider understands the patients and their families’ social and living situations, which factor into treatment decisions, explained Rosenberg, who said that she herself and her son have Gaucher disease.

Labeling information could be used for a structured way to discuss particular symptoms of interest to particular patients and treatment impact, improving informed decision making between patients and doctors, commented Elizabeth Rosenkrands Lange, executive director/head of regulatory & scientific policy at EMD Serono/ Merck KGaA.

“I could see a huge benefit in bringing this label section to the forefront for the healthcare professional, making it easier for them to navigate and learn what is the patient experience data for [a] specific drug,” Lange said.

Lange said she has heard a lot of positive feedback about the idea of a dedicated patient experience section, but also some concerns. For example, companies that don’t have the resources to collect PRO data wouldn’t be able to complete a dedicated section and there are questions about how easy it would be to get the label PRO data updated post-marketing. It’s important to bring all stakeholders into conversations on the topic, considering benefits and risks, she said.

Speaking by phone remotely, Robyn Bent, director of the FDA’s Patient-Focused Drug Development Program, noted that a new PED section would require a formal notice of rulemaking and associated following processes.

There are a number of reasons why data might not wind up in a label, including quality issues, Bent said. Also, the label is not meant to exhaustively address everything about a product – there are significant space limitations to the amount of information that can be included, she said. FDA has to be thinking about what is the most critically important information for prescribers to include in the label and in making this determination, agency officials have discussions with the drug developers.

“A lot of factors play into the final decision,” Bent said.

Haubenreisser said there are no plans in Europe to change the summary of product characteristics, which already has a home for PED in section 5.1 (titled: pharmacodynamic properties). There are clear criteria for sponsors for getting data from trials and postmarketing, including from registries, included, Haubenreisser added. Sponsors should reach out to EMA early for scientific advice and opinions on including patient experience data in trials and in their application dossiers.

“Industry knows that we have a large part to play here as well,” Martin said. “We need to make sure that we are developing the data in a way that is rigorous enough to meet the same level of standards as the other clinical trial data and make sure that we are communicating to the agency what it is, where it is and how we want them to use it, so that we can all move forward in the same direction.”

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Convergence: Regulators, experts weigh dedicated patient experience section in drug labels

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