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Label Claims


What is a label claim?

In the US, the Food and Drug Administration (FDA) is responsible for oversight of medicinal products; the concurrent European Union (EU) organization is called the European Medicines Agency (EMA or EMEA). The package inserts accompanying a drug list all of the medicine’s risks and benefits so that patients and their caregivers are informed of them. In the US these inserts are called “labels”; in Europe they’re called “SmPCs”. The label’s content represents the formal, legal representation of what the drug can and cannot do, including side effects and potential hazards or toxicities. The label establishes the legal boundaries of what the medicine’s developers can promote (or “claim”) about its effects. Given the huge investment pharmaceutical companies make in each drug, the stronger their label claim, the better their chances their drug will be received by clinicians and patients. 

How does the FDA view the use of PRO’s in label claims?

In 2009 the FDA released its Guidance for Industry, Patient Reported Outcomes: Use in Medical Product Development to Support Labeling Claims (PRO guidance) providing specific directives related to how PRO data can be used in a product’s label. A central tenet of the Guidance is that the PRO measure must demonstrate that it measures the construct on which the label claim is based. For example, if the pharma sponsor is hoping for a label claim that its drug improves patient quality of life (QoL) and functionality by reducing fatigue, it must use a PRO measure which has demonstrated it is a valid and reliable measure that is sensitive to changes in fatigue. The sponsor must concurrently demonstrate a correlation between fatigue and QoL

Where do Clinical-Outcome-Assessment (COA’s) fit into label claims?

COA’s and Patient Reported Outcome (PRO) Measures in particular, have become widely accepted as valid tools to incorporate in clinical trials to measure the patient’s perspective of his or her disease, treatment, condition or symptom. PROs are widely used in clinical trials to help sponsors differentiate their drug from others that may clinically perform similarly to other drugs. In many cases PRO’s can provide sensitive data for conditions that are not necessarily cured (like side effects), but instead might be mitigated to the point where the patient’s quality of life (QOL) or functional ability is enriched. These can be critical parameters to patients with chronic conditions; having a label claim which states that some aspect of life quality is improved while taking that drug can boost sales significantly.     

From a statistical perspective, a PRO label claim can be based upon primary, secondary or (in some rare cases) exploratory study aims. Using PRO data as a primary endpoint requires that a direct link be established between the change in patient score and the disease, condition or symptom being treated. Using PRO data as a secondary study endpoint would mean that the PRO data would be used as either secondary to improvement in a clinical indication (i.e. the disease would be cured) OR to demonstrate there are other treatment benefits. An exploratory analysis is not typically used for labeling but may result in evidence or data that another study is warranted which focuses on a new finding. Per Gnanasakthy et al (2012) the majority of PRO label claims granted have been primary endpoints focused on symptoms.

How does the EMA view the use of PRO’s in label claims?

The EMA issued a reflection paper in 2005 which did not provide specific requirements, but rather was intended more as a reference document to provide some broad recommendations and context for the incorporation of Health- Related Quality of Life (HRQL) into the drug evaluation process.


The FDA and EMA have differing standards for the use of PRO’s for labeling purposes. The FDA has strict recommendations requiring the validity of the construct being measured and also focuses more on clinical symptoms. The FDA also has more rigorous study design and analysis requirements. The EMA is more flexible with regard to open label studies and more amorphous patient-reported perspectives. Perhaps because of this, the EMA permitted more label claims in oncology trials from 2012-2016. During this period, according to Gnanasakthy et al (2019), 49 oncology drugs across 64 indications were approved by the EMA, however “no FDA labeling was identified.” It should be noted that the FDA’s Oncology Division has reviewed and approved PRO label claims for indications outside cancer, and other FDA divisions outside of oncology have approved PRO label claims.

We note that the EMA views FACIT measures favorably in oncology trials. Gnanasakthy et al (2019) note “A Functional Assessment of Cancer Therapy (FACT) measure was included in 12 (26.6%) submissions and led to labeling for 8 (38.1%) indications. Notably, FACT measures led to a proportionately greater share of EMA PRO labeling when included in a submission (8 of 12 [66.7%]) as compared to EORTC measures (9 of 26 [34.6%]).  These 2 commonly used measures with their disease-specific modules were granted PRO labeling in two-thirds of reviews (14 of 21 [66.6%]).”

What measures has FACITtrans translated that were used in label claims?

FACITtrans has performed all the translations and linguistic validation for any FACIT measure so we are pleased to have contributed to any of our clients’ label claims that included a FACIT questionnaire in their QOL endpoint. 

Confidentiality obligations do not permit us to specify which drugs used non-FACIT PRO’s translated by FACITtrans. Because we have been in this clinical trial “space” for nearly three decades, we are pleased to have contributed to many clients’ successful PRO label claims submissions. We can also state that as a preferred translation partner of the PROMIS instruments, we have collaborated in several studies’ multinational PROMIS claims submissions.

The FACIT Group would like to specifically and gratefully acknowledge the contributions of Ari Gnanasakthy, M.Sc. at RTI Health Solutions and his colleagues who publish periodically on PRO’s in label claims.

Lauren Lent, DHA, MS
President, FACITtrans, LLC
Executive Director,
About Us
© 2020 
Benjamin Arnold, MA
Executive Director, FACITtrans, LLC
Jason Bredle, MFA