In pharmaceutical research, there is a growing emphasis on the importance of the patient experience. Pharmaceutical companies are under increasing pressure to show treatment efficacy as well as the impact of treatment on patients' lives. Furthermore, there are evolving regulatory requirements from Health Authorities to put patient’s at the center of the drug development. The FDA has even released a four-part guidance series providing step by step recommendations on how to collect and submit patient experience data for regulatory decision making.
Patient Reported Outcomes (PROs) have thus become an important piece of clinical trials endpoint strategies and are gaining prominence as a way for pharmaceutical companies to support drug labeling claims.
What are PRO claims?
PROs are health outcomes directly reported by patients themselves about their health condition and its treatment, without interpretation by a clinician. Collected using a standardized questionnaire, PROs capture subjective experiences that may not be apparent through clinical testing.
A labeling claim is a statement on a drug's label or packaging that describes its intended uses, effects, or benefits. These claims inform healthcare providers and patients about what the drug can do, any side effects, and how it can improve health outcomes. The stronger the label claim, the better a pharmaceutical company’s chances that the drug will be prescribed by clinicians and used by patients.
A PRO claim is thus a statement in a drug's label that is supported using PROs and directly echoes the patient's experience with the treatment. These claims can be regarding a patient's health status, from symptoms and physical functioning to overall quality of life.
Growing importance of PRO claims
PRO claims have become increasingly important recently, given the growing importance of the patient experience in the drug development process. By putting the patient voice at the forefront of treatment evaluation, PRO claims embody the essence of patient-centricity in healthcare. These claims offer a comprehensive assessment of a treatment's impact, capturing nuanced aspects of health that may not be seen with clinical measures alone.
PRO claims also bridge the gap between clinical efficacy, captured with objective testing, and real-world impact, as experienced by the patient. This patient-centric approach is critical in a competitive market, where a better patient experience with the treatment can offer a unique selling point that resonates with both healthcare professionals and patients.
Furthermore, with regulatory bodies increasingly emphasizing the value of patient-reported data, PRO claims are not just beneficial, but often becoming necessary for regulatory approval.
Thus, PRO claims are crucial for several reasons:
- Patient-centricity: They put the patient's voice at the forefront of treatment evaluation
- Comprehensive assessment: PROs capture aspects of health that may not be captured through clinical measures alone
- Real-world impact: They help healthcare providers and patients understand the practical benefits of a treatment on daily life
- Competitive advantage: Strong PRO claims can differentiate a drug in a crowded market
- Regulatory support: Many regulatory agencies, including the FDA and EMA, increasingly value PRO data in their approval processes.
Securing a PRO claim for your drug
Obtaining a PRO labeling claim is a process that requires careful planning and execution from the early stages of the trial lifecycle. Researchers must select PRO instruments that are fit-for-purpose and relevant to their drug's intended effects, and are relevant to the patient's experience of the disease. Then, researchers must select PRO endpoints that will be measurable, clinically meaningful, and statistically robust to withstand regulatory scrutiny. The trial protocol should clearly outline how these endpoints will be analyzed, ensuring that they not only meet scientific rigor but also provide actionable insights into the patient experience.
As data is collected, analysis and interpretation must be statistically sound and present a compelling case for the drug's impact on patients' lives. The data must support not only the treatment’s efficacy but also its impact on patient quality of life.
If you’re looking to secure a PRO claim for a treatment, follow this 4 step process to ensure a patient-centered endpoint strategy (according to FDA roadmap).
- Collect patient experience of the disease
- Identify the relevant concepts of interest based on patient experience and expected therapeutic benefits, as well as the context of use for clinical trial.
- Identify the type of COAs (PROs being part of COAs) and the COAs to measure concepts of interest in the context of use. The selected COAs could be existing COAs, modified COAs or newly developed COAs
- Define the COA scores to inform the trial objectives, ensuring that the outcomes are both meaningful to patients and relevant to regulatory requirements.
By leveraging this patient-focused framework into clinical trial design, researchers can better position their product for a PRO labeling claim that resonates with both regulators and patients.
Mapi Research Trust’s expertise in PRO claims
Mapi Research Trust is the leading provider of Clinical Outcome Assessments (COA), including PROs, and COA consulting services. With its vast array of validated PRO instruments, coupled with expert guidance on instrument selection and implementation, our team can simplify the often-complex process of instrument selection and implementation.
Perhaps most crucially, Mapi Research Trust offers insights into regulatory requirements and trends related to PROs. This support can be instrumental in navigating the complex regulatory landscape surrounding PRO claims.
Mapi Research Trust offers:
- A comprehensive database of PRO instruments
- Information on the development and validation of these measures
- Guidance on selecting appropriate PROs for specific conditions or treatments
- Licensing and copyright information for PRO measures
- Existing PRO claims of FDA and EMA-approved drugs to support a successful endpoint strategy in the product approval process
For researchers looking to strengthen regulatory approval with PRO claims, Mapi Research Trust can play several important roles:
- Instrument access: our ePROVIDE™ platform offers a wide range of validated PRO instruments.
- Expertise: Our experts can offer guidance on instrument selection and implementation.
- Licensing: We manage licensing for many widely-used PRO instruments.
- Translation and linguistic validation: We can support your translation and cultural adaptation needs for global studies.
- Regulatory support: Our experts can offer insight into the regulatory requirements related to PROs and their use in labeling claims.
In conclusion, PRO claims are a powerful tool in differentiating treatments and demonstrating value from the perspective that matters most - the patient's. By developing a thoughtful PRO strategy and leveraging available resources like those offered by Mapi Research Trust, drug developers can enhance their regulatory submissions and provide valuable, patient-centric information to healthcare providers and patients alike.
Please contact us today to learn more about how we can support your PRO labeling claims.