How Do Various Stakeholders Define Clinically Meaningful Data?

Patients and Patient Advocacy Groups

One of the most critical stakeholders in the healthcare industry is the patients. At its core, clinical practice is intended to serve patients by managing diseases and improving their quality of life.

On the individual patient level, clinically meaningful data may relate to their experience with a particular medication or drug therapy. For example, they may consider improvements to quality of life, well-being, and overall health.

In addition, a patient on a drug therapy with fewer side effects than other treatments for the same condition may consider the clinical significance compared to other interventions.

Beyond the ethical and moral responsibility to develop drugs that improve clinical outcomes, understanding patient perceptions of clinically meaningful changes can help pharmaceutical and biotech companies understand which products to market and invest in.

Although clinicians are a type of consumer that drives pharmaceutical sales, a patient's perceptions about medication will be a significant factor in pharmaceutical sales and the return on investment for pharmaceutical manufacturers.

Pharmaceutical companies and investors evaluating clinical studies and outcomes for clinical meaningfulness may consider the impact of patient-reported outcome measures (PROs) such as pain levels, symptom relief, and changes in quality of life.

In the United States, the Center for Drug Evaluation and Research (CDER), under the US Food and Drug Administration (FDA), emphasizes patient-focused drug development, which heavily considers the patient perspective in the drug development and evaluation process.

In addition to individual patient perspectives or insight into the definition of clinically meaningful data, patient populations may comprise another important stakeholder: patient advocacy groups.

The Rare Diseases Clinical Research Network defines patient advocacy groups (PAGs) as “organizations that promote the needs and priorities of patients.”

While PAGs may measure clinically meaningful data comparable to how patients evaluate clinically significant data, they also consider other factors that may impact future research and advocacy efforts.

First, these organizations — like individual patients — consider PROs and disease-specific symptom relief when evaluating health outcomes. Real-world evidence that points to improvements in health-related quality of life is crucial to these organizations, as their primary goal is often to improve quality of life (QoL).

Other factors heavily weighed by PAGs include functional improvements, treatment tolerability, long-term outcomes, and patient safety.

Beyond those measures that are common across all stakeholders, PAGs may also look for a statistical analysis or data set that defines how a particular treatment impacted a patient’s psychosocial well-being.

Additionally, PAGs may consider data more clinically meaningful if it can be leveraged for advocacy efforts, policy changes, or research funding, which, over time, can impact clinical decision-making and drive innovation to benefit patients.

Healthcare Providers

Healthcare providers, including clinicians, prescribers, and nurses, may use a slightly different strategy to assess what data is clinically meaningful. Although they value clinical outcomes such as symptom improvement and other vital factors to PAGs and patients, they also consider additional factors, such as medical history, diagnostic results, laboratory tests, and more.

When looking at study data and results, providers may consider multiple factors. Primarily, providers consider the relevance to patient care. A study that identifies biomechanisms or molecular changes without explaining applications to patient care may not be considered clinically significant to a provider despite impacting future drug or treatment development.

Other data that may impact clinical relevance to providers focuses on the study population. When considering whether a specific treatment may apply to their patient, providers may look to factors such as diagnostic information, disease progression stage, participants' medical history, medication history, and allergies or sensitivities.

Like patients, providers consider factors such as treatment effects, safety, and adverse events to determine whether treatment may be a good fit for their patients.

Factors like cost-effectiveness, care coordination, and clinical guidelines may also impact a provider’s perspective on clinically meaningful data.

Pharmaceutical and Biotech Companies

Pharmaceutical and biotech companies often have a vested interest in seeing clinically meaningful data as these companies invest money and time into these studies, looking for a return on investment (ROI).

Pharmaceutical and biotech companies may define clinically meaningful data based on the following outcomes.

• Safety and tolerability: Like most other stakeholders, companies and investors look to ensure that any treatment is safe and tolerable. Without this, the study cannot have any clinical applications.
• Efficacy and effectiveness: Naturally, companies look for a treatment that accurately addresses the targeted condition. While not all treatments may cure the disease, pharma and biotech companies may look at how they improve symptoms from baseline assessments.
• Clinical endpoints: Clinical endpoints are predetermined results that have clinical significance to researchers. Often, investigators determine a primary endpoint, such as survival rate or pain reduction, to determine if a treatment is effective. Researchers may also identify secondary endpoints.
• Patient-reported outcomes: Biotech and pharma companies may heavily consider PROs, as patients are their primary consumers.
• Comparative effectiveness: Companies may ask, is this treatment more effective than other treatments? Does it provide a competitive advantage?
• Dose-response relationships: Studies identifying dose–response relationships are also vital for these companies as they can indicate optimal dose, frequency, and duration of treatment.
• Long-term follow-up: Long-term follow-up data is vital to these companies because it indicates sustained safety and treatment durability.
• Subgroup analysis: Understanding how different subgroups may benefit more or less from a specific treatment is clinically valuable for pharma and biotech companies, as it can help them market to particular patient groups.
• Clinical utility: Clinically meaningful data should also indicate the clinical utility of a treatment.

Understanding how various stakeholders define clinically meaningful data can be vital for pharmaceutical sales, marketing, and patient outcomes.