The drug development process centers on producing the desired effect, but has the process lost sight of who gets to define what the desired effect is? This got us to thinking about the difference between efficacy and effectiveness, especially in the age of calls for patient-centricity and inclusion of the patient voice.  Both terms are used in everyday language as well as technical terminology. These words are deceptively similar but, in drug development, one concerns a controlled process and environment (efficacy), while the other concerns the real world where people exist (effectiveness). Those are vastly different places, especially if you are a patient.  If a patient-centered ecosystem of individuals and advocates, pharma, and technology providers are to work inclusively in drug development, we believe it’s necessary to push for a primary focus on effectiveness so that interventions address our health priorities as we define them.

More and more entities, including regulators, biopharma companies, clinicians, policymakers, payors, and researchers, recognize the value and importance of patient reported outcomes (PROs) as an essential ingredient in developing treatments and interventions that improve people’s lives. Recently, the FDA has elevated PROs as a necessity in product validation in clinical trials and post-trials. Additionally, they have said that physical functioning MUST be measured in clinical trials. As individuals, families, and communities, it’s time to work together to ensure what is being measured aligns with what we want.

Recently, the FDA has elevated PROs as a necessity in product validation in clinical trials and post-trials. Additionally, they have said that physical functioning MUST be measured in clinical trials.

Groups can immediately act to collaborate within and across their communities and conditions to better surface and describe – quantitatively and qualitatively –common goals for treatments and overcoming the burden of disease. By preemptively establishing this – in a manner that meets the quality control and overall data rigor needed by researchers and industry to apply the information – groups can advocate for what effectiveness means to them and influence efficacy measurements.  Better yet, groups can help expand efficacy measurements beyond standard, often blunt physical measurements (e.g., reduction in number of seizures per day, increase in number of steps walked, etc.) into quality of life and other experience goals, which are increasingly becoming vital endpoints.

For example, Genetic Alliance, an organization which helps create and support advocacy groups, is launching a burden of disease study to assess the individual and population-level burden within and across diseases. The study will be administered on the Luna platform to ensure the upmost data privacy and control for all participants, and Genetic Alliance has partnered with QualityMetric, a leader in the development of validated instruments for PROs and Clinical Outcomes Assessments (COAs). The study will include QualityMetric’s SF-36v2® validated measurement instrument, a short questionnaire that measures physical functioning, bodily pain, general health, vitality, social functioning, and emotional and mental health.

Studies like this can serve to educate researchers on the burden experienced by you and people like you, drive researchers to find treatments that focus on the community’s view on critical areas of burden, identify unmet clinical needs and improper care management for your communities, and enable you to build your own custom, disease-specific surveys to the quality level required in clinical trials.

At Luna, we build technology to drive efficiency; efficiency to do the right things right the first time, and follow the fastest path to accurate answers and effective interventions for the people who need them most.

Start sharing your experiences today by joining the Cross Condition Burden of Disease study. Your experiences will form the foundation of a rigorous data set that enables researchers to focus on finding ways to ease your burden and understand the unmet medical need you and others with your disease have.
Join the Cross Condition Burden of Disease study.

About Luna

Luna’s suite of tools and services connects communities with researchers to accelerate health discoveries. With participation from more than 180 countries and communities advancing causes including disease-specific, public health, environmental, and emerging interests, Luna empowers these collectives to gather a wide range of data—health records, lived experience, disease history, genomics, and more—for research.

Luna gives academia and industry everything they need from engagement with study participants to data analysis across multiple modalities using a common data model. The platform is compliant with clinical regulatory requirements and international consumer data privacy laws.

By providing privacy-protected individuals a way to continually engage, Luna transforms the traditional patient-disconnected database into a dynamic, longitudinal discovery environment where researchers, industry, and community leaders can leverage a range of tools to surface insights and trends, study disease natural history and biomarkers, and enroll in clinical studies and trials.