Community

Leveraging Community Voice to Bring Effectiveness Back into Efficacy

by Luna

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.

Community

Blood Donations Provide Lifesaving Treatments and Potential Research Opportunities

by Luna

An ongoing partnership answers the call of blood donors by providing new clinical research opportunities. After the success of community-focused studies to develop COVID-19 vaccines and treatments, blood donors asked what more they could do as research participants to improve the health of their community.

Through an ongoing collaboration between San Diego Blood Bank and LunaPBC, blood donors are able to support health research along with providing a lifesaving donation.

“When we began collecting convalescent plasma for COVID-19 hospital patients at the height of the pandemic, donors who recovered from the virus came out in droves to donate to help those in their community and beyond,” said Nikhil Nayak, Chief Business Officer for San Diego Blood Bank. “Our blood donors often ask about additional ways they can support our mission, so we are proud to afford them the opportunity to influence the future of personalized medicine through innovative programs like this.”

San Diego Blood Bank is a platform for ensuring the community’s health by connecting its resources and diverse blood donors with research opportunities. Luna was the optimal partner because of its people-centered framework that increases participant engagement and retention, ensures interventions meet the priorities and needs of individuals, and facilitates comprehensive, longitudinal studies.

Read San Diego Blood Bank and LunaPBC Provide Blood Donors an Opportunity to Contribute to Health Research.

“Factors such as genetics, age, gender, and ethnic origin play an important role in the effectiveness of medical treatments, which is why diversity in research participants is incredibly important,” said Nayak.

Dedicated blood donor Jeff Gonka said, “One standard blood donation could save up to three lives, but one small blood sample donated for research could potentially save thousands of lives.”

Do you live in or visit the San Diego area often? Join the San Diego Research Community on Luna today.

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.

Community

Reflections for 2023

by Luna

In 2022, Luna prioritized quality research, privacy, and community. We launched a number of studies with partner organizations, established Community Driven Innovation™, and shared our thoughts on the changing dynamics surrounding health privacy. Looking ahead, Luna reflects on the previous year to predict the important trends of 2023.

Privacy and Trust Concerns Grow in Research

Dawn Barry

Dawn Barry, Luna president and founder

Unfortunately, the health space has lost ground in consumer trust, with growing criticism in areas like pandemic response, mRNA vaccines, data surveillance and selling, and cyber breaches involving medical information. This lack of trust could further hinder people’s involvement with research, especially as the industry looks to incorporate digitalization — through real-world data, decentralized clinical trials and wearables — into next-generation trial designs. To move forward, we need to get back to basics on human trust. We need to be transparent, consistent, clear in our communications, inclusive and ethical. In other words, we need to do what’s right.

Acknowledging that individuals should control the research use of their data is only a starting point. Embracing modern data privacy laws like GDPR and CCPA is another meaningful step in demonstrating respect for study participants.

Labor-Shortage Crisis in Health Care

Joe Beery, Luna chief executive officer

The healthcare worker shortage is a significant worldwide concern, and what’s also embedded in this challenge is that many of our most experienced doctors also operate as lead researchers for new medicines and therapies. We need to ensure the funnel for vital research that feeds innovation is protected and that this population of professionals is well taken care of.

Participant-Reported Research

Ian Terry

Ian Terry, Luna senior user experience researcher

Measuring patient-reported outcomes is key to ensuring medical interventions meet the needs of the people they aim to serve. But what about patient-reported goals, lived experiences and quality-of-life desires? In 2023, I want to see more research that starts with and fully integrates the patients’ goals and measures how those outcomes were met. In establishing a full continuum that’s centered on what patients need and want, the patients win because they ideally get something that improves their life — not something industry thought they needed — and researchers and industry establish more clinically relevant outcomes to meet, thereby de-risking their development efforts.

Measuring patient-reported outcomes is key to ensuring medical interventions meet the needs of the people they aim to serve.

Balancing Life, Research, and Work

Debora Thompson

Deb Thompson, Luna vice president of operations

COVID-19 spurred the necessity of virtual work on a scale not seen before. It changed people’s perspectives on work-life balance. On the positive side, the flexibility of being able to interweave work with life means fewer missed opportunities to spend time with family, exercise, and take a breath when needed. On the negative side, missed interaction with fellow employees means more missed opportunities to brainstorm, forge bonds and connect. As always, a balance is needed to garner the best of both worlds. Why, then, do we not require the same balance with research? Tests, measurements, and doctor-patient interactions are extremely valuable to research; however, they are only one side of the story. “Real-world evidence” and “real-world data” are not things that can be fully captured in a doctor’s office or clinical site that people are forced to travel to — increasing their stress, anxiety, and other negative symptoms. What are the real lived experiences, symptoms, and behaviors of people in their normal environment? Scientists and doctors must be willing to listen to the experiences of the patients themselves; they must be willing to include data captured from patients in their normal environments. Real “real-world data” is not the electronic health record; it is the voices of the people who live with a condition. We must have REAL #LifeResearchBalance.

Protecting Privacy

Scott Kahn

Scott Kahn, PhD, Luna chief information and privacy officer

De-identification of personal data has become a myth. We live in an age of re-identification because of the proliferation of data that can be collected on individuals and the power of artificial intelligence. De-identified DNA data is an oxymoron. The world must fully embrace a rights-based approach to data control that seeks targeted informed consent for personal data use. This approach is used with the EU’s GDPR, other countries and is now expanding state-by-state across the U.S.

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.

Community

Expanding the Conversation Around PCOS with Participant-Led Research

by Luna

Affecting more than 115 million women globally, polycystic ovary syndrome (PCOS) is not considered a rare disorder. However, it’s often underdiagnosed, under-researched, and under-discussed. Diagnosis usually comes after other disorders have been ruled out, a process that often takes two years or more for women. 

To advance understanding and support for people with the disorder, PCOS Challenge: The National Polycystic Ovary Syndrome Association partnered with Luna to raise the collective voice of those affected and drive patient-focused research. 

“One of our key goals is to unite the major PCOS stakeholders in the largest organized effort to address gaps in care and to find a cure for PCOS,” says William Patterson, founding Executive Board member and current Director of Public Affairs at PCOS Challenge. 

PCOS Challenge has had a long history of elevating those affected by PCOS as partners in research. “Many investigators miss a huge opportunity by not fully engaging patients as true partners in research and discovery. There is a tendency to design studies with little-to-no meaningful patient involvement and to seek a rubber stamp from patients,” he says. 

“We see the frequent misalignment between government, clinical, and patient research priorities which often stem from not listening to patients from the earliest stages of research and its impact on treatment, care, and counseling,” Patterson says. “We see this as an important means of leveraging patient insights, priorities, lived experiences, and skills to fill gaps in the literature, disrupt outdated narratives negatively impacting patient care, and accelerate discovery.”

Many problems exist regarding access to care and quality of care for those with PCOS, stemming from a lack of focus on health policy, underfunding of research, and the type of research being funded, he said. 

We see this as an important means of leveraging patient insights, priorities, lived experiences, and skills to fill gaps in the literature, disrupt outdated narratives negatively impacting patient care, and accelerate discovery.

William Patterson

“Historically, the National Institute of Child Health and Human Development (NICHD) funded about 70% of PCOS research. While the NICHD has done tremendous work and contributed greatly to our understanding of PCOS, its mission is largely limited to human development and reproductive health. When you have only one institute funding a majority of PCOS research, it ultimately creates a skew in the literature.  In this case, the skew is largely toward the reproductive aspects of PCOS,” he says. 

This equates to PCOS being viewed as a reproductive disorder, not a lifelong debilitating condition that comes with other co-morbidities, including mental health, metabolic, cardiovascular, endometrial cancer, liver disease, and other health risks.

“Through our awareness and advocacy efforts, we’ve expanded the conversation around PCOS, so patients and health professionals are more aware of the co-morbidities associated with the disorder and the impact of PCOS on people throughout their lifespan.”

This is why a patient registry is critical, says Patterson.

Propeling discoveries for PCOS research

Two studies have already been launched: the PCOS Challenge Economic Burden Study, which examines the intangible and indirect costs of having PCOS, including quality of life and work productivity, and the PCOS Pregnancy and Maternal Health Study, which aims to identify the gaps in education about pregnancy, and maternal and child health to improve awareness and counseling. 

The medical literature suggests women with PCOS are at greater risk of experiencing maternal health complications, like preeclampsia, pregnancy-induced hypertension, and pre-term delivery. These two studies will help provide a clearer estimate of the economic burden and true costs of having PCOS in terms of quality of life and work productivity and help close gaps in awareness, education, and counseling related to pregnancy and maternal and child health risks for those affected by PCOS.

“We see the Luna platform and our patient-powered registry as a means to focus on the questions that patients want to be answered,” Patterson said.

Patient-led research provides mentoring opportunities for investigators

PCOS Challenge is working strategically to reach as many individuals as possible and to empower multiple stakeholders through its patient-powered registry, The PCOS Challenge Study. One of the advantages of the PCOS Challenge partnership with Luna is that it helps the organization achieve one of its goals: getting more early-career scientists involved in PCOS research. 

“We see a lack of mentoring and development opportunities for early-stage PCOS investigators,” he says. “In connection with the PCOS registry, we are creating an early-career forum to help these emerging investigators receive mentoring opportunities from senior investigators in the field where we have relationships.” 

As an extension of the organization’s mission, the studies also help PCOS Challenge shape the future of medicine by promoting a unified, international research agenda that includes patients and scientists contributing to understanding PCOS and supporting individuals who live with the condition.

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.

Community

Community Driven Innovation Delivers Participant-Led Research

by Luna

In Duchenne muscular dystrophy research, the six-minute walk test is considered the standard endpoint to determine if a treatment would be successful. Because walking abnormalities are prominent in patients, researchers measure how far a patient can walk in six minutes to determine if a drug holds promise.

Ian Terry
Ian Terry, senior user experience researcher

When the COVID-19 pandemic occurred, most children and their families could not travel to research facilities for these six-minute walk tests. Instead, researchers began conducting phone interviews to continue their research and grant funding. They learned that, although specific treatments may not improve walk test distance, individuals were sharing stories of how their legs were getting stronger by walking their dog or standing up and playing catch—endpoints that had nothing to do with how far they walked. 

“It took the stress of a complete pandemic shutdown for researchers to begin asking the children and their families, what’s truly important to you?” says Ian Terry, senior user experience researcher at Luna. Terry was instrumental in developing a new methodology based on community-driven versus expert-led research—research driven by what individuals living with the condition determine as a priority. 

Community Driven Innovation™, or CDI, is a participant-led methodology that addresses long-standing problems with traditional research approaches while providing an unbiased, clear understanding of a community’s priorities, values, and challenges.

“Community Driven Innovation uses tools to align research around the needs and priorities of a patient group—or any health-focused community with a research question or problem to solve,” says Terry. “The complex science is built-in and “under the hood,” so to speak, making CDI accessible to anyone, no Ph.D. required.” 

Often, parents, caregivers, and family members don’t think in medical terms, but by using contextual interviews and other listening approaches, CDI was able to reveal families’ top concerns.

Ian Terry, Luna

CDI has been applied to research championed by patient advocacy groups, including KCNT1 Epilepsy Foundation, The Aicardi Goutières Syndrome Advocacy Association (AGSAA), and Bobby Jones Chiari & Syringomyelia Foundation. Luna has also utilized the methodology as the engagement foundation of the Veterans’ Health Priority and Women’s Health Priority communities.

Kaitlyn Esposito, MPH,  who leads programs and research for Bobby Jones Chiari & Syringomyelia Foundation, used CDI to determine their community’s readiness for FDA-approved treatments and clinical trials.  “We learned a lot of really interesting things, including just how complex the symptoms are in these conditions.  We knew it was complicated but were surprised at how complicated it truly was,” shared Esposito.

“Using CDI, the Aicardi Goutières Syndrome (AGS) community uncovered the importance of muscle tone management as a top priority,” Terry says.“ Often, parents, caregivers, and family members don’t think in medical terms, but by using contextual interviews and other listening approaches, CDI was able to reveal families’ top concerns.”

Historically, the clinicians or researchers would be the one who determines the research question. With CDI, the community can become research partners and advocate, in a data-driven way, for the real challenge or question they need answered.

In rare disease groups, researchers can quickly get funding to focus on a topic that may not already be supported. Those researchers often embark on a project with the most accessible endpoint to demonstrate results. However, that endpoint may not be what’s essential to that rare disease community, as shown with the walk test in the Duchenne muscular dystrophy community.

It’s easy to talk about participant-focused research. It is easy to speak about being patient-centric,” says Terry. “But CDI, for the first time in the health space, gives researchers a tool that marries true participant-focused research with patient engagement in a way that is both scientifically driven and a low burden to affected individuals, their families, and caregivers.”

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.