Leveraging Community Voice to Bring Effectiveness Back into Efficacy


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.


A Case for Progressive Institutional Review Boards


Innovation is a constant topic in the biomedical research space. The pace of new tools, new techniques, and new discoveries is often hard to keep up with. Researchers and study participants expect innovation to deliver improvements as well as compatibility with new science, new methodologies, and new priorities, all in a constantly changing, complex environment for study participants.

This progress is almost always seen in the insights and outcomes of the research, but not in how the research is designed, begins, progresses, and is managed. Anyone who has done work in the biomedical research space knows that ethical oversight – whether by internal reviewers, external Institutional Review Boards (IRBs), or Ethics Review Boards (ERBs), collectively known as “reviewing bodies” – constitutes a significant part of all study construction and, therefore, is a significant part of all new science and discoveries. Shouldn’t the processes that enable study construction also be innovative and capable of adapting to new science, new methods, and new priorities?

A careful balance must always be maintained between ensuring protection of individuals participating in research and supporting innovative methods and processes to drive more efficient and cost-effective reviews and research.

The often broad, non-specific nature of regulations that govern “human research protections”, as they are called in the US, leave reviewing bodies somewhat adrift in what amounts to a vacuum. This vacuum forces them to make their own interpretation and implementation of the exact protections to uphold and leaves them without a mechanism to cross-communicate new processes for reviews or validations of new methods for research. At their core, reviewing bodies are simply a group of people working together to determine if a research study meets certain requirements and thresholds as interpreted from these regulations. The social psychology of how people interact on a larger social scale, how people make decisions in isolation and in aggregate, and how the context of their engagements changes their behavior in those different settings is sufficiently understood today. Yet, there is a large gap in transforming that understanding into the application of how reviewing bodies function. These factors have created a space that is prone to stagnation rather than innovation, languishing as a relatively static space for decades. The same study reviews are taking place, and the same study constructions are expected, with few changes permitted, let alone the ability to include entirely new methods. How do we inject innovation into such an environment to create what amounts to an adaptive or progressive IRB?

A careful balance must always be maintained between ensuring protection of individuals participating in research and supporting innovative methods and processes to drive more efficient and cost-effective reviews and research.

“At the Genetic Alliance IRB, we’ve found that an open dialogue between the IRB members, researchers, and technology providers is essential to understand pain points and inefficiencies in our process, and incorporate new techniques that improve both our processes and the research being done. We cannot limit ourselves to past methodologies, given the pace with which new technology is developing worldwide,” says Chris Carter, chair of the Genetic Alliance IRB.

IRBs and ERBs typically do not interact with service providers on the underlying technology by which data is collected for a study. Instead, their focus is on the burden of participation, the ethical considerations of what is being asked, and the privacy, burden, and safety risks to participants. However, we have seen a marked increase in the efficiency of designing and reviewing new studies when the IRB works in conjunction with the technical service provider to understand and approve the underlying mechanisms and processes first. By utilizing standard IRB practices to review and approve new processes and methodologies leveraging newly established technology supporting the research, we can increase the speed of research by reducing the complexity of new study design. Working with the Genetic Alliance IRB, we set out to find ways where an IRB and technology provider could work together to innovate reviews and research. Here’s what we did.

Case Study #1: Luna Platform – Simplifying the IRB Process for New Studies

“For several decades, standing in the shoes of research participants, I have been outraged at the delays and extra work some IRBs cause in the name of protecting the participants. From my perspective, many IRBs act to protect an institution rather than the people. In the case of advocacy organizations and communities working to accelerate research on their condition, making this as simple as possible and ensuring conduct of the best research for their communities is paramount,” said Sharon Terry, CEO of Genetic Alliance. “Working with LunaPBC, we did a first-of-its-kind submission to the Genetic Alliance IRB to approve an entire platform based on the methods it encompasses for participant engagement and retention, data collection, and analysis. This paves the way for a streamlined review process for any study being executed using the platform. There is no need for redundant approvals or long timelines.”

The Luna platform is a tool for researchers to establish new communities or cohorts to collect data and perform analyses for their studies in a continuously participant-connected environment. The platform has IRB approval, which includes a consent individuals e-sign to share de-identified data for research purposes on the platform. This allows CDI to be deployed on Luna by groups for the benefit of their community through a simple Organization-Specific CDI Addendum. Then study-specific consents are only needed for those studies that go beyond the standard methods described above for the platform and enables the IRB to focus on specific goals of and populations included in the research, since the underlying mechanics are the same from study to study. Examples of protocols that are not covered by the approval are 1) the collection of personally identifiable information instead of de-identified data, and 2) use of bespoke instruments on topics not prioritized by the community through Community Driven Innovation.

Case Study #2: Community Driven Innovation – Innovative Methods for Research
Luna established the Community Driven Innovation™ (CDI) method for uncovering and validating the top priorities of a community or cohort, similar to methods like the Delphi technique, but without the disadvantage of inherent groupthink or expert bias those other techniques often introduce. By working collaboratively with the Genetic Alliance IRB and several researchers using CDI for their research goals, we were able to identify new ways to not only improve the research being done, but also simplify certain aspects of the IRB process itself.

 “The Genetic Alliance IRB immediately understood the value of the CDI method, not only in reducing burden on study participants, but also in reducing costs and time for their own reviews for both new studies using CDI and new data collection, typically surveys, being designed based on the insights from CDI,” shared Ian Terry, senior user experience research at Luna.

Together, we established two concepts that were integrated into the Luna platform protocol with the Genetic Alliance IRB: (1) A CDI “meta-study” design, and (2) “Related Topics” surveys.

CDI Meta-Study Design

We established a protocol that defines the CDI method leveraging the mechanisms on the Luna platform for consent, recruitment, data collection, and data analysis. New deployment of CDI can be added via an addendum to this protocol defining the specific populations and key personnel involved in recruitment since nothing else changes from CDI to CDI. This streamlines the time for review and cost to review by the IRB, and enables researchers with less scientific experience to take advantage of deploying a CDI to their communities or cohorts.

Related Topic Surveys

Building off of the CDI method protocol, we worked with the Genetic Alliance IRB to design a process to eliminate the need for researchers to submit all research questions during the initial study design and instead enable evolving content based on the insights generated from the CDI itself. Together, we established thresholds for specific topics and priorities uncovered by the CDI that could be turned into questions in follow-on surveys without requiring additional IRB reviews. We’ve now opened up the research such that the involved patient population can select the research topics using several well-documented methods from Computational Social Choice theory. By doing this, we don’t have to pre-decide what a specific population may need; we can build the very task of asking that question into the study design. This allows us to remove many steps that would have been spent attempting to figure out what the population wants and thereby speed up the time it takes to establish this study in the first place. But also gives the research population – versus the experts or researcher – the autonomy to decide where the research should be headed, a power that has been missing in the medical world for a long time.

These are only a couple of examples of how collaborative exploration of new processes, methods, and technologies can create an innovative and efficient environment for safe, ethical research. By focusing on technology and method innovation in our external and internal ethical reviews, we can explore new frontiers in the research that empower participants to help drive study design. Subsequently, elevating the participant to drive the study design ensures that the new inventions and products developed meet their needs and pain points directly, thereby expediting answers, time- to-market, and ultimately better health. We’ve turned a top-down study process into a dynamic ecosystem of iterative listening, accessible to non-scientists, that supports the privacy and safety of its members.

Learn more about Community Driven Innovation at www.lunadna.com/cdi


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.


New Variants in Alzheimer's Treatment

New Genetic Variants May Unlock Future Therapies for Alzheimer’s

By Contributing Writer Reena Jordyn


About five million — or 1 in 10 — Americans aged 65 and older have symptomatic Alzheimer’s disease (AD).

While there are medications that help slow down the development of cognitive symptoms, there’s no available treatment to prevent the disease’s progression altogether. AD has been around since the early 1900s, but research on the subject remains scarce. Previously, the National Institutes of Health (NIH) allocated less than $500 million for research focused on AD, less than half of the budget meant for research on other serious conditions like AIDS ($3 billion) and cancer ($5 billion).

Perhaps a number of other factors come into play, too, such as the gender and racial disparities of AD. Of over five million AD incidences in the U.S., two-thirds occur in women. Researchers point to women’s longevity as a major reason behind these figures. Additionally, women’s brain anatomy, function, and development may lend themselves to AD – women’s brains accumulate greater tangle burden than men’s. Furthermore, African-American women, in particular, are twice more likely to acquire AD and other forms of dementia compared to their white peers. Similarly, Hispanic women are also more predisposed to AD, the risks being one and a half times greater than in white women. Differences in health, lifestyle, education, physical activity, and socioeconomic factors are thought to be contributory factors. These disparities and their ensuing bias could’ve been limitations for earlier research— luckily, we’re experiencing societal and scientific progress like never before.

Discovering the Klotho and RBFOX1 Variants
Much of what we know today points to genes playing a major part in the disease’s development. Over three decades ago, scientists have found the gene variant ApoE4 as a main contributor to Alzheimer’s. The prevalence of copies of this gene increases the risk for AD. A recent study by Stanford University School of Medicine investigators discovered a new gene variant that could help stave off AD: klotho. The researchers contrasted the likelihood of AD development in subjects with a single copy of the klotho variant against those without. The results revealed that those carrying one copy of the klotho variant had a 30% lower risk of developing AD. It was found that a single copy of klotho substantially slowed the progression of cognitive symptoms and impairment. And klotho was also seen to lower the beta-amyloid burden in ApoE4 carriers, helping mitigate the onset of dementia.

Another study by Timothy Hohman of Vanderbilt University Medical Center and Richard Mayeux of Columbia University Medical Center was able to link a new gene variant to AD. Hohman and Mayeux were able to unearth RBFOX1, a gene variant localized around plaques and in dystrophic neurites, which present heavily in people with AD. This revelation could lead to more advances in gene-specific therapy and precision medicine.

What’s Next for AD Treatment?
Increased awareness about the disease, coupled with stronger support, enables more scientists to conduct more in-depth studies on AD. However, the success of these investigations relies on strong and vast databases, and skilled genetic specialists.

The field needs more scientists, doctors, nurses, and other healthcare professionals to cast a wider scope. A top nursing career particularly relevant to this field is genetics nursing. Professionals in this field are in a unique position where they can assist patients suffering from genetic diseases as well as conduct genetic-related research simultaneously. This gives them an invaluable viewpoint of the disease, making them ideal researchers and great educators to patients and their carers too. That being said, AD patients, people predisposed to AD, and even the everyday person should be as proactive as those in the field. You can easily do your part by connecting your personal health records with the Luna platform. By doing so, you give researchers access to genetic and lifestyle data, among other pertinent information, which could thrust health discovery even further.

Every step and every grain of information gained is a huge step forward for Alzheimer’s research. In time, there may even be a medication that could present a cure for the dreaded disease.


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.


Dawn Barry, LunaDNA

Looking Ahead Into 2021 and Beyond — a Letter From the President, Dawn Barry


Welcome to the New Year, LunaDNA Community.

Your passion and commitment to creating health solutions these past few years has inspired all of us. You’ve continuously reinforced that the LunaDNA mission is not just ours alone, and that we are all in this together. As we approach 2021 with optimism and hope, we’re confident in our pursuit of redefining relationships between people, communities, and scientists towards better health for all.

One thing we learned from 2020 is that times of emergency have a clarifying effect. They tend to surface what’s truly important to us versus what we might have assumed was important. These times have also reminded us of the importance of establishing a deeper understanding of health and disease, and how vital these understandings are for all people. Through this lens, we proudly reflect on Luna’s top guiding principles.

Consumer Data Privacy Empowers Participation in Science

From meetings with lawmakers in Sacramento, California to quotes in media articles, Luna actively maintains the position that when people have privacy protections, they lean into opportunities to share their information for research. Further, we believe that we can achieve a sorely needed level of data inclusiveness through such privacy protections.

Throughout 2019 and into 2020, we saw steady advancements in consumer privacy laws, most notably Europe’s General Data Protection Regulation (GDPR) and California’s Consumer Privacy Act (CCPA). The momentum toward rigorous data privacy regulations were no doubt necessary at a time where there nearly weekly reports on mismanagement, misuse, and consumer data breaches by big tech companies, banks, law enforcement, and healthcare systems.

The core tenant of these consumer privacy laws is quite simple: honor the individual and their intents — versus the institution that stores their data — as the one who should decide what entities holds their data and for what purposes. These laws also empower people to remove their data at will – ultimately enabling people to have control over their most personal, private information.

This evolution awoke a cultural consideration: should data privacy be a fundamental human right, and, if so, what events could supersede such a human right? The COVID emergency instigated the societal conundrum of trading consumer privacy rights in exchange for cooperative data uses that benefit the whole. Only time will tell how this exchange will impact us as individuals, families, and an international community.

Looking Ahead into 2021 and Beyond

Meanwhile, amid the chaos of December 2020, big tech companies made significant updates to their privacy policies — Apple going in a positive direction, Instagram going in a negative one. While minimally highlighted in the media, privacy policy updates to consumer data usage have many people concerned about using certain apps. Some updates, which you tacitly allow just by continuing to use the product, allow third parties to view and analyze the information captured through your phone camera; copy your address book, call log and SMS history, and reach through to other devices you own to capture your online activity.

At Luna, we know people hold the power to transform discovery by simply sharing what they already know about their experiences in health and illness. When we come together as a community of all backgrounds, ages, environments, and share this information for study in a structured way, we can surface more robust answers faster. Even better, we can advocate for what’s essential to our health, beyond just pills and doctor visits. Look no further than COVID-19 data insights: we can clearly identify that different communities have very different challenges, and science is still working to clearly understand why, while also wrestling with the community’s deeply rooted issues of trust, privacy concerns, and possible discrimination.

Luna is a new model for social and cultural engagement that empowers health for all. Just as people came together to change how we rent properties and request rides, so too can we change how we surface health answers. Together, we built Luna from a foundation of putting people’s needs for data control, privacy protections, and transparency first.

Now it’s time. While we’re all experiencing health in a whole new way — to show the world that our data is ours and it’s valuable — we can put it to good work by coming together for science.

In 2021, we invite you to continue to help us realize our collective vision by joining our ever-expanding studies and inviting others to join, too. Only together can we overcome barriers life throws our way.

Luna is bringing together individuals, communities, and researchers to better understand life. The more we come together to contribute health data for the greater good, the quicker and more efficient research will scale, and improve the quality of life for us all.  

Directly drive health discovery by joining the Tell Us About You 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.

 


Dawn Barry

Dawn Barry

PRESIDENT + CO-FOUNDER

Dawn is the president and co-founder of Luna. She is the former VP of Applied Genomics at Illumina and, for 12 years, built pioneering teams from health screening to nutritional security.


COVID-19 Study Early Insights

COVID-19 Study’s Early Insights Shed Light on New Trends 


Since the launch of our COVID-19 Study in March 2020, LunaDNA members have submitted over 340,000 responses to gather physical and mental wellness information directly from individuals during this global pandemic.

Early insights we previously released in July revealed that one in six participants had taken a COVID-19 test and nearly one-third had tested positive. Today, we can identify a new shift in trends that better represent the current state of the world.

Across the globe, members have expressed how they are impacted by COVID-19, their current health conditions, and the behaviors they have since implemented into their daily lives. Based on our recent collection of survey data, nearly 1 in 20 respondents have tested positive for COVID-19, where 25–44-year olds were more likely to test positive than any other age demographic.

Other preliminary insights from the survey data reveal that of those who have tested positive, 41.5% have an existing health condition.

The following infographic shares key findings on this unique demographic’s behaviors, including how 100% of these respondents are practicing social distancing and only 13% have sought care from a hospital of health care facility.

COVID-19 Study Excerpt
Click Image to View More Insights

Different than many institutional studies, the ongoing LunaDNA COVID-19 Study offers people and communities easy-to-use tools to capture their lived experience during this unique time, recognizing that each person, community, and geography is impacted differently and will likely experience different long-term effects.

Sharon Terry, COVID-19 Study partner and CEO and President of Genetic Alliance, presented these early insights to the National Academy of  Medicine’s (NAM) Standing Committee on Emerging Infectious Diseases and 21st Century Health Threats.

“This study is conducted by the people, for the people, and in collaboration with various communities. This inclusive approach has garnered broad appreciation both for its ability to incorporate real-world experiences but also because it’s empowering people to have a voice in what will not be a one-size-fits-all solution to COVID-19. We are responding to NAM’s recommendation to expand to at-risk individuals such as those in nursing homes and detention centers. When unique voices and experiences are represented, researchers can deliver more precise answers.”

COVID and Communities  

LunaPBC, the public benefit corporation behind the LunaDNA platform, is collaborating with various community groups to support privacy-protected COVID-19 study including Genetic Alliance and Disease InfoSearch, xCures, San Diego Blood Bank, and the Propionic Acidemia Foundation. Each community’s study has a unique focus, ranging from how the virus affects cancer, genetic disorders and co-morbidities, to general data-sharing for research studies and clinical trials.

We encourage community leaders to create their own COVID-19 study program and leverage the LunaDNA infrastructure to better understand the impact of the coronavirus in their communities. Please contact us at collaboration@lunadna.com to explore your needs and goals.

Answers Wanted  

The COVID-19 Study is an IRB-approved study. If you are a researcher interested in qualifying and/or quantifying the short- and long-term physical and mental impact of the coronavirus pandemic and/or COVID-19 disease, we want to meet you. The LunaDNA platform exists to bridge individuals, communities, and researchers for privacy-protected, socially responsible discovery that improves health and quality of life in local communities and beyond. The need to invite individuals into research from the safety of their home, and to incorporate their lived experience into discovery, has never been clearer. Please contact us at discovery@lunadna.com.

Your Voice Matters  

We invite all people over the age of 18 to join the COVID-19 Study by taking the physical and mental wellness questionnaires on LunaDNA. Together, we can surface insights to improve our current state and better prepare for future pandemics. The LunaDNA platform preserves your personal privacy, is simple to join, and is free of charge.

This is an ongoing study and is open to everyone from all over the world. We encourage you to share your experiences during this unique time to build a representative body of knowledge and help scientists better understand this global pandemic.

Take the New Vaccine Survey in the COVID-19 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.