Sana.IMPACT: IMProving Access to Clinical Trials

Accelerate medical knowledge discovery by bringing mobile-enabled adaptive clinical trials to health care providers around the world.

Kenneth E. Paik, MD MBA MMSc

Director, MIT Critical Data, MIT Sana
Research Scientist, Institute for Medical Engineering and Science
Massachusetts Institute of Technology, Cambridge, MA

Clinical trials are essential for advancing medical knowledge, however low- and middle-income countries (LMICs) are broadly underrepresented, comprising less than 5% of trial sites globally, despite their disproportionately high burden of disease. This exacerbates inequalities as treatments that have been validated for wealthy populations are consequently not generalizable to more diverse or resource-constrained environments. Several barriers prevent the conduct of clinical trials in LMICs and contribute to this disparity, including a lack of research infrastructure and trained staff, challenges in accessing the wider scientific community, difficult patient recruitment, and complex ethical and regulatory processes. Furthermore, clinicians in resource-limited settings are often time-poor and required to constantly balance competing demands, whereby immediate patient care may understandably be prioritized ahead of clinical research. To support the health of LMICs, clinical trials conducted in these contexts are needed, investigating research questions posed by local clinicians, and comprising treatments that can be feasibly administered in such settings. As emerging treatments arise, these need to be readily integrated into research and clinical practice.

There are several beneficiaries of the system, but the primary target audience for our platform are health care providers in resource-limited settings and their patients. Our platform supports them by enabling them to:

1. Improve the management of their patient rosters

2. Enroll their eligible patients into clinical trials

3. Connect to the wider academic community, while collaboratively addressing their local research priorities

Secondary beneficiaries are humanitarian NGOs or local ministries of health, by allowing them to better understand the populations they serve and monitor the performance of their operations. We will offer Sana.IMPACT to healthcare organizations in resource-limited settings as a platform for collecting operational and patient data, as is often mandated by their funding agreements. Sana has collaborated with such partners in resource-limited settings, including the International Rescue Committee and International Organisation for Migration, to better understand their needs and refine the platform. 

Thirdly are researchers, government, and corporations. Accessing the pool of untapped research participants will greatly expand the opportunity for clinical trials and facilitate trial recruitment. In the context of a global health emergency, our platform may allow more interventions to be tested, with a larger and more diverse investigatory population, and therefore potentially greater inferential power.

We anticipate that our solution will provide numerous public goods. The clinical registry division will generate novel data which will be accessible for the purpose of global health research and surveillance. Ongoing monitoring of registry data will allow the early identification of emerging global health crises, facilitating targeted public health responses and resource allocation optimisation. Our platform may support NGOs in resource-limited settings by assisting them to collect their required operational data, reducing the local resources required for data management.

Our platform will provide infrastructure allowing clinicians from resource-limited settings to conduct their own investigator-initiated trials and to enroll their patients in international clinical trials with novel study designs. Such studies may improve the likelihood that patients from resource-limited settings receive the optimal treatment while clinical trials continue. Trials conducted using the platform will contribute to the medical literature, with results uniquely applicable to patients from resource-limited settings. Finally, collecting patient-centered outcomes will generate knowledge of the outcomes that are most important to patients in resource-limited settings, which are largely unknown.

Ultimately, these benefits will offset some of the inequities in global health research, by facilitating clinical trials in LMICs and improving the care provided to vulnerable populations.

Barriers to the conduct of clinical trials in resource-limited settings include lack of infrastructure, challenges in accessing the scientific community, patient recruitment, complex ethical and regulatory processes, and time demands on clinicians. Our solution seeks to reduce these barriers. It will provide an infrastructure for the conduct of clinical trials, allow clinicians to collaborate with the international research community, provide an easy method with which to enroll patients into trials from the bedside, seek to minimise the ethical and regulatory processes that individual investigators must navigate by having pre-approved generic trial designs, and via all of these improvements, reduce the time required for clinicians to partake in clinical trials. 

Beyond assisting clinicians from resource-limited settings, the ultimate goal of our solution is to help patients in these locations. It has been demonstrated that patients who participate in clinical trials have improved outcomes, and that locally conducted research is more likely to be of benefit to patients. Outcomes which will reflect this include the number of trials conducted and the number of patients enrolled in those trials. Furthermore, novel study designs are more efficient than traditional research, allowing optimal treatments to be identified sooner and patient care improved. 

In the first year, we will expand and scale the patient management tool and clinical registry function of the platform. We will have discussions with existing and previous NGO partners (IRC, IOM, Partners in Health, Syrian American Medical Society) as well as seek out additional partners who may benefit from the platform. While individual health care providers will be able to join the platform, the scale and adoption of the solution will be greatly accelerated by partnering with NGOs. We will further seek out clinical trial partners from research, governmental, and industry backgrounds and develop the adaptive clinical trial functions based on their input and feedback. We would seek to engage with academic partners to design generic clinical trial designs and begin the process of gaining approvals for these 'master protocols'. 

Over the subsequent years, with a rapidly growing user and patient base, we would be able to initiate targeted adaptive clinical trials, supported by key industry partners. The network effects of a large and diverse user base would allow more numerous and varied clinical investigations, potentially generating revenue and accelerating knowledge discovery.

Example indicators which we will use to measure our progress include the number of:

• Countries in which our platform is utilised

• Health care providers utilising our app

• Patients included in our clinical registry

• Completeness of data collection

• Trials conducted utilising our platform, particularly investigator-initiated trials from researchers in resource-limited settings

• Patients randomised in our clinical trials

• Research publications utilising data from our platform

Examples of these metrics from previous pilot studies include:

• Adherence to treatment guidelines

• Improved data quality

We anticipate that navigating legal and regulatory requirements of each country we deploy in will be challenging. Our model of collaborating with local academic institutions, which have pre-existing international collaborations or strong ties to local government, has proven effective. We emphasize co-creation whenever possible in our projects, leveraging local knowledge and expertise to deploy a localized solution.

We have recruited clinical trialists from within academia, but intend to also seek advice from industry experts to help us navigate operational and regulatory barriers. For long-term sustainability, it will be critical to prove value to industry (e.g. pharmaceuticals), in order to fund the platform and support clinical trials. Clinical trials will require ethical approval.

Usability will be crucial for the success of Sana.IMPACT, as health care providers are already overburdened. We will focus on the user experience (UX) efficiency for the providers, without compromising data integrity, security and impact. We seek to improve uptake of the platform by providing the clinical registry division to NGOs, as a method for the collection of patient and operational data. This will help overcome an initial barrier of increasing user engagement, and allow us to rapidly grow a network of health care providers using the platform.

Massachusetts Institute of Technology

Harvard Medical School

Beth Israel Deaconess Medical Center

Emory University 

University of Melbourne

University of Toronto

Australian and New Zealand Intensive Care Research Centre (ANZIC-RC), Monash University

Australian and New Zealand Intensive Care Society, Centre for Outcome and Resource Evaluation (ANZICS-CORE)

Initially, the financial prize would allow the formal formation of the Sana.IMPACT team and allow the platform to expand as envisioned. However, long-term, relationships with Trinity Challenge Members would aid development of the solution in countless ways: 

• Validated exposure, allowing us to more easily recruit users and fundraise

• Guidance in navigating the best way in which to form and operationalize the organization, including growth and recruitment

• Navigating and easing regional regulatory concerns.

• Technical expertise in scaling solutions for production and stability.

• UI/UX expertise for usability and user adoption.

• Relationships with industry members for stakeholder insights or as users of the platform. For example, clinical trial expertise from pharmaceutical companies.

• Marketing and scale-up strategies.

We consider Sana.IMPACT to be a transformative solution and believe The Trinity Challenge can help us make it a reality.

• The Bill & Melinda Gates Foundation/Clinton Health Access Initiative, who may facilitate uptake of our platform via their Global Health Division partners, and assist in receiving feedback from health care providers in resource-limited settings.

• University partners (e.g. The University or Cambridge, Imperial College London, London School of Economics and Political Science, Johns Hopkins Bloomberg School of Public Health), who may utilise our platform for their clinical trials, assist in the design and ethical review of our proposed generic trial designs, and provide guidance to investigators from resource-limited settings seeking to conduct a trial using Sana.IMPACT

• Industry partners (e.g. GSK), who may also utilise our platform for industry-funded trials

• Technical partners (e.g. Google, Microsoft) who may assist in the development of our app and provide strategies for the protection of patient data

• Operational experts (e.g. McKinsey) who can provide advice on organizational structure and strategy