Knowledge Center

At the beginning of April 2023, the FDA published a draft of the fourth in a series of Guidance Documents entitled “Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments (COA) Into Endpoints For Regulatory Decision-Making”.  Once finalized these four documents will be combined and will replace the 2009 Guidance, “Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims”.  Part 3, which was released in draft in June 2022, provides advice around the development and validation of new COA (including the modification of existing COA).

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Since 2018, Medable has grown a network of patients, caregivers, and care partners whose job is to help improve the clinical trial experience for participants.

Known as the Patient Caregiver Network (PCN), they regularly update their best practices, including recently refreshing their technological best practices around deploying eConsent.

We’re sharing them below to help continually promote the participant's voice and to help foster more equitable, inclusive, and diverse clinical trials

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Medable CEO Michelle Longmire and Dr. David Fajgenbaum, co-founder of EveryCure, join Pharmaceutical Executive podcast to talk about their partnership adn navigating the regulatory landscape.  

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A top 10 pharmaceutical company looked to Medable to streamline participant burden and enhance site and team experience in oncology trials, while establishing a scalable template for future trials in oncology and other areas.

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For the second consecutive year, Medable has been named Leader decentralized clinical trial products per the Everest Group’s PEAK Matrix^® assessment, which evaluates DCT products from 24 companies based on vision, capability and market impact.

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Medable worked with a top 5 pharma company to increase the safety of patients as anti-cancer treatments may cause pulmonary toxicity, ranging from asymptomatic radiological changes to respiratory failure, and is considered a common side effect.

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Deployment of remote and virtual clinical trial methods and technologies, referred to collectively as decentralized clinical trials (DCTs), represents a profound shift in clinical trial practice. To our knowledge, a comprehensive assessment of the financial net benefits of DCTs has not been conducted

Research looking at mobile apps and how they may provide a meaningful access point for all stakeholders for symptom management.

In a decentralized clinical trial, part or all of the protocol occurs away from the primary study site. Instead of patients traveling, often repeatedly, to a central site for enrollment, consent, data collection or symptom monitoring, they can participate in telehealth visits from their homes, often using familiar technologies, like smartphones, tablets and wearables to transmit pertinent information. Even medications and devices can increasingly be delivered directly to a patient’s home, and a home visit from a health care professional can be arranged if necessary. 

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Patient recruitment is one of the most critical elements of a clinical trial. Without sufficient participation, studies will lack the power to demonstrate the statistical significance and efficacy of new treatments. Ultimately, poor patient enrollment will delay or prevent new therapies from reaching the market. Not only do such delays cost millions of dollars, but they also deprive patients of effective, potentially life-changing treatments. 

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Patient screening, a vital step in the clinical trial recruitment process, is when interested participants are assessed for their eligibility for a given trial using select inclusion/exclusion criteria. These criteria help characterize the target patient profile to ensure patient safety, better efficacy, and optimized signal detection. 

Traditionally, patient screening was done in person at a clinical trial site. Due to the current digital revolution in healthcare, which has recently accelerated in response to global health events, the ability to use cloud-based software and digital qualification steps to optimize patient screening and enrollment brings innovation, convenience, and sophistication to this process. 

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