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CURE ID, A Tool for Clinicians to Report New Uses of Existing Drugs with Heather Stone and Dr. Marco Schito

Q&A with FDA Podcast | Transcript

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Dr. Sara Roach: Welcome to “Q&A with FDA,” from the FDA’s Division of Drug Information where we aim to answer some of the most frequently asked questions that we’ve received from the public. I’m Dr. Sara Roach, and today’s topic is CURE ID, an internet-based repository that allows the global community to report novel uses of existing drugs for difficult-to-treat diseases through a website, a smartphone, or other mobile device. I’m here with Ms. Heather Stone, FDA’s health science policy analyst in the FDA’s Office of Medical Policy, and Dr. Marco Schito, Executive Director of the Critical Path Institute’s CURE Drug Repurposing Collaboratory, or CDRC. Heather and Marco, thank you both for joining us today!

Heather Stone: My pleasure!

Dr. Marco Schito: Likewise!

Dr. Sara Roach: So, tell me, how did your interest in repurposing drugs arise?

Heather Stone: Well, I joined FDA in 2013 after completing a master’s degree in public health, and that included fieldwork at a remote hospital that was nestled in the foothills of the Himalayas in India. While I was there, I met a man who had extensively drug-resistant tuberculosis, for whom all treatments had failed and there were no known effective treatments remaining. After searching through the academic literature for hours, a physician and I found a drug combination that had saved the life of a young girl in a similar situation. Those drugs had been approved decades earlier, although not for tuberculosis. However, at the time there was only one case report that had been published.

Since then, I’ve spoken to dozens of other patients and clinicians in similar situations whose lives had been saved by repurposed drugs. In my ten years of pioneering the CURE ID app, these patients have kept me motivated. I’d like to hope that if more case reports could be gathered in one place and systematized, then the work to bring them into clinical trials would have occurred earlier and patients like the one in India could have been saved.

Dr. Marco Schito: As for me, while working at the Division of AIDS at the National Institutes of Health, I became interested in tuberculosis as well since a large number of HIV-infected patients succumbed to TB disease in low- and middle-income countries. This led to me to joining the Critical Path Institute in 2015 as the Scientific Director for the tuberculosis public-private partnership, where I led an effort to compile global data sources to better understand the bacterial genetics of mutations associated with drug resistance.

During this time, I became involved with the pediatric inflammatory bowel disease foundation to stand up a registry to capture outcomes of children treated with new drug biologics. When the prospect of generating this type of real-world data across many different diseases was proposed, I was very interested in exploring how we can synergize with FDA and collect the information that is being generated pretty much every day to potentially inform prescribers on best treatment practices in the absence of approved therapies.

Dr. Sara Roach: Such interesting backgrounds! I’m hearing that you both had a strong interest in repurposing drugs – how did that interest materialize and lead to the development of CURE ID?

Heather Stone: CURE ID began as the brainchild of Dr. Leonard Sacks, an infectious disease clinician with whom I work at FDA who actually had a very similar experience to Marco and I, seeing the devastating toll of extensively drug-resistant TB on patients in South Africa and recognizing that clinicians were trying all sorts of existing drugs in new ways, but this information was never being captured. As a result of his idea, I began developing the website and mobile application as a pilot with a focus on a couple of infectious diseases and testing the platform’s ability to be utilized both in the US and in developing countries. We wanted to be sure we could capture data from clinicians and patients anywhere in the world, especially as we sought effective treatments for these highly neglected infectious diseases.

CURE ID was formally launched in December of 2019, just in time to help with the COVID-19 pandemic as it turned out, and immediately pivoted to focus on furthering drug development for COVID-19 treatments.

Dr. Marco Schito: As you can imagine, this project garnered a lot of interest. It began, really as a collaboration between FDA and the National Center for Advancing Translational Sciences (or NCATS), which is part of the NIH. Besides the Critical Path Institute, FDA and NCATS are also collaborating with the World Health Organization, the Infectious Diseases Society of America, and a multitude of other partners to assess the global utility of the CURE ID platform.

I became involved in early 2020. C-Path convened a public-private partnership in June of that year to support and expand the CURE ID platform. Since then, the CURE Drug Repurposing Collaboratory, also known as CDRC, has been able to engage with more than 80 additional organizations and stakeholders including patients, clinicians, researchers, policymakers, technology groups, non-profits, as well as for-profits and other regulatory agencies.

Dr. Sara Roach: Repurposed drugs, which refer to FDA approved drugs being used for new, off-label clinical uses, can potentially offer another drug-development pathway for treatments of diseases and conditions that have few or no therapeutic options, correct?

Dr. Marco Schito: Yes, repurposing drugs entails using drugs that have been shown to be safe and effective in humans for one disease and identifying their potential uses for other diseases or conditions. In recent years, drug repurposing has been gathering traction in the medical community because of its potential to benefit patient care. For diseases where there are no approved therapies, clinicians frequently must turn to repurposed drugs that try to treat their patients. It’s important that we capture and aggregate this experience.

Heather Stone: While we think the data collected from real-world treatment experiences can be really valuable, it’s also important to keep in mind that CURE ID is not meant to be a substitute for an adequate and well-controlled trial. Drugs may be good in single use but might not work in a study in more patients. However, the hope is that the real-world data collected in CURE ID will enable us to identify potential “efficacy signals.” In very rare diseases where randomized controlled trials are infeasible, strong signals that treatments are effective even from anecdotal cases may provide substantial evidence of efficacy. And in more common conditions when randomized controlled trials are possible, real-world signals of efficacy may help with the selection of drugs and patients for further study.

Dr. Sara Roach: The CURE ID app currently focuses on drugs for infectious diseases lacking adequate approved therapies, including neglected tropical diseases, emerging infectious threats, and infections caused by antimicrobial-resistant organisms. Why is CURE ID focused on infectious diseases?

Heather Stone: Great question. A significant percentage of the world’s population suffers from an infectious disease with no approved therapy. And many others suffer from infectious diseases which have one or more approved therapies, but from which they are unable to benefit. This creates a huge unmet need for treatments. Our pharmaceutical system has not always spurred development of treatments for neglected diseases, and neglected diseases lack a commercial incentive for drug development. As a result, clinical trials often do not receive funding and patients do not have effective treatment options.

For certain infectious diseases that are found in lower resource countries, the market incentives simply may not favor investment. It may also prove very difficult to identify effective therapies for some infectious diseases. Clinical experience on how existing drugs are being used can be collected, so that promising candidates, drug combinations, and treatment regimens can be quickly identified, and clinical trials can be conducted to investigate these new uses, where possible.

Dr. Sara Roach: What kinds of new information can be gleaned from looking at existing drugs?

Dr. Marco Schito: CURE ID captures treatment outcomes when drugs are used to treat new diseases or are used in other new ways. Clinicians may discover new ways of treating diseases, new combinations of drugs that are useful, new dosing regimens and durations of therapy, new populations that can benefit from existing treatments, or they may actually discover that unapproved uses do not work or are actually harming patients.

Some advantages of repurposing existing drugs include the time and cost to develop a new indication for an existing drug may be significantly less compared to developing a brand-new drug from scratch. That’s because most of the non-clinical drug development has already been done including the chemistry, the manufacturing and controls, the animal toxicology, and the clinical pharmacology. And also that there is clinical data on safety already in a population that may be relevant to the novel use.

Heather Stone: Through CURE ID’s ability to obtain and organize this information received directly from healthcare providers, patients, caregivers, published case reports, and now electronic health records, promising new therapeutic uses can be identified for formal study by institutions involved in drug development, and unhelpful or harmful uses can potentially be avoided.

CURE ID allows healthcare providers to share their real-world experiences treating patients with repurposed drugs through a simple online case report form on their smartphone, computer, or mobile device, so it provides an innovative mechanism for capturing these experiences.

Dr. Marco Schito: It really captures the clinical outcomes when drugs are used for new indications, in new populations, in new doses, or in new combinations, and captures cases both of treatment successes and treatment failures – where the repurposed drug was not able to help the patient, which is equally important to capture and understand.

Dr. Sara Roach: In other words, CURE ID essentially enables the crowdsourcing of medical information to facilitate the development of new treatments for neglected diseases.

Heather Stone: Yes, that’s right. To submit a report, a user must complete a brief registration process, and then they can enter their case report on the web or on their phone on the mobile app. Clinicians, patients and care partners must register and sign in in order to submit their case report.

Dr. Marco Schito: The app includes a case report form to capture treatment experiences, as well as access to the collection of cases that have already been reported (including successful as well as unsuccessful treatments) that users can then browse, in addition to viewing clinical trials reported in clinicaltrials.gov.

Clinicians can participate in a Treatment Discussion Forum where they can engage with fellow healthcare providers globally and can also stay up to date on the latest infectious disease news through journal articles, news articles, and events that are updated daily. CURE ID then aims to be a network connecting major treatment centers, academics, private practitioners, government facilities and other healthcare providers worldwide into a global clinical care community.

Heather Stone: Through the website and mobile app, CURE ID can help clinicians, regulators, and drug developers to share cases, where they can report their own cases as well as read cases from other clinicians and patients around the world of neglected infectious diseases with no sufficient approved therapies; communicate by engaging directly with communities of disease experts around the world; and access information – where users can view information on approved therapies for each disease as well as active clinical trials.

Dr. Sara Roach: It seems like it’s really simple to submit case reports, and also adverse event reports via CURE ID.

Heather Stone: Yes, we have tried to make it as easy as possible to submit these reports. Clinicians, patients, and their care partners can enter the basic details of the case, using either the website or the mobile application. There is then a section that is triggered if the user selects that the patient experienced an adverse event while taking one of the drugs that is reported. The additional section allows the user to enter the most pertinent details about the adverse event that was experienced and what happened as a result of the AE.

This information is captured in an identical fashion to Medwatch. CURE ID enables adverse events to be automatically shared with the FDA’s Medwatch Adverse Event Reporting system. Because this only adds a couple of questions to an already very brief case report form, it’s a way for users to report adverse events in much less time. The whole case report can typically be entered in about 10 minutes.

Dr. Sara Roach: It has been estimated that it takes an average of 17 years for a medical discovery to be translated and adopted into widespread clinical practice. Do you think CURE ID will be able to shorten that timeline?

Dr. Marco Schito: Well that’s the goal actually. We hope to shorten that timeline by collecting structured case reports and using those cases to inform the clinical community and the design of robust randomized trials. CURE ID may help reduce that time to bring those findings into a clinical trial. It can also provide highly useful clinical data to inform the trial’s hypothesis and, and even generate strategic designs.

The goal here is with the collection of these case reports, to generate signals about potential uses of existing drugs for new indications, or new combinations, and new treatment regimens that can then be studied in a randomized clinical trial fashion and potentially lead to a clear demonstration of whether or not this drug, or combination of drugs, are effective to treat the neglected disease.

Heather Stone: Historically, medical information and treatment advances have been communicated through peer-reviewed scientific publications and presentations. However, often times individual cases never make it into the published literature. CURE ID provides an easy way for healthcare providers to share information on individual cases where they used an existing drug for a difficult-to-treat infectious disease. It also then enables the aggregation of all of these cases in a systematic way, to expand their value.

Dr. Marco Schito: Completely agree. The more people who actually use CURE ID, the greater its value becomes, and that’s because there will be more case reports of novel uses of existing drugs and more fodder for discussion, which is what we’re really wanting to do. It’s all really very exciting!

Dr. Sara Roach: Is CURE ID only for infectious diseases, or are there plans to expand it to include other diseases?

Heather Stone: FDA planned on expanding the app by adding other illnesses, but as a new disease that caused a pandemic, COVID-19 expedited that process. While we didn’t create CURE ID for COVID-19, we designed the tool to respond to an emerging infectious threat, and it’s been an important tool to help us fight this public health emergency. FDA and NIH joined forces with CDC and IDSA – the Infectious Diseases Society of America (which is the professional society representing the majority of infectious disease specialists in the US) to encourage healthcare providers to share their experiences treating COVID-19 patients with existing drugs that are being used in new ways.

We also received a lot of support from the Department of Health and Human Service’s Patient-Centered Outcomes Research Trust Fund, to expand our ability to collect cases from electronic health records. While the data infrastructure being built was specific for COVID-19, it was designed in a sustainable manner so that it can be promptly deployed for future uses, including outbreaks of existing and emerging infectious diseases, and is now being trialed for sepsis and meningitis. The goal is to provide real-time access to the global clinical experience of repurposing drugs when there is an immediate need to identify potential existing treatments in the absence of novel drug development.

Dr. Marco Schito: We also have exciting plans to expand the CURE ID tool beyond infectious diseases. We are currently building out a complementary CURE ID platform for oncology, specifically focused on rare cancers, thanks to the generous support from FDA’s Oncology Center of Excellence.

We have also partnered and explored the use of the tool for special populations for whom there is often limited treatment data, such as pregnant people and neonates. The CURE Pregnancy Treatment Repository, which is part of the existing CURE ID platform was supported by the Office of Women’s Health at FDA.

The latest area of development is using the tool for chronic conditions that are a sequelae of infections, like, something like long COVID. This is also enabling us to build out the patient-reporting aspect of the platform, which is something that’s very important and began with the outbreak of MPOX that occurred last year. We have released a case report form for patients with long COVID, that will enable them to upload their own information on what treatments they are receiving and what may or may not be working for them. We really hope to hear from lots of patients about their treatment experiences.

Dr. Sara Roach: Thank you both for speaking with us today! Do you have any closing remarks or future plans you want to share with our listeners?

Dr. Marco Schito: I’ll go first. The CURE ID app is one example of FDA’s work to support the use of real-world data to create real-world evidence that can then inform clinical trials and potentially regulatory decisions, and the importance of this technology and work cannot be overstated. The case report form can now capture the most crucial information on the patient’s treatments and outcomes from electronic health records and registries, yielding informative, succinct, and easily interpretable data, as well as traditional reports directly from the clinicians and now from patients and their care partners, as well. These improvements will allow the community to have open access to anonymized case reports, including treatment outcomes, when repurposed drugs have been used for a wide range of diseases lacking adequate treatment options.

Heather Stone: We are hoping that this infrastructure may continue to be expanded to be used for the other diseases in the CURE ID app and now are beginning to pilot the EHR extraction tool’s use in sepsis and meningitis. We are also really excited about the patient case report form for long COVID. This will help provide insights into treatments that may impact patient’s quality of life — a perspective that may not be captured in the clinician case report form and will be used to inform future randomized controlled trials of long COVID treatments. We can’t wait to see the valuable information patients are able to provide, in addition to their clinicians and care partners!

Dr. Sara Roach: This is all very exciting! CURE ID can be found online at https://cure.ncats.io or it can be downloaded from the App or Play store as “CURE ID.”

Thanks for tuning in to “Q&A with FDA”. The full podcast and transcript of this recording is available at fda.gov/QAwithFDA. And if you have drug-related questions, reach out to us at druginfo@fda.hhs.gov.

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