Periodic Reporting for period 1 - REMEDI4ALL (BUILDING A SUSTAINABLE EUROPEAN INNOVATION PLATFORM TO ENHANCE THE REPURPOSING OF MEDICINES FOR ALL)
Periodo di rendicontazione: 2022-09-01 al 2023-08-31
The vast majority of the over 7000 known diseases are without effective treatments—there is thus an urgent need to make better use of the medicines that we already have in hand. These include medicines that have already been approved for human use, as well as experimental medicines still in clinical trials already showing good pharmaceutical properties and human safety. In fact, most approved drugs intrinsically have the potential to treat many more diseases than they were originally approved for, even diseases seemingly unrelated to those for which they are currently being prescribed.
The mission of REMEDi4ALL is thus to make it easier and more reliable to find these new medical uses for drugs we already know are safe and effective. We also aim to show that in many cases such “repurposed” medicines can be taken all the way into clinic faster and at the fraction of the cost of developing a completely new drug from scratch.
But with hundreds or even thousands of opportunities to repurpose approved and experimental medicines—for the thousands of diseases that remain without any approved treatments at all—REMEDi4ALL must also help to transform the very landscape of drug repurposing. This needs to encompass not only the scientific, clinical research and patient communities, but regulatory, policy and commercial drug and investment sectors as well, so that all researchers will face fewer barriers and be able to bring much needed treatments to patients faster and more effectively—to “float all boats” in the drug repurposing ecosystem.
The mission of REMEDi4ALL is thus to make it easier and more reliable to find these new medical uses for drugs we already know are safe and effective. We also aim to show that in many cases such “repurposed” medicines can be taken all the way into clinic faster and at the fraction of the cost of developing a completely new drug from scratch.
But with hundreds or even thousands of opportunities to repurpose approved and experimental medicines—for the thousands of diseases that remain without any approved treatments at all—REMEDi4ALL must also help to transform the very landscape of drug repurposing. This needs to encompass not only the scientific, clinical research and patient communities, but regulatory, policy and commercial drug and investment sectors as well, so that all researchers will face fewer barriers and be able to bring much needed treatments to patients faster and more effectively—to “float all boats” in the drug repurposing ecosystem.
Launched in September 2022, REMEDi4ALL is already off to a strong start in building a robust, multinational platform for drug repurposing, spanning 24 sites in 11 different countries across Europe and UK. At our core we have two founding principles:
1. Researchers must work arm-in-arm with patients from the very inception of a drug repurposing concept and closely together through all stages of drug development: all the way through approval by regulatory authorities and into clinical practice to ensure that the drugs are accessible to the patients who need them, and are reimbursed.
2. You can’t skip any steps in drug repurposing—even though a drug may already be approved, you still have to make sure that all the requirements for showing the safety and effectiveness for a new drug are also met for a repurposed drug. This is especially the case if the original drug must be modified (such as converting from a topical cream to an oral tablet) or if the profile of the patients will be different (such as children if the drug had previously been approved only for adults). Taking shortcuts or not working closely with patients in the development process imperils a project with failure, and with the loss of an opportunity to use repurposing to address a critical unmet medical need.
We have applied our core principles to the four drug repurposing projects that launched together with the REMEDi4ALL platform. One of these projects, in pancreatic cancer, is already actively enrolling patients, while the clinical trial for another, in “brittle bone” disease, is set to launch in the next months. A third project, in an ultrarare disease that affects multiple organ systems in the body and disrupts the normal development of infants and children, has been given a huge boost by the no-strings-attached donation of much needed data and information from a major pharmaceutical company. Finally, our project in pandemic preparedness is screening hundreds of approved drugs for potential to be used in first-line response for the next viral threat, even though we won’t know the exact identity of virus until the next outbreak hits.
1. Researchers must work arm-in-arm with patients from the very inception of a drug repurposing concept and closely together through all stages of drug development: all the way through approval by regulatory authorities and into clinical practice to ensure that the drugs are accessible to the patients who need them, and are reimbursed.
2. You can’t skip any steps in drug repurposing—even though a drug may already be approved, you still have to make sure that all the requirements for showing the safety and effectiveness for a new drug are also met for a repurposed drug. This is especially the case if the original drug must be modified (such as converting from a topical cream to an oral tablet) or if the profile of the patients will be different (such as children if the drug had previously been approved only for adults). Taking shortcuts or not working closely with patients in the development process imperils a project with failure, and with the loss of an opportunity to use repurposing to address a critical unmet medical need.
We have applied our core principles to the four drug repurposing projects that launched together with the REMEDi4ALL platform. One of these projects, in pancreatic cancer, is already actively enrolling patients, while the clinical trial for another, in “brittle bone” disease, is set to launch in the next months. A third project, in an ultrarare disease that affects multiple organ systems in the body and disrupts the normal development of infants and children, has been given a huge boost by the no-strings-attached donation of much needed data and information from a major pharmaceutical company. Finally, our project in pandemic preparedness is screening hundreds of approved drugs for potential to be used in first-line response for the next viral threat, even though we won’t know the exact identity of virus until the next outbreak hits.
The broader and long-lasting impact of REMEDi4ALL will depend on how effective we will be in transforming the landscape of drug repurposing, including in wide dissemination of our advances and learnings, and in training the next generations of researchers and patient advocates. Our ambitious goal is to be able to make it faster, easier and cheaper to repurpose drugs for all projects that follow, even those not working directly with the REMEDi4ALL consortium.
We aim to demonstrate that common approaches can be used for drug repurposing projects, thereby showing that there is no need to reinvent the wheel for every new drug repurposing project. As such, a critical part of our success is onboarding a robust and patient-centric pipeline of new drug repurposing projects, where every project is designed to show that a common problem in drug repurposing can be surmounted, and that the lessons learned can be directly applied to the next drug repurposing project that may face a similar challenge.
Through this approach our goal is to systematically tackle all the barriers that have burdened drug repurposing projects in the past, and to open the path to realizing the full potential of drug repurposing in addressing the hundreds and thousands of diseases that remain without approved treatments.
We aim to demonstrate that common approaches can be used for drug repurposing projects, thereby showing that there is no need to reinvent the wheel for every new drug repurposing project. As such, a critical part of our success is onboarding a robust and patient-centric pipeline of new drug repurposing projects, where every project is designed to show that a common problem in drug repurposing can be surmounted, and that the lessons learned can be directly applied to the next drug repurposing project that may face a similar challenge.
Through this approach our goal is to systematically tackle all the barriers that have burdened drug repurposing projects in the past, and to open the path to realizing the full potential of drug repurposing in addressing the hundreds and thousands of diseases that remain without approved treatments.