by myTomorrows | 24 Mar 2020
The role of expanded access in the coronavirus outbreak
Society is disrupted by the outbreak and trajectory of the coronavirus. Our thoughts go out to those affected. The pandemic also had its impact on our business, and we are receiving many questions on access to investigational medicine. In this article, we want to shed some light on the role of Expanded Access in this pandemic.
As with many diseases, COVID-19 does not have any approved treatments. In the face of major public health emergencies, regulatory authorities recognize that the standard process of drug development and approval is inadequate to meet a very pressing need. In the absence of approved treatments, the development process needs to be expedited. Part of this could be done with Expanded Access, but the major contributions should come from faster research, international collaboration, and scaling production and distribution processes. These are the two main reasons why:
First, the natural course of COVID-19 is uncertain. Only a tiny portion of the entire patient population will end up on the IC-unit, and estimates vary on how many recover. This makes it difficult to assess what would have happened if patients had not been treated. This is different from high-mortality diseases such as pancreatic cancer or ALS. For the vast majority of patients, COVID-19 is not a deadly disease. To find out what medication works, we need to carefully assess treatment effects. The fastest way to generate critical data that inform the appropriate use of this investigational medicine is to conduct randomized controlled trials. The WHO has launched 4 worldwide megatrials to assess the effects of four most promising coronavirus treatments. Patients who cannot participate in such trials could pursue Expanded Access pathways.
Second, if we can find a drug that is effective and safe – ramping up production to allow global distribution will take a long time. Demand will soon pass supply. This is amongst the reasons why Gilead has halted the distribution of remdesivir for compassionate use for new patients: its supply is focused on treating patients in clinical trials. This is also why off-label prescription of drugs such as (hydro)chloroquine are receiving so much attention: if found effective, it could easily be produced and distributed at scale. Bayer and Novartis are donating millions of tablets of chloroquine to COVID-19 research. Such amounts are simply not available for medication in phase 2/3 trials.
On a global level, the scientific community should quickly and concisely report results of scientific studies on COVID-19. For governments and regulators, everything should be done to facilitate research, and accelerate production and distribution processes. Those form the best chances of finding a solution to this worldwide crisis.
At the personal level, every infection with corona is a crisis. The medical community is doing everything to find individual solutions for patients, caregivers and loved ones. Expanded Access could be part of that solution.
That being said, patients suffering gravely from COVID-19 should be fully informed by their doctors on the options of Expanded Access to investigational medicine, if applicable. It would be a mistake not to collect data from patients being treated with investigational medicine, and such data should be collected to complement the results from trials. Therefore, Gilead is currently looking into launching Expanded Access programs combined with data collection as an alternative to emergency compassionate use.
myTomorrows assists and informs patients and physicians on access to treatments in development. Next to that, we also collect data from those treatments. Our partner in Real-World data collection, CastorEDC, has even made their platform freely available for al COVID-19 research – aiming to accelerate research.
THE SITUATION AT MYTOMORROWS
The operations at myTomorrows are also affected by the coronavirus outbreak. The safety and wellbeing of our personnel receives our utmost attention. Almost all our employees are working from home. Next to that, the continuation of our services and the treatment of patients without disruption is our key priority. We have taken various measures and precautions since the outbreak of COVID-19 to ensure that we remain fully operational.
We encourage everyone to follow local health measures. We do so, too.
Stay safe and look after each other,
The myTomorrows Team