Improving The FDA’s Regulatory Process Can Enhance Treatment Options

Peter Marks, director of Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research noted that “vaccination remains critical to public health and continued protection against serious consequences of Covid-19, including hospitalization and death.” But what is the protection for those Americans who can’t take the vaccine?

Approximately 3% of U.S. adults, or around 8 million people, are immunocompromised. Some are compromised because of medical conditions like metabolic diseases including diabetes, chronic kidney disease, or HIV infections. Others, such as recipients of organ transplants or people fighting cancer, are at risk of being severely immunocompromised due to the lifesaving medicines they must take.

People who are immunocompromised are disproportionately impacted by diseases such as Covid-19. They are at higher risk of experiencing more serious illnesses and at higher risk of dying. Unfortunately, they also receive less protections from standard vaccines assuming they can even take them.

Given these realities, the regulatory environment should facilitate a wider array of treatment options. Unfortunately, in the fight against the mutating Covid-19 virus, we continue to lose important options.

In the early stages of the pandemic, monoclonal antibodies (mAbs) provided benefits to vulnerable populations and six different mAbs were authorized to treat Covid-19. These treatments work by mimicking the body’s natural antibodies, which is why they are so valuable to the immunocompromised. No immune response is required. However, the virus’ mutations have undermined mAbs effectiveness, much like what happens with traditional vaccines, and the FDA has pulled the authorization for all current treatments.

Degrading protection from the current treatments is a common problem. For instance, we need annual Covid-19 boosters because the virus constantly mutates. Beyond Covid-19, an annual flu vaccine is required to ensure individuals have the right antibodies because the predominant flu strain varies from year to year.

The relevant question, consequently, is not whether mAbs are effective or ineffective. Their past success demonstrates that they can be effective for periods of time, like the annual flu vaccine, but only if cutting-edge mAbs stay one step ahead of the Covid-19 virus’ mutations.

Undoubtedly developing efficacious mAb treatments tailored to the current Covid-19 strains is a huge scientific challenge. But it should be these scientific constraints, not regulatory inefficiencies and uncertainty, that dictate whether patients have access to mAb treatments. To minimize the regulatory burdens, the FDA should adopt a regulatory structure that accounts for Covid’s dynamic disease environment and minimizes the government-created burdens that can inhibit the development of potentially efficacious Covid-19 treatment options.

The framework for such a regulatory structure – that would allow for the “update” of mAbs – already exists. Consider how the FDA approaches the development of the updated Covid-19 vaccines:

COVID-19 vaccine development may be accelerated based on knowledge gained from similar products manufactured with the same well-characterized platform technology, to the extent legally and scientifically permissible. Similarly, with appropriate justification, some aspects of manufacture and control may be based on the vaccine platform, and in some instances, reduce the need for product specific data. (emphasis added)

Simply put, the FDA does not require vaccine developers to conduct vaccine trials that simply duplicate the information already gained from past trials. There simply isn’t time if vaccines are to keep pace with the virus. The regulatory structure captures the knowledge learned creating past vaccines to lower the costs and time required to develop treatments that are tailored to the current variants.

This structure creates greater regulatory certainty, lowers the cost of updating the treatment to be effective against current virus strains, and speeds up the development time so that vaccines get to patients in time to be effective. The development of mAbs does not benefit from these more efficient regulations.

Applying this approach to mAbs would reduce the amount of time that is required to follow the current regulatory pathway. The excessive time it currently takes to develop a mAb for the current strain raises the risk that a different mutation will be the dominant one by the time an innovative mAb can finally become available to patients.

If a treatment is not efficacious against the strain that is prevalent when it is approved, then its value to patients is lost. And this is especially important for those immunocompromised patients who do not have any protection against Covid-19. The large risks that this will occur under the current regulatory structure make it exceedingly difficult, if not impossible, to develop innovative mAbs, and could disincentivize innovators and manufacturers from even attempting to enter the market. For this reason, the FDA’s current regulatory process is discouraging the development of potentially valuable treatment options.

Appropriately applying this platform approach that the FDA uses for vaccines to the mAb technology would remove a burden discouraging manufacturers from developing mAbs tailored to the latest variants. The result could improve health outcomes for vulnerable patient groups, and for all Americans, and help us stay one step ahead of the consistently mutating Covid-19 virus.