Navigating Uncharted Waters

Peters,Rita;

Navigating Uncharted Waters

July 1, 2020

By Rita C. Peters

Publication

Article

BioPharm InternationalBioPharm International-07-01-2020

Volume 33

Issue 7

Pages: 5

Data and science must guide FDA in making pressure-filled COVID-19 vaccine and therapy approval decisions.

For researchers involved in the development of new treatments and vaccines to combat COVID-19, the past five months have been a scramble to research potential therapies, collect and analyze data, accelerate clinical trials, and plan manufacturing processes. While the biopharma industry knows these development efforts are moving at record pace, for some patient groups, policymakers, and the public, the efforts are not moving fast enough.

When someone is seriously ill, family members and healthcare providers are desperate for an effective treatment. History has shown that many “promising” therapies initially raised the hopes of patients, only to disappoint when the therapy did not work as predicted. During this global pandemic, public and policymaker pressure can make the R&D task more difficult.

For example, earlier this year, hydroxychloroquine was touted as a possible treatment for COVID-19; FDA faced criticism when it issued emergency use authorization (EUA)-based on limited data-on March 28, 2020. When subsequent clinical trials showed the drug was unlikely to be effective as a treatment for COVID-19, and the cardiac side effects outweighed the benefits, FDA withdrew the EUA on June 14, 2020.

Keeping pace with the rapidly emerging research about the SARS-CoV2 virus, its medical impact on humans, and the successes and failures of therapies and vaccines to combat COVID-19 is a monumental task. Thousands of scientific papers have been released, and drug companies and research organizations have been collaborating and sharing information on levels often described as unprecedented. These findings need to be assessed and verified to ensure decisions about therapies are based on solid data rather than political motives, or undue influence.

Missteps and misinformation related to diagnostic testing, distribution of personal protective equipment from the strategic national stockpile, and the wearing of face masks have eroded public trust in the abilities of FDA and other government authorities to manage this pandemic. While anecdotal evidence points to the potential availability of a vaccine before the end of 2020, public confidence in FDA, the government, and drug companies needs to be established.

Following the “North Star”

During the BIO 2020 digital event on June 10, 2020 (1), FDA Commissioner Stephen M. Hahn emphasized the importance of science-based decision making, as well as regulatory authority independence to make approval decisions, key factors to assuring the public that the vaccines and therapies are approved with public safety and health at the forefront.

As applications for emergency use and investigational new drug applications poured into FDA in the initial days of the pandemic, Hahn says the agency reviewed all applications, and prioritized those with the highest level of science behind them and the greatest chance of making it to clinical trials.

Some bio/pharma companies are participating in and receiving funding from the federal government’s Operation Warp Speed and Accelerating COVID-19 Therapeutic Interventions and Vaccines programs. Hahn noted, however, that FDA has established a “barrier” between itself and these public-private partnerships to reinforce the agency’s regulatory independence. “We will prioritize based on science, we will not prioritize based on who comes through the door,” he said. “We are agnostic with respect to the sponsor, but we are very much aligned with where the science appears to us to be the highest level.”

Hahn said he understands the “enormous amount of pressure” related to the pandemic; however, the Agency’s “North Star” will be data and science. The agency may have to update decisions as new data are available, he noted, but will continue to make decisions based on science.