The Food and Drug Administration and the drug industry have jointly agreed to create several new pilot programs aimed at speeding drugs to market for rare or previously untreatable conditions. Announced on August 23, 2021, the agreement states the improvements the FDA will make in exchange for the millions of dollars in new drug application fees from drug makers. This agreement was negotiated throughout the fall and winter as a planned update to a prior, five-year-old agreement. The language of the present agreement stands out due to its focus on creating new programs meant to assist in drug approval for rare diseases. It is now up to Congress to ratify the goal letter before the FDA can implement the changes, as the existing agreement expires in September.
Outlined in the letter and dubbed the “Split Real Time Application Review” (STAR) Pilot Program, allows drug makers to submit data for rare disease drugs in two tranches. The goal of the program is to speed the FDA’s application review process by allowing reviewers to begin reviewing certain data before the entire application is finalized. In addition to this, the Rare Disease Endpoint Advancement Pilot Program was proposed to give drug makers a chance to work directly with the FDA in designing new clinical endpoints for rare disease drugs. The goal of that program is to improve clinical studies by discovering more measures that can be used in clinical trials to determine whether a drug actually provides meaningful benefit. The FDA plans to also develop another new pilot program focused on identifying the type of “real-world evidence'' that could be used to get their drugs approved, before the drug makers actually start their studies.
On August 23rd, 2021, the United States Food and Drug Administration approved the Pfizer-BioNTech COVID-19 Vaccine as the first COVID-19 vaccine. The vaccine will now be marketed as Comirnaty (koe-mir’-na-tee), a conglomeration of the words “Covid-19 immunity” and “mRNA,” to evoke a heightened sense of community. The mRNA vaccine has been approved for the prevention of COVID-19 disease in individuals 16 years of age and older, and under emergency use authorization, including individuals 12 through 15 years of age and a third dose for certain immunocompromised individuals. This is an expansion of prior EUA allowances, as it has been available under this provision to individuals 16 years of age and older since December 11, 2020; being extended to include those 12 through 15 years of age on May 10, 2021.
The Pfizer-BioNTech COVID-19 Vaccine underwent the FDA’s standard process for reviewing the quality, safety and effectiveness of medical products, its biologics license application (BLA) bearing extensive data and information that supported the EUA. This data includes preclinical and clinical data and information, details of the manufacturing process, vaccine testing results, and manufacturing site inspections. The FDA conducts these analyses independently to ensure the vaccine is safe and effective. To support the FDA’s approval decision, the Agency reviewed updated data from clinical trials which included a longer duration of follow-up in a larger trial population. The safety of Comirnaty was evaluated in approximately 22,000 people who received the vaccine and 22,000 people who received a placebo 16 years of age and older, having been concluded to be 91% effective in preventing COVID-19 disease.
The FDA and Centers for Disease Control and Prevention have implemented monitoring systems to ensure that any safety concerns continue to be identified in a timely manner. Additionally, the FDA is requiring the company to conduct studies to further assess the risks of myocarditis and pericarditis following vaccination with Comirnaty as well as long-term outcomes among individuals who develop myocarditis following vaccination, and safety studies, including a pregnancy registry to evaluate pregnancy and infant outcomes after receipt of Comirnaty during pregnancy.
Eli Lilly is teaming up with Lycia Therapeutics through a collaboration announced August 25th, 2021. The companies intend to use Lycia's LYTAC technology to identify and develop new drugs for up to five protein targets in Lilly's primary research areas, including immunology and pain. Lilly paid $35 million up front, and will potentially spend more than $1.6 billion later should Lycia hit set development and commercial milestones; the company may also receive tiered royalties on product sales stemming from the collaboration.
The LYTAC technology tries to encourage cells to eliminate harmful proteins by engineering antibodies that bind to the cellular organs responsible for disassembling proteins. This is a divergence from the behavior of typical drugs that act on a particular protein by blocking or inhibiting it, however there are many proteins for which that approach is not effective.
Protein degradation offers a potential loophole, and, thus, has attracted interest from drugmakers and investors alike; Nurix, C4 Therapeutics and Kymera Therapeutics — raised a combined $565 million through initial public offerings last year alone. Having invested in Kymera in 2017, Lilly is no stranger to protein degradation. Though it is receiving attention and investments, protein degradation is still a relatively new area of drug development, having yet to advance into human testing. Arvinas, is currently conducting two studies testing its experimental drugs in patients with breast or prostate cancer.
On August 23, 2021, President Joe Biden urged businesses and public leaders to implement vaccine mandates after the federal government issued its first full approval of a Covid-19 vaccine, following the Food and Drug Administration approval of Pfizer’s two-dose vaccine for people 16 and older. "If you're a business leader, a nonprofit leader, a state or local leader, who has been waiting for full FDA approval to require vaccinations, I call on you now to do that — require it. It only makes sense to require a vaccine to stop the spread of Covid-19,” Biden pressed from the White House amid stiff resistance to mask and vaccine mandates by Republican lawmakers who say they are unnecessary. In Columbus, Ohio Hundreds of supporters of a Republican-backed bill prohibiting employers from requiring workers to receive vaccinations showed up to the Ohio Statehouse on August 24th, 2021 as lawmakers heard a single additional day of testimony. Although speakers before the GOP-controlled House Health Committee were evenly divided between those for and against the measure, supportive demonstrators held the majority on the Statehouse grounds. Hundreds rallied ahead of time urging lawmakers to “Enact Vaccine Choice” and to say “No To Forced Vaccines!”, before entering into the Statehouse atrium to watch the hearing live, applauding speakers favoring the legislation and heckling those who didn’t.
Ohio State University announced shortly after the hearing that it would require all students, faculty and staff to complete the full vaccination process by Nov. 15, citing the FDA’s decision to grant full approval to the Pfizer vaccine. While the legislation has attracted opponents of COVID-19 vaccines, it does not mention the coronavirus; opting instead to call for requirements for all vaccines. The legislation would prevent employees from being fired as a result of refusing vaccination and allow them to sue their employers for wrongful termination.
The Health Committee has received more than 1,000 submissions about the bill, from supporters and opposition alike, however the fate of the bill is currently unclear. In July, however, Mike DeWine signed a bill into law prohibiting public schools and colleges from requiring individuals to receive vaccines that have not been granted full approval by the U.S. FDA. The anti-vaccination mandate bill before the House Health Committee also urges schools to provide parents information about exemptions they can seek against having their children vaccinated and would repeal a state law requiring college students to disclose whether they have been vaccinated against hepatitis B and meningococcal meningitis.