As scientists race to find a COVID-19 vaccine, policymakers and regulators face challenging questions including how to balance efficacy demands with a tight timeline, plans to pay for a potential vaccine and the best way to distribute it.

Public health experts have indicated that a COVID-19 vaccine, which will take at least a year to 18 months to develop, is important for a return to normalcy.

Last week, the National Institutes of Health announced an initiative to increase collaborative efforts to develop a vaccine and treatments for the COVID-19 pandemic. Several scientific and policy implications must be weighed as a vaccine is developed. One question is how high the standards for a vaccine’s efficacy should be. 

[NIH announces strategy for developing COVID-19 drugs, vaccines]

Supriya Munshaw, senior lecturer at the Johns Hopkins Carey Business School and an expert in commercializing new medical technologies, said it’s important to differentiate between vaccine efficacy, which is measured in clinical trials before the vaccine is approved, and vaccine effectiveness, measured after the vaccine is approved and is being administered in the general population.

“Efficacy and effectiveness can differ because clinical trials take place in controlled environments with a specific population while the general administration after approval includes a larger, more heterogeneous population,” she said. “Coronaviruses are less variable than influenza, so higher effectiveness would be expected; however, given the urgency of the situation, a less effective vaccine could be introduced at first.”

Peter Marks, director for the Center for Biologics Evaluation and Research at the Food and Drug Administration, said that so far, officials have not seen mutations with the coronavirus that would affect vaccine development.

“You’d like to have very high efficacy. If you don’t get there, we’ll probably go with the next best thing. Having some people protected is better than no people protected,” Marks said in a recent call with reporters. “It does mean that if you have a vaccine that has relatively less efficacy, you end up with a situation like we have with seasonal influenza, where you have only a portion of the population protected and you have to deal with the circulating virus.”

No magic number

Experts agreed that there is no magic number being used as a benchmark for vaccine efficacy, and it’s unknown how effective potential vaccines will eventually be.

“We want to make sure that we have a safe and effective vaccine. That effectiveness number, I think, is going to vary,” said L.J. Tan, chief strategy officer for the Immunization Action Coalition. 

Tan acknowledged that a vaccine may also not be equally effective across all adult populations. For example, a potential vaccine could be more effective for seniors compared with young, healthy adults, which would help with prioritization.

The early, ongoing conversations between developers and federal agencies will be important to ensuring that a vaccine is available as soon as possible.

“Vaccine development is not simple. Traditional vaccine development through all its phases can take 10, 15, sometimes 20 years and can cost at least $800 million if not even more,” said Tan. “You are going to require continuous and especially early collaboration with regulatory agencies to make sure that they are engaged in this whole flexibility process to get rapid, appropriate approval processes going now.”

Marks said the FDA has already been meeting with vaccine developers well before they submit their applications.

“Across manufacturers, we are engaging as early as they would like in these conversations,” he said.

He also said the agency will likely need to increase staff to deal with what he hopes will be a large number of biological licensed applications.

The FDA also may turn to emergency use authorizations, which would allow the agency to approve new drugs and vaccines during the public health emergency. Marks said it would depend on the strength of evidence to declare an EUA and if the safety and efficacy and situation on the ground justified one.

Payment and distribution decisions

Experts also emphasized the need to plan for billing and payment, among other considerations, to allow for a smooth rollout of a potential vaccine eventually.

“When these vaccines get approved, we are going to have to deal with who is going to be paying for them,” said Tan, adding that whether it’s done by the private sector or by the government, it will require different steps and billing codes.

Having those codes in place will allow a provider to put in a payment request so it can be easily processed, he said.

Progressive Democrats have pushed for including coverage for COVID-19 vaccines in legislative packages.

“The only way we’re going to be able to safely end social distancing and prevent economic disruptions permanently is going to be with the development and distribution of a vaccine to every American,” Rep. Peter A. DeFazio, D-Ore., said in a press call last week.

Esther Krofah, executive director of the advocacy group FasterCures, said its members are advocating for early and active conversations with federal officials about what resources will be needed for multiple FDA-approved vaccines. That includes discussions with the Centers for Medicare and Medicaid Services about payment.

“As we have seen with CMS and their respective agencies across federal government, they have been quite rapid in providing guidance documents and making sure that any challenges around telehealth or telemedicine … are addressed,” she said. “I’m hopeful that they would be very open to these kinds of conversations.”

Manufacturers will also need to figure out how to reinforce the supply chain. Even if there is progress on supply chain problems, there will need to be decisions on how to allocate resources and prevent confusion because not all consumers will be able to be vaccinated at the same time.

The Centers for Disease Control and Prevention has national guidelines for allocation of vaccinations during pandemics, especially influenza pandemics. Munshaw said these guidelines will likely be adapted for COVID-19. 

Krofah drew a comparison to testing issues for COVID-19. There was a disconnect, she said, between initial information claiming that testing would be available for anyone who requested it and scarcity in who could qualify for a test.

“When we see an FDA-approved vaccine, many people will think they can call their doctor and ask for it, which will likely not be the case,” said Krofah.

The FDA’s Marks acknowledged that not everyone will be able to get the vaccine simultaneously because of supply issues.

“We’re talking about a few million people in this country alone and a few billion people globally,” he said, adding there are competing pressures in developing and approving a vaccine. “One really would like to make it right to the extent we can while obviously working at a very rapid pace.”