Monkeypox vaccine: a case study for real-world data

WWith the increase in health emergencies, the world needs more efficient ways to test the safety and effectiveness of drugs, even if they have not yet completed the lengthy clinical trials that the Food and Drug Administration normally requires. Data from the real application of new drugs offer an increasingly valuable and efficient way to do this.

This allowed authorities to determine that the first large-scale vaccines against Covid-19 in 2020 were safe and effective. Because the virus was spreading so quickly, it was only a few months before the disparate results between studies began to show in participants who received an actual vaccine versus those who received a placebo. Later, in 2021, when data showed that a significant number of vaccinated people became infected, health experts quickly introduced guidelines for additional booster shots.

And now, in a welcome development that could impact future drug approval methods — and even lead to more affordable drug prices — real world data is playing an essential role in evaluating the effectiveness of monkeypox vaccines.


Following last summer’s severe monkeypox vaccine shortage, the FDA approved an alternative rescue dose regimen for the Jynneos monkeypox vaccine. This allows health authorities to administer the shot intradermally, with each dose being only one-fifth the size of the originally approved dose. The idea is to make sure as many people as possible get vaccinated.

While such an approach, in which the vaccine is only injected into the outer layers of the skin and not into muscle or other deeper tissue, has not been attempted on a large scale with this or similar vaccines, there is research that suggests it may work – and for other types of vaccines – since the skin contains many cells that defend the immune system.

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Because vaccine doses are limited and there has never been a historical opportunity to test dose changes or even the clinical efficacy of the vaccine against actual human exposure to monkeypox, the only way to demonstrate efficacy now, at least in the short term, is with real-world data to collect and analyze. In fact, the effectiveness of the original dose against an actual monkeypox infection has only been tested in animals, while its safety and levels of antibodies produced in the bloodstream are tested in humans, meaning little scientists know about how well it works. how long it takes, whether it stops broadcasting, and other factors.

While the spread of monkeypox has slowed, the United States still has about 200 new cases per day and has been the site of a third of all cases worldwide. With that in mind, researchers at the Centers for Disease Control and Prevention and elsewhere are taking the opportunity to track data to see how well the Jynneos vaccine works at its lower dose given actual exposure to the disease. The National Institutes of Health, in collaboration with the FDA, will also collect and study data on the intradermal injections to determine their effectiveness. Given the real risk of infection if the virus continues to spread, being able to see whether or not those who get vaccinated also get infected shows how well the vaccine is working (or not working) in its new dosage form.

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Real-world data (and real-world evidence) can also play a role outside of public health emergencies like Covid and monkeypox. They can help determine the long-term effectiveness of many treatments, particularly those that are subject to accelerated approvals, such as B. those used for rare diseases and can help determine the value of medicines in general. In many cases, clinical trials are not enough to understand how well drugs really work. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said the clinical trials system is “broken” and that greater use of real-world evidence could be an effective adjunct to the approval process.

The FDA has taken steps to use more real-world data outside of public health emergencies like Covid and monkeypox. In 2018, the Agency issued guidance on the use of such evidence when authorizing medicines. By 2021, when the FDA issued expanded guidance on the subject, real-world evidence was used for the approval of 90 medical devices and the new use of one drug, Prograf. But this happens too little in practice.

Not using real-world data not only misses a better understanding of the effectiveness of individual drugs, but also an opportunity to improve the entire pharmaceutical sector, including addressing issues such as rising costs. The availability of more data on real-world outcomes from the use of drugs, particularly gene therapies and other innovative and often very expensive treatments, would pave the way for pricing that takes patient outcomes into account through approaches such as value-based contracts when health insurers base drug prices on them how well medicines work in the people who take them, and not just in pre-market clinical trials.

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Former FDA Commissioner Scott Gottlieb said the ideal approach would be a hybrid system that uses both clinical trials and real-world evidence, with the real-world evidence serving as a way to further validate clinical trials, which are often limited to a small one are number of subjects and include only those participants who are likely to respond favorably to therapy.

Drugs must always go through supervised laboratory tests and clinical trials. But real data can and should play a bigger role. While changing the dose of the monkeypox vaccine due to a shortage isn’t ideal, officials taking this opportunity to collect and study data on its effectiveness amid an ongoing outbreak can show the important role that such data plays can play in evaluating drug efficacy. Properly used data and real-world evidence can help to better determine the efficacy and value of a drug or vaccine, thereby ensuring a more efficient approval process. This in turn could lead to a better and fairer healthcare system.

Girisha Fernando is the Founder and CEO of Lyfegen, a Switzerland-based company focused on software analytics for healthcare value-based contracts.


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