The pharma industry is in a mad dash to bring a Covid-19 vaccine to market, ideally before 2020 slips into 2021. This represents a dramatic acceleration from standard development timelines, which historically have often lasted 10 to 15 years. More than 100 Covid-19 vaccines are currently in development, with 21 already being tested in human clinical trials.
Regulatory agencies like the U.S. Food and Drug Administration are doing their part to accelerate the path to approval, allowing manufacturers to conduct animal studies in parallel with Phase 1 trials and outlining criteria for emergency authorization before full approval.
These efforts mean we could have a vaccine approved much sooner than was previously possible, which would be a powerful tool for resolving this health care crisis. Yet a race to the market means regulators will have a briefer period of experience with vaccine safety and effectiveness to evaluate when they are considering vaccines for approval.
Vaccine safety is a heated topic among the public. According to a Reuters/Ipsos poll, one-quarter of Americans have little or no interest in getting a coronavirus vaccine because of concerns that the speed of development could compromise safety. And unlike treatments for chronic diseases, vaccines are given to vast numbers of healthy individuals, so society’s tolerance of adverse reactions is very low. And concerns around Covid-19 vaccine safety could heighten underlying concerns with vaccines in general.
This perception is placing increased pressure on pharma companies to plan for fast and agile strategies for collecting safety data on these vaccines after approval. Post-market safety studies, which obtain information on a medicine’s safety after regulatory approval, can use direct-to-patient methods to meet this need. These methods capture patient-reported outcomes — information directly from vaccine recipients about their outcomes — as the main source of information regarding the occurrence of adverse events.
Vaccine safety surveillance: need for a flexible global approach
Post-market vaccine safety studies are designed to collect information on potential adverse events, including debilitating or life-threatening disorders like convulsions, anaphylaxis, encephalitis, and a rare condition called Guillain-Barré syndrome, as well as less-serious reactions such as fever, headache, and pain at the injection site. How often these occur among people who get vaccinated can then be compared to rates in other populations to determine if there is likely to be a causal relationship between vaccination and a given effect.
Under normal conditions, post-market vaccine studies can rely on data routinely captured during physician visits via electronic health records and claims data. However, participants in vaccine trials are generally healthy, which means they aren’t routinely returning to see their doctors in the days and weeks after vaccination, so less-serious adverse events may be missed. And Covid-19 has kept many people from making routine and even medically necessary health care visits, so the ability to reach patients directly to ask about their experiences provides additional reassurance of the completeness of safety data following vaccination.
As we saw during the H1N1 influenza pandemic of 2009, vaccine distribution in a pandemic setting may occur outside of traditional health care settings in which seasonal and childhood vaccinations are administered. Instead, temporary vaccination centers may be set up in schools, community centers, or even at public events. The result is rapid and effective distribution, but it makes data collection a challenge.
Given these considerations, direct-to-patient data collection methods offer a flexible solution well-suited for fast-track development of a vaccine during a global pandemic.
One example of a direct-to-community data collection approach is the Covid Active Research Experience (CARE) Project, a registry launched by IQVIA, the company we both work for. This opt-in registry collects self-reported data from people who have been exposed to the Covid-19 virus. Participants enroll on the website and provide insights into their symptoms, comorbidities, and self-medication outside of hospital care. This kind of registry could easily be extended or modified to collect long-term follow-up information on vaccines or treatments.
Leveraging technology that allows vaccine recipients to enroll in studies, provide consent electronically, and complete questionnaires via phone, mobile apps, or websites is a powerful complement to data collected from other sources like electronic medical records or claims data. Participants could enroll in studies at Covid-19 vaccination sites, allowing vaccine sponsors to collect short- and long-term follow-up information regarding safety and adverse events directly from vaccine recipients, and then as needed confirm that data with health records. During the H1N1 pandemic, post-market safety studies showed patient-reported safety information to be a valuable strategy for near real-time safety monitoring with high completion rates.
This direct-to-patient approach has also proved to be effective in other medical territories, such as understanding drug safety use in women during pregnancy. In this case, women self-reported drug use via the internet or telephone, and researchers compared this data to readily available pharmacy registry data and electronic health records. Researchers concluded this patient-reported data offered a more complete picture than relying on existing data from registries and electronic health records alone, a major step in better understanding the effects of prenatal exposure to new drugs.
The time to plan is now
The success of any direct-to-patient study depends on whether participants will sign up and follow through with providing information. This requires solutions that are scalable, user-friendly, and have a low burden of participation. If a study is too cumbersome, personally invasive, or time-consuming, participants will be less likely to stay engaged. In the H1N1 vaccine study we described earlier, the response rate after six months of follow-up was greater than 97%, which demonstrates both willingness from participants to provide information as well as low burden to participation.
To ensure success, vaccine developers should plan these studies now, so they are ready to launch as soon as a vaccine is approved.
Pharmaceutical companies traditionally have months to plan post-market surveillance studies. During that time, they identify study sites or data sources, choose the study design, establish protocols, develop consent forms, customize and test the electronic data-capture environment, and train staff. But in this accelerated mode, when hundreds of thousands of vaccines will likely be administered in the first weeks following approval, companies can’t afford any delays. They need to be ready to start enrolling participants the moment the vaccine authorization application is filed or risk missing the first wave of vaccine participants.
The compressed development time for Covid-19 vaccines is essential for containing this health care crisis, and monitoring individuals who have received the vaccine is a vital step in this process. Deploying innovative post-market surveillance studies will help ensure these accelerated solutions are safe and effective for the public to use and maintain public confidence in vaccination programs.
Priscilla Velentgas is a senior principal for real world evidence at IQVIA, where Karin de Haart is an associate director of safety evidence for regulators offerings.