A lasting memorial may be emerging for the millions of people who will have tragically died of Covid-19 by the time the pandemic ends: the demonstration that breakthroughs can happen fast when drug companies and regulators listen to and communicate openly with patients.
The concept of patient engagement across the health care ecosystem emerged more than a decade ago. Its core idea — incorporating patients’ actual experiences, perspectives, needs, and priorities into treatment efforts and drug-development decisions rather than taking them for granted — started a fundamental change of thinking in the drug development world.
On the regulatory side, the U.S. Food and Drug Administration (FDA) in 2012 began holding what it calls Patient-Focused Drug Development meetings to hear from people living with diseases. These meetings with patients and their advocates generated considerable amounts of raw information. That and other trends brought about changes in attitudes on the part of some health care providers, regulators, and drug developers.
But such information usually appeared late in the typically long process of drug development and approval, by which time most decisions about the characteristics and potential benefits of a new product already had been made. Some companies sought out patient insights, but too often embraced only the information that confirmed their experts’ own biases. Drug developers rarely changed their processes and structures to grapple with these insights at every step from drug discovery through clinical research to commercialization. As a result, with no ill intent, drug companies missed a lot that patients have been trying to tell them for a long time.
Then came 2020, when the pandemic focused the attention of industry and regulators on patients like never before.
Here are some key patient insights that I believe finally got a serious hearing in the last year, and what I hope will be enduring lessons learned from them:
Patients can and should be able to make informed decisions on new technologies. The messenger RNA (mRNA) technology used in the Pfizer/BioNTech and Moderna vaccines is not new. The first successful demonstration that mRNA could deliver genetic information to produce proteins in animal cells occurred three decades ago. So the delivery and acceptance of mRNA-based vaccines in nine months was as much of a patient-listening and public-education breakthrough as it was a scientific one.
Covid-19 vaccine trials gave the public a say on mRNA, and thousands of people signed on to participate in them. The result should encourage a new model of scientific transparency, dialogue, and patient engagement on the benefits and risks of new treatment technologies. For example, where earlier forms of patient engagement have been almost entirely focused on providing informed consent for a planned clinical trial, a new model should take into consideration patients’ risk tolerance and openness to new treatments long before a technology reaches human testing.
Risk tolerance for medical interventions is not constant, and it is greater when health threats loom large. Drug developers and regulators have heard inklings of this for years from the parents of children with rare diseases and from people facing untreatable cancers, but then generally defaulted to their established drug development and approval processes. One notable exception has been organizations developing rare disease therapies, though I suspect there would be an appetite for further risk tolerance within these organizations as well.
That message, however, turned unmistakable in the case of Covid-19. The remaining concerns of some about the Covid-19 vaccines should not obscure the fact that most Americans eagerly anticipated and embraced them. The Kaiser Family Foundation’s opinion-tracking efforts showed last September that 63% of adult Americans “definitely or probably” would get a vaccine, which rose to 71% in December. And 72.1% of adults age 18 and older have done just that, receiving at least one dose of the currently available vaccines.
Speed up the development processes for new therapies. The process of bringing new therapies to market seems endless to patients and their families. Anyone who has spent time with people dealing with untreatable or poorly treatable diseases has heard enormous frustration over what they perceive as the sluggish pace of R&D. In the case of Covid-19, the biopharmaceutical industry listened. There was no hesitation about sharing scientific insights and collaboratively recruiting participants for trials — and establishing outright partnerships between companies — and the effect was astonishing.
Covid-19 forced a reckoning with these three insights, likely because everyone became a patient or potential patient during the pandemic. Until then, drug development and approval systems had kept disease sufferers feeling somewhat marginalized when it came to actual decision-making. But this time, developers and regulators had to reckon with everyone as we faced a disease together.
The results of what might be called this “great listening” — vaccines developed and approved in nine months rather than nine years, a large swath of the population willingly immunized, and a global pandemic diminished if not yet defeated — are a powerful proof of concept for patient-focused development and delivery of new therapies.
Now it’s time to embrace a wider transformation. Here’s what to expect if that happens:
Drug companies will rebalance risk and speed in getting to treatments that work, will educate and listen to patients at eye level about new technologies, and perhaps will share what they learn and look for opportunities to collaborate more effectively with each other. Companies will listen for patient insights and incorporate them earlier in the R&D process.
This will make it possible to fail faster, and thereby succeed faster, by focusing squarely on what makes new treatments better for patients while more efficiently eliminating R&D programs that don’t offer meaningful benefits. Less money will be spent to bring new treatments to market by making participation in clinical trials a faster and more patient-friendly process. And because companies will focus from the earliest development stages on what patients expect and will accept, drug launches will be more about realizing potential than overcoming unforeseen and unexpected obstacles.
Nine months from the emergence of a novel, pandemic-causing coronavirus to the authorization of effective Covid-19 vaccines against it looked like a miracle. By creating better partnerships with patients and advocates, drug companies and regulators can make that example the new normal. In doing so, the biopharma industry will further honor the pandemic’s victims and heroes.
Anthony Yanni is senior vice president and global head of patient centricity at Astellas Pharma.