By any measure, this is an exciting time in biomedical science, and we are entering a golden age of discovery. Among the major advances in recent years, the sequencing of the human genome has opened up possibilities that are breathtaking, and we are learning to target specific biomarkers in patients to protect them against specific kinds of disease. Science today was science fiction in what seems like just yesterday.
The hard fact, however, is that we could be moving faster. For all of our successes, the pace of biomedical advancement is too slow for too many patients. In many cases, it takes decades—30 years or more—to move a new idea from the lab bench to the bedside. What should be an efficient system of drug development is fragmented.
New therapies and even cures could be accelerated if all of the stakeholders—academic researchers, pharmaceutical companies, government regulators, and patient advocacy groups—established better policies and procedures for working cooperatively.
The problem is not innovation; we have never had so many talented scientists doing so many amazing things. The problem is the many hurdles that innovation must cross to reach the marketplace and help the patients who need treatment.
At Faster Cures, the Milken Institute project I direct, we see three major areas where improvement is needed—and where progress can be achieved. The first need is to listen to what patients can teach the scientific and medical community. We call this elevating the patient’s voice. It used to be thought that if you asked patients what they wanted and needed in treatment, it would slow things down. We think it will move things faster.
We need to create a more permanent place at the decision-making table for disease foundations and patient advocacy groups—such as the Juvenile Diabetes Research Foundation, Parent Project Muscular Dystrophy, or the Michael J. Fox Foundation for Parkinson’s Research—that may know better than laboratory scientists what disease sufferers are dealing with and what will help them. The CEOs of such nonprofit organizations should join the national dialogue for speeding up treatments and improving health.
Some groups, notably the Cystic Fibrosis Foundation, have successfully helped produce treatments by funding research that no one else in the drug development pipeline would tackle. Even more could be achieved with cooperation across the system. We have seen the real results of this type of cooperation in the development of COVID-19 vaccines.
Patients, for instance, want to take part in trial studies for potential new drug therapies. But they often can’t find them. Pharmaceutical companies, in turn, need patients to participate in trials. But they often can’t find them easily. Closer relationships and community partnerships would help connect them.
A second area that needs to be addressed is creation of greater incentives for the pharmaceutical industry to invest in promising drugs that may not have potential for big profits—that won’t ever become blockbuster drugs, in the parlance of Wall Street. Some of these may be treatments for rare diseases with relatively small patient populations. A cancer therapy that could benefit millions of people will always get funded; a treatment for a disease with a few thousand patients might not.
In some cases, potentially helpful drugs with low profit potential end up sitting on the shelf and are never pushed to market. The insider jargon for these innovations is deprioritized assets. But what if the government could provide incentives for companies to move these ideas forward? What if a government lab or a government-funded research partnership took them on? How much funding should Congress give to the National Institutes of Health to incentivize research collaborations? What does the Food and Drug Administration need to support such efforts?
The Milken Institute has begun to innovate in this area with the Bridge Initiative, which seeks to bring together companies that have deprioritized assets with new partners in order to move shelved ideas to the marketplace.
And then there is the issue of information sharing. How can we incentivize sharing among lab scientists, drug companies, and others who may be working separately but simultaneously on the same problem? Can we start by creating data-sharing platforms where stakeholders may learn about and build on the efforts of others?
All of this is being thought out and worked on, but the effort needs to quicken.
The third area that has the potential to speed up treatments and cures is better use of technology—not only what we may develop in the future, but what we already have.
We have seen in recent years what the digitization of medical records can do. A top specialist at a university hospital in Philadelphia, let’s say, can now easily look at the MRI ordered by a physician at a community hospital in small-town Pennsylvania and quickly collaborate on a patient’s care.
The coronavirus crisis, while restricting in-person patient visits, has shown us the benefits of video appointments and telemedicine. Following the example of the Centers for Medicare & Medicaid Services, which changed its policies to better cover telemedicine, private insurers are now fairly reimbursing doctors for this kind of patient care.
Electronic health data is also helping rural and small-town patients participate in those clinical trials heretofore available only to patients near university hospitals. Not only can this help patients, but it can expand researchers’ knowledge by giving them a greater diversity of people in many situations to look at. These efforts unfortunately are too limited and not scaled across the country.
Remote participation technology, of course, is not the only area where we can capitalize on tech advances. We can point to innovative cell and gene therapies that use viral vectors to enable scientists to edit a gene and then put it back into a patient to correct for illness. Children suffering from spinal muscular atrophy who lose motor function over time, for example, now have the potential for a cure through new gene therapy that targets the underlying genetic root cause. That’s incredible, and there is so much more that we can speed to development for diseases once thought incurable.
The response to the coronavirus pandemic may become a model for what could occur in the future in many areas of biomedical science, because so much of what is happening addresses many of the issues I’ve been describing. For example, Operation Warp Speed, the federal government’s step-up-the-pace plan to produce hundreds of millions of doses of COVID-19 vaccine by early 2021, provides incentives and mandates for collaboration among stakeholders. Researchers have been sharing information among themselves. And scientists and companies are sharing their data in real time with FDA, so they can align on the design for clinical trials.
Speed, of course, needs to be accompanied by caution and ethical considerations. The overwhelming pressure for the biomedical science community to produce vaccines in the shortest possible time has made the effort seem like a race, prompting fears that shortcuts are being taken. Polls suggest that one-quarter of Americans may be leery of taking a vaccine, at least in the early stages of distribution. Trust is particularly low for many in the African American population because of many decades of neglect in health care.
The big question is, how do we ensure trust in a vaccine? How do we engender confidence that FDA is making decisions not for reasons of politics or haste, but for safety and efficacy?
Someday—we think, we hope—the coronavirus crisis will be behind us. Then the challenge will be applying the lessons we have learned about collaboration and breaking down barriers to other urgent needs in biomedicine. From Congress to regulatory agencies, to academic researchers, and among private industry, we will need to capitalize on this opportunity to change the structures that put up barriers to biomedical advances.
For despite our incredible discoveries, there is still so much to be done. The hunt for a new generation of antibiotics to fight infections is a prime example of where new thinking is required.
Because of the overuse of prescription antibiotics, dangerous microbes are becoming more and more resistant to the antibiotics on which we have long relied. We are depending on drugs developed decades ago, with few new alternatives being brought to market. Yet we also are seeing projections that superbugs, immune to antibiotics, could evolve and kill millions worldwide if we don’t create new defenses. Sadly, 700,000 people a year globally are already dying as a result.
We need greater incentives to promote the development and manufacturing of a whole new class of antibiotics. There are any number of small biotech companies that have invested in new antibiotics, and there are large companies that would be ready to do so if they saw good profit potential.
Ironically, one major hurdle is that our old reliable antibiotics are so cheap. Anyone with an ear infection or bronchitis knows these drugs are among the most affordable medications, often selling for cents on the dollar compared with what they used to cost.
When there is a new class of antibiotics, the cost of treatment could be exorbitant by comparison—in the many thousands of dollars, at first. Insurers won’t want doctors or hospitals to prescribe them except as a last resort. That may be understandable from a financial standpoint, but it’s also an example of a broken market.
Antibiotic resistance, it should also be noted, is a key area where we need to listen to the experiences of patients. Some patients already are dealing with drug resistance. Cystic fibrosis sufferers, for example, regularly get infections, and they especially would benefit from new, more effective medications.
None of what I’m describing will be easy to accomplish. But few important advances are easy—they require work and creativity and cooperation. We are witnessing those requirements now as we navigate our way through the pandemic.
The experience garnered from this difficult time as well as the obvious benefits that would result from the changes we should seek make me an optimist. I do think we are going to fundamentally change the way we approach medical research in the future.
But I am also a realist. What happens when the urgency of the COVID-19 emergency is over? That is when we will need to make permanent some of these temporary collaborations.
Will we go back to the old way of doing things when COVID-19 is gone? Or will we learn from it? Now is the time to turn the lessons of today into the practices of tomorrow. We must do this for the long-term benefit of humankind.
Esther Krofah is executive director of FasterCures, a center of the Milken Institute.