Summary

With 1,255 VA medical facilities serving over 9 million veterans each year, the VA — through its Veterans Health Administration — maintains the largest integrated healthcare system in the United States. The VA is a national leader in delivering quality health services and driving innovation in high-priority healthcare issues such as telehealth, precision medicine, suicide prevention, and opioid safety. Yet the VA remains an under-appreciated and underutilized health policy stakeholder, involved in minimal interactions with other federal health agencies and exerting limited influence on the private healthcare system. This is a mistake. The VA is a robust healthcare provider with innovative clinical and operational practices that should be firmly entrenched in the national health policy conversation.

As a remedy, we propose strategically coordinating and consolidating the healthcare innovation, demonstration, and implementation capacities of the VA and HHS in order to ensure care of the highest possible quality across urgent issues. Elevating the VA as a major healthcare policy stakeholder will demonstrate the value of government-run healthcare, promote best practices for building an effective and forward-thinking healthcare system, and advance the VA’s “fourth mission” of supporting national preparedness.

Summary

Pending bipartisan “Cures 2.0” legislation is intended to safely and efficiently modernize healthcare delivery in the wake of the novel coronavirus (COVID-19) pandemic. Such modernization is contingent on access to high-quality data to power innovation and guided decision-making. Yet over 80% of Americans feel that the potential risks of companies collecting their data outweigh the benefits. To ensure the success of Cures 2.0, provisions must be added that bolster public trust in how health data are used.

Addressing the largely unregulated activities of data brokers — businesses that collect, sell, and/or license brokered personal information — offers a budget-neutral solution to the public’s crisis of faith in privacy. Building a well-governed health-data ecosystem that the public can trust is essential to improving healthcare in the United States.

At the Senate Health, Education, Labor, and Pensions Committee hearing on Tuesday, Members of Congress and witnesses evaluated whether the U.S. will be able to manufacture enough COVID-19 vaccines to protect the population, as well as be able to distribute them equitably. Vaccine manufacturers are racing to increase their capacity to produce what will likely be billions of doses, but it might take months to years from when the vaccine is approved to produce enough doses to vaccinate the number of people necessary (about 70% of the world’s population) to achieve herd immunity.

Infrastructure for producing vaccines

Committee Chair Lamar Alexander (R, TN) asked witnesses for recommendations on what type of manufacturing capacity the federal government should have on hand to produce and distribute doses of a potential COVID-19 vaccine. Former Governor of Utah Michael Leavitt explained (1:03:30) that while the infrastructure exists to scale up U.S. vaccine manufacturing capacity, the plants were not effectively maintained after they were built. Additionally, maintaining such manufacturing facilities costs a significant amount of money, and, prior to the pandemic, pharmaceutical companies were reluctant to spend so much on facilities that are specific to only one vaccine, which may not ultimately be approved or sold.

Dr. Julie Gerberding, Executive Vice President of Merck & Co., added (1:05:00) that most pharmaceutical companies manufacturing large quantities of vaccines are nearly at capacity producing doses of other vaccines, such as those for the flu. Moderna’s CEO, Stephane Bancel, who is overseeing one of the leading experimental COVID-19 vaccine efforts, mentioned this problem in a May 12 interview with CNBC. He stated, “The odds that every [vaccine] program works are really low, obviously, but I really hope we have three, four, five vaccines, because no manufacturer can make enough doses for the planet.” To put this in perspective, pharmaceutical companies estimated they provided between 162 to 169 million doses of the flu vaccine for the U.S. during the 2019-2020 flu season. To protect the U.S. population from COVID-19, about 230 million Americans would have to be vaccinated. This also does not account for the possibility that each person may need multiple doses to be fully vaccinated or the fact that many manufacturers in the U.S. supply vaccines to other countries.

Besides overall capacity, one of the largest challenges is that the manufacturing infrastructure will differ depending on which type of vaccine(s) are the most effective. The vaccines may need to be produced by processes requiring large vats of cells or other organisms, such as tobacco plants. If a successful vaccine is based on inactivated virus, vaccine production might require highly secure facilities, of which there are very few. This means that until one or more vaccine candidates are closer to being approved by the Food and Drug Administration (FDA), vaccine manufacturers will have a difficult time tailoring their existing facilities, or building new ones, that have the necessary equipment ready to produce the vaccines.

Some pharmaceutical companies are relying on public-private partnerships to develop and scale their vaccines, such as Johnson & Johnson with the Biomedical Advanced Research and Development Authority (BARDA). These flexible agreements allow the federal government to help the pharmaceutical companies invest money and talent into the most promising vaccine candidates. They also make it possible for companies to overhaul their production facilities and build new ones to accommodate the new vaccines, a complex and costly process for these organizations by themselves.

Equitable distribution of a potential COVID-19 vaccine

Even if companies are able to develop and produce enough doses of a COVID-19 vaccine, there are concerns that the doses may not be distributed equitably among the global population. For example, during the 2009 H1N1 pandemic, Australia was the first to manufacture a vaccine but did not export it immediately because it wanted to inoculate its citizens first. Additionally, wealthier countries have the advantage of being able to purchase large quantities of vaccines at much higher rates than countries with fewer resources.

However, even in wealthy countries like the U.S., care must be taken to ensure that vaccines get to the country’s most vulnerable communities. The pandemic has devastated minority communities in cities across the country due to deep-seated public health disparities. Dr. Joneigh Khaldun, Chief Medical Executive for the Michigan Department of Health and Human Services, noted (1:06:40) during her testimony that people of color are more likely to be impoverished and more likely work in occupations that are deemed essential, but they also have the least access to healthcare. Both Dr. Khaldun (45:25) and former Governor Leavitt (40:20) emphasized that the U.S. should develop a national procurement and distribution strategy not only to reduce the competition for vaccines, but also competition for supplies in future pandemics.

Currently, pharmaceutical companies are working to adapt their vaccine manufacturing facilities to accommodate the production of the large number of doses that will be needed to protect against COVID-19, but they have a long way to go. As Chair Alexander noted (2:50:00), Congress will be reviewing the U.S. response to COVID-19 regularly, so stay tuned for more opportunities to engage with Capitol Hill.

To review the full hearing, click here.

Unlike countries such as South Korea, New Zealand, or Germany, the US has not controlled the spread of COVID-19 in a coordinated fashion, and the nation is in danger of a second surge of cases. Some experts already see indicators of the second surge. For instance, COVID-19 hospitalizations rose sharply in several states after Memorial Day, and the percentage of COVID-19 tests that are positive is rising in some parts of the country. Tragically, more than 113,000 Americans have died from COVID-19, about a quarter of all deaths reported globally. This week’s Senate Homeland Security and Governmental Affairs Committee hearing examined key aspects of US preparedness for a second surge of COVID-19.

Status of US availability of medical supplies

From mid March through April, when COVID-19 was first on the rise throughout the country – with cases especially surging in New York City and New Jersey – there were shortages of almost every COVID-19-related medical supply imaginable. N95 respirator masks. Face shields. Cleaning supplies. Drugs for patients on ventilators. Swabs for COVID-19 tests. States scrambled to obtain much-needed supplies, sometimes forced into bidding against one another. This was all due to the combined impacts of the sudden worldwide surge in demand for these goods, deficiencies with US caches of emergency supplies, domestic distribution issues, and a lack of timely, decisive, evidence-based leadership in the White House.

The US should have been ready for the intensified needs brought on by the pandemic, with the health security community recommending for many years that more resources be dedicated to infectious disease preparedness and response, but unfortunately, the country was caught flat-footed. The White House COVID-19 Supply Chain Task Force’s own estimates of US personal protective equipment (PPE) needs and availability – made public because Senator Maggie Hassan (D, NH) pressed the Federal Emergency Management Agency (FEMA) to release them during the hearing – show US supplies of N95 respirator masks, surgical masks, gowns, and nitrile gloves not meeting demand in March, April, and May. Moving forward, the Task Force projects that beginning in July for N95 respirator masks, surgical masks, and gowns, and beginning this month for nitrile gloves, supply will meet demand for hospitals, long-term care facilities like nursing homes, first responders, and janitorial, laboratory, and correctional workers.

But America’s PPE supply is in flux, and there are clearly inadequacies. For example, the Department of Veterans Affairs (VA) says it needs a six-month supply of PPE to handle a second surge of COVID-19. At the height of the initial surge, the VA’s 170 medical centers were using 250,000 N95 respirator masks every day. Right now, the VA only has about a 30-day supply of PPE, and as of June 5th, the VA had not received any of the 7.5 million “generic masks,” nor any of the 3.4 million “isolation gowns,” it had requested from FEMA’s Strategic National Stockpile program back in mid-April.

Strategic National Stockpile

The Strategic National Stockpile was intended to be America’s fallback plan. Through April 1st (the Administration changed the wording on April 2nd), the stockpile was defined as

“…the nation’s largest supply of life-saving pharmaceuticals and medical supplies for use in a public health emergency severe enough to cause local supplies to run out. When state, local, tribal, and territorial responders request federal assistance to support their response efforts, the stockpile ensures that the right medicines and supplies get to those who need them most during an emergency. Organized for scalable response to a variety of public health threats, the repository contains enough supplies to respond to multiple large-scale emergencies simultaneously.”

The stockpile was initially launched in 1999 and managed by the Department of Health and Human Services (HHS) Centers for Disease Control and Prevention. In 2018, responsibility for the stockpile shifted to the HHS assistant secretary for preparedness and response (ASPR). In March, responsibility for the stockpile shifted again, this time to FEMA.

In the face of the coronavirus pandemic, the stockpile did not offer much resilience. Government officials estimated that the US would require 3.5 billion N95 respirator masks for a severe outbreak. There were only 12 million unexpired N95 respirator masks in the stockpile in February. In early March, the stockpile contained only 16,600 ventilators, and on April 3rd, the federal government had just 9,800 ventilators available. It is unlikely that the stockpile is being replenished since PPE that becomes available is generally immediately put to use in COVID-19 hot spots or delivered to medical centers, and it is difficult to gain insight into the current inventory of the stockpile. For instance, after not receiving a response to their Freedom of Information Act request, ProPublica filed a lawsuit against the Administration to get medical stockpile records.

A group of nine former presidential science advisors warned that the US needs to build the stockpile back up by September 1st in order to be prepared for a possible COVID-19 resurgence in the fall, and that state and local supply inventories need to be stocked as well. Their recommendations revolve around increased funding for producing essential medical goods, stockpiling supplies, and improving the coordination of the supply chain and distribution.

Moving forward through the pandemic

During the hearing, the vice director of logistics for the Joint Chiefs of Staff, Rear Admiral John Polowczyk, testified that the US is ramping up domestic production of at least some critical medical supplies. Polowczyk cited the current capacity to manufacture 180 million N95 respirator masks each month, his expectation for the production of an adequate number of reusable gowns by the fall, the beginnings of at least some nitrile glove manufacturing (compared to essentially zero previous domestic capacity), and initiating the process of onshoring the making of some ventilator drugs.

Despite these signs of progress, at the moment, the US does not appear ready for another surge of coronavirus. And that surge may come sooner rather than later.

The coronavirus has killed over 108,000 people, and infected millions, in the US alone. As some areas of the country begin to lift infection control precautions, it is critical for the federal response to the COVID-19 pandemic to improve. An important part of this process is Congressional oversight of the Administration’s response to COVID-19, and during Tuesday’s Senate Finance Committee hearing, Members pressed Food and Drug Administration (FDA) officials on decision-making around the potential COVID-19 treatment hydroxychloroquine, the Administration’s commitment to withdraw from the World Health Organization (WHO), and reliance on China and other foreign countries for drug manufacturing.

Policy on hydroxychloroquine as a treatment for COVID-19

Early in the pandemic, anecdotal reports of COVID-19 patients in China and France who seemed to improve when given hydroxychloroquine, combined with laboratory findings of a possible antiviral effect, raised hopes that hydroxychloroquine, and a closely related drug chloroquine, could serve as treatments for the coronavirus. The drugs had been FDA-approved for the safe and effective treatment of malaria, lupus, and rheumatoid arthritis for many years. President Trump began publicly promoting chloroquine as a potential COVID-19 treatment, and later at the end of March, FDA granted an emergency use authorization (EUA) for both chloroquine and hydroxychloroquine to be used for COVID-19 patients.

The first study describing a large, controlled clinical trial of hydroxychloroquine for COVID-19 was released this week. It was found that people, such as healthcare workers, who experienced high risk exposures to the coronavirus, and who were then given hydroxychloroquine and monitored, developed COVID-19 just like those who experienced high-risk exposures and who were given placebo. Furthermore, the study found that hydroxychloroquine did not impact the severity of illness for these individuals. The question of whether hydroxychloroquine can prevent coronavirus infection if people take it before they are exposed to sick patients is under study in other clinical trials.

During Tuesday’s hearing, Senator Ron Wyden (D, OR), the ranking member of the Committee, said he believed FDA gave in to political pressure when it approved the EUA for hydroxychloroquine. Senator Wyden noted that the EUA led to the production of “tens of millions of pills, including some…manufactured inside facilities in Pakistan and India that have either failed FDA’s inspection or never been inspected by the FDA at all.”

The Government Accountability Office is evaluating the FDA process that resulted in the EUA for hydroxychloroquine and chloroquine to learn if the EUA was purely based on the FDA’s science-based best practices.

Plans to terminate relationship with the World Health Organization

Last Friday, President Trump announced his Administration’s intention to withdraw the US from WHO. In the global fight against disease, WHO’s roles in providing assistance, setting health standards, and collecting data are critical. Considering WHO convenes international partners to support the development of COVID-19 drugs, vaccines, and other measures to curb spread, a US withdrawal will weaken our ability to fight the pandemic. Furthermore, US absence from WHO would allow nations like China, which stalled its delivery of essential COVID-19 information to WHO, to have even more influence on the global stage.

When asked about how the US plans to address global health needs, such as the COVID-19 pandemic, or Ebola in Congo, or polio in Yemen, without being a member of WHO, the FDA’s associate commissioner for global policy and strategy Mark Abdoo responded that he had “not been privy to those conversations” and referred the Committee to the National Security Council or the Presidential spokesperson.

Meanwhile, Chinese President Xi Jinping pledged to contribute $2 billion to WHO over the next two years to mitigate the COVID-19 pandemic, as well as share any coronavirus vaccine developed in China with the world. China’s government or Chinese companies have developed five of the ten coronavirus vaccine candidates currently being tested in clinical trials around the globe.

Reliance on foreign drug manufacturing

Most manufacturing of finished drugs and active pharmaceutical ingredients is done outside the US. Throughout the pandemic, there have been shortages of certain drugs. Many members of the Committee, including Senator Pat Toomey (R, PA), asked about the security of the US supply chain for medicines, concerned that countries such as China could withhold drugs or ingredients, or that future global health emergencies could leave the US vulnerable to being without needed drugs.

FDA officials assured the Committee that the US drug supply is safe and supply chains are secure, and that, to their knowledge, no countries had held back medicines. They attributed the limited drug shortages in the US mainly to a sudden surge in demand for drugs as well as domestic distribution issues. They also advised that the US bolster its domestic drug manufacturing capacity now since the US is bound to face future pandemics. Drug companies could move more production back to the US by implementing advanced manufacturing techniques that allow for more rapid production of drugs, while at the same time reducing environmental impacts.

For the full video of the hearing, click here.

Summary

The organ-donation crisis is one of the most persistent, expensive, and yet solvable public-health challenges of our time. As of January 2020, nearly 115,000 Americans were waitlisted for an organ transplant.  The vast majority have kidney failure, which, as one of the rare conditions qualifying patients for Medicare, imposes billions of dollars of costs on taxpayers. In 2016 alone, taxpayers spent an alarming $113 billion on kidney disease — more than the entire budgets of the National Institutes of Health ($39 billion), the Department of Homeland Security ($44 billion), and the National Aeronautics and Space Administration (NASA, $21.5 billion) combined. The clear solution is to shorten the organ waiting list. For every Medicare patient who receives a kidney transplant, taxpayers save $250,000 in avoided dialysis costs.  This proposal presents a discrete set of actions for the federal government to take to quickly and decisively to address the organ-donation crisis.

Summary

The federal government can directly address the massive market failures at the center of our healthcare enterprise by establishing a new Health Advanced Research Projects Agency (HARPA)¹ modeled after the Defense Advanced Research Projects Agency (DARPA)—the agency the Department of Defense uses to build new capabilities for national defense.

The need for HARPA is twofold. First, developing treatments for disease is difficult and time consuming. HARPA will provide the sustained drive needed to push through challenges and achieve medical breakthroughs by building new platform technologies. Second, the U.S. healthcare system largely relies on the private sector to leverage national investments in basic research and develop commercially available treatments and cures. This model means that diseases for which investments are risky or downstream profit potential is low are often ignored. HARPA will step in where private companies do not, addressing market failures with direct investments that ensure that all patients have hope for a brighter future.

HARPA will leverage existing basic science research programs supported by taxpayer dollars, as well as the efforts of the private sector, to develop new capabilities for disease prevention, detection, and treatment and overcome the bottlenecks that have historically limited progress. To do this, we have to think and act differently about how we address human health challenges. HARPA would support research that directly affirms, refutes, or otherwise changes current clinical practice. It would do this using milestone-driven, time-limited contracts as the central mechanism for driving innovation. This will ensure efficiency, transparency, and optimize success.

Challenge and Opportunity

Every year, the United States spends more than $3.4 trillion on healthcare and tens of billions of dollars on biomedical research. Yet we only have treatments for around 500 of the approximately 10,000 known human diseases.² 30 million people in the United States—half of whom are children—suffer from a rare disease for which no treatment has yet been developed.³ There are no ongoing efforts to develop treatments or cures for the overwhelming majority of these diseases. That massive market failure is the big secret of the biomedical research enterprise and is simply unacceptable. We need bold action to correct this massive market failure and revolutionize how we attack disease.

In 1958, the United States created the Defense Advanced Research Projects Agency (DARPA) at the Department of Defense. This new government agency was designed to make pivotal investments in breakthrough technologies for national security and directly address market failures that were impeding innovation. The establishment of DARPA launched a new era in defense innovation that led to countless innovations, including the Internet, stealth aircraft, GPS-based precision navigation, night vision, autonomous vehicles, speech recognition, and robotic prostheses.

We need to take the same aggressive entrepreneurial approach to health innovation as we have in protecting our nation from foreign threats. Creating a new Health Advanced Research Projects Agency (HARPA) would fundamentally transform the way the United States approaches treating the majority of human diseases, and would directly address many of the shortcomings of our healthcare and biomedical research systems.

Imagine being able to predict and intervene before someone has a mental health crisis; diagnose cancers at their earliest stages when treatments are most effective; end deaths from antibiotic-resistant bacterial infections; and provide treatments for rare genetic diseases. That is the promise of HARPA.

By applying the same tools that DARPA uses to develop new capabilities for defense (Section 3), HARPA would be engineered to close the gap between basic research and real-world needs. HARPA initiatives would target the diseases that affect millions of Americans but are going unaddressed because of risk aversion and short-term, perverse incentives in academia and the private sector. These initiatives would be funded through large milestone-driven timeline limited contracts needed to take on transformational projects, and would be led by top experts recruited for focused stints at the agency. The result will be an institution designed from the ground up to finally solve the most pressing healthcare issues of our time: skyrocketing drug prices, the tragic shortcomings of our mental-health support systems, the opioid crisis, unconscionable waiting lists for organ donations, medical errors, and many more. DARPA enabled the United States to lead the world when it comes to defense innovation. HARPA will do the same for healthcare.

Function

Federal funding for medical research is primarily allocated though the National Institutes of Health (NIH). Through its $41 billion annual budget, NIH funds basic science and clinical research through grants. Grants are typically awarded to individual projects at academic institutions. Collectively, these projects form the bedrock of our knowledge about biology, health, medicine, and disease.

Importantly, NIH is not designed to develop marketable disease treatments or cures or to develop new platform technologies that are intended to revolutionize medicine. NIH funding is used to support therapeutic and technology development, but not in a way that prioritizes quick, efficient commercialization of new discoveries. Moreover, NIH does not include a mechanism for ensuring commercialization. SBIR grants flail at the challenge of commercializing innovations with woefully inadequate funding. Simply put, the current path from NIH-funded basic science to applied research to viable commercial product is too slow, and it does not address massive market failures that define health research and development today, leaving many human diseases without dedicated efforts to uncover solutions. Funds for basic science and clinical research through grants—awarded to academic institutions that pursue particular, individual interests in discovery—are great for uncovering truths about biology, but are an extremely inefficient way to drive toward therapies that make their way into the clinic.

Private companies, on the other hand, only scale up and market economically viable therapies. Therapies that are potentially effective but have a limited market remain inaccessible to the public at large or come with astronomical price tags that patients simply cannot afford.

Effectively bringing new innovations to the market requires alternative approaches to the bottom-up grant funding common to NIH programs. Again, this is not to say that the NIH dollars are poorly spent. The dollars spent on research are essential to understanding health and disease. But an alternative model is needed to advance research toward the development of necessary technologies and treatments to cure disease.

HARPA would close these gaps. Just as NIH brings federal resources to bear on basic science and early-stage research, HARPA would bring federal resources to bear on applied science and later-stage development and deployment. HARPA would have three guiding functions:

1. Launch and manage large-scale health-research initiatives. Although multiple federal entities⁴ work on health research, there is little coordination among these entities regarding research priorities, activities, or progress. HARPA would work with these entities—as well as with the private sector, academia, and states and localities—to launch and carry out targeted, multi-stakeholder research initiatives aimed at our most pressing underserved health challenges. Using milestone driven and timeline limited funding contracts, HARPA will be able to ensure rapid continuous progress. These initiatives would integrate the diverse capabilities of participating institutions to make real progress on persistent and pressing health problems.
2. Invest in transformational platform technologies. HARPA’s focus will be on projects that have direct impact on clinical care. Basic science tends to advance methodologically and incrementally. This partly reflects the nature of the field (one set of experiments informs the next) and partly reflects the nature of incentives in academia (moving too far and too fast away from an established knowledge base decreases the likelihood of publishable findings). By contrast, HARPA will only support transformative research that will substantially improve clinical practice and this is how potential impacts will be evaluated. Pushing for such platform technology breakthroughs is a high-risk, high-reward enterprise. HARPA will focus on the uncertain but potentially transformational medical technologies and therapies that tend to go underfunded today.
3. Support development of treatments and cures for all diseases. All taxpayers contribute to federally funded medical research. But not all taxpayers reap the benefits. Relying on the private sector to bridge the gap between basic research and commercially available products means that those with rare or difficult-to- treat diseases are often ignored. HARPA will correct this market failure by supporting development of treatments and cures for all diseases—especially those that are being neglected by the existing healthcare ecosystem.

Structure

HARPA would be modeled on DARPA. DARPA is considered the “gold standard” for innovation and accountability within the federal government. DARPA is also distinct from other federal agencies that fund research and development in that it is focused on building capabilities rather than simply advancing knowledge. This unique mission requires DARPA to have a unique set of attributes and operating principles, including the following:

• Contracts large enough to provide a critical mass of funding. While most federal grants for academic research are on the order of tens to hundreds of thousands of dollars annually, DARPA funds projects at $1–$5 million per year. These large contracts enable DARPA affiliates to pursue goals that would simply be out of reach at lower funding levels.
• Minimal bureaucracy. DARPA’s entire staff consists of about 220 government employees. This includes DARPA’s ~100 program managers (PMs), who collectively oversee about 250 research & development projects funded at total of about $3 billion per year. All actual research and development activities are conducted by public, private, and academic affiliates. DARPA’s small staff size and flat organizational hierarchy makes the agency effective and nimble, able to move quickly on priority issues in a limited amount of time. Moreover, the fact that DARPA is not organized around disciplines allows PMs to pursue unconventional but productive cross-disciplinary collaborations.
• No entitled constituencies. While funding from other federal grant programs may only be accessible to certain recipient classes (e.g., academic institutions), DARPA does not predetermine which types of institutions are eligible for funding. Funding projects at a wide variety of institutions—including universities, national labs, public and private companies, state and local government agencies— enables DARPA to access the full breadth of talent, expertise, ideas, and resources that the nation has to offer. For example, DARPA funding in the start- up community has yielded advances that may have been difficult or impossible to achieve in other sectors. DARPA uses flexible procurement tools like “Other Transaction Authority” to make it easy for small businesses and nontraditional defense contractors to participate in the agency’s initiatives.
• “Portfolio approach” to high-risk, high-reward efforts. DARPA understands and accepts that frequent failure is the price of success when it comes to achieving transformational breakthroughs. DARPA PMs have the resources and authority to invest in multiple approaches to a given goal. DARPA proposals are openly competed, but PMs can strategically select the winners in a way that creates a diversified, risk-mitigating project portfolio.
• Government control of contracts. DARPA negotiates contracts that enable control over performance. Contracts specify milestones and “go/no-go” decision points to ensure that scientific progress is made in an efficient and timely manner. This enables PMs to better manage funded projects and to cut funding if a project is not yielding desired results.
• Top-notch talent. DARPA attracts world-class PMs recruited from academia, industry, and government agencies. DARPA benefits from expedited direct hiring authority for science and engineering experts.
• High turnover. PMs are hired for limited stints (generally 3–5 years), and there are no career PMs. This approach keeps DARPA talent fresh—ensuring that the agency is scientifically current and flexible to new avenues of investigation—and fuels an urgency for PMs to “achieve success in less time than might be considered reasonable in a conventional setting.”⁵

Many, if not all, of these characteristics could be carried over to HARPA. HARPA could also adopt DARPA’s funding-management model. Under this model, all funding allocations would be left to the discretion of the HARPA Director while all funding oversight would be entrusted to HARPA PMs. Funds would be awarded as milestone- driven contracts that give PMs the capacity for early termination if a particular project is not yielding desired results. This almost never happens with traditional federal grants for research and development.

Because HARPA will differ in structure and function from traditional research-funding agencies, it is sensible for HARPA to have a reporting chain of command separate from NIH. We believe that HARPA would be best situated directly under the Secretary of Health and Human Services (HHS) or under the HHS Assistant Secretary for Health. The Biomedical Advanced Research and Development Authority (BARDA) provides precedent for placement directly under the Assistant Secretary for Preparedness and Response.⁶

Path to Establishment

HARPA could be established under existing authorities, but, ideally, would be established through authorizing legislation and new appropriations. There are several steps the federal policymakers could take to kick-start the establishment process. First, the president could issue a Memorandum or Executive Order directing the HHS Secretary to develop a blueprint for HARPA’s establishment as well as a strategic plan for HARPA’s activities. These documents would include identification of priorities and goals; analysis of global markets, policies and production capabilities; structure and accountability; and initial funding recommendations. Ideally, they would be developed by a short-term Federal Advisory Committee (FAC)—comprised of top physicians, health researchers, and innovative thought leaders. It is important that the FAC include avenues for external input, including providing and promoting a public comments period and convening stakeholder for a across the country. After these documents are developed, the president could urge Congress to deliver a bill establishing HARPA.

Alternatively, the President could include funds for HARPA in an annual budget proposal under the Assistant Secretary for Health or Assistant Secretary for Preparedness and Response. (If Congress appropriates those dollars, HARPA could be established without authorizing legislation.⁷) We believe that a minimum budget of $100 million for HARPA in its first year and $300 million in its second year would be sufficient to get the agency started and to establish high-impact programs, but to be truly transformational, the agency should ramp up to several billion in research expenditures annually. Throughout this process, the president should use high-profile speeches and events to publicly explain the need for HARPA, and to advocate for its creation.

Vision

With a DARPA-inspired structure, HARPA would achieve rapid translation of biomedical discoveries into patient-care capabilities. HARPA’s mission and activities would be synergistic—not duplicative or competitive—with existing federal research efforts. In particular, HARPA would use fundamental scientific understanding developed with NIH support as a foundation for developing breakthrough medical advances.

HARPA would operate in a health ecosystem that includes biotechnology, pharmaceutical, and healthcare companies, venture capital and philanthropy, academic institutions, and government and regulatory agencies. HARPA would address two of the most prominent shortcomings of this ecosystem: (1) the aversion to failure that limits the willingness of academics and the private sector to pursue high-risk, high-reward projects, and (2) profit incentives that limit the willingness of the private sector to develop therapies for rare or difficult-to-treat diseases. HARPA would provide the capital and supportive, focused research environment needed for experts from all sectors to demonstrate “proof of principle” for various medical innovations. In doing so, HARPA will drive explosive growth in the number of technologies, treatments, and cures that cross the so-called “valley of death” separating lab-scale insights from commercially available products.

HARPA would focus on developing transformational technologies that fundamentally change the way we do health research and deliver care. By focusing on the development of tools and technologies to transform the way we approach diseases, HARPA can establish mechanisms that ensure wellness and curing disease are prioritized, while correcting the perverse incentives in the market that limit the country’s ability to receive treatment.

There is a rich history of under-funding the development of such technologies even though they are often quickly engrained into the healthcare enterprise, making it difficult to imagine life without them. They enable breakthroughs that even inventors did not anticipate, create entire new fields of research, and often result in Nobel Prizes. They establish jumping-off points and serve as accelerants for progress. Such work is typically high-risk, high-reward and aims to build transformative capabilities rather than incremental discovery-based research that is commonly funded by the NIH. While NIH does a tremendous job of funding basic science and clinical research, HARPA will build new capabilities on the foundation that agencies like NIH and the Department of Veterans Affairs establish through their funding.

For instance, HARPA could drive the following:

• Technologies that allow clinicians to identify and quantify every protein in a drop of blood, completely transforming disease diagnosis, health monitoring, and care.
• A next-generation diagnostic imaging machine that makes it possible to detect a myriad of diseases at much earlier stages that is substantially cheaper, higher-resolution, and more portable than current MRI machines enabling broader use.
• A cortical eye prosthesis that communicates directly with the brain, making it possible to restore sight to the 7 million individuals (including 160,000 veterans)living with a visual disability in the United States.
• New classes of antibiotics to fight the enormous international public-health andeconomic threat posed by antibiotic-resistant bacteria.
• A series of clinical trials for the most expensive marketed drugs, aimed atdeveloping alternative treatment regimens to improve outcomes by reducing toxicities while dramatically reducing treatment costs. Such de-escalation studies of marketed oncology drugs have been shown to improve outcomes and dramatically reduce costs to save billions of dollars.
• A massive effort to repurpose already approved drugs for new applications. There have only been about 2,400 drugs ever been approved for use in humans. Exploring new applications of drugs that are already known to be safe and effective—instead of only focusing on creation of new drugs—could save billions of dollars on research and development and uncover novel uses for drugs that are already known to be safe and effective for other indications.

Beyond Health

It has not escaped our notice that the same market and institutional failures that created the valley of death and need for DARPA and HARPA exist in other areas of research and development. Our nation is facing unprecedented challenges associated with climate change and the need to provide a better world for all. We feel strongly that the federal government should establish additional Advanced Research Projects Agencies (ARPAs) to complement the efforts of other federal agencies and the private sector. Doing so would enable the government to take a leadership position in tackling monumental challenges.

We believe that, in addition to HARPA, the nation needs to establish capabilities in agriculture (AgARPA), the environment (EnARPA), and transportation/infrastructure (TARPA). Fleshing out the details for establishing each of these entities should fall upon the White House Office of Science and Technology Policy in coordination with the Office of Management and Budget, the President’s Council of Advisors on Science and Technology (PCAST), and the leadership of the appropriate federal agencies. Creating these new capabilities will kickstart new industries, create the jobs of the future, and improve our ability to be better stewards of the Earth. Without them, the nation risks continuing its piecemeal approach to addressing our most pressing challenges, while slipping further behind other nations investing heavily in innovations aimed at solving these global challenges. Establishing ARPA capabilities across the federal government would create a network of forward-thinking agencies prepared to address intractable challenges, while building an extraordinary, lasting legacy.

As the COVID-19 novel coronavirus emerged abroad and here at home, the Centers for Disease Control and Prevention (CDC) devised a diagnostic testing kit and the criteria for who should be tested. Both of CDC’s processes have come under severe scrutiny.

The ramp up in US testing capacity for COVID-19 has been sluggish, in large part due to the initially problematic COVID-19 virus test kit that was devised and manufactured by CDC and shipped to state public health labs. Four COVID-19 virus genome sequences had been deposited in public databases by scientists in China by January 12th. On February 4th, the Food and Drug Administration (FDA) issued an emergency use authorization for the CDC test kit, and kits shipped to state public health labs soon thereafter. At that point, 178 people in the US had been tested. On February 12th, it was reported that some test kits were failing quality checks at state health labs. 360 people in the US had been tested by then. As of February 26th, only 12 out of over 100 US public health labs were able to test for the COVID-19 virus; 445 people in the US had been tested.

The problems with test kit components were due to a manufacturing issue, which according to the FDA, has been resolved. FDA has also authorized public health and hospital labs to use their own tests for the COVID-19 virus as shipments of the newly manufactured CDC test kit make their way to public health labs. An investigation into the exact nature of the CDC manufacturing issue is ongoing, and by the end of the week, the CDC expects to have provided public health labs with sufficient kits for 75,000 people to be tested.

Even so, the California Department of Public Health capacity is limited to 7,400 tests through the weekend. Washington State has very high demand for testing. And state officials in Florida on Thursday said that they do not have enough test kits to evaluate all the patients who could be evaluated according to new CDC guidelines.

The CDC COVID-19 criteria guiding healthcare providers’ and local health departments’ determinations of who should be tested for the virus have gradually changed since January, and have been criticized as being too narrow. Narrow criteria may have resulted in people infected by the COVID-19 virus going undiagnosed.

At the time its test kit was authorized by FDA, CDC recommended considering testing if an individual exhibited COVID-19 symptoms and had traveled from mainland China or had contact with a confirmed COVID-19 patient within 14 days of the beginning of symptoms. In addition to China and the US, there were 22 other countries with confirmed cases of COVID-19, and 36 US states and territories had people under investigation for potentially being infected. On February 12th, the CDC guidance was updated to recommend that severely ill patients without a known source of exposure to COVID-19 virus, but who also were without a known cause for their illness, be considered for evaluation for COVID-19. On February 27th, CDC added travel from Iran, Italy, Japan, and South Korea as well as China. As of February 29th, 472 people in the US had been tested, and there were 9 cases of person-to-person spread that were not travel related.

During Tuesday’s Senate Health, Education, Labor, and Pensions Committee hearing, Dr. Anne Schuchat, principal deputy director of the CDC, explained that CDC devises testing criteria so that tests are not used up on “low-risk situations,” which could reduce testing capacity for addressing situations that are critical. After the hearing, the CDC further amended its policy to recommending that “where there is community transmission in the US, clinicians and public health officials may consider testing patients without severe illness…decisions on which patients receive testing should be based on the local epidemiology of COVID-19, as well as the clinical course of illness.” Community transmission means that there is a person infected with the COVID-19 virus, but how the person became infected is unknown. And then on Wednesday, CDC recommended that clinicians nation-wide use their best judgment based on patients’ symptoms and epidemiological factors. As of Friday morning, there have been 233 confirmed COVID-19 cases in the US, and tragically, 14 deaths.

Out of the 14 people who have died, 13 lived in Washington State. At the hearing, Senator Patty Murray (D, WA), who represents Washington, raised her grave concern (1:19:52 mark in video) about delays in testing and how the COVID-19 virus could have been circulating for weeks, undetected, in her state.

The Committee was given assurances that US testing capacity would ramp up rapidly. By the end of this week, FDA commissioner Dr. Stephen Hahn expects 2,500 newly manufactured test kits, with 500 tests per kit, to be available to hospital labs, and Dr. Schuchat said public health labs will have sufficient kits for 75,000 people to be tested. Upon arriving at labs, the test kits need to clear quality controls, and a given public health lab, for example, can run about 100 tests per day.

We will be tracking this rapidly changing situation; for more COVID-19 information, visit fas.org/covid-19.

Last week, the Senate Homeland Security and Governmental Affairs Committee queried healthcare, disease prevention, and biodefense experts during a hearing about the COVID-19 novel coronavirus outbreak. The take-home message delivered by the witnesses was that the US needs to ramp up its preparedness in a hurry so that if COVID-19 outbreaks emerge on our shores, sick Americans will receive effective care, and spread will be minimized.

COVID-19 severity and transmissibility

The world is grappling with the COVID-19 outbreak, and US public health experts are working with incomplete information to assess the situation, assist globally, and safeguard Americans’ well-being domestically. Amidst the unknowns, the witnesses at Wednesday’s hearing agreed that the case fatality ratio is ultimately likely to be less than the 2.3 percent reported by China’s Center for Disease Control and Prevention. This is because it is very likely that some people who become infected with COVID-19 virus develop no or mild symptoms and do not seek medical care, and so their cases will not be reflected in the reported number of confirmed COVID-19 cases. Indeed, models of the COVID-19 outbreak intended to account for missed cases estimate an infection fatality ratio between 0.3 and 1 percent.

However, the panel cautioned that even if the COVID-19 fatality ratio is actually less than what is calculated from confirmed case data, perhaps 0.2 or 0.5 percent, the average number of people who catch the COVID-19 virus from an infected person – the R-naught – may remain around 2 or more, as indicated by preliminary estimates. The combination of a fatality ratio at 0.2 to 0.5 percent along with a R-naught of 2 or more could make for a particularly devastating disease, causing many deaths among the large number of people the COVID-19 virus could infect.

The progression of the COVID-19 outbreak from a US perspective

There have been 15 confirmed COVID-19 cases in the US, but the panel believes that it is likely there are more that are going undetected, and that flare-ups will soon be observed. According to former commissioner of the US Food and Drug Administration Dr. Scott Gottlieb (1:23:24 mark in video):

“Modeling has shown that the time from first introduction in China, which was probably in November, to epidemic spread was about 10 weeks. If you think that cases were introduced into the US, undetected, in late December or early January, which is probably the case (statistically, it’s quite probable), we’re going to start to see those outbreaks emerge sometime in the next two to four weeks.”

Speaking on a press call last week, Dr. Nancy Messonnier, the director of the US Centers for Disease Control and Prevention’s (CDC) National Center for Immunization and Respiratory Diseases, agreed that at least some person-to-person spread in the US is probable. She said the CDC is gearing up for the COVID-19 virus to “take a foothold in the US,” continuing, “at some point, we are likely to see community spread in the US or other countries.” Later in the week, Dr. Messonnier also announced that five US cities will test patients exhibiting flu-like symptoms for COVID-19 virus as CDC works toward national surveillance for the outbreak.

What the US should be planning for

Expressing concern about a potential COVID-19 pandemic to the Committee, Dr. Luciana Borio, the former director of Medical and Biodefense Preparedness at the National Security Council, said that “we need to brace ourselves for difficult weeks or months to come” because “we’re going to see a lot more cases in the US in the near future.”

Unfortunately, funding for US biological threat preparedness has not been consistent, and the country is not in an ideal state of readiness. The witnesses lamented cyclical investment in public health that has boomed during times of crisis and waned when the biological threat landscape has been uneventful. It would be better to have sustained, well-funded public health infrastructure already in place than to scramble reactively when potential crises such as COVID-19, or Ebola, MERS, and SARS before it, emerge. And so, our healthcare system has only limited surge capacity for caring for high numbers of individuals who may become infected by the COVID-19 virus, or a different emergent pathogen. Although we have a strategic national stockpile of medical supplies and medicines, if stocks were to be expended, the resupply chain is marginal. The capability to rapidly develop point-of-care diagnostic tests – those that front-line healthcare workers could use to get results on site, reducing time to diagnosis – has not been prioritized. While the US has historically risen to the occasion when biological threats present themselves, there is no acceptable reason for being unprepared.

But what about what the US can do, proactively, right now? Witnesses stressed that public health stakeholders should review response plans and responsibilities, ensure both federal interagency coordination and federal, state, and local coordination are primed, test communication systems, assure adequate capacity of diagnostic tests, continue work on therapeutics and vaccines, and clarify how emergency funding mechanisms can be accessed.

The Bipartisan Commission on Biodefense’s executive director Dr. Asha George made the stakes for Americans very clear (1:56:43 mark in video):

“We have to plan for the possibility that we have thousands of cases. In schools of public health, they often train us to look at the data that you’ve been provided, and assume that you don’t have the data – even here in the US – that you don’t have a comprehensive set of data, and then multiply. So we’re often taught to multiply by seven or eight times what you’ve been told. For every one case you see, there are seven or eight out there that you don’t. So that means actually we’d be looking at hundreds of thousands of cases. I think that’s the scale at which we should be planning.”

COVID-19 is of concern, and the best defense is to be prepared and stay informed

Last week, the head of the National Institutes of Health Emerging Pathogens Section, Dr. Daniel Chertow, remarked that “preparedness at the individual level, and at the institutional level, both at local healthcare facilities and healthcare systems within local, state, and national levels, is warranted, and it is ongoing.” To learn about how to be prepared for potential COVID-19 outbreaks, visit ready.gov. To find some answers and stay informed about the epidemic, visit our new FAS COVID-19 novel coronavirus resource page, where we’ll be logging up-to-date information and analysis as more is learned.