Breakthroughs Blog: HHS

Representative Tom Price. Photo: Flickr/Mark TaylorThe Senate continues to consider the nomination of Representative Tom Price (R-GA) to serve as Secretary of Health and Human Services (HHS), following his nomination hearings before the Senate Health, Education, Labor and Pensions Committee and the Senate Finance Committee, which concluded yesterday. If confirmed, Price will assume leadership of a department that plays a critical role in advancing the development of new global health innovations through its sub-agencies including the National Institutes of Health (NIH), Centers for Disease Control and Prevention (CDC), and Food and Drug Administration. As head, Representative Price will play an influential role in strategy and decision-making at these agencies.Price did not directly discuss HHS’s engagement in global health research at either hearing, but he did address global health and infectious diseases; innovation for rare diseases; support for health innovation, NIH, and priority setting for NIH-funded research. Here’s what he said: Global health and infectious diseases Senator Ben Cardin (D-MD) questioned Representative Price on how the reinstatement and expansion of the Global Gag Rule under the Trump administration will affect HHS operations and active participation in international global health bodies. This rule restricts all US global health funding from being used to support international organizations that provide abortion services, counseling, or referrals; or advocate for the liberalization of abortion laws, even if they do so without US funds. Senator Cardin asked Representative Price how he will ensure the United States can “fully participate” in global health efforts to address “maternal health, to help stopping the spread and ending HIV and AIDS […] to make sure that the next Zika virus, that we [are] able to contain it so it doesn't cause the catastrophic effects if the Global Gag Rule is enforced in a way that prevents us from participating in international health organizations?While Representative Price didn’t address the impact of the Global Gag Rule on HHS’s international engagement in response, he did stress the importance    of active engagement in the global response to infectious diseases, calling the question “really important,” and noting that his goal if confirmed    is to “gather the best minds and the best talent that we have within the department and without and determine what is wisest policy for this nation    to have [as] it relates to […] infectious disease.” He further elaborated on his support for ongoing infectious disease work saying, “germs    know no geographic boundaries. And we do incredible work, the work that the CDC does and the work that's done by others in our nation that try to prevent    infectious disease, work to detect the spread of infectious disease and then provide a logical and methodical and aggressive response to the outbreak    of any infectious disease is absolutely vital to protect the American people, and we're committed to doing so.” SSenator Cardin expressed his agreement and hope that Representative Price “will look at perhaps unintended consequences from […] executive orders that could compromise our ability to be as effective as we need to, using all tools at our disposal.”While under previous administrations, the Global Gag Rule only applied to organizations receiving family planning–specific funds—which account for about US$600 million in federal funding—the expanded version of the rule applies to organizations that receive any global health money. This means that approximately $9.5 billion in US funding for global health programs administered by the US Agency for International Development, the State Department, and HHS—which address a range of issues including maternal and child health, nutrition, HIV and AIDS (including the President’s Emergency Plan for AIDS Relief), malaria, tuberculosis (TB), and other infectious diseases—is subject to the rule. This could have a dramatic impact on the ability of organizations funded by US global health accounts to team with international partners to develop, test, roll out, and monitor new global health technologies around the world. Rare diseases and the Orphan Drug ActCDC research. Photo: CDC/Dr. Scott SmithSenator Orrin Hatch (R-UT) discussed rare diseases, attesting that the Affordable Care Act prevents “investments and innovative therapies that can cure and save lives.” He asked Representative Price what steps he believes “will improve the pipeline for rare disease therapies to bring treatments and cures to patients in desperate need of hope.” Price replied that the Orphan Drug Act has “revolutionized the ability to treat rare diseases” and that it made “the United States the leader in coming forward with treatments for rare diseases.” He highlighted that there are actions the government can take “in terms of patent protection, in terms of liability, in terms of incentivization [of] resources to be able to encourage the discovery of cures for rare diseases” but stopped short of providing details about specific reforms or new policies he proposes. Price also said that the Orphan Drug Act, which Senator Hatch cited as creating “an explosion” of drugs for rare diseases is “one of the success stories truly for public policy in the country.” Rare diseases are defined as diseases that affect less than 200,000 people in the United States, or those for which investment in a new product is not expected to yield financial return. The Orphan Drug Act, and other provisions to incentivize the development of tools for rare diseases, can also be used to support the development of new technologies for neglected diseases, which affect millions of people around the world, yet few in the United States. Continued support for vehicles that advance the development of tools for orphan and rare diseases—in addition to new steps to ensure that these programs work to support neglected disease research and development (R&D)—is critical to advancing US support for global health R&D. Health innovation, NIH, and the prioritization of research funding Discussing Representative Price’s commitment to public service, Senator Rand Paul (R-KY) asked, “What are your motives? [...] What are your goals? [...] Do you want more people to have health care? Or do you think we disagree on just how we do it and not necessarily on the motives?” In listing principles that he believes are “absolutely imperative for a health care system,” Representative Price said that the system must incentivize innovation “because it is innovation that drives high-quality health care.”Senator Susan Collins (R-ME) cited biomedical research as a “passion,” noting that “if we invest in biomedical research, we have the possibility of not only improving lives for Americans and curing or coming up with effective treatments for devastating diseases, but also lowering health care costs.”When asked by Senator Collins if he supports “increases for the NIH” passed last year and under consideration this year, Price replied, “NIH is a treasure for our country, and the kind of thing we should be doing to find cures for those diseases. One of the core avenues to be able to make that happen is through NIH, and I supported the increase [as a member of Congress].”Senator Patrick Toomey (R-PA) also questioned Representative Price on his views on NIH research and allocation of resources. Citing 5.2 million Americans with Alzheimer’s—a disease for which there is no cure or treatment—Senator Toomey noted that NIH spending on the disease is $168 per diagnosed patient and asked if Representative Price would commit to “ensure that we have a better proportionality in terms of the allocation of resources and the breadth and severity of illnesses.” Representative Price responded, “I think it's absolutely imperative, Senator, and I look forward to working with you.”NIH is the largest financial supporter of global health R&D in the world. Its work to advance basic research on infectious diseases like HIV and AIDS, TB, malaria, neglected tropical diseases, and emerging diseases like Ebola and Zika—as well as research in maternal and child health—has led to important scientific breakthroughs that have transformed treatment in the United States and around the world. Continued and robust support for these research areas is critical to ongoing progress in global health.At GHTC, we will continue to monitor Price’s views on US support for global health and medical innovation and urge strong support for programs that further the development and rollout of urgently needed global health technologies.

The US Department of Health and Human Services has published new rules intended to improve compliance to a 2007 law intended to increase the public availability of clinical trial data. The law requires researchers to register and share data from clinical trials, grants the US Food and Drug Administration (FDA) the authority to fine pharmaceutical companies up to US$10,000 per day for failure to register or report results, and allows the US National Institutes of Health (NIH) to refuse to disburse funds to noncompliant grantees. Between 2008 and 2015, violations of the law warranted more than $25 billion in fines, however, no such fines have been issued to date. Many advocates have expressed concern that the new rules are only as effective as their enforcement. Neither the FDA nor the NIH plan to hire additional staff to enforce compliance of the law. Facebook CEO Mark Zuckerberg and his wife Dr. Priscilla Chan have committed US$3 billion over the next ten years to “cure, prevent, or manage all disease.” The couple is investing $600 million to launch the “Biohub” at the University of California, San Francisco, which will convene scientists and engineers, supporting collaboration in research and product development. The Biohub’s first two ventures are ambitious: the Cell Atlas, which will involve the characterization of every type of cell found in the human body, and the Infectious Disease Initiative, which will support research and development (R&D) for drugs, diagnostics, and vaccines against infectious diseases. The Initiative will establish a Rapid Response Team with the ability to rapidly mobilize scientists and accelerate R&D during an outbreak. The mosquito-borne Mayaro virus, primarily found in the Amazon, has been reported in Haiti. The symptoms of Mayaro—fever and joint pain—are similar to chikungunya, however, the pain can last for nearly a year and patients often report abdominal pain as well. The virus, which was identified in a blood sample taken from a child suffering from the illness, varied from the strain responsible for the more than 40 cases reported in South America since its discovery in 1954. Researchers are particularly concerned with the presence of Mayaro in the Caribbean in light of the ongoing outbreak of Zika virus, which was discovered several decades ago but has only recently begun to spread rapidly and have a significant health impact. A new report from the World Bank quantifying the economic impact of antimicrobial resistance (AMR) indicates that by 2050, the impact of AMR on the global economy and in particular, the livestock industry, could push nearly 30 million people into extreme poverty. Further, drug-resistant infections are much more difficult and costly to treat, and thus, AMR could increase health spending by $1 trillion per year by 2050. These findings reveal that AMR could have a much greater economic impact than the 2008 financial crisis, particularly for low- and middle-income countries. Gonorrhea, a sexually transmitted infection resulting in nearly 90 million new infections each year, has grown increasingly resistant to antibiotics. A new cluster of cases in Hawaii suggests that the emergence of untreatable strains of the bacteria are closer than ever. Over time, gonorrhea has developed resistance to several common antibiotics including penicillin, tetracycline, and fluoroquinolones; the current treatment, a combination of the antibiotics azithromycin and ceftriaxone, is considered the last resort. The samples identified in Hawaii showed unprecedented levels of resistance to azithromycin and burgeoning resistance to ceftriaxone. While azithromycin and ceftriaxone are the only remaining drugs that are effective against the bacteria and well tolerated by humans, scientists at Louisiana State University are testing a new oral antibiotic that appeared to be safe and effective against gonorrhea in phase 2 clinical trials.

Penicillin, the world’s first antibiotic, works by targeting certain proteins—appropriately named penicillin-binding proteins (PBPs)—that play a critical role in building and maintaining the bacterial cell wall. Now, scientists at Harvard Medical School have identified another class of proteins which play a similar role and could be a target for the next generation of antibiotics. The road to discovery started in 2003, when it was determined that certain bacteria could grow without PBPs. In their absence, the proteins responsible for maintaining cell wall shape, elongation, division, and spore formation (SEDS proteins) continued to function normally, enabling the bacteria to thrive. SEDS proteins are found in more types of bacteria than PBPs, and consequently a treatment targeting SEDS proteins could have even broader application than penicillin. On February 22, the Obama Administration requested US$1.9 billion in emergency funding for the Zika response, and 181 days later, Congress has yet to approve it. Consequently, the Department of Health and Human Services (HHS) has shifted $81 million from other programs to support Zika vaccine research and development—$34 million of which will go to the National Institutes of Health (NIH) and $47 million of which will be allocated to the Biomedical Advanced Research and Development Authority (BARDA). Earlier this year, HHS repurposed $589 million in funding from the Ebola response to combat Zika, however, that funding will run out by the end of this month. The NIH will use its funding for human clinical trials of its leading Zika vaccine candidate, however, the agency will need an additional $196 million for additional trials and to advance a second and third candidate. BARDA’s funding will enable the agency to secure contracts and establish partnerships for Zika vaccine development, however, an additional $342 million will be required to continue its work. New research suggests that infection with the Plasmodium parasite, which causes malaria, could reduce the severity of Ebola infection. A team of scientists at the US National Institute of Allergy and Infectious Diseases has examined 1,868 blood samples from an Ebola treatment unit in Liberia—1,182 of which tested positive for Ebola—concluding that patients co-infected with malaria were 20 percent more likely to survive. These results held true even when controlling for the patient’s age and viral load. Viral load actually correlated with survival rates: those with the highest levels of Plasmodium had an 83 percent survival rate, compared to 46 percent of those only infected with Ebola. At the treatment center, all patients were provided with antimalarials, and consequently, treatment for malaria was not responsible for survival rates. While the reason for this trend remains a mystery, Drs. Emmie de Wit and Kyle Rosenke—the study’s lead scientists—suspect that an existing malaria infection expedites the immune response to Ebola, consequently increasing survival rates.

On Tuesday, GHTC launched our seventh annual policy report through a congressional briefing and panel discussion. The critical role played by the US government in catalyzing global health research and development (R&D) was a recurring theme, as panelists discussed the interconnectedness of global and American health and emphasized the need for new health tools to protect against outbreaks and continue progress. As moderator, GHTC Director Erin Will Morton opened the briefing, reminding attendees that despite the landmark progress achieved in global health over the past fifteen years, the US government must continue to invest in health R&D to achieve the next milestones in global health. “Goal setting works. And R&D for new technologies is key to achieving global health goals,” said Morton. Morton emphasized how past investments have had a remarkable impact, highlighting that the neglected disease pipeline has grown from 365 products under development in 2012 to 485 in 2015. However, as the GHTC policy report points out, US funding for global health R&D has stagnated or declined since 2009, and with the exception of the emergency funding for Ebola, 2014 funding levels were down 13 percent. “Our dollars are best spent when [US] agencies are working closely together,” said Morton, highlighting three goals the US government should embrace to advance innovation, as detailed in the report, including the importance of sustainable, long-term funding and innovative financing mechanisms; improved coordination of global health R&D efforts across US agencies; and streamlined and strengthened regulatory pathways. Panelist Dr. Peter Hotez—president of the Sabin Vaccine Institute and US science envoy—reiterated the need for long-term, sustainable funding for global health R&D, noting that the science to develop an Ebola vaccine was first published in 2013, but the technology sat there and was not advanced to product development because Ebola is a neglected disease and the technology offered little financial incentive to the private sector. He emphasized the role of US government investment in catalyzing health research, noting that when the US government “stepped in” and put up more than US$100 million to develop an Ebola vaccine, three leading pharmaceutical companies joined in. Dr. Hotez called for new actors, partnerships, and innovative models to advance R&D, lamenting that “our technical ability to make these innovations has outpaced our social, political, and financial instruments.” Further, Dr. Hotez challenged the notion that neglected tropical diseases (NTDs) are restricted to low- and middle-income countries, noting that 70 percent of NTD cases occur in G20 countries and that “there are 12 million Americans living in poverty with a neglected disease,” adding that neglected diseases are common but are “mostly striking the poor, mostly people of color, more in the American South.” Ambassador Jimmy Kolker discusses how HHS work contributes to both helping individuals overseas and protecting the health of Americans. (Photo: GHTC/Marissa Chmiola)The other panelists concurred, echoing that global health is American health. Panelist Dr. Patrick Kachur—principal deputy director of the Center for Global Health at the US Centers for Disease Control and Prevention (CDC)—made clear that CDC’s global health work is grounded in the agency’s responsibility to protect Americans. Ambassador Jimmy Kolker, assistant secretary for Global Affairs at the US Department of Health and Human Services (HHS), added that “over the next 50 years, the cost of lives and money to the global system of the threat of terrorism, climate change, and epidemics is in the same ballpark,” however, noting that what the US government is investing in fighting terrorism and climate change “so far outstrips what we’re doing in terms of outbreaks and epidemics.” Dr. Kachur called for enhanced coordination, stating “R&D is a whole-of-government and cross-sector affair.” Panelist Dr. David Shoultz—global program leader for Drug Development and Devices and Tools at PATH—furthered this point, noting that “innovation takes place within an enabling ecosystem, and in our context, that enabling ecosystem involves the NIH [National Institutes of Health], the FDA [Food and Drug Administration], HHS, the CDC, our close colleagues and friends at other product development partnerships, and private-sector participants.”In closing, Ambassador Kolker argued that the US government must continue to invest in R&D and consider “how the public sector can be the enabler of the private sector,” both to develop new health tools and to ensure the United States keeps its “competitive edge” as a leader in science and innovation. Dr. Shoultz agreed, stating that “US leadership [in global health] has mattered, it continues to matter, it will matter” in an increasingly globalized world and that investing in health research and innovation is “enlightened self-interest.”To watch the entire discussion, see below: