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Photo: PATH/Patrick McKern Update: The Global Health Technologies Coalition is excited to announce that the Global Health Innovation Act (H.R. 2241) passed the House of Representatives on December 18. Last month, Representatives Albio Sires (D-NJ) and Mario Diaz-Balart (R-FL) introduced a new piece of legislation—the Global Health Innovation Act (H.R. 2241)—that would strengthen global health research and development (R&D) programming at the US Agency for International Development (USAID). USAID plays an important role in advancing global health in low and middle-income countries and this legislation would ensure that the agency is taking appropriate steps to incorporate innovation for new global health technologies across its programming by requiring that the agency submit an annual report to Congress on the development and use of global health innovations in the agency’s programs, projects, and activities. The Global Health Innovation Act marks a key opportunity in advocacy for the need for innovation in global health. The GHTC dissects the bill below and highlights the key provisions. If passed, H.R. 2241 would: Make permanent and improve USAID’s annual report to Congress on health-related research and development activities. The act would require a comprehensive analysis of USAID’s activities to develop, advance, introduce, and scale up global health technologies. This allows policymakers and advocates to track and understand the full scope of health R&D activities taking place at USAID, identify best practices, and zero-in on gaps that may exist. Require USAID to articulate how it sets goals for health product development and measure progress toward these goals. The legislation would require USAID’s report to describe how it sets goals for health R&D in relation to its overall health objectives and mission, and how the agency measures progress towards these goals. This provides enhanced transparency into USAID’s decision-making and review processes. Require USAID to describe and demonstrate the impact of its investment in health innovations on global health outcomes. This includes analysis in the annual report of how the agency’s investments and activities in health innovation have advanced its goals to reduce the burden of HIV and AIDS, end preventable child and maternal deaths, and protect against infectious diseases. Such an impact analysis will help magnify the importance of investment in health R&D for achieving sustainable health solutions. Direct USAID to report on how it is working with partners, donors, and through public-private partnerships to leverage US investments to accelerate health innovation. This includes a description of the how the agency is working through partnerships to develop, introduce, and scale up affordable, available, and appropriate health products and close gaps in the health innovation development pipeline. This section of the report will acknowledge the diverse coalition of private sector, nonprofit, foundation, and academic institutions partnering with USAID on health innovations, and demonstrate the importance of a coordinated, cross-sector approach to health R&D. Encourage USAID to improve collaboration and coordination, both within USAID and between other US agencies, to fill critical R&D gaps. The act requires that USAID report on how it is coordinating R&D activities across its programs and how it is collaborating with other US agencies to support global health product development and ensure critical gaps are filled. This will help promote a whole-of-government approach to health R&D, which will better leverage investment, knowledge, and expertise. The legislation would make permanent and improve USAID annual report to Congress on its R&D activities. Credit: USAID The Global Health Innovation Act would not only ensure important information about USAID’s activities is made available to global health nongovernmental organizations, advocates, and policymakers in Congress, but it would also be an asset for USAID. In codifying the report on global health innovations, the bill will ensure that the agency continues to coordinate, streamline, and organize its use and development of global health technologies—a practice area that spans many departments and bureaus. It will also help the agency gauge performance, share best practices, and understand gaps in service to more efficiently and effectively serve its mission. The legislation draws upon the 21st Century Global Health Technology Act, legislation introduced in the 113th Congress, which sought to strengthen global health R&D programming at USAID by codifying programs supporting product development partnerships. The new legislation takes a more holistic approach, and directs USAID to analyze how it is using innovation to address global health challenges and the ways in which the agency can further accelerate the development and use of health technologies. It shifts away from endorsing specific programs and instead underscores the importance of USAID’s strong partnerships that leverage investments in R&D and advance global health innovation. Passing the Global Health Innovation Act will drive home the fact that health innovations are a critical piece of the global health agenda. GHTC and our members look forward to working with Congress to champion this important legislation. To read the full text of the Global Health Innovation Act, click here.

The French pharmaceutical company Sanofi purchased a priority review voucher (PRV)—which entitles the owner to an expedited review of a future product by the US Food and Drug Administration (FDA)—for US$245 million last week. This particular sale was part of the Rare Pediatric Disease PRV, which was modeled after the Neglected Tropical Disease PRV—a program intended to incentivize research and development (R&D) for neglected diseases, as the market for drugs to treat these conditions is often limited. The voucher significantly expedites the FDA’s review process, from 10–12 months to 6–8 months. This voucher was awarded to Retrophin for approval of a drug to treat bile acid synthesis disorders, which are considered rare pediatric diseases. Only two vouchers have been awarded through a PRV program, the first of which was sold to Sanofi for $67 million and the second to Gilead Sciences for $125 million. Delegates from the World Health Organization’s (WHO) 194 member states approved a resolution at the World Health Assembly—the organization’s annual convening—to address the spread of antimicrobial resistance. The resolution establishes a global strategy to control and prevent the spread of drug-resistant infections, including through the R&D of new antibiotics. Under this new resolution, member states will be required to develop and implement national plans to combat antimicrobial resistance within the next two years. WHO Director-General Dr. Margaret Chan assured delegates at the World Health Assembly that the WHO will provide technical support to national governments as needed, stating “we are going to do our utmost to support you.” The status and progress of these efforts will be reported to the World Health Assembly in May 2017. Photo: World Health Organization The global action plan, presented by the WHO Secretariat during the World Health Assembly, called for improved surveillance; increased awareness; and adequate, sustainable R&D funding surrounding the growing threat of antimicrobial resistance. The plan notes the “serious market failure” for antimicrobial R&D, calling for new mechanisms to incentivize and accelerate the development of new antibiotics. Senators Patrick Leahy (D-VT) and Chuck Grassley (R-IA) recently introduced the bipartisan Patents for Humanity Program Improvement Act, which expands the Patents for Humanity program at the US Patent and Trademark Office (USTPO) to ensure small businesses can participate. Each year, USTPO identifies approximately ten products that have been patented through their office and that have since been used for humanitarian purposes, and provides the winners with a voucher for an expedited patent application, ex parte reexam, or ex parte appeal. The legislation would make the voucher transferable, which would encourage small businesses or individual researchers or inventors—who may not anticipate future patent applications or dealings with USTPO—to participate.

21st Century Cures.Last week, the House Energy and Commerce Committee unanimously voted to approve the 21st Century Cures Act—a bill that aims to speed the development, approval, and introduction of new health technologies.Clocking in at over 300-pages, the legislation is packed full of proposed changes to the way health products are developed by the US National Institutes of Health (NIH) and regulated by the US Food and Drug Administration (FDA). Below, GHTC breaks down a few of the proposed changes that could matter most to global health researchers, product developers, and advocates.NIH receives boost in funding The legislation would reauthorize the NIH for the next three fiscal years (FY) and boost funding over that period, starting with US$31.8 billion in FY 2015 and increasing to $33.3 billion and $34.8 billion in FY 2017 and 2018 respectively. (As a reference, current NIH funding is around $30.3 billion.) It is important to note this boost is in discretionary spending, rather than mandatory spending, so these increases could still be subject to spending caps and sequestration cuts which in the past have significantly reduced NIH’s budget.The legislation would also provide $10 billion in new NIH funding over the next five years through the establishment of a new “NIH Innovation Fund.” This pot of money is mandatory spending, and thus would NOT be subject to spending caps and sequestration. The legislation mandates that at least $500 million per year of the fund would go to a new Accelerating Advancement Program, which seeks to address unmet medical needs in the United States including infectious diseases and neglected tropical diseases and antibiotics.The legislation would also mandate that the NIH, and the centers and institutes within it, develop five-year strategic plans to guide research activities. The language defining how NIH must use portions of the funding is unlike current NIH funding allocations and has some concerned that Congress is being too prescriptive with scientific research.NCATS permitted to expand into later stage research Photo: National Institute of Allergy and Infectious DiseasesThe Cures Act would remove current restrictions that prohibit the National Center for Advancing Translational Sciences (NCATS) at NIH from supporting clinical trials beyond phase IIA—the first round of trials to test efficacy, rather than just safety of a treatment in humans.NCATS was created to help advance translational science, particularly for rare diseases in the United States. At the time it was created, Congress prohibited the center from conducting later stage clinical research out of fear that it would then be competing with and duplicating private-sector product development activities. This legislation lifts that prohibition, extending NCATS ability to conduct trials through stage III—the final pre-market stage where safety and efficacy of a treatment are tested in in large groups of individuals.Many global health conditions qualify as rare diseases under the program’s definition because they impact fewer than 200,000 people in the United States, meaning this legislation change could permit NCATS to support later stage clinical research for many neglected diseases and other global health conditions.Bill promotes pediatric research Under the legislation, NIH would be required to establish a national pediatric research network to pool resources and coordinate activities on pediatric diseases. This legislation also encourages the NIH and FDA to work with the European Union, industry, and other partners to establish a global pediatric clinical trials network.Drugs often react differently in children than they do in adults, and creating pediatric-specific treatments has proved challenging for researchers and product developers producing treatments to be used both domestically and globally. This legislation seeks to overcome some of these challenges by promoting development and testing of pediatric formulations of drugs.Congress acknowledges importance of agency participation in scientific conferences The act includes a sense of Congress acknowledging the importance of participation in scientific conferences and meetings for both NIH and FDA employees. While the legislation doesn’t go so far as to lift existing restrictions or provide funding for participation, Congress’s recognition of this importance is a critical step forward.A GHTC analysis found that many agency representatives cite a lack of funding for participation in scientific conferences as a serious hindrance to enhancing coordination and collaboration among agencies engaged in global health research and development (R&D).Increases use of biomarkers and surrogate endpoints as clinical evidence at FDA Photo: FDA/Michael J. ErmarthThe legislation paves the way for the FDA to rely on biomarkers and other surrogate endpoints to accelerate product approval for a broader category of products. Proving that a product or treatment can successfully produce the desired clinical endpoint—may not be feasible in certain circumstances or can often require clinical trials that are large, long, and costly. So in some cases, trial sponsors have proposed using biomarkers—or biological indicators—that are correlated with the clinical endpoint as a substitute to assess the efficacy of a treatment or product more easily. For example, to measure whether a treatment prolongs cancer survival—a clinical endpoint—one might assess a surrogate endpoint like whether the treatment succeeds in shrinking tumors.At the moment, the FDA permits use of surrogate endpoints for accelerated approval for drugs intended to treat a narrow category of patients with life-threatening illnesses and unmet needs. The bill calls on the FDA to establish guidance for use of qualifying biomarkers and work with sponsors to establish parameters that would allow for approval of a broader set of products using surrogate endpoints.Use of surrogate endpoints poses both benefits and challenges. It could lower R&D costs and reduce the length of time needed to conduct clinical trials, thereby accelerating the availability of treatments. However, at the same time, it can often prove difficult to determine if a surrogate endpoint or biomarker is a reliable indicator that the product has the intended clinical benefit. For example, elevated cholesterol levels are linked with heart disease, but at the same time, it is possible that a drug that reduces cholesterol will have no measurable impact in preventing deaths from heart disease.Spurring antibiotic drug development If enacted, the Cures Act would create a new expedited FDA review pathway for antibiotics intended to treat “a serious or life-threatening disease, condition, or indication” and intended for use in a limited target population. This new pathway could allow the FDA to approve a product based on earlier Phase II trials and surrogate endpoints, rather than larger, later-stage Phase III clinical trials.Some organizations support this new pathway, arguing that it would expedite patient access to new antibiotics and help tackle growing antibiotic drug resistance. Others argue that this new pathway would allow products to be approved based on lower FDA standards, which could in turn put patient health and safety at risk.The bill would also increase Medicare payments to hospitals who use certain new antibiotics as a way to financially incentivize private-sector development of critically-needed new antibiotics. However, some critics of this provision argue that it would be counterproductive to the intended goals and would instead encourage overuse of new antibiotics, which would in turn hasten the development of drug resistant strains and further exacerbate the problem of antibiotic resistance.Changing the way medical devices are reviewed and approved Photo: FDAUnder the legislation, medical devices would be eligible for “breakthrough” designation by the FDA, which allow them to receive priority, expedited review.This new priority review program for devices is modeled after the breakthrough therapies program for drugs and is intended for “breakthrough technologies” for either conditions “for which no approved alternatives exist” or that would offer “significant advantage over existing approved or cleared alternatives.”In addition to creating this priority review pathway for medical devices, the legislation would also direct the FDA to take steps to ensure clinical trial designs for medical devices are “as efficient as practicable,” by exploring options such as the adoption of shorter or smaller trials, use of surrogate endpoints, or adaptive trial design.In an effort to further accelerate approvals of medical devices, the legislation would significantly alter the type of evidence the FDA could consider when reviewing a device for approval. This bill would allow the FDA to consider as valid scientific evidence case histories, registry data, studies published in peer-reviewed journals, and data collected in countries other than the United States. While this change could help speed the testing and approval of critically needed devices, critics argue it could also undermine FDA’s ability to ensure medical device safety and efficacy— citing the fact that case histories are anecdotal and lack a control group, while journal articles can often leave out critical information or can include mistakes, misrepresentation, or fraud, which the FDA may not become aware of if it relies solely on the published articles.Expansion of market exclusivity, but less than originally anticipated The legislation would extend by six months market exclusivity for drugs approved for a new indication or use for a rare disease or condition, extending a drug’s exclusivity period not only for the new indication, but for all of the drug’s indications.The purpose of market exclusivity is to help companies recoup the investments they made into R&D for a product or therapy and encourage future private-sector investment in research and product development. However, critics also note that extending market exclusivity delays entry of generics into the marketplace which can lead to increased costs for patients and limit affordable access to lifesaving health technologies. Product price can pose a significant barrier to access particularly in low- and middle-income countries.Earlier versions of the legislation featured additional provisions extending market exclusivity that were taken out of the version approved by the Committee. These included provisions which would have permitted the sale of market exclusivity to other companies and extended exclusivity for follow-on drugs and first-to-file American-made generic drugs.FDA receives increased funding To ensure FDA has the capacity to carry out its programming, the legislation would increase FDA funding by $550 million over the next five years. The bill would also exempt FDA user fee programs from sequestration spending cuts.While passage of the legislation through the Committee is a critical milestone, the act still has a long journey before it can become law. The legislation is expected to be voted on by the full House of Representatives in June. Meanwhile, parallel legislation is under development in the Senate. If both pass their respective chambers, the two versions will eventually have to be reconciled by both chambers before the legislation can be passed and then sent to the President for consideration. GHTC will continue to monitor this legislation as it develops.

Members of the European Parliament are calling on the European Commission to adopt a resolution to combat the growing threat of antimicrobial resistance—which kills 25,000 Europeans annually. The proposal calls for both “pharmaceutical innovation” and “new revenue models whereby economic returns for companies [are] de-linked from prescribed volumes of antibiotics,” noting the role of public-private partnerships in research for new antibiotics. The proposal also includes measures to promote responsible human and veterinary use of antibiotics, including public awareness campaigns and physician training; monitoring and regulation of antibiotic prescription and sales; and improved diagnostic procedures to ensure appropriate use of antibiotics. Across the Atlantic, the United States Food and Drug Administration (FDA) wants to address the paucity of data on antibiotic use in farm animals to better assess the impact of this practice on growing antimicrobial resistance. The FDA is collecting public comment on a proposal which would require drug companies to report sales for antibiotics approved for livestock, disaggregated by use in cattle, pigs, chickens, and turkeys. While sales of these antibiotics increased by 20 percent between 2009 and 2013, several major poultry producers—including Tyson Foods and Perdue—have promised consumers significant cuts in antibiotic use. Meanwhile, Congresswoman Louise Slaughter (D-NY) is reintroducing the Delivering Antimicrobial Transparency in Animals Act which would require major farms to report on agricultural antibiotic use. The House Energy & Commerce Committee unanimously approved the 21st Century Cures Act on Thursday, a bill intended to support research for and expedite the approval of medical breakthroughs. The legislation is the result of a year-long effort, led by the committee Chairman Representative Frank Upton (R-MI) and member Representative Diana DeGette (D-CO), which involved a series of hearings, testimonies, white papers, and requests for public comment to identify the best policy approaches to advance medical cures. The committee has already developed a US$13 billion deal to pay for the legislation’s provisions, including $10 billion for research conducted by the United States National Institutes of Health and $550 million for regulatory efforts at the FDA. As these funds would be mandated, rather than subject to the appropriations process, they are exempt from sequestration budget caps. The bill’s cost will be offset by the sale of oil from the Strategic Petroleum Reserve and changes to government Medicare payments, including delayed payments to insurance companies.