Breakthroughs Blog: WHO

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.

The World Health Organization (WHO) released a statement last week calling for the publication of all data from clinical trials for drugs, vaccines, and medical devices. The pharmaceutical industry has made great strides in reporting the results of clinical trials since the establishment of the WHO International Clinical Trials Registry Platform. However, a report released in conjunction with the WHO statement demonstrates that negative or inconclusive clinical trial results often go unreported, and there is still a need to publish data from older clinical trials. The United States Centers for Disease Control and Prevention (US CDC) is partnering with the African Union to establish an African Centres for Disease Control and Prevention (African CDC) comprised of a Coordinating Center in Addis Ababa, Ethiopia—where the African Union is located—and five Regional Collaborating Centers across the continent. The purpose of the African CDC is to support and connect African ministries of health to monitor and respond to disease outbreaks. The US CDC will provide initial technical expertise and will sponsor ten African epidemiologists and two public health experts from the US CDC to staff the Coordinating Center and Regional Collaborating Centers. Dr. Jack Whitescarver, director of the Office of AIDS Research (OAR) at the National Institutes of Health (NIH), will step down in July after leading the Office for 15 years. Dr. Whitescarver first joined the NIH in 1977 and he was involved in the initial detection and monitoring of the HIV and AIDS epidemic in the United States. He was also a major player in the NIH’s HIV and AIDS outreach and education efforts, prior to the establishment of OAR in 1988. Under his leadership, OAR has supported both international and domestic research efforts, with a focus on HIV and AIDS in minority populations and in women and girls. The United States Patent and Trademark Office (USPTO) at the US Department of Commerce announced the winners of the 2015 Patents for Humanity program. The winners will be granted an expedited patent application, ex parte reexam, or ex parte appeal before USPTO. This year's winners include Sanofi—the manufacturer of a synthetic version of the antimalarial artemisinin—and Novartis, a pharmaceutical company that has discovered a class of compounds that show promise in fighting drug-sensitive and drug-resistant tuberculosis. Sanofi is partnering with GHTC members PATH and the Bill & Melinda Gates Foundation to produce synthetic artemisinin, while Novartis has provided TB Alliance—another member of the GHTC—with the compounds to continue the research and development of treatments for tuberculosis.

India is expected to add all of the drugs currently used in the government’s HIV and AIDS and tuberculosis (TB) control programs to its list of essential medicines within the next six months. These drugs are currently available for free in public clinics; many patients, however, prefer private clinics due to perceived discrepancies in quality of care. The list, which was last updated in 2011, sets price caps to ensure that treatments remain affordable—paramount in a nation where more than 70 percent of the population lives on less than US$2 per day. China’s investments in health-related research and development (R&D) are expected to surpass US funding by 2023. The government of China has invested $1.1 billion in drug development since 2011, due to a policy mandate requiring increased funding for life sciences, including the pharmaceutical industry. Meanwhile, US funding for global health R&D has declined or plateaued each year since 2009. China is currently the world’s biggest manufacturer of active pharmaceutical ingredients and is becoming a leading site for contract research organizations, which support preclinical and clinical research for major pharmaceutical companies. The World Health Organization (WHO) issued a guidance document last week regarding a leading medicine for hepatitis C. The new guidance will inform the design of bioequivalence studies, which determine whether a generic is comparable to a patented product. The drug, sofosbuvir, is patented by Gilead Sciences and can cost up to $1,000 per pill. Proof of bioequivalence will be critical for manufacturers aiming to produce a generic drug and ultimately seek WHO prequalification for their product. In her final speech as commissioner of the US Food and Drug Administration (FDA), Dr. Margaret Hamburg defended the agency’s rigorous drug approval process. “Innovation doesn’t matter if the product doesn’t work,” she noted, highlighting the risks of “unsafe and ineffective medical products.” Hamburg’s remarks come as the House of Representatives Energy and Commerce Committee is in the process of developing legislation—known as 21st Century Cures—that calls on the FDA to expedite the regulatory process using its “breakthrough therapy” designation. This designation, which ensures an accelerated review and increased guidance from the FDA, was granted to 31 drugs in 2014. The 21st Century Cures legislation would allow the FDA to review products based only on preliminary clinical trial data and would allow companies to hold off on long-term safety and efficacy trials until after the product was on the market.

On Thursday, March 26, 2015, the Global Health Technologies Coalition (GHTC) launched its sixth annual policy report titled Meeting the challenge, seizing the opportunity: US leadership can advance global health R&D with a luncheon and panel discussion on Capitol Hill. The recent Ebola crisis was a common theme as panelists discussed what lessons could be learned about the need for further global health research and development (R&D) to enhance America’s preparedness to address future health threats. Dr. Joanne Carter—executive director of GHTC member RESULTS and moderator of the event—opened the discussion by reflecting on recent successes in global health R&D, while also emphasizing the need for increased investments in tools to tackle existing and emerging public health challenges. Carter noted how years of budget stagnation and sequester cuts have stymied the development of promising technologies and eroded US preparedness to tackle global health threats like Ebola. She expressed that “as we have achieved hard fought gains, challenging fiscal circumstances have put funding for global health product development at risk. Global health crises like Ebola and emerging obstacles like antibiotic drug resistance have demonstrated that existing tools are not enough. If we are going to successfully combat these challenges and reach our goal of a healthier world, we must continue to innovate.” Panelist Daniel Bausch, MD, MP&TM—senior consultant for the Clinical Team in the Department of Pandemic and Epidemic Diseases at the World Health Organization—echoed the need for forward-looking investments in R&D and preparedness, noting that the world has spent much more money fighting Ebola than it would cost to prevent the epidemic. Bausch maintained that while the US and global response to the Ebola crisis was unprecedented, we must learn from our failures to combat future epidemics. He underscored that R&D should be treated as a preventative measure, rather than a response to a crisis, reminding the audience that the clinical trial process can take years, even decades. Bausch called for increased long-term US government investments in global health R&D and strengthening health systems and R&D infrastructure in developing nations, noting that in an increasingly interconnected world “US health is global health.” Panelist Ann Mei Chang—executive director of the US Global Development Lab at the US Agency for International Development (USAID)—echoed Bausch’s remarks admitting that the US government is “much better at responding to crises than investing long term.” Bausch further cited the challenges that budget cuts pose for global health researchers, explaining that “if you’re a researcher in a university setting, you probably spend at least 50 percent of your time looking for funding.” He further remarked that research is often stalled because researchers lack the funding to continue their work in an already long and difficult process. Panelist Captain Michael Schmoyer—director of the Office of Pandemics and Emerging Threats at the US Department of Health and Human Services (HHS)—remarked that there were bright spots in the US response to Ebola, including the ability of US agencies to collaborate and work together. He asserted that during the outbreak, coordination between HHS, the Centers for Disease Control and Prevention (CDC), USAID, the Department of Defense (DoD), and the Food and Drug Administration was unprecedented. Captain Schmoyer confessed that agency leaders surprised themselves with their ability to work effectively together and that the response to the Ebola crisis could serve as a model for US agency coordination moving forward. He cited the example of how HHS worked with other agencies including the DOD, CDC, and USAID to set up the first medical unit to care for those healthcare workings responding to Ebola. The other panelists agreed that partnership and collaboration, both across the US government and with other sectors, is essential to advancing global health R&D during times of crises and under normal circumstances. Chang discussed how the US Global Development Lab at USAID advances R&D by leveraging existing funding through diverse partnership from different sectors including the private sectors, nongovernmental organizations, universities, and local government. She emphasized the value of open innovation, through calls for proposals and ideas, to elevate previously untapped voices. Chang cited an example of the value of this collaborative model during the Ebola outbreak. USAID partnered with the White House Office of Science and Technology, CDC, and DoD to launch the Fighting Ebola Grand Challenge for Development, focused on improving the personal protective equipment used by healthcare workers on the front line. Chang said the competition received ideas from engineers, architects, and even wedding dress designers, and now USAID is working with a manufacturer to develop the winning design and introduce it into the field. Captain Schmoyer further endorsed the value of partnership noting that some of the simplest, but most invaluable, ideas generated in the Ebola response resulted from consulting new partners. All three panelists agreed that there were lessons the US could learn from the Ebola crisis to enhance our nation’s preparedness to respond to future health threats. Carter closed the event by noting that “As the world creates a new agenda for global development and seeks to confront existing and emerging health challenges, the time is right for US policymakers to strengthen the nation’s investments in research and product development and reaffirm our resolve to harness American ingenuity to save lives, improve health, and enhance economic prosperity and security around the world.” She called on US policymakers to “meet the challenge and seize the opportunity.” For more information, find the full report here and an accompanying piece on the site Medium.