Breakthroughs Blog: FDA

As the DC summer kicks into full swing, Congress has had a busy month moving forward fiscal year (FY) 2015 funding legislation for a variety of government programs including global health research and development (R&D). Global health R&D at USAID: Yesterday, both the House and Senate State and Foreign Operations (SFOPs) appropriations subcommittees held mark-ups of their draft bills. The House draft provides only topline spending numbers, with the addition of family planning/reproductive health, polio, the President’s Emergency Plan for AIDS Relief (PEPFAR), and the Global Fund funding levels. The House funded the overall account above the White House request, but below what was appropriated in FY 2014. Including the State Department and the US Agency for International Development (USAID) numbers, funding was $132 million below the numbers Congress appropriated in FY 2014, but a welcome $257 million more than what the White House had requested. For a quick comparison of numbers, check out the Kaiser Family Foundation’s SFOPs policy analysis. The other accounts called out in the House bill were $59 million for polio (the same as FY 2014, but 18 percent higher than the President’s request) and family planning at $461 million. Funding for family planning was a significant but not unexpected cut from both the FY 2014 number (24 percent cut) and the White House request (28 percent cut). As the full House appropriations committee has not yet marked up the bill (and it’s not clear that they will), details on the contents of the bill report’s priorities and other policy riders are not yet known. We do know the House bill reinstates the Mexico City Policy—which prevents nongovernmental organizations who receive federal dollars from promoting abortion services as part of family planning­—or prohibits any funding to the United Nations Population Fund (UNFPA). The full committee may take the bill up for consideration sometime next week. The Senate summary provides considerably more specific account figures for global health than the House bill. The Senate bill flat-funds the Global Fund and PEPFAR compared with FY 2014 and is even with the White House request—it’s interesting to note that for this overall account, the House bill appears to provide a higher funding level, even though we don’t yet know the exact funding distribution. Most other accounts were funded at levels either matching or slightly above FY 2014 funding. One exception is the tuberculosis (TB) account, which faces an $11 million cut from FY 2014 levels, although it is not as dire as the proposed White House funding level, which would have cut an additional $23 million. There is better news to be found for family planning and reproductive health, neglected tropical diseases, polio, and vaccine access. There’s no explicit word in either the House or Senate bills regarding the overall Maternal and Child Health account. NIH, CDC, and FDA Last week the Senate held its Labor, Health, and Human Services (LHHS) subcommittee mark-up and released a summary of their draft bill. A full Senate committee mark-up has yet to be scheduled, and the House has yet to move forward with its own draft bill. This account remains one of the most contentious, both because of ongoing efforts to alter the health care reform programs and because of a Senate plan to use the Overseas Contingency Operations account as a complicated and likely unsustainable mechanism to boost up funding for domestic priorities. The committee requested $30.459 billion for the National Institutes of Health (NIH), an increase to both the White House and FY 2014 levels by $324.0 million and $605.7 million, respectively, but still far less than the $32 billion needed. Global health programs within both NIH and the Centers for Disease Control and Prevention (CDC) also did quite well in the Senate bill—the CDC’s Centers for Global Health and Emerging Zoonotic and Infectious Diseases, the NIH’s National Institute of Allergies and Infectious Diseases (NIAID), and the Fogarty International Centers were nearly all funded at levels above both FY 2014 and the White House request. Both the House and Senate have also marked-up their legislation to fund the Food and Drug Administration (FDA). The House bill funds the FDA at $2.583 billion (this number represents budget authority and does not include user fees), which is a $23 million increase over FY 2014 and is about level with the White House request. The Senate bill funds the FDA at $2.588 billion, an increase of $36 million over FY 2014. The Senate is taking up this bill as part of a “minibus” along with the bills from the Commerce, Justice, and Science subcommittee, the Transportation subcommittee, and the Housing and Urban Development subcommittees. The Senate version of the bill also included some interesting report language around FDA’s international leadership to combat threats to global health and the global drug supply chain.  Defense The House Appropriations Committee approved their FY 2015 bill which essentially flat funds Department of Defense (DoD) R&D—which includes all research at DoD, not just neglected disease R&D—from FY 2014 levels. The American Association for the Advancement of Science provides a helpful summary of R&D funding within the draft bill which can be found here. The Senate Appropriations Committee has yet to mark-up a bill. In both the House and Senate, we see policymakers diverging from the priorities emphasized by the White House in its original budget request: there is little mention of any implementation of the infamous Opportunity, Growth, and Security account suggestions for global health or health research, although the Senate Appropriations Committee has developed its own plan for handling the declining budget caps while still supporting accounts they view as important to US health research programs. While the progress we’ve seen so far is commendable, Congress must speed its efforts to enact smart, evidence-based appropriations for global health R&D programs and their overarching accounts. As the end of the current fiscal year looms closer, policymakers must work together to avoid the stalemate that caused the government shutdown of the fall of 2013 and should support strong funding levels for global health R&D.

  Recently, there has been a surge of interest in evaluating progress made on the 2012 President’s Council of Advisors on Science and Technology’s (PCAST) recommendations that aim to accelerate research and development (R&D) of new therapeutics. Numerous policymakers and stakeholders have weighed in including contributors to the PCAST report and Dr. Janet Woodcock—director of the Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER). PCAST’s recommendations—outlined in the Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation—support an ambitious goal to double the output of new therapies for patients with unmet needs over the next 10-15 years.PCAST believes this goal can be achieved through several approaches and mechanisms including supporting federal funding for R&D, expanding use of public-private partnerships (PPPs), introducing new regulatory pathways, and improving capacity within the FDA to conduct post-marketing surveillance. The House Energy & Commerce Subcommittee on Health recently held a hearing to solicit expert testimony on PCAST’s recommendations and is seeking additional feedback on how PCAST’s recommendations have been implemented and can advance its 21st Century Cures Initiative. Launched in April, the 21st Century Cures Initiative is a bipartisan effort to bolster biomedical innovation and support the discovery, development, and delivery of new treatments. During the hearing, experts recognized Congress and the FDA’s expansion of expedited approval pathways for products that demonstrate health benefits early in their clinical development.   PCAST’s recommendations—outlined in the Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation—support an ambitious goal to double the output of new therapies for patients with unmet needs over the next 10-15 years. Photo: PATH/Patrick McKern On the FDA Voice blog, Dr. Woodcock also outlined initiatives the FDA and Congress have undertaken that are aligned with PCAST’s recommendations. She notes that CDER is involved with many PPPs—which were funded by the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA)—which include collaborative efforts to advance knowledge of diseases and develop more efficient research tools and resources. The PCAST report specifically highlights the value of PPPs in R&D, noting the instrumental role they play in accelerating global health product development. By leveraging diverse expertise—including commercial knowledge and experience navigating regulatory pathways—PPPs facilitate the development of global health innovations. In addition to funding new PPPs, FDASIA also provided the groundwork for the Breakthrough Therapy designation—an expedited regulatory pathway for new drugs and biologics that demonstrate promising preliminary clinical evidence. Eligible products must treat serious or life-threatening diseases with unmet need. Products that receive Breakthrough Therapy designation benefit from a shortened development time period and intensive guidance from the FDA to guide them through the approval process. So far the FDA has granted Breakthrough Therapy designation to 44 products—including tafenoquine—an investigational medicine for the treatment and relapse prevention of Plasmodium vivax malaria. Tafenoquine was developed by GlaxoSmithKline and GHTC member Medicines for Malaria Venture. The expedited clinical development and review times for tafenoquine have the potential to accelerate downstream access to a much needed malaria drug. Breakthrough Therapy designation has been a welcome addition to the three other accelerated FDA pathways that existed when PCAST issued its recommendations. It is evident that PCAST’s recommendations have resulted in substantial initiatives that have enhanced R&D and global health innovation. Despite this progress, there are additional opportunities to strengthen US commitment to R&D and advance the discovery, development, and delivery of new therapeutics.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. Yesterday, Senator Patty Murray (D-WA) introduced in the US Senate a significant piece of global health legislation that would strengthen global health research programming within the US Agency for International Development and improve coordination of global health research and development across the US government. The legislation—known as the 21st Century Global Health Technology Act—is companion legislation to the House version of the bill, HR 1515, which was introduced by Representatives Albio Sires (D-NJ) and Mario Diaz-Balart (R-FL) in April 2013. Knight Therapeutics—the developer of a new treatment for the parasitic disease leishmaniasis—is looking to be the first company to auction off the priority review voucher it received from the Food and Drug Administration (FDA). The company received this voucher—which it can redeem to have any drug moved to the front of FDA’s line for product review—following the approval of its leishmaniasis drug Impavido in late March. A Guardian article on the vaccine cold chain discusses the logistical challenges for the global health community in delivering and administering vaccines—which require constant refrigeration for efficacy—in environments with unreliable electricity and infrastructure. It calls for the next generation of vaccines to be developed with the goal of eliminating a burdensome cold chain and notes the success of the MenAfriVac™ vaccine for meningitis which can be used for up to four days outside the cold chain. During the World Health Assembly, UNITAID, the Drugs for Neglected Diseases initiative, and the Medicines Patent Pool launched a new initiative to develop and deliver improved treatments for children suffering from HIV and AIDS. The initiative–known as the Paediatric HIV Treatment Initiative (PHTI)–will focus on three areas: (1) research and development; (2) intellectual property; and (3) market-shaping to ensure availability of treatment regiments for children living with HIV and AIDS. The World Health Organization released a new document on its efforts to find innovative financing for neglected diseases. The documents states there is a $50 million budget line for demonstration projects and the Global Health R&D Observatory over four years, but did not make clear where the observatory would be situated.

As more detailed information about the President’s fiscal year (FY) 2015 budget requests to Congress is released, the results continue to be a mixed bag for global health research and development (R&D). The Congressional Budget Justifications—annual presentations to Congress to justify requests for funding—help shed a light into the administration’s priorities for FY 2015. National Institutes of Health (NIH) While the President made a modest request for overall NIH funding, the release of the NIH congressional budget justifications gives us a better sense of how the President hopes to fund specific institutes and programs at the agency. The National Institute of Infectious Diseases (NIAID) did include increases of $5.76 million for Biodefense and Emerging Infectious Diseases, $6.95 million for infectious and immunologic diseases, and $23.4 million for HIV and AIDS research compared with funding for FY 2014. The President’s request for the Office of AIDS Research—which covers all HIV and AIDS activities within all of the centers at NIH—was $3.004 billion. While this is nearly a $20 million increase from 2014 enacted levels, this number is still below enacted numbers for 2011 and 2012. Within this request was a decrease of 0.4 percent from 2014 funding for microbicides, an increase of 0.8 percent for vaccine research and a decrease of 1.4 percent for therapeutics research. Centers for Disease Control and Prevention (CDC) Historically, research at the CDC has led to major advances against devastating diseases, including the eradication of smallpox and advances in AIDS research. With a disappointing funding request for NIH, we were glad to see a much stronger request for the global health budget within the CDC. The President’s request included $445.29 million for Emerging and Zoonotic Infectious diseases, a $54.85 million increase from FY 2014 levels. Included in this was the $30 million requested for the Advanced Molecular Detection Initiative. The $30 million will go towards investments in advanced molecular detection technologies to help speed up outbreak detection and increase response efficacy. We’re glad to see the Administration acknowledging the need for the CDC to be cutting-edge in an increasingly global arena. US Food and Drug Administration (FDA) Amongst the government agencies involved in various stages of the development pipeline, the FDA has a vital role to play in accelerating the review of new global health products and strengthening the global regulatory system. Despite the tough budgetary times we currently face, Congress has been asking the FDA to do more with less. That said, we were pleased to see the President acknowledge that increased authority should also mean increased funding, with a robust request of $4.7 billion for the FDA, an 8.1 percent increase from Congress’ appropriation for 2014. The FDA’s Congressional Budget Justification acknowledged the unique role of the agency as an important international collaborator for global health through research and information sharing. The language specifically highlights the Biologics Program and its work to promote research and information sharing to facilitate access to vaccines and other products, harmonize regulatory standards, and establish and maintain standards for biologics. Another highlight is the news that FDA is in the process of drafting a proposed rule to add to their current list of neglected diseases for which they can allow accelerated reviews, and is specifically considering adding Chagas disease. US Agency for International Development (USAID) As we reported in our first blog post, while funding for global health at USAID and the Department of State was fairly disappointing, we were pleased to see an increase in the President’s request for malaria. Notable in this request was acknowledgement of the need for continued funding for R&D to support “the development of new malaria vaccine candidates, antimalarial drugs, new insecticides, and other malaria related research with multilateral donors.” Also in the USAID request was $153 million in central funding to accelerate science, technology innovation, and partnership. USAID describes this aim to “develop and scale breakthrough solutions and accelerate the transformation of the United States’ development enterprise by leveraging outside resources and improving the sustainability of development programs by attracting private-sector, market driven resources.” Department of Defense (DoD) DoD is the only US agency that funds global health research and product development from discovery to introduction. While we’re still sifting through all of the details in the Defense budget justifications, R&D at the agency—including health R&D—increased overall by 1.7 percent over FY 2014 enacted levels. Much of the global health research at DoD is funded through the Army’s Research, Development Testing and Evaluation (RDT&E) division as well as the Defense Health Program—including research on HIV and AIDS, malaria, and leishmaniasis. While we’re grateful the administration is continuing to recognize the need for sustained, robust support for global health R&D across several agencies, the decline of global health research funding in some key accounts remains concerning. We’ll look to Congress to equip these agencies with the necessary resources to carry out their essential role in developing new global health tools.