Breakthroughs Blog: FDA

Since our last update on the latest budget action on the Hill, two significant events happened late last week in Congress and at the White House. First, the White House’s Office of Management and Budget (OMB) released a report on the potential impact of sequestration on federal agencies. Sequestration would require a reduction in government spending of $109 billion per year for nine years, beginning in January 2013. According to the report, most defense programs relevant to global health—including global health research and development (R&D) programs at the Department of Defense—would receive a 9.4 percent cut under sequestration. Non-defense programs—including most programs at the Centers for Disease Control and Prevention (CDC), the National Institutes of Health (NIH), the US Food and Drug Administration (FDA), the State Department, and the US Agency for International Development (USAID)—that are not exempt from cuts would receive an 8.2 percent cut. It’s important to note that some parts of these programs’ funds are exempt from cuts. For instance, out of a total budget of $34.249 billion for the NIH in fiscal year (FY) 2013, the sequestration applies to $30.861 billion. The rest is exempt. As for the primary agencies that fund global health and R&D programming, the report finds that: The total cut to NIH would equal $2.529 billion. This would put NIH’s total funding for FY 2013 at $31.720 billion. At USAID, the Global Health Programs account—the account responsible for all of USAID’s global health R&D work—would be cut by $670 million, leaving the final global health program’s funding level at $7.498 billion. The FDA, which plays a key role in regulating global health products, would see a cut of $318 million, with a final funding level of $3.624 billion (not including funding from user fees). The CDC would see a cut of $470 million from its overall funding, with $464 million coming from non-defense activities funding. This leaves CDC with a final funding level of $6.861 billion. These predictions are based on current FY 2012 funding levels. Many of these agency programs are already dealing with looming cuts to their funding levels for next year under various congressional budget proposals. If FY 2013 appropriations bills in Congress reduce agencies’ funding even further, the sequestration cuts would be even steeper than what’s outlined in the OMB report. Second, the House of Representatives late last week approved a Continuing Resolution (CR) for FY 2013, in order to keep the government running through the end of March 2013. The House approved the measure by a vote of 329-91, with 70 Republicans and 21 Democrats in opposition. The bill now moves to the Senate and is expected to pass this week. The CR funds the government at $1.047 trillion, consistent with the Budget Control Act. It also includes a slight 0.6 percent increase across the board for all federal programs, essentially maintaining spending at current levels. The CR provides a total of $55.2 billion for International Affairs, slightly higher than the $54.9 billion current level and higher than either the House or Senate FY 2013 appropriations recommendations. House Appropriations Committee Chair Hal Rogers (R-KY) said that lawmakers will aim to finalize appropriations bills when they return to work after the November elections. “We intend to use the lame-duck session to the fullest extent,” he said, adding, “Just because this CR will last until March 27 of next year, we will not rest on our laurels until that time.”

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. Cipla and the Drugs for Neglected Diseases initiative recently announced that their malaria drug has been prequalified by the World Health Organization, an important step toward its roll-out across Asia. A new report from the Kaiser Family Foundation provides a comprehensive look at the Department of Defense's role in global health, including the department’s research and product development efforts. The US Food and Drug Administration has awarded GHTC member Aeras a grant worth $299,525 to spur the development of new vaccines against tuberculosis. A new study finds that the way medical research is funded in the United States has been vastly altered over the last 20 years by advocates asking that research be focused on particular diseases. A widespread vaccination campaign to protect 50 million people against meningitis with a new vaccine has been launched in seven African countries, health officials said Thursday.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. Christopher Austin has been named the new director of the National Center for Advancing Translational Sciences at the National Institutes of Health. A new analysis compares the innovation and science policies of presidential candidates Barack Obama (D) and Mitt Romney (R). A bill signed into law earlier this year that provided the US Food and Drug Administration (FDA) with funding is at risk due to federal budget negotiations. An opinion piece calls on US policymakers to “get serious” about funding scientific and research efforts. A breakthrough tool to combat tuberculosis has been launchedby the Critical Path Institute and the Clinical Data Interchange Standards Consortium (CDISC). The project was the result of collaboration between the Critical Path Institute, CDISC, the FDA, and others through the Critical Path to TB Drug Regimens. Researchers met this week in Boston for the AIDS Vaccine 2012 conference to discuss the latest in efforts to develop a vaccine against the disease.

In this guest post, Rianna Stefanakis—manager of Research and Policy at BIO Ventures for Global Health (BVGH)—writes about a new BVGH study this week in PLoS Neglected Tropical Diseases. This post originally appeared on the BVGH blog. Here at BVGH, we spend a lot of time working on incentives to mobilize companies toward pursuit of research and development (R&D) projects that tackle neglected tropical diseases. In this quest, we’ve focused a lot of our attention on the Priority Review Voucher (PRV) program—administered by the US Food & Drug Administration (FDA)—because it’s a rare instance of an innovative, cost-neutral program targeted specifically at incentivizing R&D for neglected diseases, and has actually been implemented. Because there is only one example of a voucher being awarded and used since the program was instituted in 2007, there’s a healthy debate around the impact of the PRV and its effectiveness in actually stimulating private sector investment in new neglected disease product development. One aspect of that debate is that pharmaceutical and biotech company executives often cite uncertainty around the value of a voucher as a reason for questioning the PRV’s merit. But regardless of the uncertainty surrounding its value, the PRV program has had some impact on decision-making around neglected disease product development in the private sector, as seen in recent statements by companies such as PaxVax. Investors, CEOs, and other industry executives are familiar with the program, but there has been no in-depth analysis to better understand and document the perceptions of these stakeholders, including their estimates of the PRV’s value. To address this evidence gap, BVGH recently conducted a survey of companies with active drug or vaccine programs in one of the 16 PRV-eligible neglected tropical diseases. Our results are fresh off the online journal presses this week in PLoS Neglected Tropical Diseases. Our study focused on understanding three areas: The influence of the PRV in setting priorities within biopharmaceutical companies developing medicines for neglected tropical diseases. The perceived monetary value of the PRV. Barriers toward improving the PRV as an incentive for the development of new neglected tropical disease medicines. We received responses from 12 companies, representing 27 unique neglected tropical disease drug or vaccine programs. Through the survey and follow-up interviews, we found that the majority of respondents did consider the PRV incentive when deciding whether or not to pursue neglected tropical disease R&D programs. In fact, two respondents specifically said the PRV was the controlling factor in the initiation or continuation of their respective neglected tropical disease programs. When compared to other possible motivating factors, however, the PRV ranked lower than other identified incentives, such as potential market value in the developing world or emerging markets. This result was not surprising. It is clear that the PRV is not a panacea, but one of a number of incentives meant to encourage biopharmaceutical companies to engage in neglected disease R&D. In addition to the impact of the PRV on portfolio prioritization, we also compiled data on the perceived monetary value of the voucher. Responses spanned a wide range, suggesting there is no industry consensus on the value of the PRV. This finding is not surprising since a PRV has not actually been sold, nor has there been a dollar amount attached to a voucher’s use. The bottom line is that without a better understanding of the actual market for a voucher, a PRV’s value is uncertain and difficult to assess. This was clear in our follow-up interviews with survey respondents. The respondents underscored, first and foremost, the need for a demonstrated sale of a voucher, with the purchase price disclosed. They also cited a need for the FDA to demonstrate support of the program. Finally, developers cited the need for revisions to the implementation rules governing the PRV program—changes that would have to be made by the FDA and Congress. These revisions varied among respondents and included relaxing the requirement that companies issue a one-year advanced notice prior to redeeming a PRV, relaxing the limit on voucher transferability, and providing greater clarity into the revision process for the list of PRV-eligible diseases. We hope these constructive suggestions will be considered by the FDA and other industry stakeholders. These documented suggestions should also be examined in light of the recently-passed Creating Hope Act legislation, which creates a parallel three-voucher pilot PRV program for rare pediatric diseases. Despite these uncertainties, we remain convinced that the PRV represents a valuable incentive for neglected disease research. The PRV program will ultimately generate a number of vouchers—as more PRV-eligible drug candidates are approved—which will reinforce the utility as well as the value of the PRV program. Those approved drugs will have the potential to make a real difference in the fight against neglected tropical diseases. The full paper can be found here.