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A Promising Senate Initiative on Neglected Diseases
August 20, 2009
Early this month the U.S. Senate passed an amendment to pending legislation that has the potential to strengthen and streamline regulations governing the clinical testing of drugs for neglected diseases in the developing world. The targeted diseases include malaria and TB, which annually kill an estimated 2.5 million people in the developing world, plus scores of diseases you may have never heard of (such as Chagas disease and leishmaniasis), but that nonetheless exact a large and lethal toll, especially on children and poor people in developing countries.
Senator Sam Brownback, a Republican from Kansas, introduced the amendment to the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act of 2010. As Senator Brownback explained:
What we have put forward in this amendment is a review process to try to establish a new system for neglected and rare diseases so that drug delivery can proceed, and it can proceed on an expedited basis and reduce the cost of doing it, so we can start to develop drug treatments for rare diseases and neglected diseases that happen in poorer parts of the world where the economy does not support that level of research.
In a nutshell, the amendment would require the FDA to issue regulatory guidance and develop internal review standards for products for use in the prevention, diagnosis, and treatment of rare diseases and neglected diseases of the developing world. While that sounds pretty technical—and the steps to achieve this are still more complex—the amendment has the potential to do a world of good. The global development and health community should applaud this effort and work for its inclusion in the final version of the Act. (The bill heads to the House in September.)
In an essay last year, Healthy Foreign Policy: Bringing Coherence to the Global Health Agenda and the associated White House and the World Policy Brief, my colleague Ruth Levine observed that lack of infrastructure and needless bottlenecks in clinical trial procedures may keep life-saving treatments for neglected diseases out of reach of the millions of people who need them desperately. The Brownback Amendment could help address this problem. But to be effective, the FDA mandate to review current practices and suggest improvements should include three additional stipulations:
- Coordinate with other U.S. actors: The FDA review group should coordinate its efforts with the National Institutes of Health, the Centers for Disease Control, the Department of Defense, USAID, and other U.S. government agencies that are heavily involved in the conduct of clinical trials for products for neglected diseases in the developing world. The review group should also include representatives from the public-private product development partnerships and companies that are developing, testing, and registering products for neglected diseases in the developing world. Progress on bridging the structural preclinical, clinical, and regulatory gaps that thwart efforts to prevent, diagnose, and treat these diseases can only occur with coordinated scientific and regulatory leadership and the cooperation of all relevant U.S. government agencies and others that are investing in drugs and other health technology for the poor. For the same reason, the membership of the review group should draw from all relevant FDA offices in addition to officials from the FDA Commissioner and International Programs offices.
- Don’t Forget International and Developing Country Regulatory Processes: The FDA review group should also work with the World Health Organization (WHO) and developing country regulatory authorities to explore ways to streamline the process between FDA and in-country product registration. Getting regulatory approval from the FDA is not the last regulatory hurdle in the process. Products for neglected diseases often must also clear the WHO pre-qualification program and/or developing country regulatory processes before they can be delivered to the patients who need them. Establishing a streamlined FDA regulatory pathway for products intended for use on neglected diseases in the developing world is necessary; it will not, however, be sufficient if failure to coordinate FDA efforts with the WHO or in-country regulators adds months or years of delay before products may be delivered to patients.
- Use new and existing incentives to help bridge financing gaps. Finally, the FDA review group should consider use of existing and new regulatory incentives to fund clinical trials and clinical trial capacity for products for neglected diseases in the developing world. More than 300 products for neglected disease are under development around the world. Even with expected attrition in that product pipeline, current levels of governmental, intergovernmental, and philanthropic funding are insufficient to support clinical development of these products. New sources of financing are needed. We might be able to help with this: I am working with others at CGD to design an innovative regulatory mechanism to help finance and reduce the costs of clinical trials for products for neglected diseases in the developing world.
Senator Brownback’s amendment is an excellent start on a crucial topic that is, by definition, neglected. I welcome it and hope that it will gain bipartisan support in the House. I look forward to tracking its progress and commenting further as it works its way through the committee process.
6 Responses to “A Promising Senate Initiative on Neglected Diseases”
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August 21st, 2009 at 11:31 am
Excellent. Your description of the amendment is right on target and your three additional points on representation and coordination among US agencies to make the review process more meaningful, the importance of FDA – WHO interactions to provide access to the products approved by the FDA in a timely manner, and the critical need to finance clinical trials for global diseases are topics that will hopefully be considered during further discussions of this amendment in the House.
August 24th, 2009 at 1:06 pm
This is a great initiative and the amendment is in the right direction. I wish to lay an emphasis on the component of involving regulatory institutions of the developing world and the WHO in the review process. Such an approach is very important in making any “packages” easily applicable on the ground (in the developing world) where it is meant to have an effect as well as inculcate a sense of ownership.
August 27th, 2009 at 4:24 pm
Tom Bollyky and the further comments on the main blog deserve thanks for explaining the potential of Senator Brownback’s and Brown’s proposals to bolster US efforts in the fight against neglected diseases in the developing world. The Senators’ proposals also fit elegantly into the President’s recent program commitments for global health.
Tom recommends good expansions of the proposals. The FDA, other US agencies and actors would engage in a harmonized approach to reviewing and advancing clinical trials that ask the right scientific questions and produce the answers most applicable to developing world conditions. These are areas where resources are lacking and sufficient investment for these neglected disease problems by governments or the private sector are hard to come by. Study results could then be translated for timely introduction of safe and effective products in a way that reduces the delay in getting innovation from a laboratory bench to a rural, remote bedside.
More is needed to perfect the plan, especially further discussion of efficient funding. A glitch in the definitional mechanism of the proposal means chagas disease and other illnesses Tom refers to are not yet within the scope of the law which requires cumbersome FDA rulemaking. It may also be useful to coordinate with new trial directives in the EU and other jurisdictions and to learn from trial design specialists about best practices for accumulating the data required for product approvals. Finally, the success of these efforts would also be complemented if civil society and the populations targeted for research and product use are given a meaningful chance to participate in these efforts and systems.
August 29th, 2009 at 6:25 pm
This amendment has the potential to do a world of good but we should also be aware of unintended consequences. I worked at USAID in the early 90s when the former Soviet Union broke up. We sent in teams from the FDA to “help” upgrade the regulatory systems in what had been a highly centralized system. It was extremely difficult for the FDA who arguably has the highest regulatory standards in the world to find a middle ground–one that protects the citizens but that also allows needed medicines that do not meet FDA standards to be produced. It is very hard for an agency to have two processes working simultaneously that may have very different cost/benefit ratios to consider. We saw this play out vividly with the rotavirus vaccine where almost no deaths occur in the U.S. but hundreds of thousands still die of this disease in developing countries. The FDA response based on the cost benefit analysis in the U.S. drove the actions of all other countries.
Maybe the answer lies with the FDA but maybe there is another answer out there that does not rely on an agency to have two cultures simultaneously. More time may be spent on culture change than in creating perhaps new regional systems in developing countries that could play a stronger regulatory role. Hardly a new idea but maybe one that needs more attention.
September 1st, 2009 at 3:28 pm
Thanks everyone for the excellent comments. Just a few additional thoughts.
Robert raises a good point. As it currently stands, this Amendment applies to the same diseases eligible for a priority review voucher (see 21 U.S.C. 360n(a)(3)), which includes malaria, TB, and leishmaniasis, but not chagas. The Secretary of the Department of Health and Human Services may expand that statutory language to “other infectious disease[s] for which there [are] no significant market[s] in developed nations and that disproportionately affect[] poor and marginalized populations.” I worry, however, that linking this Amendment to the priority review voucher statutory language may not be helpful. The cost-benefit analysis of extending the list of eligible diseases will be different in each case. I would much rather the Secretary be able to make a decision about the scope of the regulatory pathway envisioned in this Amendment without also having to worry about the potential costs of expanding the priority voucher program.
Melinda likewise raises excellent points about the feasibility of a two-track FDA process and cost-effectiveness. I share these concerns, but am hopeful that having a more inclusive membership of the review group, including private product development partnerships, industry, and civil society, would help raise and address these issues at the report phase of the review group process.
September 24th, 2009 at 6:18 am
Great post Tom. You emphasized good points. I am interested in Neglected Diseases and http://www.checkorphan.org is a good source to get the latest Research, Treatment and People news about neglected, rare and orphan diseases. Check this article about Human African trypanosomiasis it’s very interesting http://checkorphan.org/news/im....._available
Once more, thanks for the post.