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  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. Sarah Alexander—director of external relations for the Global Alliance to Prevent Prematurity and Stillbirth—writes about the recent groundswell of momentum surrounding maternal and child health and the role research and development (R&D) can play in helping more babies have a healthy start to life. Researchers at the University of Tokyo are bioengineering rice to transform it into a low-cost storage and delivery mechanism for drugs to combat cholera, rotavirus, and other infectious diseases and contagious illnesses. The researchers say that bioengineering the vaccines or antibodies into rice would allow them to be stockpiled and stored at room temperature, avoiding cold chain issues that exist with existing vaccines. Geno de Hostos—director of research and preclinical development at PATH’s Drug Development Program—writes about how an integrated approach to tackling diarrheal disease can save thousands of children’s lives each year. He argues that R&D efforts focused on new drugs and vaccines hold tremendous potential, and there is a need to sustain appropriate funding to achieve more progress. The Bill & Melinda Gates Foundation announced funding for 55 projects through its Grand Challenges Explorations initiative program which funds innovative ideas to address global health and development problems. Grant recipients include several projects to improve condom design, “cologne” for cows that alters their smell to mimic humans in order to divert disease-carrying insects from people, and two projects to improve the vaccine cold chain, among others. Dr. Margaret Hamburg—commissioner of the US Food and Drug Administration (FDA)—writes on the FDA Voice blog about the World Health Organization’s passage of a resolution to strengthen regulatory systems. She notes the unique challenges and opportunities faced by regulators because of globalization and the extraordinary increase in the global supply of medical products, and she discusses the increasing efforts by nations to collaborate to share information and reduce system inefficiencies.

In this post—which originally appeared on the DefeatDD.org blog and PATH’s Drug Development Program blog on June 4—Geno de Hostos—director of research and preclinical development at PATH’s Drug Development Program—reflects on themes in the organization’s new video “Defeat diarrheal disease? Together, we’ve got it covered” . He discusses how an integrated approach to tackling diarrheal disease can save thousands of children’s lives each year. The uneasy reality When you walk into a diarrheal disease health clinic in a place like Bangladesh, the reality of the disease strikes you immediately. It’s the rows of bright orange bed covers drying in the sun, long lines of women with children waiting to be seen, and the patients—most of them too young and vulnerable to be already fighting for their lives. Diarrhea takes the lives of more than 1,600 children every day before they can grow to see their fifth birthday. Photo: Jonathan Torgovnik © The onslaught of diarrhea is sudden and loss of fluids is copious. Mere inconvenience quickly turns to a daily struggle for life as you fight off dehydration that diarrhea causes. A child is rushed to a hospital where lifesaving oral rehydration solution (ORS) is administered, usually by a mother or a care provider. Spoon by spoon, lost fluids are slowly restored, though weakness can persist for several weeks, and repeated bouts of diarrhea are common. The reality is that despite rapid progress in reducing child deaths from diarrhea over the last decade—a decrease by more than 50 percent, from almost 1.3 million in 2000 to approximately 600,000 in 2012—there is a lot more to be done. And as long as diarrhea continues to take the lives of more than 1,600 children every day before they can grow to see their fifth birthday, there is no time we can allow ourselves to waste. Power of an integrated approach Another reality is that it doesn’t have to be this way. Effective, proven solutions for tackling diarrhea— safe drinking water and improved sanitation, adequate nutrition, rotavirus vaccines, ORS, and supplemental zinc, combined with continued feeding—are already in our hands. We need to work harder to scale them up and find new methods to deliver them to those who need them. Research and development (R&D) efforts focused on new drugs and vaccines hold tremendous potential, and we need to sustain appropriate funding and efforts on that front to continue with our progress forward. Together, these critical solutions form a network of approaches to diarrheal disease that reinforce one another and form a solid foundation for a healthy life. That’s why at PATH, we work to address diarrhea on multiple fronts. Our drug development team is developing new treatments to shorten the severity and duration of diarrhea before it becomes fatal, while also working to improve the effectiveness of proven diarrhea therapies like ORS. PATH is also working to develop new vaccines against the leading causes of diarrheal disease, partnering with countries to increase access to existing vaccines for rotavirus, developing and delivering safe water treatment and storage products, advancing health devices, and strengthening behaviors like breastfeeding education. R&D efforts focused on new drugs and vaccines for diarrheal diseases hold tremendous potential, and we need to sustain appropriate funding and efforts on to further progress. Let’s keep going! Having worked in nonprofit drug development for nearly seven years, I am more heartened than ever before by the impressive gains being made in R&D of innovative health technologies to fight the leading causes of child death like diarrhea. I am also reminded of the critical value of sustained commitment to our children and the need to accelerate progress. A multipronged approach can help us get there—among other factors, development of innovative health technologies, opening access to needed vaccines, drugs, clean water and sanitation, adequate nutrition, and education will continue to push us forward. It’s time we change the reality of diarrheal disease. More information Defeat diarrheal disease? Together, we’ve got it covered Defeat DD: Simple solutions to fight a common killer Targeting diarrheal diseases