Tricia Aung

[Editor's note 4/1/16:The Global Health Regulatory Requirements Database is no longer part of GHTC. The global health regulatory team's member site is now hosted on the Gates Foundation PDP forum.] Photo: FDA/Michael J. Ermarth Navigating global regulatory processes is a critical component to the development and introduction of global health technologies. New drugs, vaccines, medical devices, and diagnostics must be evaluated and registered by national regulatory authorities before they can reach patients who need them. These technologies are typically intended for use in low- and middle-income countries (LMICs) which often have limited regulatory capacity and evolving regulatory requirements. As a result, securing clinical trial and product registration approval from national regulatory authorities can be a considerable hurdle for product developers. The GHTC’s Global Health Regulatory Requirements Database, developed with support from the Bill & Melinda Gates Foundation, is a tool that helps product developers and global health advocates better understand and navigate regulatory requirements of LMICs. The database serves as a critical information tool to help facilitate the testing and launch of new, lifesaving global health technologies. Here are four reasons why you should check it out: 1. It’s easy to use. Organized by country and technology type, the database features high-level summaries of regulatory requirements for 25 countries in Africa, Asia, Europe, and Latin America. The database conveniently consolidates regulatory requirements for these countries in one place, so users don't have to spend as much time tracking down regulatory information for different countries. Think of it as Cliff Notes for global health regulatory processes. 2. It’s comprehensive, but still comprehensible. Users can access top-line information on regulatory authority structure, relevant harmonization initiatives, ethics review processes, clinical trial and product registration application requirements, and pharmacovigilance requirements. You can also find links to key guidances and other relevant resources. All the information is presented in an easy-to-scan format so you can find what you seek. 3. It’s regularly updated. In August 2014, the Global Health Technologies Coalition (GHTC) completed its annual verification of the database. All information in the database is systematically reviewed on an annual basis, and significant changes to country regulatory requirements also are added on an ad-hoc basis. 4. It’s constantly expanding. During the last year, the GHTC introduced eight new countries to the database—Bangladesh, Ghana, Malawi, Malaysia, Nepal, Peru, Russia, and Ukraine! The GHTC also recently added country-specific labeling requirements and expanded information on regulatory authority collaborations with the World Health Organization and stringent regulatory authorities like the US Food and Drug Administration and European Medicines Agency. Whether you are a global health product developer or an advocate, you’ll find the database to be a useful tool. It can help product developers access necessary information to formulate a regulatory strategy, and can help advocates identify areas to provide and champion technical assistance for capacity development, harmonization, and other ways to advance global regulatory processes.

  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 guest post, Tricia Aung—the GHTC’s regulatory associate—writes about a new database that provides easy access to information on global health regulatory information. Before they can improve the lives of patients and communities, new global health technologies must undergo rigorous testing and evaluation to ensure that they are safe and effective. National regulatory authorities—like the US Food and Drug Administration (FDA) in the United States—are responsible for protecting the public health throughout this process, making sure that clinical trials are conducted safely, and reviewing trial results to determine whether the benefits of potential products for the population outweigh the risks. Since many global diseases are endemic in countries with limited regulatory capacity, getting regulatory approval can be a huge roadblock to getting products clinically tested, manufactured, and delivered. Regulations vary by country, so developers of new vaccines, drugs, and other health products often face the challenge of understanding and juggling numerous sets of regulatory requirements. This is further complicated by the fact that in many developing countries, it can be difficult to find information on regulatory requirements. The Global Health Regulatory Requirements Database provides consolidated access to country-level regulatory requirements, thereby aiding product developers, multilateral organizations, and advocates with getting products through clinical trial approval and designing programs to strengthen regulatory capacity. Photo: Richard Franco. The Global Health Regulatory Requirements Database, a new tool developed by the GHTC and supported by the Bill & Melinda Gates Foundation, is designed to help address this regulatory information gap. The database provides consolidated access to country-level regulatory requirements, thereby aiding product developers, multilateral organizations, and advocates with getting products through clinical trial approval and designing programs to strengthen regulatory capacity. Currently featuring 17 countries in Africa, Asia, and Latin America, the database is a central repository of high-level regulatory information and links to key guidance documents and regulatory resources. The database also offers users easy navigation from country to country and separates technology-specific information into three categories: drugs, vaccines, and diagnostics. Key data points include: Contact information for national regulatory authority representatives. Overview of clinical trial and product registration requirements, including data requirements, costs, estimated timelines, and pharmacovigilance requirements. Description of ethics review processes. Background on collaborations with other regulatory agencies, multilateral organizations, and capacity-building and harmonization initiatives. For example, the FDA offers trainings to enhance regulatory capacity building in developing countries. The agency also contributes to global efforts to improve regulatory harmonization in conjunction with partners like the World Health Organization. The database was designed to include a broad base of information, making it useful to many audiences. For example, researchers and regulatory affairs professionals can find the information they need to formulate effective regulatory strategies to gain approval to conduct clinical trials and market products. Creating a more informed, strategic regulatory plan that satisfies country requirements can help reduce regulatory delays that inhibit products from reaching people. The database also promotes systemic change by capturing the current state of national regulatory authorities in developing countries. It can help guide advocates in efforts to develop advocacy strategies to improve and streamline global regulatory processes. Further, stringent regulatory authorities, such as the FDA, and public health organizations can use the database to identify opportunities to provide technical assistance at the country level to strengthen the capacity of regulatory authorities, and encourage harmonization and policy reform. To deliver new health technologies to people as quickly and safely as possible, there needs to be a strong plan for regulatory approval and an enabling environment that supports regulatory review. The database can help address these needs and inspire lasting changes in the global health regulatory space.