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Medicines for Neglected Diseases Caught in Regulatory Labyrinth
October 31, 2011
WASHINGTON, D.C. (October 31, 2011) — Scores of new medicines and other medical products to treat deadly diseases in poor countries are caught in a regulatory labyrinth that slows approvals, raises costs and sometimes puts patients at risk, according to a new report from the Center for Global Development.
Buoyed by public and private funding for global health research, nearly 90 new compounds for the prevention, diagnosis, and treatment of diseases such as malaria, TB, and other afflictions are now in the pipeline, with many approaching clinical trials, when costs soar, according to the report.
But funding for global health is now contracting, even as regulatory and clinical procedures have become more costly and time-consuming. Clinical trial costs are sometimes as high as $30,000 per patient and it can cost billions of dollars to develop a new medicine and bring it to market in poor countries, according to the report.
“Increased funding for late-stage clinical trials and regulatory capacity building in low-income countries is needed but it will not be enough,” says Amanda Glassman, CGD director of global health. “To get these treatments to people who desperately need them, the world needs a streamlined approach that will trim unnecessary costs and improve patient safety,” she adds.
The report, Safer, Faster, Cheaper: Improving Clinical Trials and Regulatory Pathways to Fight Neglected Diseases, was prepared by a CGD working group comprising 22 experts from diverse backgrounds including medicine, law, ethics, government, industry, public-private partnerships, and international development.
The upsurge in new products—including vaccines for diseases like TB, cholera and dengue fever—are due in large part to the funding from major donors such as the Bill and Melinda Gates Foundation, Wellcome Trust , Médecins Sans Frontières (MSF) and the World Health Organization. These funders now face huge costs in bringing the new compounds they helped to develop to market.
“There are now dozens of candidate products in the pipeline,” says Thomas Bollyky, a former CGD fellow and expert on regulatory issues who led the group. “For many neglected diseases, these drug and vaccine candidates would be the first new therapies in a generation. For others they are the first ever. Delays in approval are literally costing lives.”
Malaria and TB alone kill an estimated 2.1 million people annually, nearly all in low-and middle income countries. Lesser known diseases, like human African trypanosomiasis, chagas disease, leishmaniasis, dengue fever and leprosy kill another half a million people.
These diseases take their largest toll in the world’s poorest countries, precisely where the regulatory capacity to test new therapies is most lacking. Moreover, many of the victims are children, exacerbating complex ethical issues such as the meaning of “informed consent” for trial subjects. In addition, to assure scientific validity, new compounds must often be tested in several countries, each with their own regulatory authorities and ethical review boards, which are often weak and lacking in transparency.
In response, a clinical trials support industry has arisen to help product sponsors navigate the regulatory and clinical trials labyrinth. These companies “are increasingly part of the standard overhead for conducting clinical trials; it has become difficult to conduct global trials without their assistance,” the report notes.
The CGD report urges those most closely involved—regulatory authorities in low-income countries, product development partnerships, private firms developing new products, and regional and global institutions—to work together on a fresh regulatory approach that pools scarce country regulatory resources and provides a sustainable platform for clinical trial oversight.
Specifically, the CGD report offers two recommendations for bringing down costs, improving the reliability of the studies, and ensuring patient safety:
Establish Regional Regulatory Pathways
Develop regional approaches to clinical trial regulation and product registration. Countries should be encouraged to pool resources and then opt to accept regional procedures in place of their own. This would increase regulatory capacity, reduce inconsistences across national requirements, and speed product development and delivery to patients.
Streamline Clinical Trial Practices
The regional entities could then make systematic improvements in the design and conduct of clinical trials to decrease cost, increase efficiency, and improve monitoring; for example, by simplifying and placing greater emphasis on identifying problems in trial design early in the process, when they can still be addressed.
CGD president Nancy Birdsall strongly endorsed the recommendations. “This report continues a proud tradition at CGD of pushing to the forefront new approaches that are technically sound and politically feasible,” she says.
One step that Birdsall and Glassman would like to see: the African Union working with the World Health Organization and the World Bank to put in place an effective regional regulatory institution to approve and oversee clinical trials and the speedy registration of new products.
“The coordination challenges are daunting, to be sure. But experience shows that when there is a strong technical consensus among the experts and a clear path forward, diverse players can come together in support of a fresh approach,” Birdsall adds.
For example, she says, a CGD report on the idea of an Advance Market Commitment (AMC) to provide incentives for investment in vaccines opened the way to a $1.5 billion AMC for a vaccine to prevent pneumococcal disease, which annually kills about 3 million children in developing countries. As a result of the AMC, a vaccine suitable for use in developing countries is now in use.
“I’m hoping that this report on safer, faster, cheaper clinical trials will be like our work on AMCs, that it will lead to policy changes that can greatly improve the lives of poor people,” Birdsall says.
Notes for Editors:
Clinical Trials and Regulatory Pathways Working Group: Realizing the promise of the neglected disease products currently being developed requires improved clinical trial practices and regulatory pathways more favorable to trial subjects and current and future innovation. The working group investigated practical strategies for meeting those needs. Members served as volunteers in their personal capacity, independent of their institutional affiliation. A list of working group members is available here.
The Center for Global Development: CGD works to reduce global poverty and inequality through rigorous research and active engagement with the policy community to make the world a more prosperous, just, and safe place for all people. As a nimble, independent, nonpartisan, and nonprofit think tank, focused on improving the policies and practices of the rich and powerful, the Center combines world-class scholarly research with policy analysis and innovative outreach and communications to turn ideas into action.