This is a joint post with Cindy Prieto.
Technology is not the answer to all the world’s problems, but advances in medical diagnostics and therapeutics have the potential to improve the lives of millions of the world’s poorest people who suffer from diseases that have few, if any, effective treatments. While recent efforts by product development partnerships (PDPs) and donors like the Bill & Melinda Gates Foundation and the National Institutes of Health have made tremendous progress in building a pipeline for health products for neglected diseases, we have a long way to go before many of these life-saving therapies reach patients.
Developers must next demonstrate the safety and efficacy of these candidate technologies in a series of clinical trials and register these therapies for use in disease endemic settings. These products must be manufactured, distributed, and supported for effective use by target populations. As many candidate technologies for neglected diseases move to late stage clinical development, significant challenges await. There is not enough research and regulatory capacity in many of the countries where the clinical trials will need to occur. There is also not enough money available, under current cost assumptions, to pay for all the clinical trials that will need to happen before delivering these therapies to the people who need them.
Addressing these related challenges will require not only more funding for clinical trials and capacity building, but also increased attention to how trials and their regulatory pathways can be improved to reduce unnecessary costs, delays, and risks to trial subjects. If we can do these trials better, cheaper, and faster, we will not only deliver more of the current candidate technologies to those most in need, we will improve the sustainability of R&D for neglected diseases overall.
These are the goals of CGD’s new Clinical Trials and Regulatory Pathways for Neglected Diseases Working Group and our resolution for 2011. Fortunately, we have tremendous partners in achieving that resolution. Participants in the Working Group include PDP and industry representatives, regulators, donors, clinical trial investigators, and other thought leaders in clinical trial practice, regulation, economics, operations, and global health. The Bill & Melinda Gates Foundation funds the working group and will provide much of the data used in the group’s analysis. John Hurvitz, Dick Kingham, and their legal team at Covington & Burling LLP are assisting with pro bono legal and institutional analysis. ClearTrial is lending its innovative clinical trial budgeting and forecasting modeling software and support to the effort, allowing us to better shape our proposals and build the evidentiary case for their adoption.
As CGD’s working group continues to refine its recommendations for the final report due to come out this spring, we hope that others will join in supporting our new year’s resolution to ensure that the pipeline for neglected disease products can sustainably deliver for the world’s poor.