Enhancing FDA Information Sharing for Neglected Tropical Diseases

Many countries look to the US FDA for guidance on approval decisions. In fact the FDA will sometimes receive and evaluate drugs and vaccines whose primary market is in less developed countries. Fexinidazole, for example, is a drug for treating African trypanosomiasis, i.e. sleeping sickness. We don’t get many cases of sleeping sickness in the US but there are many such cases in the Democratic Republic of Congo.

Thus, the US FDA is providing a useful service, both to US pharmaceutical firms and especially to developing countries. That’s great. But Jacob Trefethen of OpenPhil notes that for odd bureaucratic and legal reasons we redact a lot of information that could be useful to the countries that actually will use these treatments. Here, for example, is an excerpt from the approval decision for Fexinidazole:

Second excerpt from fexinidazole review documents

What’s especially strange here is that as far as Trefethen, or I, can tell, no one wants this! The FDA has no reason to hide this information, the company submitting the proposal surely wants as much information as possible sent to the countries where they will ultimately need to get approval (remember this is successful applications!) and the medical agencies in the developing countries would like to get context to have confidence in the FDA’s decisions. Instead, it seems that these drugs are getting caught in rules intended to protect pharmaceutical firms in other contexts. Thus, Trefethen makes two suggestions:

let’s create a track for products on the Neglected Tropical Disease list, sharing assessments with few or no redactions with the WHO Pre-Qualification (PQ) system, and allow PQ to share those documents further with regulators in partner countries.

Such an approval track already exists in the EU:

[The EU] have an approval track for products that are mostly going to be used elsewhere. If you apply using that track, they loop in regulators from those countries too. They share the documents assessing your clinical data and inspecting your manufacturing site with the WHO prequalification (PQ) team – the team whose stamp of approval speeds things up for many countries with less experienced national regulators. Gavi and the Global Fund need a product to be prequalified in order to buy it through the UN procurement agencies (e.g. UNICEF, for children’s vaccines).

Even without an approval track there are other small changes in priority and emphasis that could improve information sharing. The FDA is not unaware of these information sharing issues, for example, and there are procedures in place for confidentiality agreements with other countries. Trefethen suggests these could be given greater priority.

FDA leadership should set aggressive goals to complete more two-way Confidentiality Commitments with lower- and middle-income country regulators.

Extend the scope of existing commitments, when they’re limited, to allow sharing in more areas – especially related to drug approvals.

Extend 708(c) authority to more country agreements, not just those with European countries, to allow sharing of full documents that include trade secrets.

I’ve long advocated for peer approval, Trefethen gets into the weeds to point to specific ideas to make this a more useful idea, especially for developing countries. See Trefethen for more ideas!


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