The European Medicines Agency (EMA) is considering lowering the minimum number of patients required in clinical trials of potential hemophilia treatments because of “dramatic” increases in hemophilia research and concerns about the availability of patients for rare diseases like hemophilia A.
“EMA began the process after becoming concerned that the minimum data requirements of its current guideline are incompatible with the ‘historically unique’ pace of progress in hemophilia,” the regulatory agency for the European Union said in a press release. As such, it “is planning to revise its guideline on development of drugs to treat hemophilia to cut the risk of patient enrollment bottlenecks slowing progress.”
Currently, the clinical development of factor VIII products requires that companies provide data on at least 100 trial patients in their marketing submissions, and collect post-authorization data on at least 200 patients within a specific time frame. Those numbers were introduced in 2012 and left unchanged in a May revision, but the EMA reconsidered this and released a draft concept paper in July proposing further changes.
One option being studied is to shift the burden of evidence generation away from clinical trials and toward patient registries.
Specifically, changes the Blood Products Working Party (BPWP) recommends include:
(1) the reconsideration of clinical trial requirements in PUPs (Previously Untreated Patients), such as applicability, concept, patient numbers, exposure days, statistics, consequences, etc.
(2) the establishment of a minimum core parameter set on data collection in hemophilia registries to address regulatory expectations, specifically for safety concerns regarding inhibitors (neutralizing antibodies).
(3) addressing non-replacement therapies being developed for hemophilia treatment, such as monoclonal antibodies, small peptides, and gene therapy products
Regulatory and public discussion on these proposed changes is expected to begin soon, with the planned release of a revised draft guideline early in 2017. This draft will be open to public comment for three months following its release and prior to a vote for adoption.