A call for registries independent of the industry for post-authorisation assessment of orphan drugs
The unsustainability of the high cost of orphan drugs along with lack of clarity on its effectiveness has been a concern of many rare disease stakeholders. The authors of a commentary in Lancet believe that the system of post-authorisation assessment for orphan drugs needs to be reformed to address these problems. They illustrate the pitfalls in the post-authorisation studies of agalsidase alfa that is recommended for the treatment of Fabry Disease to explain their case. To change the systems that are currently in place the authors propose the launch of collaborative registries that are independent from the pharmaceutical industry based on the features mentioned below, to promote appropriate use of orphan drugs and management of costs and to conduct adequate post-authorisation assessment of orphan drugs.
a. Disease-centred registries, instead of drug-centred registries b. Registries supervised by relevant stakeholders, independent of corporate activity c. Analysis of data by independent statisticians d. Obligatory data entry for all doctors which include extended trial data and natural history data e. launched early in the development process f. key factors needed for cost-effectiveness studies
