Quinacrine has been reported as an antiprion agent and proposed as an immediately applicable treatment for Creutzfeldt-Jakob disease (CJD). The authors report the results of an open compassionate procedure to which 32 CJD patients had access. In some genotypic subgroups, a slight but nonsignificant increase in survival was observed, likely due to biased inclusion of long-term surviving patients. There was no pathologic evidence of a beneficial effect of quinacrine treatment.

In 2000, regulation on orphan medicinal products was adopted in the European Union with the aim of benefiting patients who suffer from serious, rare conditions for which there is currently no satisfactory treatment. Since then, more than 850 orphan drug designations have been granted by the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products (COMP), and more than 60 orphan drugs have received marketing authorization in Europe. Here, stimulated by the tenth anniversary of the COMP, we reflect on the outcomes and experience gained in the past decade, and contemplate issues for the future, such as catalysing drug development for the large number of rare diseases that still lack effective treatments.

Objectives The aim of this article is to describe the process, results, and experiences of European Network for Health Technology Assessment (EUnetHTA) Joint Action 3’s (JA3) Early Dialogue (ED) activities and to highlight opportunities for improving the processes. Methods A descriptive analysis of the steps of the EUnetHTA ED process and evaluation of the data from the EDs conducted by EU health technology assessment (HTA) bodies, published guidelines, and documents, as well as internal statistics. Results In JA3, an Early Dialogues Working Party (EDWP) was established, responsible for developing and improving processes and providing advice to pharmaceutical companies, supported by the ED Secretariat. From June 2017 to May 2021, 113 requests for pharmaceutical EDs were received and 38 conducted. The process was continuously optimized, and different approaches for involving patients were tested. Finally, a centralized procedure was chosen with the key documents produced by two responsible agencies and reviewed by the EDWP. Patient involvement was primarily done by interviewing a national patient representative to obtain general feedback on the disease and the planned study design. Conclusions During JA3, EDs were established as an efficient, successful product. Pharmaceutical companies benefited not only from the positions of the individual agencies for the national HTA, but also from the recommendations that were common to all HTA authorities. In addition, regarding the European HTA Regulation, it will be important to conduct Joint Scientific Consultations with a view toward future Joint Clinical Assessments and to further develop processes aligned with the high demand for consultation.

No abstract