… patient registries as key instruments to overcome challenges … as industry-driven single
purpose drug registries. There is an … Networks for Rare Diseases requires the establishment of …

… , as with many rare diseases, patient registry data may provide … In 2015, EMA established
a Patient Registries Initiative to … Recognising the benefits and challenges inherent in using …

… for rare disease therapy development and describe clinical progress so far in developing
drugs … of clinical trial data, electronic medical records and/or postmarketing surveillance data …

… a specific RWD source is fit for purpose; second, whether the trial or … are children, but there
are often ethical issues when enrolling … The efficacy of cerliponase alfa was established in a …

… rare diseases, while only 8% are well-established conditions (… , a European network of
registries for the surveillance of … hosted at iucr.org is unavailable due to technical difficulties. …

… to create a comprehensive and in-depth overview of rare … The observed differences are
challenging for rare disease patients, … is currently considering the use of MCDA for this purpose. …

… of these groups and the challenges that they have faced. The … , and a preparatory call was
set up to brief him or her on the … transcribed verbatim for the purpose of analysis. Occasionally…

… Reasons for discontinuation were extracted from the registry, and … Challenges in performing
rare disease trials include small … issues in developing drugs and biological products for rare …

… of issues in order to represent rare disease patients and be … in accessing funding and
developing new treatments [25]. … information to patients regarding the purpose and outcome of the …

… of issues that hinder drug repurposing for rare diseases from … abatacept off-label and the
rationale for its use based upon its … We established that developments that start in academia …