ER1: When numbers are small – clinical trials in rare diseases

ACPE UAN: 0475-0000-19-028-L04-P. A knowledge based activity.

Linked to EAHP Statements:

Section 2 – Selection, Procurement and Distribution: Statement 2.3
Section 6 – Education and Research: Statement 6.5

Abstract

New therapies for rare diseases are always foreseen. Although clinical research methodology has set its standard since almost 60 years, paradigms for conducting clinical trials in rare diseases are dramatically different, due, by definition, to lack of sufficient numbers of subjects to enrol in order to reach statistically significant results.

Hospital pharmacists face the question when new drugs come on the market, and there is the need of their evaluation for inclusion in hospital formularies. 

The seminar will approach on one side the issue from the methodological standpoint, e.g. which are the rules and correct ways for conducting research for new therapeutical entities in rare diseases; on the other hand, the issue will be described from the patient standpoint, e.g., which are his/her expectations when they are asked to participate in clinical trials.

Learning Objectives

After the seminar, participants should be able to:
• recognise whether methodology of clinical trials in rare diseases is reliable;
• be critical when evaluating literature on a new drug for rare diseases;
• take into consideration patient’s needs and expectation when participating in a clinical trial for rare diseases.

Educational need addressed

Hospital pharmacists need to know differences in the process of evaluation of new medicines for rare diseases in order to contribute to proper decisions when such new technologies become available to be included in hospital formularies.

Keywords | Rare diseases, research methodology, patient involvement.