Of the 7000 rare diseases, most do not have treatments or effective treatments. Most patients with rare diseases will need to take part in a clinical trial if they are to see the development of a new medicine in their lifetime. In my last Blog post I told you how to find clinical trials for your rare disease, or your child’s rare disease. Many clinical trials are listed on ww.clinicaltrials.gov. If you have not yet read that Blog, please go back and read it. The purpose of today’s Blog is to help you to understand if the clinical trial may be appropriate for you or your child with a rare disease. The way to do this is by reviewing the “Inclusion Criteria” listed under specific protocols on www.clinicaltrials.gov.
Inclusion Criteria are criteria that identify the people that can take part in the clinical trial. These criteria are provided for clinical trials for a number of reasons. One of the most important reasons is to ensure that you will be safe if you are included in the clinical trial. The second reason is to ensure that the clinical trial has a good chance of being successful. For instance, all clinical trials will have Inclusion Criteria that seek to ensure that you have the disease that is being studied. The Inclusion Criteria may require that you take a diagnostic test to ensure that you have the disease that is being studied. If you do not meet the Inclusion Criteria you will not be included in the clinical trial. If you do not meet these criteria, this is not a rejection of you as a person.
Some examples of Inclusion Criteria are as follows:
- Patients with SMA Type 1 as determined by gene mutation analysis
- Patient is greater than 6 months of age
- Patient has passed a swallowing evaluation.
You will note that the Inclusion Criteria are specific to the disease and the study that is to be conducted.
If you meet the Inclusion Criteria, you may be included in the Clinical Trial, if you do not also meet any of the Exclusion Criteria, which I will write about in another Blog post.
Lorna Speid, Ph.D.
Founder and President, Putting Rare Diseases Patients First!®