Clinical trials and data for laypeople, Part 1

Hey, MastAttackers,

We are going to take a short break from the 107 series to address a topic I get asked about constantly: which drugs are best for advanced systemic mastocytosis and how they compare to one another.

Before we get started, there are some things we need to get out of the way. While my second life revolves around educating about mast cell disease and helping patients (and my third life involves having mast cell disease and living with it), my first life and real world job is as a senior scientist for the biomedical research division of a large pharma organization. My job is to figure out ways to test for things that will tell us if a patient is likely to get benefit from the therapies we are testing in studies and clinical trials to treat diseases. While my job largely focuses on supporting trials for cancers like lung cancer and melanoma, I also contribute to trials for rare diseases. One of the rare diseases we have trials for is systemic mastocytosis.

Nothing I say here or in my capacity as Lisa Klimas from MastAttack or Lisa Klimas, a human with systemic mastocytosis, should be taken as representing the organization I work for. I do not ever speak as an employee about the things I just mentioned unless I am at work working. Ever.

Obviously, I have systemic mastocytosis. Everyone knows that. This is not a secret. Systemic mastocytosis is the center of the Venn Diagram of my three lives: they all touch there. For this reason, I avoid talking about certain things about my health because it triggers questions about things that relate closely to my job. For the same reason, I am also very restricted in what I can say about certain therapies for systemic mastocytosis. Specifically, I am very restricted in what I can say about therapies for advanced systemic mastocytosis, like tyrosine kinase inhibitors and multitarget kinase inhibitors.

However, I can talk about how to compare two therapies to one another using science, and you can apply that however you wish. So for the next several posts, I’m going to give a crash course in drug development, clinical trials and data interpretation for laypeople. If you have specific questions, please comment on this post. I will answer any question in a post provided it does not violate my obligations I mentioned above. First post goes up tomorrow night and covers how clinical trials work, what the phases mean, and how people with no science background can understand what the results mean.

Hope this helps clear things up. This will be fun.