2 posts tagged “prescription drugs”

Epidiolex: First FDA-approved drug made from cannabis component (CBD)

Posted 3 months ago by

In a historic move, the U.S. Food and Drug Administration (FDA) recently approved Epidiolex — an epilepsy medication made from purified cannabidiol (CBD), found in cannabis. Read all about it and what it could mean for the future of cannabis-based treatments. As background, see these other recent write-ups we’ve done on medical cannabis/marijuana and CBD.

What is Epidiolex?

Epidiolex “is a liquid formulation of pure plant-derived cannabidiol as a treatment for various orphan pediatric epilepsy syndromes,” according to GW Pharmaceuticals, the U.K.-based company that markets the medication (an oral solution).

Some other info to keep in mind:

  • Limited scope of approval — The FDA approved the treatment specifically for two rare and severe forms of child-onset epilepsy — Lennox-Gastaut syndrome and Dravet syndrome — in patients 2 years of age and older.
  • No THC — Epidiolex is made from purified CBD and does not contain tetrahydrocannabinol (THC), the component of marijuana that causes a euphoric “high.”
  • Thorough research — Researchers studied the treatment’s effectiveness in three randomized, double-blind, placebo-controlled clinical trials involving 516 patients. Epidiolex (taken along with other medications) helped reduce the frequency of seizures when compared with a placebo. Research also found the medication has a very low potential for abuse.
  • Side effects — The FDA says the most common side effects that occurred in Epidiolex-treated patients in the clinical trials were: sleepiness, sedation and lethargy; elevated liver enzymes; decreased appetite; diarrhea; rash; fatigue, malaise and weakness; insomnia, sleep disorder and poor quality sleep; and infections.
  • Cannabis and CBD still controlled substances — Despite the approval of Epidiolex, other CBD and cannabis products are still currently (July 2018) “Schedule I” controlled substances under federal law, the FDA says (again, check out our CBD report for more info on laws and other FAQs).

Some reports say that Epidiolex could be prescribed for off-label uses (for patients with other forms of epilepsy), and its approval could open the door for other cannabis-based treatments

“This approval serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies,” says FDA Commissioner Scott Gottlieb, M.D. “The FDA is committed to this kind of careful scientific research and drug development.”

GW Pharmaceuticals lists other possible treatments they’re studying or developing. GW also markets Sativex (nabiximols) in several other countries to treat cancer pain and multiple sclerosis spasticity, Forbes reports, and a U.S. phase 3 trial is planned to test Sativex for MS spasticity.

What’s your reaction to the approval of Epidiolex? Join PatientsLikeMe or log in to discuss Epidiolex and CBD with others in the forum, and explore treatments members have tried.

Share this post on Twitter and help spread the word.


A Day in the Life of Health Data and Drug Information Clinical Specialist David Blaser

Posted January 11th, 2013 by

What’s it like to work at PatientsLikeMe?  We are continuing to reveal just that with our ongoing blog series “A Day in the Life,” which features PatientsLikeMe employees from different departments.  Today we’d like to introduce you to David Blaser, PharmD, a registered pharmacist who decided to trade his white lab coat for the more casual dress of the startup world in early 2011.  Find out what drew him to PatientsLikeMe, how his pharmacy background factors into his work and more.

1.  What led you to join PatientsLikeMe?

My journey had a few twists and turns, but now that I’m here, I can’t imagine working anywhere else.  I started studying pharmacy at Northeastern in 2003. Toward the end of my time there, I started to consider the career paths I could take and didn’t find any of the traditional ones particularly compelling. Maybe it was part of being young and naïve, but I continually was disappointed and perplexed by our healthcare system in the US. I felt like there had to be a better way.

David Blaser

Then I took a great class called Pharmacoeconomics and Health Outcomes. During this class, you take a medical question (e.g., Should I take drug A or drug B for this problem?) and develop computer models that take into account how it would affect the overall health of the population. It made perfect sense to me, and I didn’t understand why this wasn’t done in our system.

Fascinated with this area of medicine, I started a two-year research fellowship at UMass Medical School to learn more about it. During this time, I worked on lots of models examining conditions from insomnia to hepatitis C. Toward the end, I was looking at career opportunities in this area and got an alert about a posting on PatientsLikeMe. I was amazed how the system PatientsLikeMe had put together was even better than the models I was working on and how it could revolutionize our healthcare system. So I immediately contacted Paul Wicks, the head of R&D at PatientsLikeMe, and was able to set up an internship to work a few days a month on various projects. This eventually turned into a position on the Health Data Integrity Team with Christine Caligtan, Sally Okun and Shivani Bhargava.

On a more personal note, during this time my family and I went through the death of my brother due to substance abuse. This has had a deep impact on me and made me reflect on how can I help others avoid a similar fate. One of my long-term goals at PatientsLikeMe is to develop a better support community for other patients with substance abuse disorders.

2.  What’s surprised you the most about the health startup world?

The majority of my previous work experience was in pharmacies, which is one of the most heavily regulated professions. The amount of documentation, guidelines and laws you have to follow is staggering. When I started at PatientsLikeMe, I would find myself asking, where are our guidelines or what is the protocol?  I remember asking Co-Founder Jamie Heywood, and his response really changed my way of thinking.

He told me that no one else has ever tried to do what PatientsLikeMe is doing and there is no rule book. When you reflect on it, it is amazing to be part of the first company to try to accomplish our mission and develop a rule book for something that’s never been done.  Besides this, there is nothing better than having a job where you can have a beer in the office at the end of a stressful day and others join in with you.  (This is frowned upon in hospitals!)

3.  How does your doctoral and fellowship training inform your work?

While at Northeastern, I completed a doctor of pharmacy degree (PharmD). This gave me the knowledge needed to maintain our drug database and think about how medications should be added to our user profiles. There is still a lot to be done in this area, but I’m looking forward to improving it as we continue to develop our site.

David Blaser (second from left) at play

While at UMass, I studied the different ways that ‘health’ can be measured. This seems like something that should be straightforward, but I found a whole new way of evaluating medicine and health. In theory, you give one group a drug and give another group a sugar pill and see who lives longer, but many patients don’t have the time for that. We need to get answers now, so how can we measure more intermediate outcomes to give us a clue about which medications work better? And what about medications that don’t make you live longer, but make your life better? It’s a difficult process that will never be perfect, but I think that the surveys and tools PatientsLikeMe has developed do an excellent job of measuring these things.

4.  What are the challenges of overseeing the wealth of drug information on the site?

People love sharing information! I recently talked with a member who entered information related to a hand injury they experienced while cutting some fruit, including every nerve and tendon that was injured and the different surgeries and operations to heal it. We love that people share such detailed information, but it can create some difficulty in designing profiles so that they are not overwhelming.

As for the drug information on the site, there are many ways that medications are formulated and taken that are difficult to show in the system. Medications can have different dosages, different formulations (e.g., creams, syrups, pills, injections), different schedules (e.g., take one daily, take one every six weeks, etc.), and they can come in a variety of combinations with other drugs. Not to mention the same medications may be available as a prescription drug, over-the-counter drug and supplement all at the same time. The medication databases that are available don’t always meet the needs of our users, but I do my best to put the right information and options in front of them.

~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~

Interested in making a difference in patients’ lives?  Check out our Careers page to see our current job openings.  Headquartered in Cambridge, Massachusetts, PatientsLikeMe is looking for a Senior Visual Designer, Client Services Program Director, HEOR Research Scientist and more at the moment.