Phase 1 Clinical Trials – An Early, But Key Step, in Drug-approval Process
We wait in anticipation for multiple sclerosis (MS) drugs to clear Phase 3 clinical trials and get to the Food and Drug Administration (FDA) for approval, but I honesty knew little of the process that takes place before the large-scale testing of drugs in Phase 3. I was selected to the Drug Industry Association (DIA) Patient Fellows program and attended its DIA 2016 convention in Philadelphia in late June, and learned much about what happens before the FDA receives a drug for approval. I had not given much thought to what Phase 1 for a clinical trial might look like — I just assumed the process involved those of us with a particular condition getting a drug and others passing through Phase 2 and Phase 3 as a treatment meets the necessary benchmarks of showing effectiveness.
Phase 1 trials don’t involve people with the particular disease, such as MS, being tested — these participants are healthy people with clean medical records and in excellent shape. The trials are designed to make sure the proposed drug is safe for people to take, and there are companies that specialize in doing nothing but assisting with Phase 1 trials. One such company is Celerion, and I had the chance to talk with David Misiak, Celerion’s senior director, Business Development, at DIA 2016.
The company has clinical test facilities in Arizona and Nebraska as well as in Belfast, Ireland, and Zurich, Switzerland. Celerion has been involved in clinical testing for over 40 years and has a total of 750 clinical beds available. People who do the Phase 1 trials and take drugs to prove they are safe or are subjected to other medical tests are compensated nicely for their time. Misiak says they are usually paid about $250 per day, but some trials may pay more, depending on the risks to the participants.
He said there is a network of people who do Phase 1 testing as a full-time job, earning somewhere between $40,000-$60,000 per year by participating as human test subjects in assorted Phase I trials. An online community for these people exists, he added, a place where they share test opportunities and discuss the pros and cons of how they are treated at particular test sites.
I went looking online for some of these communities, and found several commercial sites recruiting Phase 1 participants. Study Scavenger is one such enterprise, and its site has extensive listings for many different companies looking for subjects. It offer an app that allows people to put in their testing preferences and geographical information, and will connect them to the study leader if they have questions. Study Scavenger even offers to send email updates when new recruiting notices for trials that meet a person’s profile are listed. I also found patient-run sites, such as Guinea Pig Zero, that offers insights on being a Phase 1 trial participant. Finding subjects for, and participating, in Phase 1 clinical trials are big business opportunities.
While the prospect of the money sounds good, I have to point out that Phase 1 trials are risky because it is the first time a particular drug has been given to a human, having only been used on laboratory animals before then. I recognize that these people are mainly doing Phase 1 tests for the money, but I still appreciate their willingness to be engaged in the research process. Whether they are medical pioneers or just finding ways to earn an income, Phase 1 participants are crucial to the research process and I am glad they are engaged in this critical first step in proving the effectiveness of new drug therapies.
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Steve Johns
I strongly believe that the whole process needs re-evaluation and reform. I have PPMS and have been involved in a phaze3 trial of Gilenya/Fingolimod by Novartis. This drug was approved for RRMS 1n 2008 but PPMS was withheld from those trials so that it could be trialled later and if found effective used to claim another application and so extend their patent and the time that they can continue to charge $3000/month or $50/capsule for it for about 7 years. I see in MS News Today that MediciNova is doing the same thing now with Ibudilast.
Quote: "Next Steps For Ibudilast
MediciNova completed a Phase 2 multicenter, randomized, double-blind, and placebo-controlled clinical trial of MN-166 in 297 patients with relapsing MS in April 2008, demonstrating significant positive outcomes in three measures indicative of a potential disease-progression modifying effect — slower rates of disability due to nerve fiber damage, reduction in brain volume loss, and a reduced relative risk of new inflammatory lesions converting to persistent black holes, which are associated with relapses. The treatment was well-tolerated over the trial’s two years.
In 2013, MediciNova announced the initiation of a similar Phase 2b clinical trial in 250 patients with progressive forms of MS. This trial, NCT01982942, is funded by the U.S. National Institutes of Health (NIH), and expected to conclude in May 2017."
Another incidence of this corruption is in an information release from Genenteck about positive trial results for Ocrelizumab, a drug treatment for PPMS, again this has been used to treat other forms of the disease for some time but PPMS was kept out of previous trials to be used to extend the patent. The way the system works it will be something like 10 Years before it is available here.
I am supportive of research and trials, I have been involved in 3 to date, as I understand that this, and my illness, is a learning opportunity for all concerned and necessary for the development of treatments. I do understand though that there are aspects of the system that range from out dated and ridiculous to the sort of thing that would normally be associated with organized crime.
Laura Kolaczkowski
I hear your frustration about the lack of attention to the PPMS community (and SPMS as well). Fortunately this has changed and there are some great projects taking place to better understand these non-RRMS forms of MS.
I want to clarify some points you have made - Genentech has applied to the FDA for approval of Ocrevus, their brand name for ocrelizumab, for use in PPMS, and a response from the FDA is expected by late December. This is the first drug request for PPMS to make it this far through the various studies. Genentech has NOT yet filed for use of Ocrevus for RRMS. This drug is a variation of rituximab, which is currently used off-label by neurologists to treat some people with MS. Rituximab is used to treat rheumatoid arthritis. I am very excited to know there will finally be an approved treatment for PPMS.There are limits on the expectations of this drug but it is at least a start.
How drugs are developed and make the way through phase II and phase III trials and FDA approval is extremely complex and could use a good reform. My article here on Phase I talks about what happens before that point and is a process very few people are aware of happening.
Keep watching for the news on Ocrevus and I hope it will be a treatment option for you. -Laura