A Clinical Trial – Patient Journey – Part I

Background

After discovering last November (2018) that my second DMARD (disease-modifying antirheumatic drug) had to be retired from my treatment regime, I did two things. I tried going medication free. I also started researching drug trials. If being medication free didn’t work, what was going to be my next step? What choices did I have that might be outside the standard treatment pathway?

Why research drug trials in particular? The first DMARD I was prescribed was developed in the 1930s. The second was introduced in 1955, originally to treat malaria but was later found to have immunomodulatory properties. These drugs are as old or older than I – surely modern medical science had something better by now? Something I wouldn’t have reactions to?

Well, yes, there are newer drugs available. Biologics are available on the PBS (Pharmaceutical Benefits Scheme) – IF the patient qualifies. That qualification bar can be high. As a example, this link to the PBS has the qualification requirements for one biologic that may be used to treat psoriatic arthritis. Click on the red “Authority Required” link to see the qualification requirements. When would I qualify? When I was 80? Not really a very positive thought for me. Yes, a private script can be given, but at $1,049.69 for four injection pens that is a little outside my price range long-term. And that is one of the cheaper ones. $40.30 with PBS subsidy. The schedule is usually inject once a week, so that is a monthly cost.

After much researching I found a drug trial that appealed to me. I don’t know who thinks up drug names, but this is risankizumab. Before I learnt how to say it, I referred to it as the “Kazakhstan drug“! I had researched the drug extensively, then looked for trials in Australia. The drug had already been approved in some jurisdictions to treat the skin condition psoriasis, so there was plenty of information available. On July 23, 2019 it was announced this drug received TGA (Therapeutic Goods Administration) approval for use in Australia, but is not yet on the PBS.  Published results from those clinical trials were very informative and positive.

The trial in which I am a participant is to assess the efficacy for psoriatic arthritis.

So I had found a drug trial I was interested in for a drug that looked very promising.

What Was I Looking For?

At this point you may be asking what was I looking for by considering doing a drug trial. As odd as this may sound, I was looking for treatment stability. I am now on my fourth DMARD. Each one has not been the right medication for me. As noted above, some of these were not the newest drugs around. Plus there was this encouraging line on the patient information sheet of one: “It is not clear how [drug X] works.” Or this one: “It is not clear how [drug Y] works in inflammatory conditions; however it is thought to have an action on the immune system.”

There is my personality type to take into consideration. I’m one of those annoying yellow quadrant HBDI people – we are the experimental thinkers. This might make me a radical patient, I suspect! It means I’m always looking “outside the box”.

I’d also had the experience of having a Synvisc injection when it was very new – that experience had been extremely positive for me. Trying new stuff is not something that scares me in the slightest.

I have faith in medical science. No, we don’t always get it right, thalidomide being the classic example as I have discussed before. I don’t seem to qualify for the biologics currently on the PBS, yet I was desperate to at least try a medication younger than myself. A medication where the therapeutic action was actually known.

I wanted something that worked without the myriad of side effects I’d experienced to date. I was also looking for a medication that might address the fatigue that goes hand-in-hand with many chronic conditions, including psoriatic arthritis.

A Third and Fourth DMARD

There was an event that drove me even more in the direction of being involved in a drug trial. In March this year, while I was still mulling over the prospect of being a lab rat, I suffered an adverse reaction to my third DMARD. I was prescribed a fourth DMARD. It seemed if there was a reaction to be had from these DMARDs, I was unfortunately a person who was going to react. By this stage I had lost faith in DMARDs as a treatment for me. I must stress, they work perfectly well for many, many people. I’m just not one of those people, it seems. One of the DMARDs had worked wonderfully for the arthritis, but ruined my skin, for example. There was something with each of them.

Again, I stress, all these were the standard treatment pathway. I wasn’t being “mistreated” in any way.

Next Steps

I discussed the proposal with my GP, my dermatologist, my urologist and my pharmacist. All four were very supportive. I have since also brought my gastroenterologist up to speed and he is also supportive. I must make clear I was not asking them from a rheumatological perspective of course, I was seeking any concerns they may have for me in their field of speciality and/or any concerns they may have about MABs in general.

I signed up and waited for the screening appointment. While waiting I bit the bullet and got a second opinion. This rheumatologist was also supportive of my desire to do the trial. Disclosure time. Through sheer coincidence this professor happens to be involved with the drug trial. My GP had written the referral well before I’d found the trial I was interested in. My GP had not even known I was researching drug trials, I had never mentioned it. The fact there was a connection was serendipitous.

Screening

My initial screening appointment was on my birthday! That was in June. So. Much. Blood! They do take a lot of tubes of blood, let me tell you. At least for this trial. I failed the first screening. Probably because I’d been taking prednisolone to try to control the inflammation while DMARD four ramped into gear, my CRP (C-reactive protein) had dropped 0.15 below the cut-off. A month earlier it had been way over. Luckily there was a sister trial with different patient qualification criteria and those criteria I did meet. That meant a second screening appointment. That took place in July. Of course, at this later screening my CRP was back up again, well over the cut-off for the first trial, but the rules are the rules. I am on the sister trial.

You are weighed, height measured, vitals taken, blood tested, joint and enthesitis assessments are done. The skin is assessed. There is paperwork, lots of paperwork. A medical history is taken (I took in a typed history from birth to now). A consultation with a site doctor. All up the screening process takes about two hours.

When I cite visit durations, these are for this particular trial. Other trials for other medications may be longer or shorter.

Active or Placebo?

This is a double blind clinical trial. Those wanting further information on clinical trial protocols in Australia can read this link at Australian Clinical Trials. Yes, there is a placebo arm. While the trial goes for four years, the placebo arm only goes for six months, after that all participants are on the active intervention. My rationale was six months of placebo could not be more difficult than the last six months I’d been through and I would only have to manage for six months if I lost the 50/50 lottery. I had nothing to lose.

I also knew that results from the trials for treating the skin condition showed very little placebo effect and that results had been seen quite quickly. I felt I would have a very good indication before the six months was up of whether I was on the placebo or the risankizumab, based on my skin.

Baseline

My Baseline appointment, the appointment where you actually receive the drug or the placebo, also took place in July. This was a much longer appointment. Essentially all the steps of the screening appointment are repeated, plus the injection, plus the patient has to wait for two hours post injection to ensure there is no adverse reaction. The coffee is good.

How is it going?

I was originally not going to write about this until after the six months. The fact I am writing earlier should be a slight hint. This will be a series as there is way too much information for one article! Tune in later in the week for Part II!