As I psyche myself up for the first post DBS round of Botulinum toxin (using the term BOT to avoid any branding) treatment for my cervical dystonia (CD), I’m plagued with horror at this quest. BOT injections go like this – I premed with one dose of a steroid and hope, head to my doctors and jump into the injection chair. Two people snake wrestle the cords of the EMG and ultrasound and the third is the BOT executioner. My job is to not move and stay conscious. Imagine tight bands of muscle, never relaxed, pierced with thick needles. My palms get sweaty, the pain akin to being vampire stabbed with a wooden stake to the neck. In an hour, I leave with giant neck bubbles of BOT, wobbling out the door. It’s a race to make it home before my body poison-crumbles. My husband is on call, the superhero that he is, to collect me. He practically carries me up the stairs and tucks me in bed. I feel like a caterpillar entering its chrysalis and emerge after days of feeling like an elephant napped on me, while my bones are undergoing heavy construction.
The results of the BOT are far less dramatic. It whispers its presence and just when I think, only a bit more and it could be awesome, the effects fall off like a base jumper stepping off the cliff. The cost / benefit to me as a patient can be summed up as MEH. Its an option for my CD, which I am grateful for, but still MEH. In a very unscientific manner, I have sampled the BOT buffet on the market. They all receive MEH reviews.
Which brings me to my copy / paste drug rage. I keep track of CD research and options entering clinical trials and to my chagrin, I see another BOT in development. I can confirm, the thing I don’t need is more BOT. I am their poster child for a CD patient with unmet need. And there is no way I would waste the time and energy on a copy / paste drug. I am sure somewhere in the world, there are conversations wondering what magical intervention would help their copy / paste study recruitment. I can picture the talk of booster visits, investigator webinars, EMR queries, patient recognition. The simple fact is the drug will limp along development and if it makes it to market with CD on its label, will limp there too. The market access team will have to work magic for disappointing reimbursement and uptake, the why of the drug lost in the BOT buffet.
While many in clin ops would say it’s a recruitment issue, I argue it’s a value issue. No amount of risk mitigation can make a copy / paste drug more valuable to patients. I dream of funding to understand the biology behind CD. What’s making my brain want to look right all the time? What makes the tug of war with my neck end? If there was a drug that offered a new MoA, I would travel anywhere to be a part of that study. That’s patient value; that’s what breaks the mold of the 0.2 patients / site / month. That is what changes my life.