What’s Next After Your Ship Comes In?
“What do we do now?”
That line from the 1972 movie “The Candidate” came to mind recently. The movie stars Robert Redford as a political neophyte. Redford’s character, Bill McKay, is offered as a sacrificial lamb against an incumbent thought to be unbeatable in a fictitious senatorial race. In the final scene, McKay poses the aforementioned question to his campaign manager after pulling off a stunning upset on election night.
What prompted that recollection was the positive press regarding AMX0035, a novel combination of compounds developed by Amylyx Pharmaceuticals. In a phase 2/3 study, the formulation was shown to have “significant benefit compared to placebo” in delaying ALS progression for the patients enrolled.
The results were so encouraging that the ALS Association initiated, and is still soliciting sponsorship for, a petition calling for the approval process of AMX0035 to be urgently accelerated.
That hubbub prompted my mind to fast-forward to the eventual approval of a breakthrough ALS therapy. Clearance from the U.S. Food and Drug Administration (FDA), particularly in light of the dismal success history to date, may be characterized as an against-all-odds triumph.
The question then becomes: In order to introduce the substance to every member of the ALS population as soon as possible, what happens next?
Although it may seem a no-brainer, the therapy has to be manufactured. The quantities consumed during clinical studies are relatively small and, compared to market penetration numbers, much easier to control.
The FDA has published a guidance titled Scale-Up and Post-Approval Changes. Increasing the batch size from pilot scale to small or large production batches after the approval of a new drug application requires submission of additional information.
Having run this gauntlet, established pharmaceutical companies are well-positioned to expeditiously manage the process. However, much of the research and development for ALS therapies is being driven by start-up, or near-infant, entities. They will likely need to contract out manufacturing, at least initially.
That is why I research the company as well as their promising development. For example, Amylyx has a global head of supply chain. I presume that office has a production strategy teed up.
Parallel with the plan to feed the demand, the acquisition mechanism for the patient must be anticipated. For the vast majority of us, that means prescription medication insurance coverage. Further, the medication must be on our insurer’s formulary.
Medications on a formulary are chosen by a panel of experts, independent of the insurance company. These panels include doctors, nurses, pharmacists, and other clinical experts. The group meets regularly to discuss new therapies, safety data, FDA-approved prescribing information, clinical trial results, and doctors’ recommendations to keep the formulary up to date.
Like manufacturing ramp-ups, it sounds rather turnkey on the surface. Except when it isn’t. Some payer coverage decisions come with qualifications, such as a requirement that patients meet specific criteria. In the case of ALS, that has meant excluding coverage based on time-since-disease-onset or symptom progression.
When Radicava (edaravone) came to market, some insurers imposed restrictions modeled after the clinical study population. Similar to nearly every ALS medication trial, no mid- to long-term survivors or rapid progressors were studied.
If, and when, that were to happen, the insurance company can be contacted through a doctor to request a formulary exception. Your plan will review the request to see if they’ll cover it. If the insurer does not agree to an exception, an appeal may be made. For those in life-or-death situations, the review must be conducted immediately.
Not to be lost in the “devil’s in the details” hassle of scale-up and insurability is the bestowal of a suitable name. On Pfizer’s website, an explanation of the medication-naming method may be found. The names are intended to tie into the therapy in some way.
“They can range from abstract ideas, tonality, strong sound or gentle sound. It can be imagery. You hear a word and it brings something positive to mind, or a nice association that isn’t a claim,” said Michael Quinlan, senior manager, trademark development, within customer analytics and insights at Pfizer.
The name Lyrica, given to a medication used to treat nerve and muscle pain, was selected because it evokes calming lyrics or music.
In addition to the prohibition against making an overt claim about what the medication does, the name can’t be promotional. For any given formulation, a list of over 200 candidate proprietary names may be forthcoming.
With that in mind, following are some of my ideas on a name for an ALS game-changer:
We already know “that too many people have died.” Now, we need to make sure that, when the time comes, we don’t offer the Bob Dylan reply to the Bill McKay question. Our answer must not be “blowin’ in the wind.”
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