The recently enacted federal Right to Try Law (RTT) is really a “Right to Ask” law. And, for the pharmaceutical companies, it’s a “Right to Say No.”
Which they certainly almost always will.
Case in point: BrainStorm Cell Therapeutics pre-emptively announced that NurOwn, its ALS stem cell treatment candidate, will not be made available under RTT. The company cited in a June release a lack of funding alternatives for patients unable to afford NurOwn on their own.
Other therapy developers may well follow suit for a host of reasons, such as:
- An unsupervised adverse effect, risking future FDA approval.
- Diversion of resources from the approval process.
- Inadequate assets to manufacture and distribute the requested therapy.
- Pharmaceutical companies cannot market any medication until it’s been FDA-approved, so their only compensation may be reimbursement of cost. They may be reticent to share the true manufacturing cost to avoid jeopardizing future market value.
- Investor concerns.
In response to that dynamic, what do we do?
- Hold our breath (a precarious choice, given ALS), hoping that an oxymoronic philanthropic pharma company will emerge?
- Leave the U.S.? Genervon, whose investigational medication was stuck in a regulatory quagmire, has distributed its formulation abroad. “Genervon can legally export the investigational drug GM604 to physicians treating ALS patients in 35 countries under various special access programs,” the company states on its website. And, it adds: “A broad range of ALS patients from around the world have been treated with GM604.”
- “Roll our own?” In the case of GM604, since it’s an endogenous human, neural-regulating signaling peptide, out of complete desperation, can we have this peptide synthesized and begin self-trialing on our own? Plenty of peptide synthesis labs are around. Universities with spare synthesis capacity offer services via Science Exchange, an online research market.
- Tumble the actuarial dice and wait out a full Phase 3 clinical trial, followed by a traditional new drug application approval — which would have a combined average duration of 3 1/2 to four years — betting that our survival will defy the disease’s cruel math?
I believe a surer, and far faster, path is potentially available.
In 1992, in response to HIV, the FDA instituted its Accelerated Approval Program (AAP) to allow for earlier approval of medications that treat serious conditions and that fill an unmet medical need. Clearly, ALS fits both criteria. Since 1992, the FDA has granted accelerated approval to more than 100 medications, according to the Center for Drug Evaluation and Research, so the action has precedent. The conditions that medications were indicated for include HIV, various forms of cancer, leukemia, tuberculosis, and Crohn’s disease. ALS ranks equal to, or above, any from that list in terms of dire prognosis, time-to-mortality, and a dearth of meaningful treatments.
With that as a backdrop, I suggest the following:
- Modify the FDA’s authorizing laws to allow any ALS therapy, having passed a Phase 2 clinical trial, to be immediately eligible for AAP.
- Require that the FDA proactively consult with entities developing ALS medications prior to the commencement of a Phase 2, thereby establishing an agreed-upon pathway for AAP.
- Jettison the hackneyed elements of the clinical trial process, bringing it into the 21st century.
- Adopt a methodology for faster, less expensive, and more accurate trials. Specifically, employ state-of-the-science predictive algorithms and data enrichment tools. This will reduce, or even eliminate, the need for a placebo group while reducing the number of participants required to achieve the FDA’s required measure of statistical significance.
- Make it clear to the FDA that terms like “expedited” and “accelerated” mean what they imply: speed. Add transparent, patient-relevant, disease-specific metrics to their charter, and performance reviews.
Anticipating your concern that systemic change may increase the risk for negative side effects or other unanticipated outcomes, may I ask what side effects you are most worried about? Partial paralysis? Choking? Respiratory distress? Inability to talk? Death? We already have all that. Are you fearful that maybe we’ll get worse? We get worse every day. As for unanticipated outcomes, having ALS is the epitome of the worst unanticipated outcome ever. We understand the risks well. In the grand scheme of things, the risk of doing nothing is devastating.
I realize that my analysis and suggestions may oversimplify a complex situation. That is where you, as lawmakers, come in. Purportedly, as evidenced by your elected office, you are among our country’s best and brightest. Plus, you have access to subject-matter experts. In short, you possess all the tools. PLEASE FIX THIS!
The tragic irony is that this plea comes at a time when ALS awareness has never been more pronounced. The sad reality is that, in the aftermath of an unprecedented display of hope and compassion, the current guardianship of that trust offers none for those struggling today, or soon to be struggling tomorrow.
To wait on any treatment of promise means certain death to thousands of people. To act means giving all of us a puncher’s chance.
Note: ALS News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of ALS News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to ALS.