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When President Trump was recently hospitalized for COVID-19, at least one element of the treatment he received raised eyebrows.
According to Trump’s doctor, the Commander-in-Chief received “a dose of an experimental antibody cocktail being developed by the drug maker Regeneron, in addition to several other drugs, including zinc, vitamin D and the generic version of the heartburn treatment Pepcid.”
The experimental Regeneron antibody cocktail in question was REGN-COV2, a SARS-CoV-2 virus multi-antibody therapy for the prevention of infection in people who have been exposed to COVID-19 patients.
Now, while Trump received the drug on or around October 2, REGN-COV2 is unapproved by the FDA and is currently being assessed in a Phase 1/2/3 trial. Regeneron published some initial data from the trial on September 29 claiming that REGN-COV2 “rapidly reduced viral load and associated symptoms in infected COVID-19 patients,” but the trial is ongoing.
How then was Trump able to gain access to an FDA-unapproved therapy, and what benefit is it to companies to have their drug candidates used in this way?
The answer to the first question is simple. According to the New York Times, Trump and Regeneron’s CEO, Leonard Schleifer, had known each other casually for years, due to the latter’s membership of the former’s Westchester County golf club.
Trump’s medical staff reached out to Regeneron for permission to use the drug, the company agreed, and that use was then cleared by the Food and Drug Administration. Furthermore, Regeneron’s spokesperson, Hala Mirza, stated the company had a limited amount of REGN-COV2 “…available for compassionate use requests that are approved under certain exceptional circumstances on a case-by-case basis.”
Typically, if there is a benefit to them, companies grant access to their experimental therapeutics under “compassionate use” guidelines only when all other options have failed, and a patient would otherwise die. That is the bar set by the FDA for approving such treatment. Drug companies are not required to make their drug candidates available in this manner, although one can easily see why Regeneron did so in this case.
What is interesting is that Trump’s life was, so far as we know, unlikely to have been in grave danger. CNN’s Dr. Sanjay Gupta put the odds of Trump surviving his COVID-19 infection, given his age, weight and pre-existing conditions, at 90-95%. As such, the downside risk to Regeneron providing him access to REGN-COV2 was small. That is not usually the case however and therein lies the rub for most biotechs when they consider using compassionate use schemes (sometimes called expanded access schemes, eINDs, or single-patient INDs – the terminology differs from country to country around the world). Compassionate use schemes can allow companies to tangibly demonstrate the efficacy and safety of their drug candidates in the real world, sometimes even when a clinical trial for a drug is not ongoing, but usually only in a population that is incredibly sick.
Thus, over a large number of patients, all undergoing compassionate use of a potential new drug, there are likely to be success stories intermixed with deaths, all of which can muddy the waters for biotech investors. Can we be sure the drug worked? Can we be sure it is safe? What did this compassionate use program actually prove?
That being said, where clinical benefit can be observed, successful compassionate use of development stage drugs can draw the interest of biotech investors. Note, however, that there is a declining marginal financing/credibility benefit from more and more patients receiving a drug under compassionate use guidelines. A handful may prove that a drug works or has benefits and is safe. But it is no substitute for a controlled clinical trial, even if compassionate use patient numbers approach that which might typically be seen in a Phase 1 trial.
The compassionate use of development stage drugs is also subject to the law of small numbers. To paraphrase Sir Richard Branson’s comments shortly after founding Virgin Atlantic, which he made on the airline’s maiden flight on June 22, 1984 from the UK to the U.S., “By the end of the day, we will either have the best safety record in the airline industry, or the worst.” The same fate can sometimes befall biotechs who place too much faith trying to cure small numbers of patients and finding out too late that these patients were too sick or otherwise untreatable for various reasons.
In conclusion, unless they have the chance to save a president’s life, or unless the clinical development path for a drug candidate is otherwise unclear, biotechs typically avoid the cost and potential downside associated with compassionate use schemes, instead focusing their very limited resources on their FDA-agreed clinical development programs as the best way to bring their therapeutic candidates to patients as quickly as possible. Generally, biotech investors prefer this conventional approach.
Have any questions about the compassionate use of development state drugs? Contact the Gilmartin team today.
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Laurence Watts, Managing Director
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