Updates

I’m staying pretty occupied with work these days.  However, I’m feeling more physically tired than ever as I try to manage work along with life’s current emotional demands. I’d like to share some items that made me feel a glimmer of relief:

(1) Hydroxychloroquine and azithromycin as a treatment of COVID-19: results of an open-label non-randomized clinical trial – *Disclaimer: This paper doesn’t mean that HCQ + Azithromycin (an antibiotic) is a “treatment” for the virus. It’s just some preliminary findings based on a couple of patients that followed 3 varying course of treatments (including HCQ + azithromycin) in France. There is no available safety data in the context of large a clinical trial. As I explained in a recent post, the clinical trial process is a lengthy one to ensure patient safety (determining the correct dose, potential adverse effects, patient medical history, concomitant medications, etc.).

(2) Trump calls anti-malaria drug a ‘game-changer’ for coronavirus, but the FDA says it needs study (Thanks for responsible reporting, Washington Post).

This article packs a lot of information in it. I’d like to expand a bit on the “compassionate use or expanded access” portion.

What is this concept of “compassionate use”? Let’s look at the FDA guidelines. Per the FDA guidelines, expanded access is defined as follows: “Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.” {Guidance Source} For additional information, refer to 21CFR312 Subpart I (Expanded Access to Investigational Drugs for Treatment Use)

Traditionally, when the FDA approves or clears a drug it is for a particular indication (for a particular use). For example, in 2016 , I started working on a study that was looking at the potential disease modifying effects of a drug approved for use for the indication of hypertension (high-blood pressure) in subjects with Parkinson’s Disease. What does this mean? The drug that we were studying was approved by the FDA to help patients with high-blood pressure control their disease. It’s what we call an anti-hypertensive drug. Some animal studies, suggested that this same drug that was used to treat patients with hypertension could also have a disease modifying (therapies aimed to prevent, slow, or halt disease progression) effect in Parkinson’s Disease. Although we had collected safety and dosing data for this drug for in patients with high-blood pressure, we needed to collect data on dosing, safety, and efficacy on using the drug in Parkinson’s Disease patients. This is because hypertension and Parkinson’s Disease have patient populations with different health profiles.  The drug ended up being ineffective for Parkinson’s Disease patients. Gathering and analyzing this data took a long a time to do. This is how clinical trials are normally conducted. However, we’re currently in the midst of public health emergency. There are instances, as I described above that the FDA allows, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. Per the information in this Washington Post article, “the FDA has approved the use of remdesivir for about 250 seriously ill patients under an emergency provision of the agency’s “compassionate use” program.”

In addition to the remedesivir, there’s preliminary data that suggests that using chloroquine, an anti-malarial drug, could be helpful to patients with COVID-19.  However, in order for that to be confirmed, additional safety data needs to be collected for the FDA to call it a treatment or to consider it an indication for the drug.

(3) Some remedesivir papers: (A) First 12 patients with coronavirus disease 2019 (COVID-19) in the United States, (B) Remdesivir and chloroquine effectively inhibit the recently emerged novel coronavirus (2019-nCoV) in vitro

Now having shared all that, I’ll close this off by sharing an article I find fitting:

Don’t rush to deploy COVID-19 vaccines and drugs without sufficient safety guarantees

Make no mistake, it’s essential that we work as hard and fast as possible to develop drugs and vaccines that are widely available across the world. But it is important not to cut corners.

Hope you and all your loved ones are all staying safe and healthy.