It’s okay to be skeptical

During pharmacy school there were several landmark research studies that greatly impacted the way healthcare was delivered. Studies about hormone replacement therapy (HRT), angiotensin converting enzyme inhibitors (ACEI) and statins. Don’t prescribe combination HRT. Let’s put ACEIs and statins in the drinking water because everyone will benefit. These were high quality randomized control clinical trials conducted with the utmost rigor and integrity. They helped shape the way we provided care.

Over the years new clinical trials have been conducted, guidelines have been updated and the way we practice medicine and pharmacy have been revised. I have lost count how many times the guidance around aspirin use has changed. We have come to expect this approach as we seek to advance healthcare. We rely on our education and training that has taught us as long as there is high quality, replicable evidence we should feel confident implementing the findings. After all, we are trusting the evidence in the guidance we provide to our patients.

An anecdote I want to share about when skepticism proved to be wise:

Recently a fourth-year student pharmacist was presenting on a new medication being considered for FDA approval. The student did a really nice job describing the process of Phase 1, Phase 2, and Phase 3 clinical trials and the data this drug has from each trial. As I reviewed the notes and questions I jotted down to ask after the presentation I notices that I had questioned the sample size of each trial. This medication is on the verge of being FDA approved in under 1 year start to finish and they have tested the safety in less than 300 people, majority of which are healthy, white and 18-60 years old?

Another stunning fact which raised the hairs on the back of my neck was that governments from several countries all over the world have already pre-ordered millions of doses each before the Phase 3 trial was complete. As a quick refresher, the main focus of a Phase 3 trial is to demonstrate and confirm the preliminary evidence from the previous trials that the drug is safe, beneficial and effective. The only evidence available prior to these purchases was that the drug was safe and projected to be effective at a particular dose.

The student went on to explain that as of the date of the presentation, no evidence from the completed Phase 3 trial had been released. And limited data from the phase 1 and 2 trials had been released. I have been around high-quality clinical research for long enough to know that when positive results occur, a manuscript can’t be written fast enough. Yet, limited data has been released and not so much as a press release touting the effect.

During the Q&A session, the student was inundated with questions about the quality of the 3 trials and their results. To his credit, he reflected on his drug literature evaluation training and suggested the trials and subsequent results may not be to the standards he has come to expect. When asked if he would recommend this medication be prescribed to a patient with the condition it is meant to treat, he responded by saying he didn’t believe there was sufficient evidence to recommend it at this time. He too was a bit skeptical on how this drug made it this close to FDA approval given the results.

I believe trusting broad claims can be naive as a healthcare professional:

Maybe it’s because I am getting older. Maybe I am getting wiser. Whatever the reason, I am more skeptical now than when I first graduated pharmacy school and I will not apologize for that. I no longer trust claims of “Always” or “Never”. We are all the compilation of our experiences. Value the importance of dissenting opinions and closely assess the claims you are presented. It is our responsibility as healthcare providers.