A little less than 8 years ago, I started out wide-eyed and naïve struggling to master the top 200 drugs during pharmacy school. My challenges today are new drug approvals, those on the horizon in drug development, and the increasing use of off-label therapies. Pharmacy and medicine are continuously evolving disciplines where conflicting thoughts occur of reveling in the progress of the past to identify directions for the future, while also simultaneously being dissatisfied with the status quo to help advance treatment paradigms and outcomes. At times, even during the course of graduate studies, certain disease state algorithms have changed from when you learned it to the time your professors are teaching those a year behind you depending on the timing of drug discoveries, landmark trials, and/or new guideline releases.
My first experience of being impacted by a new category and drug approval was with ivacaftor for the treatment of cystic fibrosis. Fortunately, I was on a pulmonary/academic rotation at the time and the narrow yet specific niche of the drug made it easier to incorporate into my mental cystic fibrosis treatment template. In the hematology and oncology world, the rate of new drug approvals is even more exponential leading to constant shifting and restructuring of known treatment standards.
When a new drug is approved or a promising trial is published, each institution likely responds to it differently. There is a sense of anticipation from the drug information department, the practitioners in the disease state where the drug will be approved, and even the patients that stay abreast of drug developments and available clinical trials. The excitement for FDA approval and direct utility is almost palpable. This is especially true for off-label use where its mechanism may be promising in other malignancies or benign hematological disorders. Many are hoping that it will be the answer to their dried up armament for the patient that has exhausted all conventional lines of therapy.
As this is very much the foreseeable future, discussing this with colleagues through the ever-changing times is of utmost importance for each of us to stay abreast and learn from the data, physician practices, and maybe even gain insights from each other’s perspectives. Not much in my training has prepared me on how to methodically consider use of newly approved drugs off of Phase 1 data or for completely off-label indications, but there are many thoughts that arise that I think create good discussion points. I would love to hear thoughts that others more experienced in the profession may have:
-
What comfort level do we have as the pharmacist counseling on safety, efficacy, and taking on the financial burdens of these therapies that are yet not well studied?
-
What are the ethical considerations of giving therapy simply because the patient is willing to pay for a drug, where the benefit is likely theoretical and not strongly supported by evidence?
-
Is there concern for practitioners in private practice that don’t necessarily have an advisory board like P&T committees to regulate formularies? Is there an increased likelihood for them to provide therapies off-label if patients are willing to pay out of pocket?
-
At what cost of therapy-related complications should subjects be given the hope of benefit (theoretical or actual)?
-
Given our title as pharmacist, are we then to be heavily involved in drug acquisition and prior authorizations for insurance approvals with these new drugs/off-label uses? Is any barrier or lack of assistance from the pharmacist seen as a barrier to medical progress?