I informed him of the vast price difference and asked which he would prefer. This veteran did not hesitate for a second: “I will take the cheap one, doc…. I am not the only one in this foxhole.” This sentence broke my heart, and, to this day, its recall gives me goose flesh and unavoidable flowing tears. This intense and unforgettable experience, although long ago and far away, remains highly relevant to today’s cancer medicine.
Read the full article at: https://ascopost.com/issues/september-10-2023/perspectives-on-cancer-therapy-development The Food and Drug Administration (FDA) approves about two thirds of new cancer drugs on the basis of clinical trials that use surrogate end points, such as laboratory values or radiographic findings, rather than clinical end points that assess survival or how patients feel or function. The accelerated approval program allows drugs designed to treat serious conditions for which there is an unmet medical need to be approved on the basis of changes in surrogate measures that are only reasonably expected to predict clinical outcomes. Because in certain fields of medicine, such as cancer, a drug’s effects on surrogate measures such as tumor size (see table) are often more pronounced and occur more rapidly than effects on a patient’s clinical status, trials focused on surrogate measures can enroll fewer patients and can be completed more quickly than trials with clinical end points, thereby enabling products to reach the market earlier. Since clinical end points such as survival are generally what matter to patients, however, the FDA requires that the clinical benefits of drugs granted accelerated approval be confirmed in subsequent trials.
Read the full article at: https://www.nejm.org/doi/full/10.1056/NEJMp2306872/
It’s time to abandon the term “specialty drugs,” argues Alex Jung, founder of Alex Jung Consulting at a forum sponsored by the Midwest Business Group on Health. When 53% of drug spend is considered specialty and new drugs launch at high prices, specialty drugs have become the norm. “When it’s more than half of spend, is it really special,” she asks. I don’t think specialty pharmacy exists anymore.”
The term specialty drugs, she pointed out, was coined 15 years to isolate that 2% to 3% of spend. Now most of the newer drugs are not oral solids. They are injectables or infusions and require special handling, storage and administration.
NOTE: Alex Jung is a VIVIO Advisory Board Member and Former Partner–Strategy/M&A at Ernst & Young.
Read the full article at: https://www.formularywatch.com/view/alex-jung-it-s-time-to-think-differently-about-specialty-drugs