“It’s very much a change. In areas of high unmet need, FDA seems to be committed to getting medicines to these people as fast as possible.”
–Alethia Young, biotechnology analyst, Cantor Fitzgerald
As the EU MDR full roll-out readies for enforcement in March and ISO 11607-2 new packaging requirements threaten to extend launch timelines, teams everywhere are trying to quantify the impact. Yet, just as we discuss “how much longer,” a story comes along that seems to upend our focus.
In this case, the story centers around the surprisingly fast pace at which the FDA is approving new drugs—touting the potential benefits for people living with rare or untreatable diseases. The first example is Global Blood Therapeutics Inc.’s Oxbryta sickle cell disease drug, which passed Go three months ahead of schedule in 2019. Informal calculations noted if a standard medicine takes 10 months for an FDA ruling, certain drugs like those in the high unmet need category showed five medicines approved “in as little as eight weeks.”
So, should we be happy or concerned? The story gives a nod to biotechnology breakthroughs and genetic science as yielding more precise data, for one. Yet, patient safety watchdogs are wary, concerned that speed can kill. Patients who might receive these therapies are dismissive, even elated. When you have no viable options, there is nothing to lose and potentially everything to gain.
Those of us who work with drug makers on a daily basis should remain committed to safety and compliance regardless of requirements that may lengthen the journey. We should follow the unfolding outcomes of medicines and devices that receive these fast-track approvals. The Bloomberg story explores several avenues of consideration for this high-speed trend. All worth pondering, even if it's too soon to know how the story ends.