Some believe unmet need refers only to rare diseases and tiny patient populations. But it is more than this. In 2020, the term sits at the heart of decision-making for drug makers – along with cost, access and value, of course.

Over the past few years, frustrating instances of shortages and recalls – particularly generics – have been blamed on inferior quality control at countless suppliers of active pharmaceutical ingredients and finished products, especially those based in India and China.

In case you have not noticed, a new gold rush appears to be under way. Buoyed by surging interest in medical cannabis, big and small companies from around the world are racing to take advantage of the potential for the marijuana plant to cure numerous diseases and take its place in the pantheon of pharmaceutical products.

On a mild day in April 2016, a Marriott Hotel conference room in Hyattsville, Md., was the scene of one of the most politically charged panel meetings the US Food and Drug Administration held in years. A throng of parents and young boys in wheelchairs hoped to convince a committee to endorse the use of a controversial drug for Duchenne muscular dystrophy, a rare and fatal muscle-wasting disease.

On a mild day in April 2016, a Marriott Hotel conference room in Hyattsville, Md., was the scene of one of the most politically charged panel meetings the US Food and Drug Administration held in years. A throng of parents and young boys in wheelchairs hoped to convince a committee to endorse the use of a controversial drug for Duchenne muscular dystrophy, a rare and fatal muscle-wasting disease.

As controversy continues over pharmaceutical pricing, more drugmakers are eyeing deals that peg health plan cost to outcomes to boost volume and win formulary placement. But these deals are challenging to construct and to date there is little evidence that they reduce costs for patients.