There is one certainty about the current regulatory process for drug approval in the United States and Europe: No one likes it.
Manufacturers are frustrated by the need for large, complex, and lengthy clinical-development programs that often hinge on meeting a single endpoint in one pivotal clinical trial. As a result, the cost to bring a drug to market has been estimated to be well over $1 billion — and it may be much higher. Patients and providers are disturbed by lack of timely access to medicines that show early promise in addressing significant unmet needs. Even regulators, who are responsible for enforcing the current structure, chafe at what manufacturers typically present to them: successful trial results in patients who are carefully selected to show the drug offers benefits but who are not very representative of the broader population likely to receive it. Payers then have a mess on their hands: pressure to pay for premium-priced medications that, when broadly employed, don’t offer much therapeutic benefit over existing alternatives.