Shots on Goal
Dr. Christopher McCabe
The ASSESS Project Chief Scientific Officer
‘The evidence generation system is broken….we need more shots on goal.’
Robert Calif’s recent comments on the state of pharmaceutical development hit the nail on the head – the current system is broken. And if I am honest, it would be great if the FDA put their hand up and acknowledged their partial culpability for this state of affairs. Regulators gradual abandonment of conventional evidence requirements because the science was so exciting, combined with the failure to hold companies accountable when technologies failed to deliver on their promise, has made drug development little more than an expensively regulated wild west. There are lots of expensive processes that have to be followed, but the products don’t have to be any good. Commissioner Calif’s diagnosis provides the opportunity to refocus drug development on the real goal – improving the health of patients and populations, and ensuring everyone has access to the treatments that they need.
So how do we get more shots on goal? One way, is just to take more shots and hope that sheer volume results in more of them hitting the goal. Another is to widen the goal – and to be honest that is not an unfair description of what the international drug regulators have been doing for much of the last two decades. The first strategy is likely not feasible given the cost of developing new drugs (whether it is $0.68 , $0.98 or $1.2 Billion), and the latter is not financially viable for health care funders delivering comprehensive population health care . Paying premium prices for incremental benefits is simply not feasible if we want to ensure that everyone has access to the treatments they need.
A third approach is to design the research and development process to ensure that by the time you take a shot at goal, you are highly confident that it will hit the target of delivering major improvements in individual and population health at prices that are viable for health systems and commercially attractive for investors. It’s time to be honest with investors and innovators that this is the real goal for biotherapeutic development, and then design the R&D process to maximise the chance that a drug that gets approved will be paid for. No one benefits without a paid invoice. It’s the pharmaceutical industry’s turn for disruption. The 20th Century model is not fit to deliver on 21st Century needs.
The ASSESS Project (AP) builds on 30 years of research and practice in health technology assessment around the world, to identify the key questions that university life sciences spin-outs and early stage SMEs should be asking at each stage of the R&D process, to maximise the probability that regulatory approval will convert into paid invoices.
As an eco-system, life sciences commercialization needs to define the goal correctly (hint “affecting the underlying disease process” is not enough), assess whether a technology has the potential to hit the value target which will translate into paid invoices, design a de-risked clinical development process that creates the evidence to support that claim, and stops investing in technologies when the evidence suggests the goal will be missed.
The AP way is less Louis Van Gaal’s long ball hit and hope, and more Pep Guardiola’s keep possession until you have scored. The solution is not shooting more, it is shooting more accurately. The AP way is prospective HTA, where you ask the market access questions at the beginning, not the end.
