The U.S. needs a more constructive and inclusive approach to the challenge of affording high-quality medicines. In a column this summer, I argued for an agenda focused on reducing out-of-pocket costs to patients, elevating value as the preeminent criteria in pricing medicines, and increasing competition among medicines. I’ve been offering some ideas along these lines in recent columns, and in this final installment will look at how to boost competition by improving and accelerating drug development.
My premise on this one is simple. Our problem is not that we have too many effective treatments but rather that we have too few. More breakthrough treatments targeting similar diseases will mean greater competition, lower prices and a higher likelihood that there will be an effective answer for patients who seek better treatments or cures.
Where the development of new medicines is concerned, the rate-limiting factor is very often the complexity of clinical research: the approval, conduct, and analysis of trials that often involve hundreds or thousands of patients worldwide. There’s a lot that "we" – and by that I mean biopharmaceuticals companies, academic researchers, physicians, and government regulators – can do to pick up the pace, if we can work together. Broadly speaking, we need to make sure that the right kinds and the right numbers of patients are found for individual trials. Then we need to enroll those patients, get trials running, and act on the results as quickly as possible.
Towards the first goal, the much greater use of biological and statistical models to predict the course of clinical trials will be a big step forward in optimizing patient selection. Modeling is increasingly possible – thanks to advanced computing – and will become even more effective once we encourage the creation and use of larger data repositories. They must tap so-called "meta-data" on previous clinical-trial patients – which includes their health histories and other individual characteristics – as well as real-world data on the outcomes of existing treatments. We know a lot more than we think we know – if only we gather and make use of it.
New technologies also can help to speed up clinical-trial enrollment and conduct – alongside changes in attitudes. At my company, we’ve helped patient-advocacy groups establish user-friendly, online clinical-trial finders for people trying to understand and then take part in clinical research. Such tools need to be the norm. The use of social media in recruitment and electronic informed-consent documentation at enrollment also are no-brainers to improve efficiency, but still far too uncommon in practice.
There’s more. Drug companies sponsoring trials on diseases such as cancer – where genetic variations and other differences between patients play a large role – need to develop research protocols jointly whenever possible and share in the pool of patients willing to take part in clinical research. It’s a "win-win" approach: pushing trials more quickly to full enrollment while increasing the chance that the right patient finds the right trial in the right place. Companies also can share in the qualification of clinical sites and the training of investigators.
We also have increasing ability to speed up clinical trials and, even more importantly, to avoid dead ends. In particular, we can use what are known as "adaptive trials" – in which things such as dose levels and treatment combinations can be adjusted in mid-stream based on what’s being learned in small groups of patients – to push drug development much more quickly to the finish line rather than back to the drawing board. That’s revolutionary. The rise of modeling techniques, growing knowledge of genetic variation, improved understanding of tell-tale patient outcomes (often called "biomarkers") that show whether a treatment is working or not, and other new insights should allow regulators to green-light more and more trials that are adaptive in nature.
Patient-advocacy groups are showing the way – as they should – acting as the hubs of complex, adaptive trials linking multiple research institutions and companies. A recent and dramatic example is the "Precision Promise" trial announced by the Pancreatic Cancer Action Network, initially involving a dozen clinical trial consortium sites. Under one overarching trial design, it will study a range of treatment options for patients with pancreatic cancer – assigning them to the most appropriate option based on their molecular profiles and in some cases shifting them quickly to other options based on the results. Currently, only about four percent of pancreatic-cancer patients take part in clinical trials – a tragedy if there ever was one. Precision Promise almost certainly will improve that number while gleaning insights on treatments more quickly.
My "call to action" once again is not for a heavy political lift in Washington. The FDA appears to be encouraging much of what I’ve described in proposals submitted to Congress for renewal of the Prescription Drug User Fee Act (known as PDUFA VI). The not-for-profit TransCelerate consortium serves as an ideal vehicle for drug manufacturers to work together across company lines to accelerate R&D. Precision Promise and similar patient-advocacy efforts serve as further models.
So, for innovation-minded regulators and drug developers, the call to action is pretty straightforward. Let’s just get on with it!
John Lechleiter serves as chief executive officer of Indianapolis-based Eli Lilly and Co. This column originally appeared on the Forbes website.