The need to transform the clinical trial landscape

An Expert View from Rajan Jethwa, chief executive of Ellipses Pharma.

The road to success in developing cancer treatments can, as the oncology sector knows only too well, be long and strewn with obstacles – many of them unavoidable.

Yet can we all say, hand on heart, that – across the sector – absolutely everything is being done to streamline a process that should ultimately be to the benefit of patients? The answer, I fear, is “no.”

If the answer is “no”, then what is it that can be improved?

Since US President Richard Nixon gave his famous “war on cancer” speech in 1971, pump-priming the oncology discovery industry that we know today, the global oncology community has followed a familiar trail from novel discovery to drug production.

On average, it takes 10 or more years - from the point where a treatment is discovered - to reach patients who need it. In the world of cancer, where cases and mortality are constantly on the rise, this pace is just too slow.

A cursory glance at where the money is spent reveals so much. Globally, ~$83 billion is spent on funding research and discovery. At the other end of the journey, more than $100 billion is spent annually on the manufacture, marketing and distribution of new medicines. These eye-watering sums are a credit to those committed to developing new treatments. But what of the all-important stage of clinical development – the critical journey that every marketed medicine must make? Annual expenditure on clinical trials in oncology is currently running at only ~$11 billion per year – a hefty amount of money, but it pales in comparison with what is being spent at either end of the development process.

We all know the necessary hurdles that have to be overcome along the road to developing a new medicine that can be prescribed by doctors, manufactured efficiently and reimbursed by payers - safety, toxicology, efficacy, scaled manufacturing and regulatory to name a few. Overcoming these hurdles is part of ensuring that a drug is both effective, safe to use in its chosen indication, will be prescribed by doctors and affordable to payers. But by and large, the most significant rate limiting step in the drug development process is the clinical stage.

For many reasons the rate of failure of drug development is high, but the greater contributor to this rate of failure is the complexity, cost, and rigidity of the current clinical trial model.

Patient recruitment, particularly for rarer types of cancer, is a significant challenge. Add to that early-stage investors looking to exit and the loss of patent life due to delays created when investors exit, new investors enter plus time lost through fundraising and you have a combination of factors that come together to erode time through the clinical development stages.

On the flip side, we know the elements that drive success in trials – active patient stratification, using targeted medicines, biomarker and omics-driven decision-making and engaging a suitable range of KOLS in the provision and review of real-world evidence to shape a trial in real time. Combine this with deploying the right amount of capital to advance a drug at the start with a dedicated approach to support clinical trial sites and investigators directly - that have a tough job to do in an often resource-constrained environment - and it is possible to enhance success rates for clinical trials, recruit patients faster and ensure that more of the right patients are recruited into the right trials. This saves substantial amounts of time.

It struck me at this year’s JP Morgan Healthcare conference in San Francisco that amid all the discussions of myriad new discoveries and ample capital to follow them, there remains a lack of debate and innovation around how to improve the long-term success rate from oncology clinical trials.

Sponsors of clinical trials need to form better, more productive relationships with clinical research organizations (CROs) whose business model is often diametrically opposed to those of sponsors.

Aligning outcomes is critical to achieving efficiencies, and it is not all about money! Recruiting for a trial on time – instead of the usual delays that add years as well as cost – ensures that commercializing pharmaceutical businesses have longer periods of exclusivity to sell each approved drug.

The longer the period of exclusivity for an approved drug, the better value (and lower risk) it offers to commercializing pharmaceutical businesses.

Overall, improving the success rate of oncology clinical trials alongside the efficiency of running them will mean that capital can be deployed across more clinical trials, including the rarer indications. This in turn increases the number of options for patients in the future – something that is desperately needed across all cancer types and in all countries.

References:

https://www.mckinsey.com/industries/life-sciences/our-insights/pursuing-breakthroughs-in-cancer-drug-development

https://www.cbo.gov/publication/57126

https://arstechnica.com/science/2019/01/healthcare-industry-spends-30b-on-marketing-most-of-it-goes-to-doctors/

https://www.grandviewresearch.com/industry-analysis/oncology-clinical-trials-market#