|19th July 2016
Normally in our blogs and articles at Blue Latitude Health we offer up snapshots of things we have learned from our experience working in a therapy area. I would love to be talking about our latest thinking around launching new therapies in Alzheimer’s disease (AD) but I can’t – no one can. This is because in the last 13 years, only one new drug has been approved for patients with AD. The most recently approved drug was in 2014, bringing the total number of agents approved across AD to 5.
In this article, I’m going to explore the success rate of trials in AD and the reasons for this incredulous situation, as well as look at the some of the glimmers of light at the end of the tunnel.
99.6% of all trials in Alzheimer’s disease (AD) between 2002 and 2012 failed. This is one of the highest clinical trial failure rates in healthcare. During this period, 413 trials were performed using 95 compounds with 8 unique modes of action (MOAs). 35.1% of these studies were with disease modifying agents, 36.6% were symptomatic agents aimed at improving cognition, while 18% were disease modifying immunotherapies. Failures in these trials have been associated with lack of efficacy, excessive side effects, or challenges in trial execution; leaving patients and HCPs with 5 therapies to choose from:
These therapies have shown significant, but clinically marginal, benefits in relation to cognition, daily function, and behaviour. Patients on these therapies remain stable for approximately a year or more, with a slower subsequent rate of decline to untreated patients.
So what is the problem? Has there been sufficient investment from industry? Why haven’t we optimised the activity of proven active agents for better results? Why haven’t we identified more potent targets in the disease pathology with all the recent developments in genetics?
They have - 78% of all trials in AD from 2002 to 2012 were funded by the pharmaceutical industry. If we consider that the mean duration of a phase 3 trial in AD is 90.9 weeks, with an average of 833 patients, and a 0.04% likelihood of success - the cost of AD trials and the amount of investment that has been made with little to no return is shocking. When you consider the success rate of a new oncology therapy is 19%, any company continuing to invest in this area deserves credit because the unmet need in AD is hard to fathom. The number of deaths in AD per year is on par with cancer, and the annual cost of care actually exceeds that of cancer - which is surprising considering there is 10x more research funding in cancer than AD. This is an area in dire need of innovation, and with significant reward for whoever can make that breakthrough given the market valuation at $6.5billion.
|27th August 2020
Precision and personalised medicines are more than products, they are services in their own right. So, how should pharma approach this uncharted territory to ensure targeted therapies work for patients?