Why Clinical Trials are Akin to "Trying to Build a Tesla in a Model T Ford Factory" for Emerging Industries Such as the Microbiome Health Sciences!

I was asked this question by a friend and colleague who attended a global microbiome conference In California, because of several businesses in that selfsame space with whom I’ve been working. Those entities process biomass via fermentation and other technologies to create new bio actives and compounds for the customers of the burgeoning Microbiome Health Science (MHS) Industry- whether diagnostics, ingredients, functional foods, supplements. biotics, therapy delivery, or therapeutics.

THE STORY SO FAR - Microbiome research presents a multitude?of opportunities and challenges. It is an extremely?exciting field with multiple successes.

To?increase that number of potentially successful clinical?development projects in the microbiome, it is important to recognize standard drug development principles and global drug or biologic development?requirements. Understanding what normal flora is, what the?confounders are, and what are clinically meaningful endpoints is an important step in developing a successful clinical development program for products affecting the microbiome. Additionally, a full evaluation of manufacturing processes and materials are critical in realizing the potential for microbiome platforms.

Put simply clinical trials are essential for advancing MHS, improving patient/consumer care and finding better ways to prevent, diagnose, and treat health conditions – and they rely on collaboration of researchers, participants and ethical oversight to drive progress for our health globally.

It’s a stamp of approval to say the product is safe and efficacious and has passed strict ethical standards to ensure patient/consumer safety and conducted appropriately – it does what it says on the container, pack, tube or bottle.

Yet even in the Pharma industry that helped mold and create these processes over time, and who have been running trials for decades and have vast experience - there is still a lot of risk throughout each phase, and significantly cost, 80% of phase 2 trials fail – at stage 3 there is still a 30+ % failure rate.

Given these parameters the door is ajar for others to shake this up. ?

ENTER STAGE LEFT COVID.

One of the positive impacts of covid was to shake the whole clinical trials process up in terms of planning, developing and executing at accelerated rates, but equally it has created room for new disruptors who recognize the shortfalls of the former and see opportunity as to how it could be improved in the present, due to advances in AI, data analytics and common sense - speeding that up, bringing greater efficiency to the area and driving down cost.

CONSIDER THIS

Unlike other therapeutic areas, MHS isn’t dominated by Big Pharma and Big budgets where current estimates for research and development of new medicines range from$40m to $3.9bn, the average over the last 10 years being $2.3B. Instead, we see innovative SMEs dominating with more modest means, all focused on driving the development of a single or a small pipeline of bio-therapeutics.

This has resulted in an impressive range of innovative products in development, often targeting conditions for which conventional “small molecule” drug discovery projects have failed to adequately address.

PHARMA AWAKES

In 2018 we saw the first acquisition of a microbiome company by Big Pharma, with the purchase of Rebiotix by Ferring. Most Big Pharma are more tentatively venturing into the microbiome field via collaborations (e.g. MSD and 4D Pharma, Takeda, and Finch Therapeutics). and significantly as Ferring and others have success at stage 3 with more to follow given the exponential increase in trials in the space and patents filed it behooves us to evolve our thinking and processes of how we do trials more efficiently and drive down costs.

POIGNANTLY

A recent study by Médecins Sans Frontières (MSF) – indicated that Doctors who have for the first time released details of their spending on a major clinical trial, demonstrated that the true cost of developing a medicine may be far less than the billions of dollars claimed by the pharma industry. The extortionate cost of trials is oft used to justify high prices of new medicines, but companies do not publish either the topline or a breakdown of their spending. This opacity should end says MSF and I think most people would concur.?

FOR THE NEW INVESTOR

The MHS industry, people will tend to be more cautionary. So, it pays to look beyond some of the long-established CROs and look for those that bring a new twist to the area and can peel back the layers and mystique that surrounds trials. I personally like the approach of Citrus Labs and what it’s doing for supplements,?skincare,?biotech,?medical devices, and even?pet health.

FOR AN EVOLVING INDUSTRY

The more successes at stage 3 and trials full stop, the more money it will attract to each phase of the process.

Put simply - risk and cost with an increase in supporting data around efficacy, safety and commercial viability/positive outcomes are key factors – so it pays to have people who have been through this process working with new disruptive teams. AI and data analytics will improve hit rates in the future.

The point is a nascent industry must fight to establish these new processes that more closely reflect our time as opposed to those created for another age, but need to be an improvement on what has gone before less costly and achieve better outcomes/hit rates.

It seems to come down to culture, and the desire to manage risk out by many , and querying the very science that smaller companies are pioneering , so it pays to have your strategy right and to find the right partners to work with to find a path through, what can be a clinical and regulatory minefield, while ensuring that you make it affordable, robust, and remove inefficiency for your customers and improve outcomes – after all its to everyone’s benefit that that we push the boundaries to improve health! ??????