Lost in Translation

The Great and the Good gathered to celebrate the launch of our latest medicinal product. With enormous fanfare the project team were hauled onto the stage. The Conference Centre rang to the sound of rapturous applause. There was a holiday, ticker-tape parade atmosphere.

Jack turned to me as we applauded. Barely audible, he muttered in my ear, "Nineteen years. Nineteen! Not the finest example of 'Development Speed' is it?"

Needless to say, Jack made no mention of this during his speech. Instead he praised the team lavishly for dragging this one over the line. Their superhuman efforts in getting the product over the many regulatory hurdles to full marketing authorization approval had not gone unnoticed.

This stuck with me. Our drug had made it to market. Huzzah! But, in the euphoria that surrounds a product launch, it can be easy to forget the mistakes that got it there.

While companies might aspire to be 'learning organizations', many suffer instead from 'corporate amnesia'. They lose touch with the lessons that were hard-won along the way. They confabulate 'success' stories to maintain a corporate narrative.

As part of this process there is a tendency to lionize key individuals central to that narrative. In Western Culture we make much of the individual - originality and precedence are highly valued. Each scientific or technological breakthrough comes with an origin story. But any muppet can come up with an idea. Translating that idea into something real, making it happen. That's the key.

Any muppet can come up with an idea.

The goal of translational medicine is to bridge the gap between basic scientific research and clinical practice by converting laboratory discoveries into practical applications that improve human health. This interdisciplinary field focuses on moving research "from bench to bedside," ensuring that breakthroughs in areas like molecular biology, genetics, and drug development lead to real-world therapies, diagnostics, and treatments.

Key Goals of Translational Medicine:

• Accelerate the development of new treatments: Speed up the process of translating scientific discoveries into new drugs, therapies, medical devices, and diagnostic tools.
• Improve patient outcomes: Apply research findings to improve patient care by developing more effective treatments and interventions for diseases.
• Enhance collaboration between disciplines: Foster cooperation between scientists, clinicians, and healthcare professionals to ensure that innovations address real-world medical needs.
• Personalize medicine: Use insights from research to tailor treatments to individual patients based on their genetic makeup, environment, and lifestyle.
• Reduce the time and cost of bringing innovations to the clinic: Streamline the process of taking a discovery from the lab to clinical trials, ultimately improving the efficiency of healthcare innovation.

Translational medicine focuses on making sure that scientific discoveries have a direct and positive impact on patient care, closing the translational gap between research and practice.

The journey from the lab to the patient is hazardous and fraught with scientific and technical challenges. It's fascinating, challenging, interesting, engaging. It's also fun. That's why we do it. But it can be a bumpy ride 'from bench to bedside'.

Until recently, academic scientists and clinical researchers would massively underestimate the time and resources required to make this happen. Things have improved. Now, they just grossly underestimate the time and resources needed to make this happen. You might think that once you've discovered a new drug - biological entity, treatment, device, diagnostic tool, prognostic tool, other health intervention - your future is assured? It's just a question of time before the riches, accolades and awards accumulate? Perhaps not a Nobel Prize - at least, not every time - but what about the New Year's Honours List? Freedom of the City?

In reality, of the thousands of potential new treatments emerging from discovery and entering clinical development, just 10% will make it all the way to the market place. The majority fail. Lack of efficacy or a poor safety profile account for most failures. Technical challenges, such as a poor formulation, lack of stability, inadequate manufacturing controls, etc. account for the rest. Some get right to the end of this process only for the developers to learn there isn't a market for their product.

To the development scientist or engineer these challenges are fascinating in themselves and problem solving is what makes them tick. But most of us enter biopharma because we want to save lives, make people well again, or make their lives better.

Sadly, there is no guarantee that the project you are working on will be the one to save lives.

With a success rate of just 10%, the expected number of failures before the first success follows a geometric distribution with mean 1/p where p is the probability of success. For those working in early development, the expected number of failures is 10. If you're unlucky though, you may be assigned to rather more. In a career spanning forty years you might be associated with just one or two successful launches. And some people will go their whole working life without ever being assigned to a project that makes it to market.

Even those drugs that make it to full marketing authorization approval will not necessarily turn a profit. In the pharmaceutical industry less than half of new products will recover their R&D costs. The so-called blockbuster drugs - those generating more than $1bn per annum in sales - make up less than 20% of new drugs. Do the math. Those working in early clinical trials may never be around long enough to end up working on a commercially successful medicine.

Sometimes you get lucky. I worked on four drugs that made it to market. Two did not recover their R&D development costs. One broke even. Just one was a bona fide commercial success. Patented in 1982, it was approved by the FDA in 1997. By 2011 - the last year before the patent expired - it was one of the top-selling drugs worldwide with cumulative sales of $70bn. But mine was a bit part in a success story that was not of my making.

Still, it covered my salary - just about.

We cherish the wins.

Such is the roulette wheel of R&D.