Rethinking Pharma Part 1: How Precision Medicine is Changing the Game
Dr Andree Bates looks at the impact of precision medicine on pharma and the change in mindset necessary to fit this new reality.
I admit I have been critical of pharma leadership, and my frustrations are starting to show in articles such as No More Lip Service – Just Do It. But pharma is at a crossroads. And these companies can’t afford to continue with business as usual.
There are several interrelated issues that will either be their death knell or slingshot them to the head of the pack. This series will explore those issues.
The first is the very nature of the pharma business. For years pharma companies have chased after the next blockbuster to drive growth. Given the extraordinary time and cost associated with clinical trials, it’s easy to understand the mindset.
And when you combine that with a limited budget, on the surface, it makes sense to react by restructuring, cutting costs, desperately seeking pipeline via M&As or in-licensing, outsourcing where you can, focusing on big therapy areas, and trying to find ways to expand.
But these are not long-term solutions. The blockbuster model cannot sustain itself indefinitely. The industry is slowly moving to alternative models (although many old school CEOs are still clinging to the blockbuster model).
For instance, some companies have started to adopt a more diversified model, expanding their focus to encompass more rare diseases, generics, biosimilars, OTC, and vaccines. And there is a growing movement towards a value/outcomes model, where the focus is on value and health outcomes. In this model, the industry is not simply delivering drugs, but providing products and services planned for health improvement and engaging up close and personally with their stakeholders to meet their needs.
But the transition is proving painful for some. While the values/outcomes model makes sense, it looks at the issues a little too simplistically. There are radical changes taking place that are challenging everything we thought we knew about pharma.
We had it all wrong
Precision medicine is a game changer in the truest sense of the term. So much has been discovered recently in medicine that soon all the textbooks will have to be rewritten.
Nature Medicine describes precision medicine as referring to the idea that molecular information improves the precision with which patients are categorized and treated. With an accurate diagnosis of a patient at the molecular level, the treatment can be precisely matched to the underlying molecular pathway and relevant gene expression, and therefore will work as it is meant to. This in turn will provide strong value and outcomes for the patient, the payer and ultimately, the pharma company.
This approach is eye opening, as we are now discovering that we know so little about most conditions. For example, recent discoveries in oncology demonstrated a breast cancer and a bladder cancer as being identical. And interestingly, leukaemia was found to be in fact 51 separate independent diseases!
This shows that we have been on the wrong track for a long time in oncology, with some notable star exceptions that focus on the molecular pathways and gene expression—namely Herceptin, Iressa and Gleevec. It is all about the molecular pathways and gene expression, rather than the old-fashioned body part classification, such as CRC, BC, LC, etc.
Mara Aspinall further validated this theory at a 2008 conference. She asserted that if you presented with cancer to a hospital which organized departments by body part (gastrointestinal, breast, lung, etc.), then the chances of you responding to treatment are left to chance. If you want proper treatment you can trust, you would need to go to a hospital organized instead by molecular pathway, as the diagnosis otherwise will be inaccurate.
And precision medicine is not unique to oncology. We can now see many—if not all—conditions are about the molecular pathway and gene expression rather than the body part the condition is in. With Tysabri (natalizumab), Elan pharma uncovered that multiple sclerosis is actually one of several symptomatic expressions that result from an underlying disease pathway promoting excessive entry of white blood cells into the Central Nervous System. This leads to nerve inflammation and nerve cell damage. What is even more interesting, is they found that this underlying pathway is shared with Crohns disease (an inflammatory bowel disorder seemingly completely independent of MS). It’s hypothesized that this underlying pathway may also be responsible for ulcerative colitis and rheumatoid arthritis; things all considered quite different with very different treatments.
While I am on Rheumatoid Arthritis, Robert Plenge, VP of Merck Research Labs in Boston, stated in a blog in 2013:
“For a disease such as RA, many skilled physicians prefer the word ‘syndrome’ over ‘disease’, to emphasize this point exactly. RA is not one disease; rather, it is a collection of distinct but overlapping diseases which make up a crude category we currently call rheumatoid arthritis. Intuitively, most physicians believe that if we could understand these disease subsets, then we could treat patients more effectively by picking the right medications (most of the time, but not all of the time) and predicting the right outcomes (with greater precision).”
What does this mean for pharma?
What many in the industry don’t appear to realize yet, is that it soon won’t be just about the drugs alone, but about the successful linked pairing of a diagnostic and a drug. Companies that do this combination successfully will be the winners of the future.
Having the diagnostic not only got it approved and saved lives, but also got it done much faster (the typical 5-10 years was reduced to 2 years due to having the right patients in the trial), and the cost of the trials was also significantly reduced due to the precision of the diagnosis and applicable drug".
My poster child for precision medicine and diagnostic pairing is Herceptin. I love this drug for so many reasons. Almost everything I see about the future of pharma, I can point to something around Herceptin and things they did right (including using our analytics, but that is another story).
To accurately be treated with Herceptin, you have a diagnostic test, and if you have the HER 2 positive gene expression, then Herceptin is your drug. And it works reliably in these patients. However, if you have breast cancer without this gene expression, you would be wasting both time and money on Herceptin.
Essentially a diagnostic and a drug are paired like a lock and key. If Herceptin had done clinical trials without the diagnostic, it probably would not have got approval as not all the patients would have been HER2 +ve in which case the lives of the hundreds of thousands of people it saved would not have been possible. In fact, having the diagnostic not only got it approved and saved lives, but also got it done much faster (the typical 5-10 years was reduced to 2 years due to having the right patients in the trial), and the cost of the trials was also significantly reduced due to the precision of the diagnosis and applicable drug.
When you change your mindset to fit this new reality, you can see the massive impact on nearly every aspect of pharma.
Clinical trials: Currently many patients in clinical trials are not actually good candidates for that treatment, so it takes a long time to ensure you have enough good data to show safety, efficacy etc. Better targeting will change this.
Operations and organizational structure: The constant restructuring and outsourcing are products of the outdated blockbuster model. In the new era, pharma will be able to reduce costs, increase efficacy and reduce side effects while increasing value, outcome, profit and share - without giving up control of important aspects of their business.
Marketing: If you’re not hitting the right audience with the right message, you’re wasting your marketing dollars. Pharma companies that embrace change will have access to better analytics and information and services that goes beyond the pill and the only way to really get this right is using analytics.
I’ll elaborate more in upcoming articles. Stay tuned.
Precision creates value
Precision medicine is changing much of what we thought about several conditions, and along with it, the drugs used to treat people who have those conditions.
By pairing diagnostics with specific drugs, pharma companies are able to offer more value to all parties. From patient treatment and hospital costs, to clinical trials to the reduction of lawsuits, the potential savings to the system is staggering.
Consider Herceptin. The drug costs $79,181 per patient when a diagnostic not used. But when it is used only with patients who will respond, the cost drops to $53,738—a difference of $25,443.
And think of all those drugs that failed to get approval after clinical trials did not show efficacy in enough patients. If you could find the gene expression and molecular pathway of the subgroup of patients that responded, those drugs could possibly to be resurrected and, in fact, save many lives.
This approach of testing for precision with a diagnostic adds more cost initially - which is probably why it is not already being embraced widely. But that’s short-term thinking. Pharma companies should be considering how they can better pair a diagnostic with a drug if they want to succeed in the long term.
If you have any questions, please contact the author, Dr Andree Bates at Eularis http://www.eularis.com
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