Marrying Targeted Therapies with Companion Diagnostics: The Novartis MDx Model

May 31st, 2011
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In late 2008, Novartis established its molecular diagnostics unit, to be known as Novartis Molecular Diagnostics (MDx) and to function as an integrated unit within the Novartis Pharmaceuticals Division.  Led by Michael J. Nohaile, Ph.D., Global Head of MDx, the unit is designed to leverage the global pharmaceutical company’s strengths and capabilities in research, development and commercialization to translate identified biomarkers into high-quality diagnostic tests. As Novartis explains via its global website: Novartis MDx strives to become a world leader in developing and commercializing diagnostic tests to optimize patient outcomes and to transform the practice of medicine.
 
Recently, I spoke to Novartis MDx Global Head of Diagnostics Development Michael C. Little, Ph.D., an old colleague of mine from Becton Dickinson and Company, and to Director of Business Development and Licensing Yves Dubaquie, Ph.D., who presented on behalf of Novartis MDx at the Personalized Medicines Partnerships Conference last month. Both gentleman arrived in their current Novartis MDx roles about two years ago and both have impressive industry experience.
 

While many of the leading international pharmaceutical companies have embarked upon research and business strategies to bring companion diagnostics to market alongside targeted drug therapies, Novartis has created a unit dedicated to the development of molecular diagnostics within its pharma division. Below, Michael and Yves share some of their insights into this unique model and its promise.
 
CP: Explain the Novartis MDx model and how it differs from other big pharma approaches?
 

ML: To date, big pharma has taken three general approaches to the development of personalized medicines alongside companion diagnostics. First, companies like Abbott and Roche have set up internal diagnostics R&D (based on legacy diagnostics businesses), but in separate business units from their pharmaceutical research. Separate diagnostics and pharma businesses must compete in their legacy businesses, which may reduce their opportunity for collaboration and integration.

 

In the second approach, some large pharma companies have created arm’s length arrangements with diagnostic companies (e.g., Amgen and DxS). This model may be convenient for each entity, but arm’s-length relationships prevent true integration and may inhibit upstream opportunities for the two companies to work together.

 

Third, there’s our model. Novartis began with a fresh piece of paper. The company placed the molecular diagnostics unit within our pharmaceutical division to do what is best for the business. We work to integrate the efforts of our Biomarker Development unit in the Novartis Institutes for Biomedical Research with our clinical drug development while ensuring regulatory alignment on both the diagnostic and drug development sides of the equation. Our process is cohesive and internally integrated. This is an innovative approach, and many in the industry seem to be watching us closely.

 
CP: During his presentation at the Personalized Medicines Partnerships Conference, Yves spoke about companion diagnostics becoming a NEED to have, rather than just a NICE to have. Please explain.
 

YD: In the future, more payors and regulators are likely to require companion diagnostics to accompany some new targeted therapies. At Novartis MDx, we recognize this fact and that we need to develop more precise medicines to improve patient outcomes. Once we have proven that a targeted drug works for a select patient subset, there’s no stepping back when FDA requires concurrent filing of a companion test. We will aim to move the therapy and the diagnostic down the regulatory path together so that the companion diagnostic doesn’t hold the drug hostage, which could cause delays in drug approval that could be costly.

 
CP: Is there a disease area that you are most optimistic about in terms of where your company can make a difference in healthcare?
 

YD: In the near term, Novartis is a big player in oncology and this is one therapeutic area where there is a big push to create targeted therapies because of the potential to improve outcomes for sub-groups of patients facing life-threatening diseases. About 50 percent of our MDx portfolio is in oncology. But, MDx supports all our General Medicines efforts and infectious disease and transplant are two particularly important areas for targeted therapies. In the long term, we may have collaborations with our newer Novartis divisions, such as through the company’s acquisition of Alcon, a global leader in eye care.

 
CP: What are some of the biggest challenges to bringing the promise of personalized medicine to the next level?
 

ML: First, I’ll address the U.S. regulatory process and the product development processes. It’s complicated to bring a diagnostic to market. It’s complicated to bring a drug to market. You need to manage both pieces effectively and in an integrated fashion (our model recognizes this, hence our structure) if you aim to co-file the diagnostic and the drug together for a targeted therapy review process. The stakes are high to do it well.

 

Another challenge is intellectual property (IP). If the U.S. higher courts uphold a recent decision that says that certain biomarkers are no longer “patentable,” then this will change the way companies think about IP. There will need to be a new model for funding innovation if you can’t protect biomarkers.

 

I’d add that the identification of potential biomarkers is only the beginning of the process. Once research with the biomarker can be reproduced, formatting and creating FDA-acceptable tests is a lot of hard work. For example, you need to develop analytically validated tests before you assess the biomarker performance. This is to ensure that the apparent change in the biomarker in the clinical realm is not change that is due to your measurement system or assay. You also need enough clinical samples for the development work, but these samples don’t always exist in abundance. The hurdles that exist – plus the work that needs to be done to surmount them – make it challenging to “deliver” a real biomarker baby.

 
CP: What keeps you optimistic and focused?
 

YD: For me, it’s the prospect of breaking new ground. There aren’t many successful examples in the marketplace today of companion diagnostics and targeted therapies. We are at the beginning of a new revolution in the drug industry, and I’m excited about the prospect of delivering improvements in patient outcomes.

 

ML: I think about two things: past successes and living into the future.  As far as the past: Ten years ago CML, a rare form of leukemia, was a fatal diagnosis. With medical developments, including by Novartis, we have been able to drastically improve the overall survival rate. Today, we are improving upon the tests that manage these CML patients. It is a very powerful success story. Looking forward, the people in Novartis Pharma and MDx are ultra-dedicated to patients. They know we are making a difference for real patients with real names.

 
As a life science professional who has been involved in the diagnostics industry for many years, I’m closely watching the model of Novartis MDx and the other approaches our industry is taking to develop companion diagnostics. Are you a fan of one development model versus another? Where will the next drug/diagnostic companion pairings make the biggest impact in healthcare? Please share your thoughts and experiences with us here.


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About the Author:

I founded Popper and Company with Ken Walz more than ten years ago to address inefficiencies in health care by helping life science companies develop and commercialize new technology. Today, the members of our growing team leverage their extensive knowledge of the tools and trends shaping all aspects of health care and its participants. Send me an email.


4 Responses to “Marrying Targeted Therapies with Companion Diagnostics: The Novartis MDx Model”

  1. Paul Sonnier Paul Sonnier says:

    Hi Caroline,

    Great interview!

    A quick question: The R&D marriage between companion diagnostics with targeted drug therapies is fascinating, but I’m curious if there are any examples of adding a third element to the equation insofar as also integrating innovative new non-molecular diagnostics technologies into the fray (e.g. biological dynamics-based diagnostics)? Or, perhaps this is outside the core competency of pharma companies, hence not really applicable at this time or in this burgeoning new model?

    Thanks,
    Paul

    Paul Sonnier
    VP, Partner Development, Wireless-Life Sciences Alliance http://www.wirelesslifesciences.org/
    Founder, 6,000+ Member Wireless Health group on LinkedIn
    http://www.linkedin.com/groups?gid=2181454

  2. Ken Walz Ken says:

    Thanks Paul. Yes, once treatment is initiated it is important to be able to monitor disease progression in order to determine if the drug is working . This is particularly important in cases where it might not generally be possible to accurately measure anything (such as a reduction in tumor mass) for perhaps several months. For many types of cancer it is difficult to get an early read on disease progression because of the difficulties associated with measuring tumor mass or other morphological changes. CD4 counts and viral load are good measures of disease progression in HIV patients. Molecular markers can be more precise and they may start to change (or not…) long before gross morphological changes become apparent. In many cases blood pressure, glucose, weight and other simple parameters are important to track in patients undergoing therapy and many such “markers” can now be monitored and recorded using handheld and portable devices. While this may be, as you suggest, outside the core competency of pharma companies, it represents a significant opportunity for wireless health companies.

  3. Ken,

    Thanks again for your response and I really enjoyed our phone conversation. Here’s another angle: Pharma producing mobile/wireless companion diagnostic devices. An example: “Medical Diagnostics via Cell Phone – Cell Phone Counts Cells” http://pubs.acs.org/cen/news/89/i31/8931scene2.html. I had the pleasure to see this project presented by Aydogan Ozcan during the UCSD von Liebig Center’s TATRC/Qualcomm Wireless Health Innovation Challenge, held earlier this year here in La Jolla.
    Again, medical device development is a bit of a stretch for pharma, but this is thinking outside the box – also, this is where strategic partnering can be of value. Of note is that this is a core focus of the Wireless-Life Sciences Alliance. (Note that I shared the above news in the now 6,500+ Wireless Health group, here: http://lnkd.in/gj-jnS)

    Lastly, as we discussed, here’s a reference for my first response to your blog post: Early Cancer Diagnosis Startup Wins Entrepreneur Challenge
    http://www.jacobsschool.ucsd.edu/news/news_releases/release.sfe?id=853
    http://www.xconomy.com/san-diego/2009/06/02/early-cancer-diagnosis-startup-wins-entrepreneur-challenge/
    Company website: http://www.biologicaldynamics.com/home/Welcome.html

    Best,
    Paul

  4. Ken Walz ken says:

    Thanks Paul.

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