May 31st, 2011
Posted by Caroline Popper, M.D., M.P.H.
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.