LAWS & REGULATIONS: Genomic BiomarkersA genomic biomarker is any gene-related information used as an indicator of a particular disease state. Over the next decade, regulations regarding genomic biomarkers promise to change the way in which pharmaceutical companies determine the economic viability of their drug discovery process. This is especially true in view of the recently proposed regulations put forth by regulatory agencies. In the European Union

The European Medicines Agency (EMEA) introduced the “Note for Guidance on Genomic Biomarkers Related to Drug Response: Context, Structure and Format of Qualification Submissions,” in June of 2009. The EMEA guideline seeks to implement a uniform structure for the qualification of genomic biomarkers and to foster consistency of applications across regions and promote joint discussions within the European regulatory authorities. The Biotechnology Industry Organization, (BIO) requested that the guideline be “fully aligned and complementary with the IVDMIA (In Vitro Diagnostic Multivariate Index Assays) guidance of FDA.” Final publication of the guide is expected in 2010.

In the United States In April 2005, the US Food and Drug Administration (FDA) issued its first white paper on Rx/Dx co-development: “Drug-Diagnostic Co-Development Concept Paper.” The document was intended to elicit feedback, from which the FDA would draft guidelines. Development of the guidelines has been slow. In an effort to restart development, a multi-center, multi-disciplinary working group was formed by the FDA in December 2008.

To find out what The MarkeTech Group consultants can do for you, visit our Web site at www.themarketechgroup.comUSA: +1 (530) 792-8400, EU: +33 (0)2 41 88 41 44

CASE STUDY:Personalized Cancer Treatment

The Client: A cancer treatment center team of scientists seeking to understand the value of an innovative predictive test. The research done by the cancer treatment team led to the development of an in-vitro test which would assess a patient’s sensitivity to fluoropyrimidines and facilitate a personalized approach to patient treatment.

The Challenge: To provide the team with insights about the future of companion diagnostic to fluoropyrimidine treatments and how the project could fit into that future. The MarkeTech Group also helped the company formulate an operational and tactical position to effectuate a quick and effective launch of the final product.

Our Solution: The MarkeTech Group, (TMTG) conducted a series of in-depth interviews with oncologists, hospitals administrative stakeholders, and pharmaceutical industry chemotherapy product managers in France, Germany, and the USA. The interviews were intended to define needs and expectations in terms of prediction testing for fluoropyrimidine sensitivity. Extensive market data was also collected allowing precise quantification of the potential market.

The Impact: TMTG’s client received detailed information enabling complete and relevant filing of the start up business plan, including market quantification and drivers and barriers to adoption of the prediction test. TMTG also evaluated the envisioned business models and recommended the most relevant development path to take, including the best actionable short term sales tactics.

Winter 2010VOLUME 10, ISSUE 1

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The Emerging Role of Genomic Therapy in Clinical Medicine

The term “genomics” came into practice 15 years ago to denote the study of the functions and interactions of all the genes in the genome. The term ushered in a new era in medicine, where boundaries will continue to expand as our understanding of the cause of conditions such as breast cancer, colorectal cancer, human immunodeficiency virus (HIV) infection, tuberculosis, Parkinson’s disease, and Alzheimer’s disease grows. These conditions are examples of multifactorial disorders which are being examined through the lens of genomics as they are all caused by underlying interactions of multiple genes as well as environmental factors.

The implications for this emerging era in the practice of medicine are far-reaching. A patient’s genome will soon help determine the optimal approach to care, whether it is preventive, diagnostic, or therapeutic. As such, all physicians will need to understand the concept of genetic variability, its interactions with the environment and its implications for patient care.

In this issue, we examine genomics as it applies to clinical medicine, regulations, and business trends. We will also provide some insight into the world of theranostics, which combines therapeutics and diagnostics to help find the right therapeutic decision for the patient.

of the Quarter:Mr. Marcelo Cloes, Senior Director of Strategy, Bacterial Infections, bioMerieux

Q: The term “theranostic” is fairly new; can you explain what it is and how the term came about?A: It is a combination of two words: therapeutics and diagnostics. There are several definitions of theranostics, but the most agreed upon definition is that theranostics is a

diagnostic test that helps a clinician make the right therapeutic decision for the right patient. At bioMerieux, we like to say that theranostics is the right therapy for the right patient given at the right time. This personalized approach to therapy is the reason that theranostics is also known as personalized medicine. It gives the patient the opportunity to only receive the drug he or she needs as opposed to receiving a generic approach to their condition.

Q: What are the main benefits for patients who are treated with theranostics compared to traditional treatment methods? A: In short, to maximize therapeutic benefits and minimize unwanted adverse events. It is important to understand that the benefit of theranostics is evident for all stakeholders:

patient, physicians and regulatory agencies, but also for the pharmaceutical industry, the diagnostic industry and payers. For instance, by implementing companion diagnostics in drug development, pharmaceutical companies can drastically improve success rates and reduce clinical development costs. The drugs that were previously considered ineffective are being reanalyzed by pharmaceutical companies and can be resubmitted for FDA clearance hand-in-hand with the companion diagnostics. For payers, a lot of unnecessary healthcare costs can be eliminated if the right drugs are used on the right patients at the right time. So there are multiple benefits.

Q: How widely adopted is theranostics? Why?A: For the association of drugs with diagnostics, which we will refer to as companion diagnostics, where the value has been very clearly defined, the adoption has been quite

good. In order to establish the value proposition, four elements are critical: analytical validity, clinical validity, clinical utility, and health economics. Analytical validity dictates that a diagnostic test delivers its analytical performance consistently. Clinical validity means that the test being validated in clinical studies must demonstrate its intended use. Clinical utility highlights how the test will be used as a part of standard of care in disease management. Last, but certainly not least, the economic equation must still make sense for payers.

Q: Which clinical areas do you think are the most promising for applying theranostics in the near future? What additional clinical applications will benefit from theranostics in the long term?

A: The number one application that is taking off is oncology. Another application that has great promise is cardiovascular. We are hearing more and more potential companion diagnostics being asked for with Clopidogrel, which is being called the “super aspirin.” Because the possibility of bleeding events in those antiaggregation therapies is significant, predicting the response of those drugs in a given patient is an important piece of information. Finally, infectious disease is one avenue that bioMerieux is pursuing very aggressively. Such companion diagnostics could target pathogens and host elements; the latter is seeing more and more interesting breakthroughs. For example, measuring the antibody levels in certain patients for certain infectious disease conditions can be one way to provide valuable prognostic information while at the same time stratifying patients for more aggressive treatment for hospital-acquired infection.

For more information about bioMerieux, please visit: www.biomerieux.comLong version of this interview: www.themarketechgroup.com/minute/tmtg-min25-cloes.pdf

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THE ASSOCIATE CORNER: Wallonia, a “Cell Therapy Valley?”The region of Wallonia, in the southern part of Belgium, has a long tradition in the field of biotechnology and health care. The region is highly advanced in areas of cell therapy and regenerative medicines. Indeed, there are already three cell therapy product companies present on this small territory that has a population of 3.4 million. The companies, Bone Therapeutics, Cardio3 Bioscience, and Promethera Bioscience all face a similar problem: existing production capacities are insufficient. In order to resolve this challenge, the region of Wallonia has begun to build a cGMP production center. The facility will be designed in such a way that it can accommodate the current companies in the region as well as other companies who have an interest in manufacturing in this part of the world. BioWin, (Biotechnologies Wallonia Innovation) was created by the Walloon government in an effort to promote innovation and training in the field of biotechnology in health care. The first phase of the project will be to create a center of excellence that will attract other companies, either directly involved in cell therapy product development, or active in the different support activities that are required for this discipline. The existence of this production platform for cell therapy in Wallonia, which can support several companies active in the cell therapy industry, indicates the viability of the cell therapy market and its importance globally.

Dr. Patrick Stragier, BioWin – Director of the Cell Therapy Platform For more information, please visit www.biowin.org.

The Role of Theranostics in Healthcare

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HOT TREND

ON THE HORIZON: Patient Influence on Care Delivery

HIGHLIGHTSGenomic testing is increasingly being used to eliminate potential drug recipients whose genes do not make them a good match for a particular drug.7% of all patients in US hospitals suffer from adverse drug events. In psychiatry, that percentage jumps to 16%. Efficacy of drug treatment is low, especially in oncology with an average of 25%.

AT STAKE!The FDA has recognized theranostics as an important tool in the development process, but has not fully outlined the guidelines yet. Opportunities to comment and influence future guidelines will become more limited as these guidelines become established. Protection of intellectual property and patent extensions are critical to secure investment into research and development of new drugs and to resurrect existing drugs, which may only target a small sub-section of the population.

SO WHAT?The use of theranostics will raise the bar for pharmaceutical companies in their efforts to gain market access because there will be narrower markets as potential customers are weeded out through genomic testing.Though the technology of theranostics allows for improved patient-drug interaction, market access will most definitely become more difficult for pharmaceutical companies as the emerging body of law surrounding the use of Genomic Biomarkers evolves in the EU and the US. Insurance companies could benefit from the application of theranostics by reducing the number of re-hospitalized patients and optimized dosing.A general genetic screening could potentially put too great of a strain on the present health care system. Alternative models should be considered.