The Specialist Forum Volume 13 No 11 November 2013 | Page 72

ADVERTORIAL Companion diagnostics lead to improved safety, efficacy and outcomes Dr Deepa Maharaj, head of Regulatory Compliance and Health Economics, Roche Products: Diagnostics Division D uring the last 15 years an increasing number of oncology pharmaceuticals have failed to garner regulatory approval because response rates have been too low to justify broad patient exposure to those drugs. This development spurred the healthcare industry to find means to identify patient subgroups which might experience a treatment benefit while excluding nonresponders, thus heralding the age of personalised therapy. This growing interest in personalised healthcare is reflected by a number of reports indicating that numerous pharmaceutical companies are entering or reinforcing partner programmes with diagnostic companies. Associated with a specific pharmaceutical impact, personalised therapy improves the benefit-risk ratio of medical decision making and treatment. For safer, more effective therapeutics to emerge, drug development needs to fully leverage the progress made in genome analysis, and reflect an improved understanding of disease etiology and interindividual genetic variability. A new drug development model pairing therapeutics with molecular diagnostic tools is known as companion diagnostics. Companion diagnostics Companion diagnostics are assays or tests that are intended to assist physicians in making treatment decisions for their patients based on the best response to therapy. This is called ‘personalised therapy’. There are two types of companion diagnostics: Tests that have been developed after a drug has come to market. (For example the presence of acquired Kirsten rat sarcoma viral oncogene homolog gene mutations in tumor tissue as a measurement of response to targeted therapies such as cetuximab.) Tests that are being developed as a companion to the drug that is currently in development. In contrast to other in vitro diagnostics (IVDs), which may also be used to guide treatment decisions, companion diagnostics do not only provide additional information, which might increase the chance of a positive treatment outcome, but generate information which is crucial for a positive benefit/risk assessment of the respective medicinal product. Essentially the data obtained via the IVD provides a means to either select patients who are suitable for the respective medication, to optimise the dose of the medicinal product throughout the treatment process or to exclude patient populations who are expected to experience adverse events. Beyond the codeveloper relationship, personalised therapy impacts healthcare providers at the points of testing, prescribing and payment and to deliver more valuable outcomes to patients when they are receiving the appropriate care. Personalised therapy incorporating companion diagnostic solutions also creates new commercial opportunities for strategic pricing and affordable reviews by cost effectiveness committees, both at the state and private levels. Companion diagnostics allows for more timely access to safe, highly effective patient-stratified therapies moving healthcare towards an era of predictive medicine, as opposed to reactive medicine, with the undoubted benefits of earlier treatment translating to better long term outcomes. In this respect, it may well be feasible to prolong life, but the challenge of future healthcare is to ensure that quality of life remains commensurate with this longevity. Challenges It is clear that a number of uncertainties and hurdles face stakeholders, and regulatory entities worldwide and many are currently undertaking efforts to provide guidance on companion diagnostics. One of the major points of concern is how the different regulatory entities involved in the assessment of either the therapeutic or the IVD part of the product pair may align their review processes in order to facilitate a swift and reliable approval of these novel therapeutic concepts. All the different stakeholders perceive different value propositions of the therapeutic agent versus the companion diagnostics. In some cases those interests may be radically divergent, as in the case of the cost/price of the companion diagnostic test. Stakeholders also agree that there are still a few problems to be solved. Firstly, it is not easy to describe the costs of diagnostics. Secondly, the evaluation of cost effectiveness for companion diagnostics is difficult. Thirdly, the regulatory pathway is fragmented. Cost effectiveness Studies on the cost effectiveness of personalised therapy are ongoing, but promising results are available. Personalised therapy could provide more value for money because of improved drug effectiveness and reduced toxicity, and it could also decrease the average research and development costs for new medicines, however the efficiency gains may show only in the long term. Conclusion From research it is clear that companion diagnostics can enable significant improvements in safety, efficacy, outcomes, and cost effectiveness associated with many existing and new therapies. Payers interested in providing access to such personalised therapy tests by covering and paying for them can be of great help by supporting the following: standardisation necessary to create a clear road map for evidence-based development, commercialisation, payer coverage, and value-based reimbursement of companion diagnostic tests; and development of pathways for the assessment of new innovations, such as next-generation sequencing and other emerging companion diagnostic technologies. 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