Policy - Biomarkers in Drug Development

Catalyzing the timely and efficient development of innovative medicines

September 14, 2016

Biogen is committed to leading research in the discovery and development of biomarkers to accelerate the availability of new medicines to patients in need. Biomarkers are indicators of a biological process, used to show that a person is healthy, has signs of disease or is responding to a medicine. Biomarkers have been integrated into medical practice for decades – for example, a doctor uses cholesterol as a biomarker to monitor a patient’s risk of developing heart disease or to look at improvements in cholesterol levels as a patient starts a new medication. A biomarker can also be the level of expression of a critical protein that causes a rare, genetic disease, like spinal muscular atrophy, where disease results from low or absent levels of a protein called “survival motor neuron” or “SMN.” In addition to their use in clinical care, biomarkers are important tools for accelerating drug development. The use of biomarkers to inform decisions during drug development and regulatory review is essential to the timely advancement of innovative, safe and effective medicines. However, demonstrating that a biomarker may be relied upon for regulatory decision-making can be a complex and lengthy process. 

Not all patients with the same disease will respond to therapeutic intervention in the same way – differences in underlying disease biology can mean that some patients may respond well others may fail to respond. In developing new medicines, Biogen conducts research to understand the characteristics of a disease from before symptoms start to late-stage disease, which helps us to identify the best therapeutic targets to pursue and the patients most likely to benefit from a potential treatment. Biomarkers are important tools used throughout our research and development process as we look for ways to measure where a patient is in their disease course and how they are responding to treatment. For example, when the role of a biomarker in disease is well-understood, it can serve as an objective measure of how a treatment is working to modify, reverse, or prevent disease. 

As part of the discovery and development of our therapies, we conduct the foundational research that is necessary to advance the identification, development and use of biomarkers for decision-making. Biomarkers can inform our decisions to advance a candidate drug into development, the design of clinical studies and the assessment of whether or not a drug is safe and effective. A constellation of well-characterized biomarkers can help shorten or smooth the journey to the discovery and development of a medicine. The effective use of biomarkers is at the core of our science-driven process to bridge the understanding of disease to the innovation of a new therapeutic drug.

The scientific understanding of the role of novel biomarkers in complex diseases, like Alzheimer’s disease, is growing rapidly. Therefore, we need a policy framework that keeps pace with this scientific knowledge and biomedical advances. Currently, there are gaps in the U.S. policy framework for use of biomarkers in the development of innovative medicines, which include uncertainty in intellectual property protection for biomarkers and limited clarity and predictability related to regulatory expectations for acceptance of a novel biomarker.

Confidence in a clear, predictable and efficient pathway to regulatory acceptance of biomarkers will encourage biopharmaceutical companies to continue to invest in the research and development required to identify and validate novel biomarkers. A robust policy framework for biomarker use in drug development should include a mechanism to advance from exploratory biomarkers used in clinical research to validated biomarkers that can advance a specific drug development program to qualified biomarkers that can advance multiple drug development programs across a therapeutic area. Recent policy development has focused on the last step – the qualification of biomarkers. However, a comprehensive and durable framework requires a bridge from exploration to validation to qualification of biomarkers. Biogen is committed to working with stakeholders to ensure that the policy environment keeps pace with the rapidly evolving science that will enable pharmaceutical companies to provide innovative medicines that are safe and effective to people suffering from serious and complex diseases.

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