The Rewards and Challenges of Biomarkers in Pricing & Market Access
The Rewards and Challenges of Biomarkers in Pricing & Market Access
The application of biomarkers in therapy areas beyond oncology will likely play an ever-crucial role in developing more targeted therapies in high unmet need indications, while providing payers with new attributes by which to assess a new treatment’s value.
Biomarkers are defined as quantifiable biological characteristics that can be used to diagnose conditions, predict treatment response, and monitor safety and tolerability of drugs. [1-2] While historically used for diagnostic purposes, biomarkers have been leveraged within the last 15 years to create treatments targeting gene expressions, most notably in oncology in which such treatments (i.e., the PDL-1s, HER2s) have largely replaced chemotherapy as first line options due to their improved levels of patient response. In particular, the HER2 (breast cancer), ALK (lung cancer) and BRAF (melanoma and colorectal cancer) biomarker targeted therapies have been able to guarantee certain levels of response within exclusive patient sub-populations. As such, these treatments have been able to secure high prices that, in the United States, regularly hover around 6 figures.[3]
Unsurprisingly, the use of biomarkers in drug development is of increased interest in other therapy areas, such as multiple sclerosis or immuno-inflammation.[4] In fact, in 2019, over 50% of new treatments approved by the FDA and EMA throughout dozens of therapeutic classes (antivirals, immunosuppressants, antibacterial etc.) included biomarkers.
Despite opportunity for commercial success, there are significant challenges in developing and implementing a successful biomarker. For one, it is difficult to discover a biomarker, and have it validated by the relevant regulatory bodies [5], let alone accurately demonstrate that it can predict the safety, efficacy, and durability of a treatment. Indeed, biomarkers require multiple clinical trials, which can be very expensive and do not guarantee conclusive evidence. In addition, the cost and challenges associated with biomarker testing can be an additional practical hurdle which makes their use less compelling, especially in disease areas which are not life threatening and where overall payer willingness-to-pay is low.
Windrose’s take
Windrose supports clients that have clinical assets with biomarkers for indications outside of oncology and seeks to probe the added value of the attribute for their product’s pricing & market access potential. While each indication offers unique challenges and opportunities, we feel that the following general considerations are widely applicable:
Payers tend to only value treatment predictive biomarkers that are binary in their forecast i.e., produce a yes / no answer on whether a subset of patients will have a significantly higher rate of response for a particular treatment. This helps facilitate implementation of stopping rules to strictly control access and mitigate budget impact concerns.
Diagnostic biomarkers tend to offer less compelling value since they do not provide any information on the efficacy of a treatment within a biomarker designated population.
Premium pricing would largely be contingent on biomarker products having a higher efficacy than current standard-of-care within the biomarker designated sub-population.
The size of the eligible biomarker population is also a key factor in informing pharmacoeconomic models and cost-offsets for price negotiations.
Therefore, while biomarkers will likely be evermore important in the pharmaceutical industry’s quest to develop innovative and personalized medicines, their implementation in indications beyond oncology is far from being a guarantee of increased payer value perception.
Article written by William Noumba Um, Analyst, Windrose Consulting Group
Sources
Mattes WB, Goodsaid F. Regulatory landscapes for biomarkers and diagnostic tests: qualification, approval, and role in clinical practice. Exp Biol Med (Maywood). 2018;243:256-261.