Value is in the Eye of the Beholder

Therapeutics must demonstrate efficacy and safety to relevant regulatory authorities before they are approved for use in clinical practice. This is a relatively straightforward process, assuming the treatment positively impacts the relevant therapeutic area (e.g., ability to prevent major adverse coronary events [MACE] in coronary heart disease, ability to maintain/improve lung function in respiratory disease). While necessary, efficacy and safety alone are insufficient for a successful launch, as decision makers who safeguard access to therapies must have evidence of the value-for-money associated with new therapies before they allow for widespread use.

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Unlike regulatory hurdles of efficacy and safety, demonstrating value is more challenging. Why is this the case? One reason is “value” can mean different things to different stakeholders. For example, patients likely perceive value primarily in terms of effectiveness and safety (and potentially convenience). Conversely, payers may be more focused on economic implications, especially if less expensive and well-known alternatives exist. Physicians likely fall in the middle, wanting to provide optimal patient care while thinking about pricing/profit.

To maximize uptake, manufacturers must demonstrate value that resonates with each relevant stakeholder. However, it has been our experience that manufacturers tend to focus primarily on payers, risking optimal market access after regulatory approval because such evidence, while important, may not resonate with all relevant decision makers.

We will look at how value is perceived by different stakeholders, using case studies to illustrate differences. We will also provide suggestions to guide evidence generation efforts that incorporate multiple perspectives. Our recommendations are intended to help manufacturers generate evidence to inform discussions on value, both internally and externally, from early in the development process through loss of exclusivity.

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