The assessment of value within healthcare is undergoing a major transformation. Gone are the days when a new mechanism of action alone would be regarded as a high value innovation. Today, there is much more of an emphasis on what are the outcomes (i.e., mortality and morbidity benefits), how does this compare with the current standard of care and what is the impact to the usage of healthcare resources. This emphasis is quite understandable given the financial crisis we are experiencing with an ever growing and aging population that is placing considerable strain on the healthcare system.

There are few Eureka moments in science. Incremental innovation such as a new mechanism of action is vital for furthering the scientific understanding and fostering the development of future innovations, especially when it is linked to targeted patient populations and coupled with optimization of the disease management process. It should come as no surprise that healthcare systems are asking “what is the value for money we are receiving?” It is very human of us to ask this simple question, “is it worth the money?” We do this every day when we purchase a consumer good and consider other expenses.

The facts are very simple: innovation comes at a cost. The attrition rate for compounds from initial exploration through to the market is more than 999 of 1,000, and these 999 abandoned compounds and their associated costs are just part of the cost of innovation (Forbes 2011). If innovation is not rewarded the advancements we have seen within the pharmaceutical space would not have taken place or would not have taken place so quickly. Assessors of value need to ensure that innovation is evaluated in a transparent manner that allows for constructive dialogue to take place for future advancements.

This is where health technology assessment (HTA) can play a significant role. Although the mandates of HTAs may vary from one to the other there is always one constant, HTAs are evaluating the current treatment paradigm and estimating how it will change with the introduction of new interventions. This is a fair question that the pharmaceutical industry also asks itself when it begins the development of a new treatment. Where differences in opinion can arise is the criteria used to define value. In some instances, value is assessed by HTAs with a limited scope. Aspects such as productivity, social benefits, benefits of efficient healthcare delivery, and improved patient relevant outcomes can often be overlooked by HTAs. But all of these factors in addition to the core elements of value (e.g., efficacy and safety) provide a more complete picture of what the introduction of a new pharmacological treatment represents as a healthcare solution.

Establishing the benefit of a new treatment primarily resides with the pharmaceutical industry and we can often fall into the trap that the benefit can be rather intuitive. Significant efforts have and are being made to establish this benefit with evidence. Given the constraints of the current operating model for delivering evidence (i.e., randomized controlled trials) which can limit the type of information the pharmaceutical industry can provide (i.e., efficacy vs. effectiveness), we must find more efficient ways of generating the evidence to convey the value of our treatments. Evidence from a multitude of sources—whether it be concrete data or a formalized opinion from various stakeholders—should be given consideration when assessing value as it can project the credible promise of the treatment.

These issues lead to some recommendations by the pharmaceutical industry to ensure that HTAs can robustly assess and allow access to value in a timely manner. Such considerations include:

• • Ensuring the HTA decision-making process is transparent – HTAs should be clear and explicit on the methodology they are using and the criteria and data they need to reach their decisions

• • Encouraging dialogue with all stakeholders throughout the process – HTAs should be available for active engagement with various stakeholders (patients, physicians, industry) from clinical development to real world usage. The development and usage of treatments is a dynamic process but is currently subjected to a static assessment. We need to allow for continuous dialogue throughout the process of value assessment so that multiple perspectives can be shared across the life cycle of an asset

• • Reflecting all aspects of value – Improved productivity, convenience, reduced burden of care on caregiver/family support are just some examples of value that are considered as wider elements of value that are not routinely evaluated by HTAs. There can be an insistence that hard evidence be provided to substantiate these elements but many issues can arise which prevent such data collection. Such data must be collected in an ethically correct manner and current constraints that exist within the system need to be acknowledged (e.g., convenience cannot be readily assessed in a double blind randomized controlled trial as the patient does not experience the benefit as they do not know what they are receiving).

Significant strides have been made by industry to demonstrate value and by HTAs to assess value. A cooperative and collaborative spirit has emerged between both parties to begin and continue the dialogue on assessing value in a direction where the potential ground for consensus can be reached. Given the challenges posed by demographic change and budgetary constraints, we (all healthcare stakeholders) can only succeed by working together to ensure optimization of healthcare to the wider society.