It is no longer adequate to proceed through the early clinical development phase with just an assessment of safety and tolerance. Many development programs strive to demonstrate value assessments for the development candidate and along with the pharmacokinetic profile of single and multiple doses. Although it would be developers delight to find a robust treatment effect in their development Phase II program(s) results. Now developers are faced with providing evidence of the value this candidate provides to the world of health care management.
A robust phase II efficacy result provides the data to utilize predictive analytics and obtain the probability estimate of the success of a Phase III program for the development candidate. This will likely lead to regulatory approval success based on the efficacy. However commercial success will critically depend upon demonstration of the value the product provides.
For a new product to be supported by national health care bodies such as NICE (National Institute for Health and Care Excellence in the UK) and IQWIG (Institute for Quality and Economic Efficiency in Health Care in Germany) it needs to provide evidence of the cost savings to the national health care system. One can see a future in the US where the FDA approval will be coupled with a CMS (Center for Medicare & Medicaid Services). The latter would be anticipated to identify what medications/treatments would be supported by Medicare and other government medical support programs.
Value is not just in the eyes of the beholder. Commercial interests of the development candidate's firm need to be participants in the early clinical development program. Their input is essential to identify competitor profiles for both safety and efficacy. The commercial input can identify what real world evidence that can be collected in the early clinical development program. Early identification of what pharmacy benefit manages criteria are for their decision to classify a treatment as a tier 1- 3 for reimbursement purposes are needed.
A value assessment needs to be addressed in the early development program to find value that can be fully developed in a Phase III program. If it involves a patient reported outcome (PRO) it can be validated and approved by SEALD (Study Endpoints and Labeling Development) like units in world-wide regulatory bodies and thus available for the commercial arms of the company at launch of the product. Estimating value in a Phase IV program is no longer a viable path for industry to pursue. Estimating value early in development is a challenging task, but defining it is critical for the commercial success of the candidate.