Health Technology Assessment in Clinical Development: When to Bridge Research and Decision-Making

Related Reading: Supporting Decisions in Clinical Development

Health technology assessment (HTA) is a field of analysis that evaluates the medical, social, ethical, and economic implications of development, diffusion, and use of health technology. The aim of HTA is to aid decision-making by providing information about the likely outcomes associated with different courses of action, and it’s increasingly used to aid decisions regarding resource allocation with respect to new technologies. HTA comprises three core activities:

• the identification and review of evidence,

• the synthesis of evidence from multiple sources,

• and the development of decision-analytic models that seek to explicitly predict the outcomes of different courses of action in terms of endpoints that decision-makers can value and trade-off (such as resource use and measures of how patients feel, function and survive).

In addition to aiding decisions regarding resource allocation, HTA can aid decision-making during the clinical development process. The value of any clinical development process is a function of the likelihood that it will affect change (e.g. the adoption of new technology) and the perceived value of that chance. HTA activities can an aid decisions regarding investment, RCT design, and the need to conduct ancillary, non-experimental studies to confirm the value of new technologies. HTA aids these decisions by improving estimates of the value of candidate research programs.

However, failure rates are high during clinical development, and there may be a reluctance to invest in additional activities outside the core RCT program given the risk that an individual product may not make it to market and generate revenue. This can lead to HTA activities only being commissioned late in the clinical development process. However, this means that the results of HTA activities are not available when decisions need to be made during the clinical development process.

The concept of Net Present Value (NPV) is useful when evaluating potential HTA activities during different stages of the clinical development process. The NPV of a project is the sum of the present values of the incoming and outgoing cash flows associated with a project. If we consider early HTA activities, the outgoing cash flows are the costs of the proposed activities. There will be little discounting of these as they are incurred now and in the near future. The incoming cash flows are the difference in expected profits from the new technology if we conduct and do not conduct the proposed HTA activities. These will be heavily discounted as they both occur in the future, are subject to time discounting and are inherently uncertain. The earlier in the clinical development process HTA activities are undertaken, the greater the discount.

This suggests that HTA activities should be left as late as possible. There is however one important constraint; the HTA activities must be completed before the decisions they are intended to inform. If we wish to use the results of HTA activities to inform trial design decisions, they must be completed early in the clinical development process. If the HTA is being used to inform decisions regarding the need for ancillary studies to support reimbursement, HTA activities must be completed in sufficient time to allow such studies to be commissioned and completed.

Overall, early HTA activities need to be fit for purpose, and no more. If they are 'over-engineered', they are likely to be judged not cost-effective and their potential value will be lost.

-Neil Hawkins | Vice President, Health Economics, ICON Commercialization & Outcomes