Anatomy of a Decision

It’s easy to think of health technology assessment (HTA) reimbursement decisions as boiling down to a simple a “Yes” (Recommend) or “No” (Do Not Recommend). In fact, there are often multiple dimensions to the outcomes of HTA agency reviews. Failure to take the nuances of these decisions into account when developing a market access strategy can have significant impact on everything from clinical trial design, building an economic model, to projecting revenue and beyond. Working with an accurate and precise classification of a decision, and taking into account the context of that decision, enables manufacturers to use decision qualifications, such as restrictions, to develop a well-informed strategic blueprint for market access. This blueprint can take into account:

  • The types of decisions that an agency may render
  • How and why different decisions are used
  • The influence of HTA agency decisions within their specified healthcare systems

The context that a decision occurs within has a large impact on what that decision ultimately means for a company. For example, HTA agencies carry the responsibility to contain costs and optimize the available reimbursement budget. The decision to not recommend a drug for reimbursement at a given price is often based on reasons such as:

  • Lack of efficacy
  • Inadequate efficacy
  • Lack of need
  • Excessive budget impact

While a “Do Not Recommend” decision may seem like an impenetrable barrier to market access, it is not always a dead-end. The impact of an HTA agency’s decision on market access is dependent on the healthcare system for which they have jurisdiction. Agencies fall into two categories: those with binding decisions and those with non-binding decisions. 

In the United Kingdom, the National Institute for Health and Care Excellence (NICE) administers HTAs and issues legally binding decisions. Under standard procedures, if a drug is rejected by NICE the National Health Service (NHS) will not reimburse it. The submitting manufacturer will either have to resubmit or explore other available payer options, such as the Cancer Drug Fund (CDF) or private payers. However, in the Canadian system, a drug that has received a non-binding “Do Not Recommend” decision from the Canadian Agency for Drugs and Technologies in Health (CADTH) may still be accepted for use in an individual Canadian providence, where the final decisions are made. In one analysis, 43% of drugs that received a negative CADTH reimbursement recommendation were available in British Columbia, and 51% were still available in Ontario. For more on this variability in Canada, click here

While a “do not recommend” decision does not always block market access, a decision to recommend a drug may not necessarily translate into full market access to the entire population. Restricting the population that is recommended to use a drug to a subset of the total population for which it has received regulatory market authorization (i.e., FDA or EMA approval) is one method that HTA agencies and their countries use to maximize efficacy within their populations and to control costs. To reflect these decisions accurately, Context Matters classifies these decisions as “Recommend with Restrictions.” Common restrictions fall into two primary categories:

  • Who will get the drug – determined by population markers such as genotype, age group, etc.
  • How the drug is used in therapy – after failure of primary therapy, last line of therapy, as a rescue medication, with another drug, etc.

Population restrictions allow the drug to be used only in the segment of the population for which it has proven to the agency to have the greatest health impact. For example, the presence of certain mutations can greatly reduce the efficacy of certain cancer drugs, and as a result these drugs are commonly restricted to patients without those mutations.

Restrictions also allow the drug to be used only where the agency determines it is most cost-effective, such as after a less expensive drug has failed to control the disease (or conversely, before a more effective, but more costly drug has been tried). Our recent webinar provides a more in-depth look at the challenge of identifying restrictions and how a rigorous analysis process can provide the information needed to assess restrictions and use them strategically in positioning a drug on the market.

Restrictions are not the only way that HTA agencies meet the demands of their remits and best serve the needs of their populations. Another qualifier a decision can receive is an agency-specific score. In Germany, the Federal Joint Committee (G-BA) uses a comparative benefit scale in all of its decisions to determine where a drug falls in both treatment and reimbursement pathways, which can have a significant impact on the size of the end-user patient population. This comparative benefit scale assesses both the extent and proof of added benefit the drug demonstrates versus the appropriate comparator selected by the G-BA. 

If a drug demonstrates no additional benefit, or no proof of an additional benefit, then it can only be reimbursed on a cost-minimization basis versus the existing standard of care. A manufacturer can still demand a higher price, but the patient will have to face the financial disincentive of paying for that premium out of pocket, and may choose the fully reimbursed alternative instead. As a result, in Germany, you must look deeper into the nuances of a decision—even a positive one—in order to determine its ultimate impact on a drug's market share. 

The Haute Autorité de Santé (HAS) in France uses Service Médical Rendu and Amélioration du Service Médical Rendu (SMR and ASMR; in translation, Actual Benefit and Improvement in Actual Benefit) scores to assess a drug’s relative effectiveness compared to the current standard of care.

The SMR, or Actual Benefit, describes the actual benefit of a medicine in terms of severity of disease, efficacy/safety ratio, and its public health benefit on a scale of "substantial", "moderate", "low", and "insufficient". These ratings, which loosely correspond to reimbursement rates of 100%, 65%, 35%, and 0% of a drugs’ list price, are used by HAS to determine the reimbursement rate of the drug prior to price negotiations. 

The ASMR, or Improvement in Actual Benefit, score describes the therapeutic advance of the medicine over other available forms of treatment on a scale of 1-5, with 1 indicating major improvement and 5 indicating no improvement. This rating is used by the agency in charge of pricing negotiations (the Economic Committee on Health Care Products, or CEPS), with medicines with a level 5 ASMR being reimbursed only on a cost-minimization basis, much like in Germany.

Decisions are multifaceted and complex. While they might appear to be simple "Yes" or "No" responses, factors such as where that decision is made, what system it is embedded within, the degree of approval it receives, and restrictions all have serious implications. A drug can receive a "Do Not Recommend" decision and still attain full market access in Canada, or it can receive a positive recommendation in Germany, but due to limited reimbursement only be used by a portion of the population that falls under its original marketing authorization. Similarly, restrictions can be either used by HTA agencies to limit the populations a drug has access to, or be strategically used by drugmakers to help them move into a market, target an unmet need, or justify a price premium. In short, not all positive (or negative decisions) are equal, and knowing the full context of a decision matters.