Differences Between Establishing Value for Approval Versus Coverage?

Yes, but maybe not irreconcilable.

Many of the educational sessions on the first day of the 2014 BIO International Convention touched on one essential question: how can safe and effective treatments get to the patients who need them faster?

Panelists in the session Irreconcilable Differences? A Balanced Approach to Establishing Value approached this key question through a discussion of how to shorten the time between—and improve the predictability and expectations around—regulatory approval and coverage and reimbursement.

Currently in the U.S. and Europe, these processes are sequential: first, a sponsor develops clinical evidence to win a market approval and then they turn to the process of using this evidence to obtain broad coverage and favorable reimbursement. But Panelist Charles Gerrits, Vice President, Global HEOR, Pricing, Reimbursement & Market Access at Pfizer noted that the discrepancy between the standards of evidence for regulatory approval and those for coverage/reimbursement decisions often lead to wasting limited research funding and fail to provide timely access to new medical technologies for patients who need them.

Hans-Georg Eichler, Senior Medical Officer at European Medicines Agency stressed the EMA’s emerging model of bringing regulatory and HTA agencies and sponsors to joint discussions early in clinical development. This has been particularly helpful, Dr. Eichler noted, in areas where these agencies often place different weights on different types of evidence, including when it comes to: the choice of a comparator for a clinical trial (e.g., placebo, standard of care); choice of clinical endpoints (e.g., progression-free survival, quality of life, impact of treatment on caregivers); duration of a clinical trial; and what evidence must exist for initial approval and coverage and what can be collected post-marketing.

The primary benefit of these joint discussions, said Dr. Eichler, is that they will prevent against sponsors being told at the end of a long, expensive clinical trial or pharmacoeconomic data development that how they developed that evidence—rather than what the evidence says—is problematic or insufficient. However, he cautioned, they are not promises of approval for licensing or agreements to cover a therapy or pay a specific price.

Dr. Gerrits cautioned the audience that while joint agency discussions can be beneficial, it is not a comprehensive solution to the issue of complicated and costly evidence generation for regulatory review and coverage/reimbursement decisions. For example, many of these joint-review processes don’t have mechanisms to adjudicate differences in opinion between regulatory authorities and HTA agencies regarding a sponsor’s proposed clinical development program. Additionally, Dr. Tunis, Founder and Director, Center for Medical and Technology Policy, noted that HTA agencies often have a different tolerance for the certainty of evidence: while regulatory authorities are usually concerned with the safety and efficacy of a product, HTA agencies also must consider how provide for individual patients and entire populations while acting as a steward of scarce resources. Thus, panelists encouraged the audience to view joint health authority discussions early in clinical development as an investment that will eventually lead to better understanding between the agencies on each’s evidence needs.

At the conclusion of the session, Dr. Tunis contextualized the discussion through the lens of an undeniable trend: payers are increasingly putting importance on the body of evidence that sponsors use to make a case around the value of a new treatment. Thus, while joint deliberations of regulatory and HTA agencies may not hold all the answers, the questions of how much must be known before approval, how to translate clinical trial data to real-world coverage decisions, and what standards of evidence can be agreed to for specific types of treatment are not going away. To address these crucial questions, regulators, sponsors, and other stakeholders including patients, will need to balance the demonstration of real-world effectiveness with the need to protect a robust innovation ecosystem in the context of ever-increasing downward pressure on spending.

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