CBSA is committed to keeping our community updated on issues that impact life sciences. Our Policy + Advocacy team continues to monitor and review legislation that could directly affect life sciences companies in Colorado, including an FDA program designed to speed needed medicines to market.

What is the Accelerated Approval Pathway?

The Food and Drug Administration (FDA) Instituted an Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint. The use of a surrogate endpoint can considerably shorten the time required to receive FDA approval.

If a drug goes to market with accelerated approval, the manufacturer is still required to conduct studies to confirm the anticipated clinical benefit, known as phase 4 confirmatory trials. If the confirmatory trial shows that the drug provides a clinical benefit, then the FDA grants traditional approval for the drug. Alternatively, the FDA could remove the drug from the market if the confirmatory trial does not demonstrate a clinical benefit.

Accelerated Approval Program Under Review

The Accelerated Approval Program has come under review by multiple states, Congress, and HHS due to concerns about insurance coverage and drugs introduced to the market that did not perform as anticipated. Congress and HHS have put pressure on the FDA to review the status of a select number of drugs due to their performance indicators. FDA advisers agreed, saying it was imperative to withdraw drugs without a proven benefit from the market or risk setting a bad precedent.

State Insurance Coverage Impacting the Accelerated Approval Pathway

State proposals surrounding insurance coverage are also leading to changes to the Accelerated Approval Program, which could ultimately alter and reduce the pathway. For example, Oregon recently applied for a Section 1115 Medicaid waiver that would allow the state to refuse Medicaid coverage for Accelerated Approval drugs. That application was ultimately declined but other states are considering similar waivers.

Vital Transformation Study and Findings

The recently published Vital Transformation Study identified an important a flaw in the reasoning behind state waivers. It indicates the average budget impact of Accelerated Approval therapies as a percentage of state Medicaid spending is one-half of one percent, 0.5%, across all 50 U.S. states and D.C.

Key Findings from Vital Transformation Study

· State-level proposals to delay Medicaid coverage of Accelerated Approval therapies would negatively impact between 66,000 and 319,000 patients and provide minuscule savings.

· 75% of these drugs “have their evidence pack and are approved by the FDA within four years,” said Duane Schulthess, CEO of Vital Transformation, which examined all 206 drugs that received or are waiting for confirmation since the pathway was established in 1992. The few trials taking more than five years generally involve drugs for rare diseases with small populations, which can delay trials.

· 82% of Accelerated Approvals are for orphan indications, rare cancers, and other diseases with fewer than 200,000 U.S. patients, according to Schulthess. With a limited pool of patients, putting together clinical trials takes longer.

· Orphan drugs for rare diseases are also likely to have a lower net present value (NPV), which impacts whether companies can invest in developing the drugs at all. Patients with rarer conditions, including many types of cancer, are therefore the ones who will suffer from changes to Accelerated Approval.

· Limiting the Accelerated Approval pathway would particularly impact patients living with rare disease and “could render the development of those therapies economically untenable.”

Read more about Accelerated Approvals and the Vital Transformation Study from CBSA’s National Partner, BIO.