Evidence or Opinion: what is really driving decisions following accelerated approvals?

Delivery of confirmatory studies following any accelerated approval has been an area of growth and contention. Sue O’Leary, Senior Vice President of Prime Access, and Jenny Blackham, Vice President of Client Partnerships, take a closer look at what is happening.

The number of products now approved on the basis of limited datasets is significant and dominates the rare disease and oncology space. The changes seen over the last decade, enabling patient access to new therapies where no other options exist, have given clinicians new tools and given patients new hope. Payers, whether they be reimbursement authorities or private health insurers, have dug deep in efforts to fund treatments, which are sometimes regarded as ‘experimental’.

The spring 2021 Oncologic Drugs Advisory Committee (ODAC) meeting focused on reviewing six “dangling” immune checkpoint inhibitors (ICIs) that have received US Food & Drug Administration (FDA) accelerated approvals without meeting their trial primary endpoint. However, the accelerated approval process came under fire. As a result of the decision to keep four out of the six on the market, criticism flooded the headlines. A host of articles were published on topics from evidence or lack of evidence, the role of opinion, to how “value” or “unmet need” is really defined.

These headlines over recent debates between the FDA and the ODAC have provided a real-world case study on what evidence is driving decisions. As market access and RWE communication experts, we found the ODAC meeting results and all the supporting articles and perspectives very interesting.

The table below considers recent PD-1/PD-L1 inhibitor decisions that have been an emphasis of FDA confirmatory study reviews this year, and maps the data presented against the decisions taken.

To view a larger size, please click the table.

Table showing recent evaluation of PD-1/PD-L1 Inhibitor Indications

Despite having accelerated approval maintained following ODAC evaluation, following consultation with the FDA Roche voluntarily withdrew its US accelerated approval for Tecentriq® in PD-L1 positive advanced or metastatic triple-negative breast cancer on 27th August 2021.

We think these inconsistencies are too important to ignore and the current evidence throws up more questions than answers!

  • Why aren’t the studies delivering data that aligns with the experts’ opinions?
  • Is it worth all the investment from the Pharma industry in collecting evidence?
  • Is efficacy being captured correctly by the defined endpoint?
  • How are we defining “value” or “unmet need”?

Ensuring that the best evidence is available is critical to proving value for all stakeholders, but we believe from assessing the recent decisions that a main factor is how the evidence is interpreted and communicated. It is clear that ‘value’ is a subjective measure and perspectives of key stakeholders go well beyond numbers and levels of statistical significance.

As a consultancy, we will continue to assess these questions moving forward as we believe some clarity is required. In the meantime, we are focused on helping clients communicate their evidence in a compelling way to drive patient access.


Prime Access is a strategic and tactical market access, value evidence and patient voice consultancy. To find out how we can help you, click here > https://primeglobalpeople.com/contact-us/ for more information.

Prime GlobalEvidence or Opinion: what is really driving decisions following accelerated approvals?
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