#8: Special Protocol Assessment

November 9, 2016

The Food and Drug Administration (FDA) released a draft guidance on the Special Protocol Assessment (SPA) in May 2016, updating the original SPA guidance issued in 2002. SPAs are meant to give Sponsors and FDA a framework to meet, discuss, and agree upon certain clinical trial designs before the initiation of a study. This guidance discusses several key considerations for Sponsors who are contemplating the submission of a SPA.  First, it is necessary to understand what type of protocols are eligible for a SPA. Animal carcinogenicity or efficacy protocols, drug substance or drug product stability protocols, biosimilarity or interchangeability protocols, and human efficacy protocols all qualify for a SPA request. It should be noted that only human efficacy protocols are only eligible if they are intended to be relied on as the primary basis for an efficacy claim. Additionally, the Sponsor should meet with the FDA prior to the submission of a request. The meeting type varies based on the product being developed, but can include an End of Phase 2 (EOP2) meeting or a Type 2 biological product development meeting.

In the guidance Sponsors are given criteria required to be included in a SPA submission. Those materials are:

  • Information regarding the role the trial will have in product development;
  • Reflection of the study and disease population;
  • Justification for the design of the trial, including but not limited to: dosing, endpoints, controls, and statistical plan.

Sponsors must give the Agency adequate information to thoroughly critique the SPA and decide if it addresses the key scientific concerns. Therefore, any potentially problematic features of the study protocol should be addressed with sufficient information for the FDA to come to a consensus.

Despite the provided justification, the FDA does not always agree that the submitted protocol will satisfy all questions. Subsequently, a nonagreement letter can be issued. When a nonagreement letter is reached, the Sponsor has three general options. They can initiate the clinical trial without an agreed upon SPA, send a written response to the FDA regarding the nonagreement letter, or request a meeting to discuss the nonagreement letter. To ensure the most effective utilization of resources, reaching an agreed upon SPA is recommended before initiating the clinical trial. Depending on how many outstanding protocol issues are presented, a meeting or written response may be preferred.

An agreed upon SPA can be critical to ensure the clinical trial design is appropriate to support a regulatory application. Achieving an agreed upon SPA with the FDA signals that the Agency agrees that the clinical trial will adequately address the study outcomes, consequently driving development forward. If your company has been advised to submit a SPA or if you believe a SPA will move your product development forward, please contact us today to learn more about our services and how we can help you achieve a successful interaction with the FDA.

Agency Alerts Regulatory Sciences

December 14, 2018

FDA Proposes Changes to the De Novo Pathway

On Tuesday, December 4th, FDA published the De Novo Classification Proposed Rule. If finalized, the rule would establish procedures for the De Novo classification process by facilitating appropriate...

Read the Full Article
Agency Alerts General Regulatory

November 27, 2018

FDA Takes Steps to Promote Digital Tools for Prescription Drugs

On Tuesday, November 20th, FDA announced the establishment of a public docket to solicit comments and feedback on a proposed framework regarding the regulation of prescription drug-use-related...

Read the Full Article
Agency Alerts General Regulatory

April 18, 2016

FDA Reopens Comment Period for Proposed Rule Regarding Regulation of Fixed-Combination Drugs

On December 23, 2015, the FDA announced a proposed rule regarding the regulation of fixed-combination drugs.  In its announcement, FDA proposed that its requirements for prescription...

Read the Full Article