Before a phase 3 trial can be launched, approvals must be obtained from regulatory authorities. It is advisable to involve a regulatory expert to help plan regulatory interactions with regulatory authorities.

End of phase 2 meeting with the FDA

An end of phase 2 meeting with the US Food and Drug Administration (FDA) is not a formal requirement, but is strongly recommended. The meeting:

  • Enables a sponsor to obtain FDA input into phase 3 plans and protocols.
  • Ensures that the FDA agrees that the available safety data support proceeding to phase 3.
  • Helps to identify any additional information necessary to support a marketing authorization application and/or a desired label.
  • Serves as a forum to discuss any issues encountered in the development programme.

There is no defined timing for an end of phase 2 meeting, but it is important to present phase 2 data and a thorough phase 3 plan and target indication to ensure a productive meeting.

Further details can be found on the FDA website.

Trials involving children: If a drug is to be evaluated for use in children, a paediatric study plan (iPSP) must be developed. In general, the FDA expects that a paediatric plan (iPSP) should be submitted no later than 60 days after the end of phase 2 meeting, unless specific regulatory agreement is obtained in advance.

CHMP Scientific Advice (EMA)

In Europe, developers can request scientific advice from the Committee for Medicinal Products for Human Use (CHMP), established by the European Medicines Agency (EMA). This advice is designed to provide guidance on the most appropriate way to generate required evidence on a treatment’s benefits and risks.

Scientific advice can be requested at any time during development and on an aspect of drug development (e.g. CMC, non-clinical, clinical, methodological/statistical issues). It complements regulatory guidance documents by providing tailored input into a specific development programme.

Scientific advice can include review of a phase 3 plan and study designs. Advice is intended to ensure that the planned development programme meets EMA requirements for data quality and adequately evaluates the safety and efficacy of the investigational drug.

Trials involving children: The EMA recommends that a paediatric investigational plan (PIP) be submitted when PK and safety data are available in adults. The appropriate time for submission should be determined in consultation with a regulatory expert.

Limits of regulatory input

Obtaining regulatory input does not guarantee that an investigational agent will be approved, even if the advice is followed. The input is focused on the appropriateness of the intended plan. During the regulatory review of licensing applications, the results of all studies will be evaluated to determine if the data adequately define the efficacy and safety of the product and support a favourable benefit/risk assessment.