120 and 180-day questions

Requests for additional information from regulatory agencies during the review process of a biotech product. These questions are typically issued at the 120-day and 180-day marks of the review process, and the applicant is required to respond to these questions within a specified timeframe to continue the review process.

Abbreviated New Drug Application (ANDA)

A submission to the FDA for the approval of generic drugs.

Accelerated Approval

A regulatory pathway established by the FDA that allows for earlier approval of drugs that treat serious conditions and address unmet medical needs.

Annual Reports

Serve as essential regulatory documents that include summaries/reviews of any ongoing clinical studies.

Biologic License Application (BLA)

A BLA is submitted to the FDA to request permission to introduce a biologic product to market in the US. BLAs include comprehensive information gathered during the development of the biological product.

4 months Safety Update Reports (4MSU)

Contains any new safety information learned about the drug that may reasonably affect the statement of contraindications, warnings, precautions, and adverse reactions in the draft drug labeling. Completed after the NDA or BLA has been submitted and is being reviewed by the regulatory authority.

Briefing Book

A document that provides relevant information to the members of an advisory committee before a meeting with a sponsor or applicant of a drug or biological product. The purpose of a briefing book is to help the committee members understand the product, the disease, the clinical development, the efficacy, the safety, and the benefit-risk assessment of the product.

Breakthrough Designation

Document to request breakthrough designation to a drug/device intended to treat a serious or life-threatening condition and has shown preliminary clinical evidence of substantial improvement over available therapies.

Case Report Form

A document used to collect standardized data from each participant during clinical trials. It can either be paper-based or electronic.

Clinical Development Plan (CDP)

A document that outlines the clinical trials that will be conducted to evaluate the safety and efficacy of a new drug or medical device.

Clinical Evaluation Plan (CEP)

A document that outlines how the clinical performance and safety of a medical device will be assessed throughout its lifecycle. It is a required document in order to obtain a CE marking for a medical device in the EU (according to the EU Medical Device Regulation (MDR) 2017/745).

Clinical Evaluation Report (CER)

A document that summarizes clinical data and evidence related to a medical device. Used to demonstrate that the device is safe, effective, and has an acceptable benefit-risk profile when used according to its intended purpose. Required for all medical devices marketed in the EU to support the CE marking process. Must be updated regularly with new data from post-market surveillance and clinical follow-up.

Clinical Study Report (CSR)

A CSR is a comprehensive and detailed document that outlines the results and findings of a clinical study, including the safety, efficacy, methods, design, data analysis, evaluation, results, appendices, etc. The CSR presents what was planned, what changed, what happened, why, and what was observed/concluded.

Clinical Trial Protocols

A document describing objectives, design, methodology, statistical considerations, and aspects related to the organization of clinical trials. They must meet a standard set out by the Good Clinical Practice and are used to obtain ethics approval from local Ethics Committees or Institutional Review Boards.

Clinical Trial Redactions

This involves selectively masking sensitive information within clinical trial documents.

Common Technical Document (CTD)

A standard format for submitting information about a medicine to regulatory authorities for approval or licensing.

Development Safety Update Reports (DSUR)

A DSUR is a document that provides an annual update on the safety of a drug during its clinical development. It includes information on serious adverse events, new safety risks, and changes to the risk-benefit profile of the drug. 

Fast Track Designation

A request to the FDA for an expedited review of a drug that treats a serious or life-threatening condition and fills an unmet medical need. It is usually requested during the writing of an IND.

Gap Analysis

Involves assessing the differences between the current state (such as existing clinical pharmacology data) and the desired state (what’s needed for regulatory approval).

General Investigational Plan

A document that forms part of an IND and outlines the overall clinical development plan and helps the regulatory authorities anticipate the needs of the future program.

Global Value Dossier (GVD)

An internal document designed for use by local market access teams in pharmaceutical and biotechnology companies. Its purpose is to establish the product’s value proposition with decision-makers responsible for adoption, access, reimbursement, and funding.

Grant Writing

The process of crafting a written proposal to receive grant funding from a grant-making institution.

Health Technology Assessment (HTA) Reports

A systematic evaluation of the properties of health technologies and interventions covering both their direct and indirect consequences.

Informed Consent Form

An informed consent form is a document that is used to inform participants about the details of a study or research project, and to obtain their voluntary agreement to participate.

Integrated Summaries of Safety and Efficacy (ISS/ISE)

A document that summarizes and analyzes the results of multiple clinical trials of a drug or biological product. It is required by the FDA for new drug applications (NDAs) and biologics license applications (BLA).

Investigator’s Brochure (IB)

A comprehensive compilation of clinical and nonclinical data on the investigational product maintained by a drug developer or investigator that contains the body of information about the investigational product obtained before and during the clinical trial. It is updated with new information as it becomes available.

Investigational Medicinal Product Dossier (IMPD)

A document containing information about an investigational medicinal product (IMP) to be marketed in the EU.

Investigational New Drug (IND)

A request for authorization from the FDA to administer human clinical trials.

Manuscripts

A manuscript is a written document that presents the findings, methodologies, and implications of a scientific study or research project intended for publication in a scientific journal or presentation at a conference.

Marketing Authorization Application (MAA)

An MAA is submitted to the EMA to request permission to introduce a medical product to market in Europe. MAAs include comprehensive information gathered during the development of the medicinal product.

Medical Device Directive (MDD)

Introduced in 1994 to harmonize laws related to medical devices within the European Union (EU). It provides regulations for medical devices sold in the EU, ensuring standardized technical requirements, testing, and certification procedures.

Investigational New Drug (IND)

A request for authorization from the FDA to administer human clinical trials.

Orphan Drug Designation

A status assigned to a medicine, intended for use against a rare condition. The medicine must fulfill certain criteria for designation as an orphan medicine to benefit from incentives, such as protection from competition once on the market.

Paediatric Plans (PSP/PIP)

PSP - a proposed plan for conducting pediatric studies that sponsors of new drugs and biological products submit to the FDA during the IND phase of drug development.

PIP - a development plan aimed at ensuring the necessary data are obtained through studies in children, to support the authorization of a medicine for children. A PIP is required for all applications for marketing authorization for new medicines in the EU and Great Britain unless the medicine is exempt due to a deferral or waiver.

Periodic Safety Update Reports (PSUR)

A PSUR is a pharmacovigilance document that provides an evaluation of the risk-benefit balance of a medicinal product at defined time points after its authorization. It includes information on the safety of the product, including adverse reactions, the effectiveness of risk minimization measures, and any new risks that have been identified.

Pharmacovigilance Plan (PV Plan)

A document that includes identification, collection, and assessment of adverse events/reactions. Its purpose is to protect the public from medicine-related harm.

Plain Language Summary

Plain language summaries are short and simple descriptions of scientific research for laypeople.

Population Pharmacokinetics (PK) Report

This document describes the application of population PK in drug development.

Previous Human Experience (PHE)

A high-level summary of prior human experience with the study drug. It includes the status of the drug in other countries (if applicable), and if the drug was withdrawn from the market for any reason, must identify the country(ies) where the drug was withdrawn along with the reason for withdrawal.

Priority Review

A request to expedite the review process for drugs that are expected to have a significant impact on the treatment of a serious disease. It could be to greatly improve the safety, efficacy, diagnosis, or prevention of a serious disease.

Protocol Amendments

Sponsors are expected to submit protocol amendments for new protocols or changes to existing protocols before implementation of any respective changes to the IND application.

Protocol Synopsis

A summary of the procedural method and design of a study.

Rare Pediatric Disease (RPD) Designation

This is granted by the FDA for treatments intended to address serious or life-threatening diseases affecting children under 18 years old and fewer than 200,000 patients in the U.S.

Risk Evaluation & Mitigation Strategies (REMS)

Focuses on preventing, monitoring, and/or managing a specific serious risk by informing, educating, and/or reinforcing actions to reduce the frequency and/or severity of the event.

Risk Management Plans (RMP)

A document that outlines the potential risks associated with a project and the strategies to mitigate or manage those risks. It is a critical component of project management, as it helps to identify potential issues that could impact the success of the project and outlines the steps that can be taken to minimize their impact.

Safety Narratives

A required part of the clinical study report (CSR) that is provided to drug/device regulators. It describes deaths, serious adverse events, and adverse events of special clinical interest (usually defined in advance by the sponsor or regulator).