Administering an investigational drug to humans is subject to prior approval from regulatory authorities.

Investigational New Drug

An IND is a request for authorization to administer an investigational drug or biological product to humans with the objective to collect clinical data (e.g. safety, PK, efficacy, …).

IND in the U.S.

The FDA’s primary objectives in reviewing an IND are, in all phases of the investigation, to assure the safety and rights of subjects, and in phase 2 and 3 trials, to help assure the quality of the scientific evaluation of the drug’s effectiveness and safety.

For submission to the FDA, the IND application must contain manufacturing information on the product, data from animal pharmacology and toxicology studies with the product, clinical protocols and investigator information.

The central focus of the initial IND submission should be on the general investigational plan and the protocols for specific human studies.

The general principles and content/format of the IND application are dependent upon the applicable legal basis – legal basis is set out in the Code of Federal Regulations (CFR) Title 21 Part 312.

Latest Glossary Definitions

Efficacy-Effectiveness Gap

The observed discrepancy between effects of a health intervention in routine clinical practice as compared with the effects demonstrated in randomised controlled clinical trials.

Briefing Book (or briefing materials)

Documentation submitted to facilitate an Agency meeting, for example to request scientific advice in relation to a proposed drug development pathway. It should contain company questions, justifications or company positions, and appropriate summary information.

Clinical Impact

The effect that something is likely to have on the treatment of a particular group of people, or on the results of treating that group.

Commercially Confidential Information (CCI)

In relation to Policy 70, CCI shall mean any information contained in the clinical reports submitted to EMA by the applicant/MAH which is not in the public domain or publicly available and where disclosure may undermine the legitimate economic interest of the applicant/MAH.

Clinical Evaluation Report (CER)

Required under the new MEDDEV 2.7.1 Rev. 3 guidelines. Document containing information and results generated in conducting an evaluation of the clinical safety and performance of a medical device.

CE-Marking Technical File

A CE Technical File is a comprehensive collection of documents providing medical device composition, specifications, manufacturing process, rationale for qualification and classification, non-clinical testing results and clinical evaluations, risk analysis and instructions for use/Labeling.

Comparative Effectiveness Research (CER)

The conduct and/or synthesis of research comparing different benefits and harms of alternative interventions and strategies to prevent, diagnose, treat, and monitor health conditions in routine clinical practice (i.e. the real-world setting).

Core Value Dossier

Core value dossiers are used in the frame of reimbursement negotiations and to support HTA submissions, as well as national and local payers.

Core Reimbursement Dossier

A core reimbursement dossier is submitted during the late stage of development of a product, in order to be considered for reimbursement.

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