Participate In Initial Industry-FDA Meetings

Overview

Talk to the Sponsor about participating in their early meetings with the U.S. Food and Drug Administration (FDA). In most circumstances, before an Industry Sponsor can administer an investigational therapy to humans, they must submit an Investigational New Drug (IND) Application to request authorization from FDA to transport or distribute the investigational therapy across state lines. The Sponsor may request pre-IND meetings with FDA to request feedback on their development program. FDA regulators are increasingly interested in having patients and patient advocates participate in these early discussions. However, it is up to the Industry Sponsor to invite patient groups to participate because the FDA cannot invite patients or patient advocates directly. 

  • The biologics license application (BLA) is similar to the IND application and is used by Sponsors for investigational biologics. Pre-BLA meetings with the FDA have similar goals as pre-IND. 
  • The FDA approval of medical devices prior to marketing the device is very dependent on the classification of the device and is not addressed specifically in this section. To learn more about the medical device marketing approval process, you may wish to reference FDA: How to Study and Market Your Device, which is written for small business and industry Sponsors, and FDA: The Device Development Process, which is written for patients.

Learn about Pre-IND Meetings

A pre-IND meeting is a Type B FDA meeting which occurs early in the therapy development process to help to guide trial strategies. These meetings can help facilitate faster therapy approval processes. Pre-IND meetings greatly increase the likelihood of a program’s success by allowing concerns to be addressed early in the trial process. Pre-IND meetings are also a great opportunity to build a relationship with the FDA, minimize costs, and identify preclinical studies that have already been conducted. They are especially important when the therapy is intended to treat a serious or life-threatening disease.  

Note: A pre-IND meeting is for drug development. A pre-BLA meeting is for licensing of biologics. For device development, the process, when required  is called “pre-submission”, but is very similar. 

  • Understand the purpose of pre-IND meetings:
    • Identify supporting preclinical trials. 
      • A preclinical trial is a trial that tests the proposed therapy in animal or cell models. 
      • A preclinical study is required before clinical trials in humans can be started. 
      • By identifying these studies, you and the therapy Sponsor can gain access to feasibility, testing, and safety data that has already been generated.
    • Identify possible designations and development and review statuses that may accelerate or enhance the clinical trial or marketing review process. The FDA provides information about each of the available programs:
  • Consider the main goals of a pre-IND meeting:
    • Reduce time to market.
    • Address novel qualities of application.
    • Provide guidance to industry/small business Sponsors new to process.
  • Be aware of recurrent problems at pre-IND meetings, especially problems that your group may be able to address prior to the meeting. Identified problems include:
  • Review the list of frequently asked questions the FDA has prepared for Sponsors about pre-IND meetings. Although written for small business and industry, the information answers many of the questions you may be asking as well: Small Business and Industry Assistance: Frequently Asked Questions on the Pre-Investigational New Drug (IND) Meeting (updated periodically).

Find Sponsors

Pre-IND meetings are organized by industry/small business Sponsors of the investigational therapy. Because involvement of patient and patient groups in these types of meetings is still evolving, it is important to stay aware of the scheduling of any pre-IND meetings focused on treatments relevant to your disease to maximize the opportunity for input.

Securing an Invitation

FDA regulators are increasingly interested in having patients and patient group representatives participate in these early discussions, but the Sponsor is the one who will invite you to a pre-IND meeting. It is your job to make certain the Sponsor is aware of the benefits of your participation. Studies are often designed with hard data and statistical measures in mind, but this approach may not produce results that the patients and caregivers find valuable. Your participation in pre-IND meeting can ensure the FDA that the study objectives are designed with the needs of the patient community in mind.  

Participation by your group representatives in initial meetings between the Sponsor and the FDA can reduce therapy time to market by:

  • Ensuring that studies are designed to provide useful information.
  • Enhancing the understanding of data proposed to be gathered by clinical trial.
  • Providing a platform for the patient voice.
  • Creating or further growing a relationship between your group and the FDA.
  • Minimizing clinical holds on therapy development (an order issued by FDA to delay or suspend an ongoing trial/investigation).

Prepare for the Meeting

Pre-IND meetings are the most productive when they are focused on specific issues, like regulatory requirements, previous studies, IND application requirements, and so on. Therefore prior to the meeting, it is important that you:

  • Identify the purpose and goal of the meeting from the Sponsor’s point of view.
  • Become familiar with the information provided to the FDA by the Sponsor in the meeting packet.
  • Understand the role of the FDA:
    • Review Working with Regulators: A Focus on the FDA (2015) is a resource developed by the Cancer Support Community to provide patient groups with information about working with the FDA. The resource contains information about the role of the FDA, the relationship between the FDA and patient groups, and new initiatives that allow patients to have better access to therapies.
    • Be aware that the FDA does not conduct medical research or regulate the practice of medicine. 
    • Reach out to the FDA Patient Affairs, available at +1-301-796-8460. The FDA Patient Affairs Staff can help you understand more about pre-IND meetings and how your participation may be helpful.
  • Know what is included in an IND application (specific to therapies classified as drugs):
    • Chemical composition of the drug being studied in the clinical trial.
    • Studies performed in animal or cell models supporting safety and the potential benefits of the investigational treatment.
    • Methods to assure safety, including range of possible doses.
    • Recommendations for size and scope of clinical trials.
    • Controls, i.e. what the investigational therapy will be compared to.
    • Study endpoints throughout the different phases of the clinical trial process.
    • Multiple designated points of interaction between the FDA and the Sponsor during the clinical trial process to assure that every clinical trial is addressed in detail, the development program is well designed, and to establish safety profile and determine efficacy.
    • To learn more about the comparable application process for biologics, you may reference FDA: Biologics License Applications (BLA) Process (CBER).
  • Learn about Target Product Profile (TPP) and how it may be used in a pre-IND meeting.
    • The TPP is a template that summarizes the information that a Sponsor hopes to include in the drug (biologic or device) label after FDA approval. In other words, it is beginning the therapy development process with the goals in mind. 
    • A TPP is filled out by the Sponsor of the investigational therapy and submitted to the FDA, often with the pre-IND packet. It is however not a required document.
    • A TPP has been found to increase constructive discussions with the FDA in the pre-IND meeting and throughout the clinical trial process.
    • A TPP helps focus a Sponsor’s therapy development team and FDA review staff on the therapy development goals in terms of labeling. 
    • A TPP can help address issues early on in the therapy development process thereby preventing late stage failures and decreasing the total time involved with treatment development.  
    • The final version of the TPP will be similar to the annotated draft labeling submitted with a New Drug Application (NDA) or BLA.
    • To learn more about the TPP, you may wish to read the draft provided by the FDA: Guidance for Industry and Review Staff Target Product Profile — A Strategic Development Process Tool (2009).
    • The U.S. Department of Health and Human Services (HHS) provides an example of a TPP: Target Product Profile
    • The Center for Biologics Evaluation and Research (CBER) Office of Cellular, Tissue and Gene Therapies (OCTGT) webpage for industry education also has a 15 minute webinar on The Target Product Profile.
  • Recognize the difference between 505(b)(1) and 505(b)(2) New Drug Applications (NDA) and how they affect the IND for investigational drugs (similar standards apply to biologics and medical devices).
    • 505(b)(1) application is for new drugs that have not previously been studied. It requires the Sponsor to conduct all necessary safety and efficacy studies. The IND application of a 505(b)(1) must contain a report that includes the results of the pre-clinical trial studies performed in cell and/or animal models.
    • 505(b)(2) is for new drugs containing previously approved active ingredients. This allows the Sponsor to submit an IND (and later a New Drug Application) which uses some of the safety and efficacy information on the active ingredient from studies not conducted by or for the applicant. The 505(b)(2) can be a less expensive and faster route of approval compared with the 505(b)(1) process. Examples of investigational drugs that may be eligible for 505(b)(2) include:
      • A new dosage form that is faster acting.
      • A combination drug using 2 active ingredients in a novel way.
      • A drug delivery mechanism that patients or doctors prefer over previous versions (for example, a pill form of a drug previously only available in liquid form).
      • A new use of a drug that already has FDA approval to treat a different disease or symptom.

Tips for Success

  • Discuss with the Sponsor how you can contribute during the meeting.
  • Be prepared to present analyzed patient data from patient registries, natural history studies, and patient preference studies, and discuss how this data informed or supports required elements of the IND application.
    • Making Data Talk: A Workbook by the National Cancer Institute (2011) is a workbook describing how to communicate public health data to the public, policy makers, and the press. Although written for a researcher/scientist familiar with statistical analysis, the definitions and tips within the workbook can help you understand the analysis of your own data and learn about the best way to communicate the information to others. 
  • Contact the FDA Patient Affairs, available at +1-301-796-8460, to understand the type of information or specific presentation formats that may be most helpful from the FDA’s perspective.
  • Reach out to other group leaders who have recently been involved in pre-IND meetings for investigational therapies being developed for their disease.
  • During the meeting, listen for ways you can help the Sponsor overcome any uncovered obstacles or missing information that may delay the submission of the IND application.
  • Schedule a follow-up meeting with the Sponsor to debrief the information learned in the meeting and to discuss next steps to move forward. 

Resources

Overview
How to Study and Market Your Device U.S. Food and Drug Administration (FDA) (link)
The Device Development Process U.S. Food and Drug Administration (FDA) (link)
Learn about Pre-IND Meetings
Developing Products for Rare Diseases & Conditions U.S. Food and Drug Administration (FDA) (link)
Prepare for the Meeting
FDA Patient Affairs U.S. Food and Drug Administration (FDA) (link)
Biologics License Applications (BLA) Process (CBER) U.S. Food and Drug Administration (FDA) (link)
Target Product Profile Template Health and Human Services (HHS) ASPR (link)
The Target Product Profile Webinar Center for Biologics Evaluation and Research (CBER) Office of Cellular, Tissue and Gene Therapies (OCTGT) (link)
Tips for Success
FDA Patient Affairs U.S. Food and Drug Administration (FDA) (link)

Talk to the Sponsor about participating in their early meetings with the U.S. Food and Drug Administration (FDA). In most circumstances, before an Industry Sponsor can administer an investigational therapy to humans, they must submit an Investigational New Drug (IND) Application to request authorization from FDA to transport or distribute the investigational therapy across state lines. The Sponsor may request pre-IND meetings with FDA to request feedback on their development program. FDA regulators are increasingly interested in having patients and patient advocates participate in these early discussions. However, it is up to the Industry Sponsor to invite patient groups to participate because the FDA cannot invite patients or patient advocates directly. 

  • The biologics license application (BLA) is similar to the IND application and is used by Sponsors for investigational biologics. Pre-BLA meetings with the FDA have similar goals as pre-IND. 
  • The FDA approval of medical devices prior to marketing the device is very dependent on the classification of the device and is not addressed specifically in this section. To learn more about the medical device marketing approval process, you may wish to reference FDA: How to Study and Market Your Device, which is written for small business and industry Sponsors, and FDA: The Device Development Process, which is written for patients.

A pre-IND meeting is a Type B FDA meeting which occurs early in the therapy development process to help to guide trial strategies. These meetings can help facilitate faster therapy approval processes. Pre-IND meetings greatly increase the likelihood of a program’s success by allowing concerns to be addressed early in the trial process. Pre-IND meetings are also a great opportunity to build a relationship with the FDA, minimize costs, and identify preclinical studies that have already been conducted. They are especially important when the therapy is intended to treat a serious or life-threatening disease.  

Note: A pre-IND meeting is for drug development. A pre-BLA meeting is for licensing of biologics. For device development, the process, when required  is called “pre-submission”, but is very similar. 

  • Understand the purpose of pre-IND meetings:
    • Identify supporting preclinical trials. 
      • A preclinical trial is a trial that tests the proposed therapy in animal or cell models. 
      • A preclinical study is required before clinical trials in humans can be started. 
      • By identifying these studies, you and the therapy Sponsor can gain access to feasibility, testing, and safety data that has already been generated.
    • Identify possible designations and development and review statuses that may accelerate or enhance the clinical trial or marketing review process. The FDA provides information about each of the available programs:
  • Consider the main goals of a pre-IND meeting:
    • Reduce time to market.
    • Address novel qualities of application.
    • Provide guidance to industry/small business Sponsors new to process.
  • Be aware of recurrent problems at pre-IND meetings, especially problems that your group may be able to address prior to the meeting. Identified problems include:
  • Review the list of frequently asked questions the FDA has prepared for Sponsors about pre-IND meetings. Although written for small business and industry, the information answers many of the questions you may be asking as well: Small Business and Industry Assistance: Frequently Asked Questions on the Pre-Investigational New Drug (IND) Meeting (updated periodically).

Pre-IND meetings are organized by industry/small business Sponsors of the investigational therapy. Because involvement of patient and patient groups in these types of meetings is still evolving, it is important to stay aware of the scheduling of any pre-IND meetings focused on treatments relevant to your disease to maximize the opportunity for input.

FDA regulators are increasingly interested in having patients and patient group representatives participate in these early discussions, but the Sponsor is the one who will invite you to a pre-IND meeting. It is your job to make certain the Sponsor is aware of the benefits of your participation. Studies are often designed with hard data and statistical measures in mind, but this approach may not produce results that the patients and caregivers find valuable. Your participation in pre-IND meeting can ensure the FDA that the study objectives are designed with the needs of the patient community in mind.  

Participation by your group representatives in initial meetings between the Sponsor and the FDA can reduce therapy time to market by:

  • Ensuring that studies are designed to provide useful information.
  • Enhancing the understanding of data proposed to be gathered by clinical trial.
  • Providing a platform for the patient voice.
  • Creating or further growing a relationship between your group and the FDA.
  • Minimizing clinical holds on therapy development (an order issued by FDA to delay or suspend an ongoing trial/investigation).

Pre-IND meetings are the most productive when they are focused on specific issues, like regulatory requirements, previous studies, IND application requirements, and so on. Therefore prior to the meeting, it is important that you:

  • Identify the purpose and goal of the meeting from the Sponsor’s point of view.
  • Become familiar with the information provided to the FDA by the Sponsor in the meeting packet.
  • Understand the role of the FDA:
    • Review Working with Regulators: A Focus on the FDA (2015) is a resource developed by the Cancer Support Community to provide patient groups with information about working with the FDA. The resource contains information about the role of the FDA, the relationship between the FDA and patient groups, and new initiatives that allow patients to have better access to therapies.
    • Be aware that the FDA does not conduct medical research or regulate the practice of medicine. 
    • Reach out to the FDA Patient Affairs, available at +1-301-796-8460. The FDA Patient Affairs Staff can help you understand more about pre-IND meetings and how your participation may be helpful.
  • Know what is included in an IND application (specific to therapies classified as drugs):
    • Chemical composition of the drug being studied in the clinical trial.
    • Studies performed in animal or cell models supporting safety and the potential benefits of the investigational treatment.
    • Methods to assure safety, including range of possible doses.
    • Recommendations for size and scope of clinical trials.
    • Controls, i.e. what the investigational therapy will be compared to.
    • Study endpoints throughout the different phases of the clinical trial process.
    • Multiple designated points of interaction between the FDA and the Sponsor during the clinical trial process to assure that every clinical trial is addressed in detail, the development program is well designed, and to establish safety profile and determine efficacy.
    • To learn more about the comparable application process for biologics, you may reference FDA: Biologics License Applications (BLA) Process (CBER).
  • Learn about Target Product Profile (TPP) and how it may be used in a pre-IND meeting.
    • The TPP is a template that summarizes the information that a Sponsor hopes to include in the drug (biologic or device) label after FDA approval. In other words, it is beginning the therapy development process with the goals in mind. 
    • A TPP is filled out by the Sponsor of the investigational therapy and submitted to the FDA, often with the pre-IND packet. It is however not a required document.
    • A TPP has been found to increase constructive discussions with the FDA in the pre-IND meeting and throughout the clinical trial process.
    • A TPP helps focus a Sponsor’s therapy development team and FDA review staff on the therapy development goals in terms of labeling. 
    • A TPP can help address issues early on in the therapy development process thereby preventing late stage failures and decreasing the total time involved with treatment development.  
    • The final version of the TPP will be similar to the annotated draft labeling submitted with a New Drug Application (NDA) or BLA.
    • To learn more about the TPP, you may wish to read the draft provided by the FDA: Guidance for Industry and Review Staff Target Product Profile — A Strategic Development Process Tool (2009).
    • The U.S. Department of Health and Human Services (HHS) provides an example of a TPP: Target Product Profile
    • The Center for Biologics Evaluation and Research (CBER) Office of Cellular, Tissue and Gene Therapies (OCTGT) webpage for industry education also has a 15 minute webinar on The Target Product Profile.
  • Recognize the difference between 505(b)(1) and 505(b)(2) New Drug Applications (NDA) and how they affect the IND for investigational drugs (similar standards apply to biologics and medical devices).
    • 505(b)(1) application is for new drugs that have not previously been studied. It requires the Sponsor to conduct all necessary safety and efficacy studies. The IND application of a 505(b)(1) must contain a report that includes the results of the pre-clinical trial studies performed in cell and/or animal models.
    • 505(b)(2) is for new drugs containing previously approved active ingredients. This allows the Sponsor to submit an IND (and later a New Drug Application) which uses some of the safety and efficacy information on the active ingredient from studies not conducted by or for the applicant. The 505(b)(2) can be a less expensive and faster route of approval compared with the 505(b)(1) process. Examples of investigational drugs that may be eligible for 505(b)(2) include:
      • A new dosage form that is faster acting.
      • A combination drug using 2 active ingredients in a novel way.
      • A drug delivery mechanism that patients or doctors prefer over previous versions (for example, a pill form of a drug previously only available in liquid form).
      • A new use of a drug that already has FDA approval to treat a different disease or symptom.
  • Discuss with the Sponsor how you can contribute during the meeting.
  • Be prepared to present analyzed patient data from patient registries, natural history studies, and patient preference studies, and discuss how this data informed or supports required elements of the IND application.
    • Making Data Talk: A Workbook by the National Cancer Institute (2011) is a workbook describing how to communicate public health data to the public, policy makers, and the press. Although written for a researcher/scientist familiar with statistical analysis, the definitions and tips within the workbook can help you understand the analysis of your own data and learn about the best way to communicate the information to others. 
  • Contact the FDA Patient Affairs, available at +1-301-796-8460, to understand the type of information or specific presentation formats that may be most helpful from the FDA’s perspective.
  • Reach out to other group leaders who have recently been involved in pre-IND meetings for investigational therapies being developed for their disease.
  • During the meeting, listen for ways you can help the Sponsor overcome any uncovered obstacles or missing information that may delay the submission of the IND application.
  • Schedule a follow-up meeting with the Sponsor to debrief the information learned in the meeting and to discuss next steps to move forward. 

Resources

Overview
How to Study and Market Your Device U.S. Food and Drug Administration (FDA) (link)
The Device Development Process U.S. Food and Drug Administration (FDA) (link)
Learn about Pre-IND Meetings
Developing Products for Rare Diseases & Conditions U.S. Food and Drug Administration (FDA) (link)
Prepare for the Meeting
FDA Patient Affairs U.S. Food and Drug Administration (FDA) (link)
Biologics License Applications (BLA) Process (CBER) U.S. Food and Drug Administration (FDA) (link)
Target Product Profile Template Health and Human Services (HHS) ASPR (link)
The Target Product Profile Webinar Center for Biologics Evaluation and Research (CBER) Office of Cellular, Tissue and Gene Therapies (OCTGT) (link)
Tips for Success
FDA Patient Affairs U.S. Food and Drug Administration (FDA) (link)