Meetings with the Office of 
Orphan Products 
Development 
 
 
Guidance for Industry, 
Researchers, Patient Groups, 
and Food and Drug 
Administration Staff 
 
 
Document issued on July 9, 2015. 
 
The draft of this document was issued on April 9, 2014. 
 
Written comments and suggestions may be submitted at any time for Agency consideration to the 
Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers 
Lane, rm. 1061, Rockville, MD 20852.  Alternatively, electronic comments may be submitted to 
http://www.regulations.gov.  All comments should be identified with the docket number listed in 
the notice of availability that publishes in the Federal Register.  Comments may not be acted 
upon by the Agency until the document is next revised or updated. 
 
For questions regarding this document contact Mr. James D. Bona at 301-796-8673 or 
james.bona@fda.hhs.gov. 
 
 
U.S. Department of Health and Human Services 
Food and Drug Administration 
Office of Special Medical Programs 
Office of Orphan Products Development 
  Contains Nonbinding Recommendations 
Meetings with the Office of 
Orphan Products 
Development 
 
 
Guidance for Industry, 
Researchers, Patient Groups, 
and Food and Drug 
Administration Staff 
 
 
Additional copies are available from:  
 
 
 
Office of Orphan Products Development  
Office of Special Medical Programs  
Food and Drug Administration  
10903 New Hampshire Ave.  
Bldg. 32, rm. 5295  
Silver Spring, MD 20993  
www.fda.gov/orphan  
E-mail: orphan@fda.hhs.gov 
Phone: 301-796-8660 
   
  ii  Contains Nonbinding Recommendations 
TABLE OF CONTENTS 
 
I.  INTRODUCTION............................................................................................................. 1 
II.  BACKGROUND ............................................................................................................... 1 
III.  MEETING TYPES:  INFORMAL AND FORMAL ..................................................... 2 
A.  Definitions ....................................................................................................................................... 2 
B.  OOPD Program Areas ................................................................................................................... 4 
IV.  MEETING REQUESTS FOR INFORMAL AND FORMAL MEETINGS ............... 4 
V.  MEETING PACKAGE CONTENT FOR FORMAL MEETINGS ............................. 6 
VI.   MEETING PROTOCOL ................................................................................................. 7 
VII.   DOCUMENTATION OF MEETINGS........................................................................... 7 
 
  iii  Contains Nonbinding Recommendations 
 
Meetings with the Office of Orphan Products Development 
 
Guidance for Industry, Researchers, Patient Groups, and Food and 
Drug Administration Staff 
 
 
 
This guidance  represents the Food and Drug Administrations (FDAs) current thinking on this topic.  It 
does not create or confer any rights for or on any person and does not operate to bind FDA or the public.  
You can use an alternative approach if the approach satisfies the requirements of the applicable statutes 
and regulations.  If you want to discuss an alternative approach, contact the FDA staff responsible for 
implementing this guidance.  If you cannot identify the appropriate FDA staff, call the appropriate 
number listed on the title page of this guidance.  
 
 
 
I.  INTRODUCTION  
 
This guidance provides recommendations to industry, researchers, patient groups, and other 
stakeholders (collectively referred to in this guidance as stakeholders) interested in 
requesting a meeting, including a teleconference, with the Food and Drug Administrations 
(FDAs) Office of Orphan Products Development (OOPD) on issues related to orphan-drug 
designation requests, humanitarian use device (HUD) designation requests, rare pediatric 
disease designation requests, funding opportunities through the Orphan Products Grants 
Program and the Pediatric Device Consortia Grants Program, and orphan product1 patient-
related topics of concern.  It is also intended to assist OOPD staff in addressing such meeting 
requests.  This guidance describes procedures for requesting, preparing, scheduling, 
conducting, and documenting such meetings. 
 
FDA guidance documents, including this guidance, do not establish legally enforceable 
responsibilities.  Instead, these guidances describe the Agency's current thinking on a topic 
and should be viewed only as recommendations unless specific regulatory or statutory 
requirements are cited.  The use of the word should in Agency guidances means that 
something is suggested or recommended but not required. 
 
II.  BACKGROUND 
 
OOPD staff regularly participate in meetings with stakeholders who seek input from OOPD 
relating to orphan-drug designation requests, HUD designation requests, rare pediatric disease 
designation requests, funding opportunities through the Orphan Products Grants Program and 
                                                 
1 As used in this guidance, orphan product may include a drug, device, biological product, or medical food for a 
rare disease or condition.  
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the Pediatric Device Consortia Grants Program, and orphan product patient-related issues.  
These meetings vary from general information-gathering meetings, where stakeholders ask 
basic questions about the designation or grants process, to more specific and complicated 
discussions involving, for example, questions related to orphan drug exclusivity or deficiencies 
related to a designation request.   
 
In some cases, these meetings may represent a critical point in the orphan product development 
process and may even have an impact on the eventual availability of products for patients with 
rare diseases and conditions.  It is therefore important that these meetings be scheduled within a 
reasonable time, conducted effectively, and documented where appropriate.  This guidance is 
intended to provide consistent procedures to promote well-managed meetings between OOPD 
and stakeholders.   
 
III.  MEETING TYPES:  INFORMAL AND FORMAL 
 
A.  Definitions  
 
Meetings between OOPD and stakeholders can be informal or formal.  
 
1.  Informal Meetings 
 
Informal meetings provide an opportunity for OOPD to address:   
 
  general questions about OOPD policies and procedures;  
  standard designation and grant process questions (e.g., how to submit a designation 
request or grant application, what information to include);  
  definitions of basic designation terms (e.g., rare disease or condition, orphan subset of a 
non-rare disease or condition, prevalence vs. incidence);  
  general advice on calculating the population estimate of a disease or condition; 
  patient group initiatives related to orphan products; and  
  other general questions.   
 
In some instances, an informal meeting may be appropriate to discuss a designation deficiency 
letter if the stakeholders questions can be readily addressed and do not require extensive 
analysis within OOPD and/or extensive discussion between OOPD and the stakeholder.   
 
Informal meetings usually take the form of a brief telephone conversation between OOPD and 
the stakeholder.  For informal meetings, stakeholders do not need to provide a meeting package 
to OOPD; the information contained in the meeting request and any subsequent 
communications should suffice (see Section IV).  If an informal meeting fails to resolve the 
stakeholders questions, OOPD may recommend that the next meeting be a formal meeting.  In 
some instances, if a stakeholder requests an informal meeting, OOPD may advise that the 
matter be addressed through a formal meeting.  Other times, OOPD may be able to resolve the 
stakeholders questions through e-mail without having to schedule an informal meeting. 
 
  2  Contains Nonbinding Recommendations 
 
2.  Formal Meetings 
 
Formal meetings provide an opportunity for OOPD to address: 
 
  specific and often complicated questions related to designation requests such as 
questions arising from a deficiency letter related to a complex orphan subset issue; 
  denied designation requests; 
  questions related to orphan-drug exclusivity; and  
  other matters that require preparation by OOPD and extensive discussion between 
OOPD and the stakeholder.   
 
Formal meetings can take the form of an in-person meeting or teleconference and usually 
involve more participants than an informal meeting.  Before a formal meeting occurs, 
stakeholders should provide a meeting package to OOPD that includes detailed background 
information and a proposed meeting agenda, among other information (see Section V).  In some 
instances, when a stakeholder requests a formal meeting, OOPD may advise that the matter be 
addressed through an informal meeting.   
 
It is important to understand that the formal meetings described in this guidance are not pre-
submission or interactive review meetings.2  That is, they are not a forum for stakeholders to 
obtain feedback from OOPD on product development protocols or planned studies with 
appropriate reviewing Centers within FDA or for OOPD to determine whether information in a 
pending or forthcoming original or amended designation request or grant application is 
complete and adequate.  Rather, formal meetings are a forum for stakeholders to obtain 
clarification from OOPD, and discuss disagreements with OOPD about OOPD policies, 
positions, and statements, including previous correspondence (e.g., a deficiency letter on an 
orphan-drug or HUD designation request), which may assist stakeholders with planned 
resubmissions to on-going designation requests.  
 
                                                 
2 For more information on pre-submission meetings, see FDAs guidance Requests for Feedback on Medical 
Device Submissions: The Pre-Submission Program and Meetings with Food and Drug Administration Staff 
(http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM311176.
pdf).  For more information on interactive review for devices, see FDAs guidance Types of Communication 
During the Review of Medical Device Submissions 
(http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM341948.
pdf).  For more information on formal meetings for drugs or biological products, see FDAs guidance, Formal 
Meetings Between the FDA and Sponsors or Applicants 
(http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm153222.pdf).  
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B.  OOPD Program Areas   
 
Stakeholders may request meetings with OOPD about any of the following orphan product 
programs: 
 
  OOPD Designation Programs 
    Orphan-Drug Designation (including Orphan-Drug Exclusivity) 
    HUD Designation 
    Rare Pediatric Disease Designation (for Priority Review Voucher Program) 
 
Many stakeholder questions about orphan product designations can be addressed through 
informal meetings, with the exception of orphan-drug exclusivity questions.  In OOPDs 
experience, most exclusivity-related questions are best addressed through formal meetings.  
Formal meetings are also appropriate for complicated designation questions that require 
extensive analysis within OOPD and/or extensive discussion between OOPD and the stakeholder 
(e.g., an orphan subset question that has resulted in several rounds of deficiency letters). 
 
Orphan Product Grants  
  Orphan Products Grants Program 
  Pediatric Device Consortia Grants Program 
  Orphan Product Outreach Program  
Patient-related Topics 
 
In OOPDs experience, questions about Orphan Products Grants, Pediatric Device Consortia 
Grants, and patient-related topics generally can be addressed through informal meetings.  For 
example, informal grant topics may include general questions regarding how to apply for a grant 
(e.g., what types of products qualify) and the grant review process, as well as questions about 
ongoing funded grants (e.g., how to handle enrollment issues or reporting requirements).  
Patient-related topics may include, for example, questions about various orphan product 
incentives for rare disease product development. 
 
IV.  MEETING REQUESTS FOR INFORMAL AND FORMAL MEETINGS 
 
Before requesting a meeting with OOPD, stakeholders should consult OOPDs website, which 
contains general information about orphan product designations and grants:  
www.fda.gov/orphan.  This website includes links to relevant FDA regulations and guidance 
documents, as well as responses to Frequently Asked Questions (FAQs).3  The information on 
this website may provide the answers and clarification the stakeholder seeks.   
 
                                                 
3 These regulations include 21 CFR Part 316 (Orphan Drugs) and 21 CFR Part 814, Subpart H (Humanitarian 
Use Devices).  Relevant guidance documents include FDAs guidance Humanitarian Use Device (HUD) 
Designations 
(http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/DesignatingHumanitarianUseDevi
cesHUDS/LegislationRelatingtoHUDsHDEs/ucm283517.htm). 
  4  Contains Nonbinding Recommendations 
If, after reviewing this publicly available information, the stakeholder still seeks a meeting 
(including a teleconference) with OOPD, the stakeholder can request a meeting in one of several 
ways: 
 
  If the requested meeting concerns a designation request or grant application that has 
already been submitted to OOPD, the stakeholder should request a meeting by e-mailing 
the identified point of contact for the designation request or grant application, with the 
subject heading Meeting Request.  This point of contact will be identified in the latest 
correspondence from FDA, whether an acknowledgment letter, deficiency letter, or other 
correspondence about the request or application. 
  If the requested meeting does not concern a designation request or grant application that 
has already been submitted, the stakeholder can request a meeting by e-mailing the 
general OOPD inbox at orphan@fda.hhs.gov, with the subject heading Meeting 
Request.  Alternatively, the stakeholder can send an e-mail to the point of contact for 
each OOPD Program Area listed in the Contact FDA section of OOPDs website 
(www.fda.gov/orphan), again with the subject heading Meeting Request.  
  OOPD strongly encourages that meeting requests be sent by e-mail to readily enable 
back-and-forth exchanges with the stakeholder about scheduling and other details.  
However, a stakeholder who prefers not to request a meeting by e-mail can submit a 
written request to: 
 
Office of Orphan Products Development 
      Attention: Program Contact Name  Meeting Request 
      Food and Drug Administration 
      10903 New Hampshire Avenue 
      Building 32, Room 5295 
      Silver Spring, MD  20993 
 
At a minimum, all meeting requests should include: 
 
  a brief statement of the meeting purpose, including identification of the product to be 
discussed and any applicable designation request or grant application number; 
  whether the stakeholder prefers an informal or formal meeting (see Section III);  
  suggested dates and times for the meeting;  
  preferred format of the meeting (i.e., teleconference or in-person meeting); and 
  the e-mail address(es) to which OOPD should send a response to the meeting request 
(if different from the e-mail address from which the request was sent) and telephone 
number for the primary contact for the stakeholder.    
 
OOPD will aim to respond to a meeting request within 5 working days of receipt; OOPD will 
determine the appropriate meeting type and will work with the stakeholder on identifying a 
convenient date and time for all participants in a timely manner.  Before scheduling the 
meeting, OOPD may ask the stakeholder for more information about the proposed meeting to 
help determine whether an informal or formal meeting is most appropriate and who from 
OOPD should attend.  This additional information may include a request for a brief list of 
questions that the stakeholder hopes to have answered. 
  5  Contains Nonbinding Recommendations 
 
Once a meeting is scheduled, OOPD will notify the stakeholder by e-mail or phone of the 
meeting type, date, time, duration, place or call-in number, and expected OOPD participants.  
If the meeting is to be a formal meeting, stakeholders should submit a meeting package to 
OOPD that includes detailed background information and a proposed meeting agenda, among 
the other information described in Section V.  For informal meetings, the information in the 
meeting request may suffice, although OOPD may ask for supplemental information via e-
mail or phone.  This supplemental information may include a request for a list of questions 
that the stakeholder hopes to have answered (if not already provided) and a list of individuals, 
with their titles and affiliations, who are expected to participate on behalf of the stakeholder.  
 
V.  MEETING PACKAGE CONTENT FOR FORMAL MEETINGS  
 
If a formal meeting is scheduled, OOPD should receive the meeting package at least 2 weeks 
before the meeting.  This meeting package is intended to help OOPD prepare for the meeting and 
enable a productive discussion between OOPD and the stakeholder.  We encourage stakeholders 
to submit the meeting package electronically by e-mail to the OOPD program contact who 
scheduled the meeting, with the subject heading Meeting Package.  If a stakeholder chooses to 
submit a paper copy, the stakeholder should send the meeting package to the following address: 
 
  Office of Orphan Products Development 
  Attention: [Program Contact Name]  Meeting Package 
  Food and Drug Administration 
  10903 New Hampshire Avenue 
  Building 32, Room 5295 
  Silver Spring, MD  20993 
 
We expect the length of the meeting package to vary depending on the issues to be discussed, 
the product, the indication, and the phase of product development.  The meeting package should 
contain the following information:   
 
  The date, time, and subject of the meeting; 
  An explanation of the meeting purpose (this explanation should generally be more 
detailed than the meeting purpose described in the meeting request); 
  Basic information about the product to be discussed, including: 
  Product name or identifier;  
o
  Designation or application number, if applicable; 
o
  The relevant rare disease or condition; 
o
  Brief background about the product, including how it may be used for the rare 
o
disease or condition; 
  A proposed meeting agenda, including a detailed list of questions that the stakeholder 
hopes to have answered along with any supplemental explanation or context that may 
help OOPD address these questions (this list should generally be more extensive and/or 
detailed than any list provided as part of a meeting request);  
  6  Contains Nonbinding Recommendations 
  Any data, information, or presentation materials to support the discussion, if needed;4 
and 
  A list of all individuals, with their titles and affiliations, who are expected to participate 
in the meeting on behalf of the stakeholder.  Note that a stakeholder may include patients 
and/or other subject-matter experts at the meeting with OOPD.5 
 
If a formal meeting is scheduled and OOPD fails to receive a meeting package at least 2 weeks 
before the meeting, OOPD may choose to postpone the meeting given the lack of adequate 
preparation time to review the meeting package.  OOPD will notify the stakeholder of this 
postponement and will work with the stakeholder to reschedule the formal meeting for a later 
date. 
 
VI.   MEETING PROTOCOL 
 
Meetings will be chaired by an OOPD staff member and will begin with introductions and a 
statement of the meeting purpose.  The stakeholder will then take the lead, for example, by 
summarizing the meeting agenda, giving a presentation, and asking questions.  Presentations 
should generally be kept brief to maximize the time available for discussion.  If a stakeholder 
requires any audio-visual requirements for the meeting, this should be discussed ahead of 
time.  OOPD advises that stakeholders limit their presentations and questions to the 
information previously submitted to OOPD (whether as part of the meeting request for 
informal meetings or in the meeting package for formal meetings), with added explanation 
and clarification as necessary.  If any new information or questions are presented, OOPD 
may not be in a position to substantively engage with the stakeholder on the new topics.   
 
At the close of the meeting, OOPD and stakeholder attendees should summarize the 
important discussion points, meeting outcomes, and any action items.  If meeting minutes are 
warranted (see Section VII), OOPD will remind the stakeholder to prepare and submit a draft 
summary of meeting minutes within 15 working days after the meeting.  
 
VII.   DOCUMENTATION OF MEETINGS 
 
Documentation of meeting outcomes, agreements, disagreements, and action items is critical to 
ensuring that this information is preserved for meeting attendees and future reference.  
Summary meeting minutes should be prepared for all formal meetings and certain informal 
meetings, if appropriate (e.g., where disagreements arise between OOPD and the stakeholder or 
where the stakeholder and OOPD agree on specific action items).  Within 15 working days 
following the meeting, the stakeholder should provide a draft of summary meeting minutes to 
the OOPD program contact by e-mail, with the subject heading Draft Meeting Minutes.  This 
draft should summarize the meeting discussion points, agreements, disagreements, and action 
items.  OOPD will review the draft and provide revisions to the stakeholder via e-mail in a 
                                                 
4 If information has previously been submitted to OOPD, stakeholders may reference that information in the 
submission or they may choose to submit the information in the meeting package for ease of discussion. 
5 Stakeholders may seek assistance from any source for meetings with the OOPD.  No registration as a special 
government employee is required prior to meeting with OOPD. 
  7  Contains Nonbinding Recommendations 
timely manner (generally within 15 working days).  The stakeholder will then either accept the 
version as final and notify OOPD to that effect or follow up with questions and/or further 
revisions, which OOPD will subsequently review and respond to in a timely manner (generally 
within 15 working days).  If continued disagreement on the meeting minutes exist, OOPD and 
the stakeholder may want to have a teleconference to further discuss the issues. 
 
Once the meeting minutes are finalized, OOPD will provide copies by e-mail to all OOPD 
attendees and to the stakeholder.  These minutes will serve as OOPDs record of the meeting 
and be included in all relevant files.   
  8