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 In this section:

                Development & Approval Process (CBER)

Development & Approval Process (CBER)

                        2023 Biological Approvals
          2022 Biological Approvals
          Biological Approvals by Year
          Expanded Access to Experimental Biologics
          Vaccine Development – 101


                        Biologics License Applications (BLA) Process (CBER)
          Investigational New Drug Applications (INDs) for CBER-Regulated  Products
          Device Submissions for CBER-Regulated Products
          Labeling for CBER-Regulated Products
          Master Files for CBER-Regulated Products
          New Drug Application (NDA) and Abbreviated New Drug Application (ANDA) for CBER-Regulated Products
          Formal Meetings for CBER-Regulated Products Home
          Vaccines, Blood & Biologics
          Development & Approval Process (CBER)
          Center for Biologics Evaluation and Research Patient Engagement ProgramDevelopment & Approval Process (CBER) Center for Biologics Evaluation and Research Patient Engagement Program
  
  					Bringing the Patient Voice into Medical Product Development
	
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Advancing Patient Engagement and Patient-Focused Drug Development at CBER
	CBER Engagement with Patient Organizations
	Request a Patient Organization Meeting with CBER StaffPatient input is a critical part of the Center for Biologics Evaluation and Research’s (CBER) understanding of diseases and conditions. Engaging with patients and listening to their perspectives on their diseases and treatments may help the Center advance patient-focused medical product development. Patient engagement, and the development and application of patient input methods are included in the CBER 2021-2025 Strategic Plan (Objectives 1.2, 1.3, and 2.2). Supported by provisions in both the 21st Century Cures Act of 2016, the Food and Drug Administration Reauthorization Actof 2017 and the Prescription Drug User Fee Act VII CBER’s patient engagement program is committed to incorporating patient involvement in the Center’s regulatory work.

Advancing Patient Engagement and Patient-Focused Drug Development at CBER

**CBER supports Agency-wide efforts to advance patient engagement and patient-focused medical product development**

CBER interacts with patient groups on Center-specific topics and actively engages in Agency programs, initiatives, and events to gather patient input and share best practices for involving patients in medical product development and regulation. CBER’s patient engagement program collaborates closely with the Center for Drug Evaluation and Research (CDER), the Center for Devices and Radiological Health (CDRH), the Office of the Commissioner and other FDA offices and programs to coordinate patient engagement activities and patient-focused medical product development efforts, and to share best practices.

Programs at CBER that support advancement of patient engagement and patient-focused medical product development include CBER’s Science of Patient Input (SPI) initiative and CBER’s Rare Disease program. SPI initiative activities include supporting studies on methods and tools to obtain robust patient input to support biological product regulatory reviews and providing CBER reviewers with assistance in the regulatory review of patient input and patient-reported outcomes data. CBER’s Rare Disease program works to facilitate the incorporation of the patient perspective in regulatory decision making for biologics for rare diseases.


FDA Offices and teams CBER works with on cross-cutting patient engagement issues

CBER collaborates with FDA offices and patient engagement teams on cross-cutting patient engagement meetings, workshops, and activities:

CBER Rare Disease Program
	FDA Patient Affairs Staff
	FDA Stakeholder Engagement Staff
	CDER Professional Affairs and Stakeholder Engagement (PASE)
	Center for Devices and Radiological Health (CDRH) Patient Science and Engagement Program
	CDER Patient-Focused Drug Development
	Oncology Center of ExcellenceBack to Top

FDA Guidances on Patient-Focused Drug Development and Patient Preference

FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making*CBER and CDER are collaborating to develop methodological PFDD Guidances in accordance with 21st Century Cures Act and The Food and Drug Administration Reauthorization Act of 2017 Title I*
	Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data (Draft Guidance)*A CBER and CDER draft guidance on how a person seeking to develop and submit a proposed draft guidance relating to patient experience data for consideration by FDA may submit such proposed draft guidance to the Agency**CBER and CDRH collaborate on the following guidances about including patient input in the development, evaluation, and post-market surveillance of medical devices:*

Patient Preference Information – Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling
	Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications
	Factors to Consider When Making Benefit-Risk Determinations for Medical Device Investigational Device Exemptions
	Patient Engagement in the Design and Conduct of Medical Device Clinical Investigations
	Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device EvaluationBack to Top
 

CBER Engagement with Patient Organizations

**CBER engages patient organizations on disease- and therapy-specific issues.**

CBER staff make every effort, subject to staff resources and availability, to hear directly from patients about the burden of disease, the impact of treatment on patients, and other information that could inform regulatory decision making. This includes:

Participating in FDA-led patient-focused drug development (PFDD) meetings
	Attending externally-led PFDD meetings
	Bringing together important stakeholders and FDA staff through the RegenMedEd series to discuss foundational information about regenerative medicine therapies, such as gene and cell therapy products, and explore opportunities for patients, caregivers, and advocates to engage with FDA to help advance product development.
	Participating in or attending external conferences dedicated to patient engagement issues
	Coordinating with CBER’s Rare Disease Program on rare disease patient stakeholder engagement as well as participating in rare disease listening sessions organized by FDA’s Patient Affairs Staff and the National Organization for Rare Disorders (NORD) with patient stakeholder organizations.
	Responding to and facilitating meetings with patient stakeholder organizations on disease- and treatment-related issues
	Engaging with individual patient and caregivers through the FDA Patient Representative ProgramWays for patients to engage with the FDA on disease- and therapy-related issues

Learn About FDA Patient EngagementInformation about how patients and stakeholders can engage with FDA centers and offices, including CBER
	FDA-Led Patient-Focused Drug Development MeetingsMeetings held by CBER and CDER between 2013 and 2017 to systematically obtain the patient perspective on specific diseases and their treatments
	Externally-Led PFDD MeetingsCBER participates in this FDA program that welcomes patient stakeholder organizations to generate public input on disease areas using FDA-led PFFD meetings as a model
	Condition-Specific Meeting Reports and Other Information Related to Patients' Experience*This webpage provides links to certain publicly available external reports and resources relating to patient experience data. The patient community, patient advocates, researchers, drug developers, and federal agencies may find these materials useful. For specific questions related to a report or resource, FDA recommends reaching out to the point of contact listed on the cover page of the report.*Back to Top

Request a Patient Organization Meeting with CBER Staff

Meetings between patient organizations and CBER staff present opportunities to enhance CBER’s understanding of disease burden and the impact of treatment on patients. These meetings may also help to inform efforts by patient organizations working to advance drug development such as developing or conducting natural history studies or patient input studies to support medical product development.

To request a meeting with CBER staff, a representative from a patient organization can email CBERPatientEngagement@fda.hhs.gov. CBER will review requests and make every effort to hear directly from patient organizations according to staff availability and resources.

If a patient organization requests a meeting with topic that applies to more than one medical product type, the organization can submit a request through the Patients: Ask FDA page. Patients: Ask FDAcan also help patient organizations that are unsure which FDA center or program to contact about engaging with FDA.

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What CBER can learn from patient organizations

**Your organization’s activities, for example**

Natural history studies
	Patient-reported outcomes studies
	Patient surveys on risks, benefits, and preferences related to treatments**Impact of the disease and its treatment**

Chief complaints (most bothersome signs and symptoms)
	Burden of living with and managing a disease or condition
	Impacts from a disease or condition on activities of daily living and functioning**Perspectives about current and potential treatment approaches**

Expectations of benefits
	Tolerance for harms or risks
	Patient preference
	Unmet medical needs**Clinical trial considerations**

E.g., views on various therapies, such as gene therapy
	Burden of participating in clinical studies
	Risk tolerance

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                                                                                  Content current as of:
      09/08/2023
              
              
Regulated Product(s)
		 BiologicsDevelopment & Approval Process (CBER)

          
                        2023 Biological Approvals
          2022 Biological Approvals
          Biological Approvals by Year
          Expanded Access to Experimental Biologics
          Vaccine Development – 101


                        Biologics License Applications (BLA) Process (CBER)
          Investigational New Drug Applications (INDs) for CBER-Regulated  Products
          Device Submissions for CBER-Regulated Products
          Labeling for CBER-Regulated Products
          Master Files for CBER-Regulated Products
          New Drug Application (NDA) and Abbreviated New Drug Application (ANDA) for CBER-Regulated Products
          Formal Meetings for CBER-Regulated Products
 
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