SUPPLEMENTARY INFORMATION:

I. Background

FDA regulates drugs, including those that are licensed as biological products, under the Federal Food, Drug, and Cosmetic Act (FD&C Act), the Public Health Service Act (PHS Act), and relevant implementing regulations to promote and protect the public health.

The central mission of CDER's Office of New Drugs (OND) is the proper and, where appropriate, flexible implementation of review authorities to ensure that safe and effective drugs and biologics are available to the American people. Rapid scientific and technological advances have enhanced our understanding of disease pathologies and, in many cases, have identified potential actionable targets that translate into drug development programs with an increased emphasis on precision medicine, rare diseases, and defined subsets of common diseases. FDA can help to ensure these innovations reach patients through the development and dissemination of clear policies regarding our expectations and standards.

FDA is engaged in multiple, high-priority policy initiatives to facilitate effective drug development. Effective drug development programs leverage the best available scientific knowledge to characterize the benefits and risks of a potential product and generate the data necessary to support product approval. Current FDA endeavors in this area include evaluating the potential use of real-world evidence in regulatory decision-making and the incorporation of the patient perspective into drug development and review. FDA has previously released comprehensive plans or frameworks for the advancement of these efforts; therefore, these topics are not the focus of this meeting. Instead, stakeholders are encouraged to provide input on these initiatives at topic-specific public meetings or in response to draft guidance.

Consistent with FDA's broader initiatives and modernization efforts, the clinical and scientific leaders of OND are seeking suggestions on where we can provide regulatory clarity to promote innovative and effective drug development across multiple therapeutic areas. FDA will hold a public meeting on November 7, 2019, from 9 a.m. to 5 p.m., to solicit input from stakeholders regarding where the Office of New Drugs can focus policy priorities while those broader Agency- and Center-wide efforts, described above, continue. In particular, OND welcomes specific policy suggestions that can be implemented in the near-term.

The format of the meeting involves presentations from the public to a panel of leaders from OND review divisions. The Agency will not be inviting specific presenters; rather, with this document, FDA is soliciting presentations from interested stakeholders. FDA also invites interested stakeholders to submit written comments to the docket on the topics described below.

II. Topics for Discussion at the Public Meeting

FDA is soliciting specific, actionable policy suggestions that could be implemented in the near-term by the review staff of CDER's Office of New Drugs to promote effective drug development programs without compromising our regulatory standards for the assessment of safety and effectiveness.

The Agency welcomes any relevant information that stakeholders wish to share at the meeting or in a submission to the docket, but we emphasize that the focus of this meeting is to seek input that is distinct from parallel, topic-specific initiatives related to real-world evidence and patient-focused drug development. Furthermore, to best inform policy priorities, we anticipate that the most informative suggestions would not be specific to a therapeutic area or disease but rather apply across multiple therapeutic areas or diseases. We are particularly interested in the topics that follow.

1. We are interested in input from stakeholders about where OND can provide additional guidance or prioritize additional scientific discussion in the near-term to improve clarity and encourage effective drug development. Given that OND's portfolio includes a diverse spectrum of drugs and diseases, such input should focus on specific policy needs for various clinical areas linked by a shared therapeutic context (e.g., drugs intended to treat serious, life-threatening rare diseases; non-serious, self-limited conditions; etc.), rather than focusing on any specific disease or condition.

2. Over the past decade, advances in scientific knowledge have led to unprecedented targeting of drugs to the underlying genetic or molecular pathophysiology of a disease. For many diseases, however, the current state of knowledge does not provide opportunities for such precise targeting, but patients living with these diseases require therapeutic innovation as well. Recognizing that each disease has unique considerations, we are interested in specific suggestions for guidance or policy development that OND could undertake to facilitate drug development for diseases not currently amenable to targeted therapies.

3. Some therapeutic areas, particularly those that include serious and life-threatening diseases, have begun to implement novel trial designs, such as the use of master protocols to study multiple therapies and/or multiple diseases under a common infrastructure. We are interested in stakeholders' views regarding the advantages and disadvantages of extending these approaches to additional therapeutic areas, and what guidance development would be most useful.

4. FDA has published many guidances intended to explain the Agency's current thinking regarding drug development topics that are not specific to a particular disease or indication. If stakeholders believe that OND review divisions are implementing these guidances in different ways, which are not explained by case-specific features, this may reflect a need for guidance revision or additional policy development. We are interested in hearing specific recommendations for topics where further clarity of the Agency's current thinking may be warranted.

5. Innovative approaches can bring additional uncertainty to drug development, since the advantages and disadvantages of the approaches may not yet be fully understood by either the Agency or sponsors because of their novelty. Sometimes, a well-understood development pathway may be chosen solely because of existing precedents in the therapeutic area. We would like to hear how OND can promote effective drug development programs when this tension exists. ( printed page 39858)

III. Participating in the Public Meeting

Registration: Persons interested in attending this public meeting must register online at promotingeffectivedrugdevelopmentprograms.eventbrite.com by midnight on October 10, 2019. Please provide complete contact information for each attendee, including name, title, affiliation, address, email, and telephone. Please also indicate whether attendance will be by webcast or in person. Early registration is recommended because seating is limited; therefore, FDA may limit the number of participants from each organization. If registration reaches maximum capacity, FDA will post a notice closing registration at www.fda.gov.

If you need special accommodations due to a disability, please contact Eithu Lwin (see FOR FURTHER INFORMATION CONTACT) no later than October 30, 2019.

Requests for Oral Presentations: During online registration you may indicate if you wish to present. To facilitate agenda development, registrants requesting to present will be contacted to provide information regarding which topics they intend to address and the title of their presentation. We will do our best to accommodate requests to present. Individuals and organizations with common interests are urged to consolidate or coordinate their presentations, and request time for a joint presentation, or submit requests for designated representatives to participate. All requests to make oral presentations must be received by the close of registration on October 10, 2019.

Following the close of registration, we will determine the amount of time allotted to each presenter and the approximate time each oral presentation is to begin, and we will select and notify participants by October 24, 2019. If selected for presentation, registered presenters planning to use an electronic slide deck should submit an electronic copy of their presentation (PowerPoint or PDF), to ONDPublicMTGSupport@fda.hhs.gov with the subject line “Promoting Effective Drug Development Programs: Opportunities and Priorities for FDA's Office of New Drugs” on or before October 31, 2019. If presenters choose not to use a slide deck, they are requested to submit a single slide with their name, affiliation, title of presentation, and contact information. Persons registered to present are encouraged to arrive at the meeting room early and check in at the onsite registration table to confirm their designated presentation time. No commercial or promotional material will be permitted to be presented or distributed at the public meeting.

Streaming Webcast of the Public Meeting: This public meeting will also be webcast. To join the meeting via the Webcast, visit collaboration.fda.gov/​ond110719/​.

If you have never attended a Connect Pro event before, test your connection at collaboration.fda.gov/​common/​help/​en/​support/​meeting_​test.htm. To get a quick overview of the Connect Pro program, visit www.adobe.com/​go/​connectpro_​overview. FDA has verified the website addresses in this document, as of the date this document publishes in the Federal Register, but websites are subject to change over time.

Transcripts: Please be advised that as soon as a transcript of the public meeting is available, it will be accessible at www.regulations.gov. It may be viewed at the Dockets Management Staff (see ADDRESSES). A link to the transcript will also be available on the Agency's website at www.fda.gov.

84 FR 39856