In general, breakthrough therapy designation requests should not be submitted to a PIND. Sub-Contractors and Professional Services List. In addition, products that have been designated as fast track can obtain rolling review. Table 1: Fast Track Designation Products Statistics Since Inception. Upon designation, the FDA and sponsor would collaborate in a dynamic and cross-disciplinary process to determine the most efficient path forward. A breakthrough therapy designation can apply for a combination product (drug-device, biologic-device) as long as the primary mode of action in the combination product is a drug or biologic. Even if [Sponsors] request preliminary BTDR advice, the Division may not have enough information to determine if a BTDR is appropriate at this time. If a sponsor is in need of help with their BTD application, the FDA does offer optional guidance prior to the formal submission. Note that a drug that has received a breakthrough therapy designation or a fast track designation can be eligible for the accelerated approval pathway, if the relevant criteria are met. After a recommendation from the U.S. Food and Drug Administration (FDA), CytoDyn is planning to ask the regulatory agency for a preliminary meeting to discuss the granting of breakthrough therapy status to leronlimab for the treatment of metastatic triple-negative breast cancer (TNBC). Upon designation, the FDA and sponsor would collaborate in a dynamic and cross-disciplinary process to determine the most efficient path forward. Home; Uncategorized; breakthrough therapy designation guidance; breakthrough therapy designation guidance The breakthrough status for a given drug is not disclosed by the FDA until it receives final approval. However, other observed clinical endpoints that may showcase medical superiority can include: The BTD is just one of several drug designation programs that the FDA offers in an effort to expedite certain therapies to market. 2) Request for designation- The sponsor of a drug may request the Secretary to designate the drug as a breakthrough therapy. If you are in the process of applying for Breakthrough Therapy Designation, contact us today to learn how we can help you submit a successful application. to learn how we can help you submit a successful application. Therefore, in deciding which of these designations to apply for, as well as considering the associated benefits, Sponsors must examine the requirements in light of the specific data package available for the product. 200 S Anaheim Boulevard 1st Floor, Suite 145 Anaheim, CA 92805 Map. If a drug is denied breakthrough therapy designation, is it automatically reviewed for fast track designation? The review division managing the investigational new drug application (IND) for the drug in question is the sponsors first resource for questions related to the development program of the specific drug, and its potential for breakthrough therapy qualification. A drug development program may qualify for more than one expedited program. Can a request for a breakthrough therapy designation be submitted for a combination product? The preliminary clinical evidence that the drug may demonstrate substantial improvement over available therapies; FDA will respond to Breakthrough Therapy designation requests within 60 calendar days of receipt of the request. The site is secure. FDA. Scendea recommends that this information is captured in approximately 10 to 20 pages. %
By submitting a preliminary Breakthrough Therapy designation advice form, sponsors can obtain preliminary, non-binding advice from the FDA about whether a formal request is appropriate, based on available data - or whether the request would be too preliminary because it does not currently meet the criteria. The Sponsor submitted a letter requesting Preliminary Breakthrough Therapy Designation Request Advice on November 4, 2016. The Preliminary BTDR Advice Request appears to be a way to formalize these inquiries and encourage Sponsors to open a dialogue about BTD eligibility before submitting an official BTDR. Requesting breakthrough designation - March 2018 - Cardinal Health Is there a deadline for a sponsor to submit a request for breakthrough therapy designation? In this two part interview, Dr. Rich Moscicki takes a deep dive into FDA's Breakthrough Therapy designation to help clarify common misunderstandings around this expedited . If the Agency determines that a Breakthrough Therapy designation request was incomplete or that the drug development program failed to meet the criteria for Breakthrough Therapy designation, the Agency will send a non-designation letter to the sponsor. Requests for breakthrough therapy designation should not be submitted to inactive INDs or INDs that are on partial or complete clinical hold. Section 506(g)(5) of the FD&C Act specifies that these early interactions may be used to discuss potential surrogate or intermediate endpoints to support accelerated approval. 10 FDA Designations First-in-Class: Drugs with a new and unique mechanism for treating a medical condition Orphan Drugs: Drugs approved for small populations of patients with rare diseases (<200,000 patients)* Fast Track: Drugs that can treat an unmet medical need for a serious condition Breakthrough: Drugs for a serious condition with preliminary clinical evidence . Drugs which are designated as a breakthrough therapy showcase significant improvement upon current therapies and its planned outcome. Some of the key benefits of Breakthrough Therapy designation include: Sponsors should consider requesting Breakthrough Therapy designation only after they have preliminary clinical evidence demonstrating that the therapy has a clear advantage over available therapies. Ideally, a designation request should be requested prior to the end-of-Phase-2 meetings. Their enterprise value is $2.0 billion but their numerous cancer indications make them an ideal . Last month, the Company reported that the FDA had recommended such a meeting to provide preliminary advice prior to resubmission of a Breakthrough Therapy designation request. City Hall. in some cases, fda may suggest that the sponsor consider submitting a request for breakthrough therapy designation if: (1) after reviewing submitted data and information (including preliminary. A therapy intended to treat a serious condition and nonclinical or clinical data shows the potential to address an unmet medical need, A drug that has been designated as a qualified infectious disease product, A drug that is intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinical endpoint over currently available therapies, As soon as sufficient data is available to show the drugs potential to address an unmet medical need, Actions to expedite drug development and review, Intensive guidance on an efficient drug development program, beginning as early as Phase 1, Organizational commitment involving senior managers, Post reviewing the submitted data and information (including preliminary clinical evidence), the FDA believes the drug development program may meet the qualifying criteria for Breakthrough Therapy designation, The remaining drug development program can benefit from the designation. So, it is unclear that Sponsors will use the Preliminary BTDR Advice Request since they are able to get an actual determination on an official BTDR with fairly limited effort, within 60 days of FDAs receipt of the request. Fast Track designation can be requested with nonclinical data and/or preliminary clinical evidence. An overview of PRIME, Breakthrough Therapy & Fast Track procedures for designation of a drug as a breakthrough therapy ". . If youre interested in pursuing a Breakthrough Therapy designation for your product, here are some of the basics youll need to know. Expedited drug review process: Fast, but flawed. CytoDyn Files Request with FDA for Preliminary Meeting for Breakthrough Guidance for Industry . Learn more about how Cardinal Health is improving healthcare. 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The Preliminary BTDR Advice Request appears to be a way to formalize these inquiries and encourage Sponsors to open a dialogue about BTD eligibility before submitting an official BTDR. $7Q=.zkxxHj%34U Breakthrough Therapy Designation (BTD) is a rather new drug designation category that was signed into law with the approval of the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012. This will greatly increase the chance of earlier approval if the product qualifies for accelerated approval or priority review, and expedite patient access to the drug. Address an emerging or anticipated public health need. Drugs which are designated as a breakthrough therapy showcase significant improvement upon current therapies and its planned outcome. At that time, DPP had concerns about the plan for long-term human exposure due to neurotoxicities observed in the non-clinical studies in dogs. A request for the designation may be made concurrently with, or at any time after, the submission of an application for the investigation of the drug under section 355 (i) of this title or section 262 (a) (3) of title 42 . Nevertheless, even after the drug enters the market, the sponsor may be required to conduct post-marketing trials to verify and describe the drugs clinical benefit. The Food Drug and Cosmetic Act (21 USC 356) states that a request for a breakthrough therapy designation may be made concurrently with, or at any time after, the submission of an application for [the IND]. Breakthrough Therapy Designation Application Timeline. Huntersville, NC 28078 An official BTDR may be required to make a determination. The .gov means its official.Federal government websites often end in .gov or .mil. Discover resources on how to navigate the regulatory affairs process, including medical writing, FTE models, eCTD and marketing applications. However, there are some limited circumstances where this may be acceptable, if done at the request of the FDA. Preliminary, non- binding advice on whether an official request for breakthrough therapy designation is appropriate may be requested when an . A medication qualifies as a breakthrough therapy if it offers new benefits over existing treatments. Introduction. No written documentation of the advice provided by the Division or minutes of the telecon will be issued to the Sponsor. May a sponsor submit a request for Special Protocol Assessment (SPA) for a drug that has breakthrough therapy designation? The program aims to facilitate the development and expedite the review of drugs and biologics to treat serious conditions and fill an unmet medical need. Since its implementation in 2012, more than 100 drugs have obtained this designation and already 39 . Requests for Breakthrough Therapy designation should be submitted to Module 1, Section 1.12.4 Request for Comments and Advice of the IND. This template should then be submitted as a formal amendment to the IND and a subsequent teleconference between the sponsor and the review division will be set-up by the RPM. The FDA response time for BTD is within 60 calendar days of receipt of the request.