Breakthrough Therapy Designation is introduced as a new designation with the passage of the FDA Safety and Innovation Act of 2012 (FDASIA) . According to section 902 of the FDASIA, the general criteria according to which this new designation can be applied are (1) serious or life-threatening disease or condition and (2) the drug also demonstrates substantial improvement over existing therapies on one or more clinically significant endpoints. Once a drug is designated as a Breakthrough Therapy, the FDA and the drug sponsor work closely to determine the most efficient pathway for generating additional evidence about safety and efficacy. As of March 31, 2015, a total of 293 requests for Breakthrough Therapy designation have been submitted, out of which 82 requests are granted .
This document outlines the application process for Breakthrough Therapy designation for novel drugs. It then proceeds with outlining the supporting data needed to apply for a Breakthrough Therapy designation for a novel anti-cancer molecule that has evidence of efficacy to treat patients with Chronic Lymphocytic Leukemia.
When to apply for designation request
The Breakthrough Therapy designation is granted on the basis of evaluating preliminary clinical evidence obtained from clinical trial data in humans. The data from animal studies or studies conducted in vitro are insufficient to defend this Breakthrough Therapy designation  . Therefore, a sponsor usually submits a Breakthrough
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1-A new investigational new drug application (IND). Expanded access with the new investigational new drug application (IND) involves submitting a new IND or there is an existing IND in effect for the drug, but the sponsor of the existing IND declines to be the sponsor of the access use through a licensed physician (21 CFR 312.310). There is a review period of 30 days, and during this period the treatment cannot proceed (21 CFR 312.40 and 312.305(d)(1)). However, the FDA could notify the sponsor to begin the treatment process earlier. The investigator should have an agreement with the sponsor to supply the investigational product to the patient and the health care professional for treatment.
Under this act only treatments for unmet medical needs would be eligible. Some helped to improve outcomes and reduce risk more often than those approved by the FDA. All of these drugs could not already be on the market or they would not be eligible. To do this it is required of sponsors to give up all other IP protections for indications protected under dormant therapy exclusivity (Usdin).
A drug can get priority Review designation if offer major advances in treatment, or provide a treatment where no adequate therapy exists. A priority Review means that the time it takes FDA to review a New Drug application (NDA) is reduced. The goal for completing priority review is six months whereas Standard review process takes ten months. The priority review status can apply both to drugs that are used to treat serious disease and drugs for less serious illness. The FDA is giving additional attention and resources towards the drug approval process which have potential significant advances in a treatment. In the period 1999–2011, 100 FDA-priority review pharmaceuticals were approved by both the FDA and the EMA. The majority of the products were ﬁrst submitted to and approved by the FDA. The FDA has a signiﬁcantly shorter drug application review time than the EMA.
The purpose of clinical development is to bring new drugs and therapies to patients. These drugs and therapies are studied and researched to be used for humans. Clinical development is very expensive and time consuming. The process of discovering a new drug, to having it approved for patient use, is approximately twelve years. The average cost to complete this process
* No foreseeable new product in pipeline. Currently takes up to 11,000 compounds to be screened to find one (1) compound to send through final testing (human trials). And even then it’s not guaranteed to make it past the FDA.
How does the Orphan Drug act affect the drug industry’s viability in continuing research and development of medications for the rare disease population?
“It is critical to the nation’s future health that innovative practices and biomedical and clinical research be promoted and incentivized” (Torrens & Williams, 2009). It is the medical research team that has to develop the drug, device or technology and make it accepted and available for patients and clinicians to use. “This phase involves private companies and private capital almost entirely and calls for a mixture of scientific promotion to the technical expert community and broader general marketing to the health care system that must ultimately approve it for purchase and clinical use” (Torrens & Williams, 2009). It is through clinical trials that will eventually gain the FDA approval, if a drug or medical device is deemed safe for the community to use. “Once the appropriate regulatory approvals are obtained for a new device or drug, the development process moves into an active marketing phase designed to encourage the use of the product by individual clinicians and
(i) On 2009, the FDA promulgated a program called the “Expanded access” or“Compassionate use” ( 21 CFR 312.1) which allows the patients to have an access to a Investigational Medicinal product which is subjected to Investigational New Drug (IND) application (according to an IND 21 CFR 312.8). The FDA in 2009 revised the regulation of “Expanded access” because there was no clear regulation related to different types of patients, and the access to
This is the outcome the community is trying to avoid. Scientists, doctors, and family are worried that there will not be a trial with the numbers to get a drug approved by the Food and Drug Administration (FDA) for either treatment nor a possible cure. The FDA shared that 17 of the 41 new drugs it approved in 2014 for rare diseases.
In 2002, data on gefitinib/Iressa, the first targeted therapy subsequently to win approval in NSCLC and the first drug developed simulataneously in the US, Europe and Japan were submitted.
The 505(b)2 mechanism has notably changed how pharmaceutical companies can submit to the Food and Drug Administration. Through the 505(b)2 mechanism, sponsors can apply to market a drug without preclinical studies and Phase 1 through 4 studies. If sponsor handles the process correctly, the Food and Drug Administration will approve their drugs rapidly and the sponsor will incur less cost in developing the drug. However, use of the 505(b)2 mechanism poses its own set of challenges. If it is mishandled during the early stages, it can result in a product failing to be approved. As such, sponsors should understand the common issues associated
CLL is caused by mature, monoclonal B-cells that exhibit round nuclei, condensed chromatin and scant cytoplasm. These B-cells co-express Cd5, CD19, as well as CD38. They have a dim expression of CD 20, CD22 and CD23 and have weak surface immunoglobins. In severe cases Zap-70 will be expressed. In addition these cells are typically lacking certain antigens that would normally be present (CD10, FMC7, and CD79b). An interesting little tidbit: All B- cell CLL lines are positive for Epstein Barr virus (Leukemia).
Prior to the amendments, only safety of the drug needed to be proven to be approved by FDA. The amendments now required new drugs to show efficiency as well as safety--which means drugs need to show that their benefits outweigh risks in order to be approved. The three phases of clinical trials were now done to prove the drug’s safety and efficiency--phase I trials typically assess data on safety; phase II trials evaluate efficacy; and phase III trials compare the potential drug with standard therapy, usually on a randomized, double-blind basis (Jonsen & Stryker, 1993).
Identification of device, drug, biologics, or combination product status and the appropriate regulatory classification and pre-market submission pathway in jurisdictions where the product will be marketed