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Rare Diseases Drug Development
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Shein-Chung Chow, Shutian Zhang, Wei Zhang
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). To determine whether the improvement over available therapy is substantial is a matter of judgment and depends on both the magnitude of the treatment effect, which could include the duration of the effect and the importance of the observed clinical outcome. In general, the preliminary clinical evidence should show a clear advantage over available therapy.
Rare Diseases Drug Development
Published in Shein-Chung Chow, Innovative Statistics in Regulatory Science, 2019
FDA’s incentives program include (i) fast track designation (ii) breakthrough therapy designation (iii) priority review designation and (iv) accelerated approval for approval of rare disease drug products. In its recent guidance, however, the FDA emphasizes that the FDA will not create a statutory standard for approval of orphan drugs that is different from the standard for approval of other, more typical drugs (FDA, 2019). For approval of drug products, the FDA requires that substantial evidence regarding effectiveness and safety of the drug products be provided. Substantial evidence is based on the results of adequate and well-controlled investigations (21 CFR 314.126(a)).
Challenges Facing the American Healthcare System
Published in Kant Patel, Mark Rushefsky, Healthcare Politics and Policy in America, 2019
Breakthrough Therapy designation expedites the development and review of drugs that are intended to treat a serious medical condition and where clinical data indicates that the drug may demonstrate significant improvement over available therapies. A drug with this designation is also available for fast-track designation.
The use of ibrutinib before and after allogeneic stem cell transplantation
Published in Expert Opinion on Orphan Drugs, 2019
Massimo Martino, Anna Ferreri, Virginia Naso, Tiziana Moscato, Barbara Loteta, Massimo Gentile, Antonella Morabito, Fabio Provenzano, Michele Cimminiello, Angelo Michele Carella, Giuseppe Console, Anna Grazia Recchia
Ibrutinib has demonstrated considerable efficacy in a variety of B-cell malignancies [18–23] and the US Food and Drug Administration (FDA) formally designated the drug as a breakthrough therapy [24], approving for the treatment of patients with relapsed and refractory (R/R) CLL and as first-line therapy for patients with del(17p) or TP53 mutation ith CLL, patients with CLL 17p deletion, MCL, and Waldenstrom’s macroglobulinemia (WM). In August 2017 the FDA approved ibrutinib as a second line treatment for cGVHD. Breakthrough therapy designation is meant to expedite the development and review of drugs that are intended to treat a severe condition and have sufficient preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement in at least one clinically significant endpoint over currently available therapies (Table 1) [25].
Early clinical trials of Toca 511 and Toca FC show a promising novel treatment for recurrent malignant glioma
Published in Expert Opinion on Investigational Drugs, 2019
Brandon D. Philbrick, D. Cory Adamson
The FDA has granted the Toca 511 and Toca FC combination therapy the ‘Breakthrough Therapy’ designation for rHGG. This status is given to drugs under development to treat serious conditions and have demonstrated early clinical evidence that the drug may offer a substantial improvement and advantage in the treatment of a disease compared to standard of care therapies. Other glioma drugs with the Breakthrough Therapy designation included rindopepimut (phase III trial terminated for failure to display clinical benefit) and the currently under-development PVSRIPO (an oncolytic polio:rhinovirus recombinant). Drugs designated with the Breakthrough Therapy designation are eligible for expedited review by the FDA to facilitate development. Toca 511 and Toca FC has also received Priority Medicine (PRIME) designation from the European Medicines Agency in July 2017 (https://www.prnewswire.com/news-releases/tocagen-receives-european-medicines-agency-priority-medicines-prime-designation-for-toca-511-in-high-grade-glioma-300492648.html), Orphan Drug designation, and an associated grant award from the FDA to support the Phase III trial (https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm579366.htm).
Esketamine for treatment resistant depression
Published in Expert Review of Neurotherapeutics, 2019
Jennifer Swainson, Rejish K Thomas, Shaina Archer, Carson Chrenek, Mary-Anne MacKay, Glen Baker, Serdar Dursun, Larry J. Klassen, Pratap Chokka, Michael L Demas
IN esketamine HCl, marketed by Janssen Pharmaceuticals as Spravato, was approved for use in conjunction with an oral antidepressant on 5 March 2019 by FDA of the United States of America for the treatment of depression resistant to prior antidepressant medication treatments. The application for approval was given Fast Track and Breakthrough Therapy designations. Ketamine, including its enantiomer esketamine, does not have an approved indication for Major Depressive Disorder or Suicidality at present in other countries. However, in Canada, esketamine is being reviewed under the Priority Review Policy of Health Canada, which fast tracks the review of the compound by the agency. Janssen also has a current Marketing Authorization Application for the European Union for esketamine.