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Off-label use of medicines between clinical research and practice
Published in Andrea Parziale, The Law of Off-label Uses of Medicines, 2023
The outlined medical and legal definitions of off-label use share a similar structure. This consists of two cumulative elements. The first is that the medicine used “off-label” must be already authorised for a given indication. This means that off-label uses do not include the uses of medicines that have not been authorised for any indication whatsoever in a specific jurisdiction. In fact, the use of unauthorised medicines is generally prohibited and is only allowed under exceptional circumstances, for example, under so-called expanded access in the USA17 or compassionate use programs in the EU and the UK.18 In essence, such programs enable the use of unauthorised medicines to address severe conditions for which valid therapeutic alternatives do not exist.19 This book focuses on off-label uses and, therefore, does not elaborate further on the use of unauthorised medicines.20
Drug Repurposing and Novel Antiviral Drugs for COVID-19 Management
Published in Debmalya Barh, Kenneth Lundstrom, COVID-19, 2022
Shailendra Dwivedi, Aakanksha Rawat, Amit Ranjan, Ruchika Agrawal, Radhieka Misra, Sunil Kumar Gupta, Surekha Kishore, Sanjeev Misra
Another trial on MSCs in March–April 2020 was reported by Pluristem Therapeutics Inc., a leading regenerative medicine company in Haifa, Israel. Successful treatment was obtained in patients suffering from COVID-19 complications in the United States [59]. The treatment was conducted according to the US FDA Single Patient Expanded Access Program, also called the Compassionate Use Program. This is part of the US Coronavirus Treatment Acceleration Program (CTAP), an emergency program for encouraging therapies that explore every existing method to move new treatments to patients as quickly as possible.
The Food and Drug Administration
Published in Stanley R. Resor, Henn Kutt, The Medical Treatment of Epilepsy, 2020
The IND may be commercial or noncommercial. A sponsor who is a pharmaceutical firm will be collecting efficacy and safety data with the goal of submitting a New Drug Application (NDA), which, if approved by the FDA, permits marketing of the drug for specified uses. A noncommercial IND allows the sponsor to use the drug in research or early clinical studies designed to advance scientific knowledge. Both the commercial and noncommercial IND must supply specific data, some of which may be difficult for a noncommercial sponsor to provide. Express consent must be obtained from the original sponsor of the IND to cross-reference the data. The FDA is prohibited from acknowledging or disclosing the existence of an IND. Thus, a researcher seeking access to a new AED must, after identifying the holder of the IND, either obtain permission to cross-reference an existing IND or seek permission form the original IND sponsor to be added as an investigator to the existing IND. The issue of treating individual patients as a “compassionate use” or “treatment IND” will be discussed later.
Educational needs, perception, and perspectives of oncologists regarding compassionate use programs in Asia
Published in Current Medical Research and Opinion, 2021
Manmohan Singh, Ankita Jain, Wade Fang, Peter Ong, Roberto Uehara, Jingming Zhong
The importance of simplifying the application process and providing guidance on CUPs is demonstrated by the success of FDA initiatives in this regard. The FDA has been supportive of the compassionate use of drugs to eligible patients. In a step aimed at helping oncologists with the application process, Project Facilitate was launched on 31 May 2019. Under the aegis of this project, a call center was set up to guide oncologists through the expanded access process and collect data provided by oncologists on outcomes of expanded application. In the initial 3 months itself, there was a 20% increase in the number of applications for oncology drugs received by the agency compared to the same period in the previous year. Apart from Project Facilitate, the FDA.gov website also provides information, including relevant FDA contact information, to assist in applying for expanded access to investigational drugs in other disease areas24. Similar initiatives may help other parts of the world.
Remdesivir emergency approvals: a comparison of the U.S., Japanese, and EU systems
Published in Expert Review of Clinical Pharmacology, 2020
A Saint-Raymond, J Sato, Y Kishioka, T Teixeira, C Hasslboeck, SL Kweder
A Compassionate Use opinion can be issued at the EU level for medicines intended for patients with a chronically or seriously debilitating disease or whose disease is considered to be life-threatening, and who cannot be treated satisfactorily by an authorized medicinal product. A compassionate use opinion may be requested by any EU Member State to the European Medicines Agency Committee for Medicinal Products for Human Use (EMA CHMP) for medicines undergoing clinical trials or under review. Each Member State is then free to make use of this opinion at the national level and only needs to inform EMA when it does so. The request for a centralized opinion does not come from the company, but the company must submit available data, and is obliged to adhere to a set of general conditions related to manufacturing, distribution, and safety monitoring. An opinion by the CHMP does not prevent the Member States from recommending the medicine as part of their national systems of compassionate use, yet the Regulation does mention ‘ … a common approach should also be followed, whenever possible, regarding the criteria and conditions for the compassionate use of new medicinal products under Member States’ legislation,’ with the aim to meet the legitimate expectations of patients in the Union. Compassionate use programs complement the possibility of enrolling patients in clinical trials.
Systematic review of extracellular vesicle-based treatments for lung injury: are EVs a potential therapy for COVID-19?
Published in Journal of Extracellular Vesicles, 2020
Kasra Khalaj, Rebeca Lopes Figueira, Lina Antounians, Giuseppe Lauriti, Augusto Zani
Lastly, patient selection is crucial and should be examined closely. Clinical parameters and possibly available biomarkers may be helpful to identify the inclusion and exclusion criteria. Ideally, patients should be enrolled in carefully designed clinical trials, where phase 1 would inform on the safety of EVs (escalating doses), phase 2 would test EV efficacy and side effects (therapeutic doses), and phase 3 would determine EV efficacy, effectiveness and safety [114]. Following this systematic approach, an EV-based therapy might eventually be approved by national regulatory authorities for use in the general population. Alternatively, especially during a crisis period like this pandemic, patients could be offered an EV therapy outside of a trial through compassionate or named patient use. Compassionate use is restricted to patients who are not responding to conventional treatment strategies and have developed an immediate life-threatening clinical status, who do not meet the inclusion criteria for a trial if one is available, and whose caregivers agree that the benefit justifies the potential risks of the treatment [115]. Named patient use refers to the treatment of a single individual with an unauthorized product, under the direct responsibility of their physician [116]. We anticipate that during these unprecedented times, every effort is made by ethical boards and regulatory agencies to streamline and expedite the process [117]. However, the high quality of research should be maintained by respecting regulatory standards.