Compassionate use of cell products

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Compassionate use of cell products

Investigational medicinal products are available to patients through two pathways. The first is by participating in a clinical trial, the second as part of a compassionate use program, which is the use of an investigational medicinal product outside a clinical trial in patients with serious or life-threatening diseases for which no other treatment is available or expected to be effective. Usual...

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Everolimus for Compassionate Use in Multiple Basal Cell Carcinomas

Everolimus is an inhibitor of the mammalian target of rapamycin (mTOR) and has been shown to have antineoplastic activity in addition to its use as an immunosuppressive agent for the prevention of organ transplant rejection. We report the use of everolimus for the compassionate treatment of four elderly, nontransplant patients presenting with multiple basal cell carcinomas (BCC). All patients h...

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Compassionate use of orphan drugs

BACKGROUND EU regulation 726/2004 authorises manufacturers to provide drugs to patients on a temporary basis when marketing authorisation sought centrally for the entire EU is still pending. Individual Member States retain the right to approve and implement such 'compassionate use' programmes which companies will usually provide for free. Nevertheless some companies have opted not to partake in...

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Ethics review in compassionate use

BACKGROUND Compassionate use is the use of unapproved drugs outside of clinical trials. So far, compassionate use regulations have been introduced in the US, Canada, many European countries, Australia and Brazil, and treatment on a compassionate use basis may be performed in Japan and China. However, there are important differences between relevant regulations in individual countries, particula...

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Compassionate use of medicinal products in Europe: current status and perspectives.

The medical treatment of patients with chronic, life-threatening or seriously disabling diseases can be very disappointing both for the suffering patient and their physicians in cases where patients cannot be treated satisfactorily with currently authorized medicines. It may be possible to successfully treat such patients with new pharmaceuticals that have not yet been authorized by suitable cl...

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ژورنال

عنوان ژورنال: Revista Brasileira de Hematologia e Hemoterapia

سال: 2013

ISSN: 1516-8484

DOI: 10.5581/1516-8484.20130036