 Innovative Therapies for Serious Diseases
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Innovative Therapies includes correcting a defective gene, inserting a gene-drug, grafting cells capable of regenerating healthy tissue. In patients with rare, severe immune-mediated inflammatory diseases (IMIDs), biologics are often the last treatment option when standard therapy with classical immunosuppressive drugs fails. However, clear guidelines on biologic prescription in these diseases are often lacking. A major reason is that proper randomized controlled trials (RCTs) are difficult to design due to the low prevalence, heterogeneity of patients, and the severe medical condition of patients. Since RCTs are usually required for registration of therapy for a specific disease, physicians often prescribe biologics off-label in these diseases. Off-label prescription can be defined as the prescription of a drug for conditions other than current registered indications. This means that the drug is approved by either FDA or EMA for a certain disease, yet there is no definitive proof for the efficacy of the specific drug for other conditions. The physician’s decision to prescribe off-label in such cases is often based on the pathophysiology of the disease or shared symptoms with other diseases in which the biologic has proven to be effective. This is called rational prescription and may lead to innovative treatment options, especially in rare diseases.
- Types of inovative therapies
- Problems involved in innovative therapies
- Breakthrows
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 Innovative Therapies for Serious Diseases Conference Speakers
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- Advancements in Biotherapeutics
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- Â Immunopharmacotherapy
- Â Innovative Therapies for Serious Diseases
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