Managed Access Program
As a company, we are dedicated to developing innovative therapies for patients suffering from rare and life-threatening neurological genetic diseases. Before these therapies can be approved and commercially available to patients, they must undergo clinical trials to evaluate their safety and effectiveness.
While it is generally preferred that patients gain access to investigational therapies by participating in clinical trials, patients may not be eligible or able to take part in these studies. Patients with serious or life-threatening diseases or conditions sometimes seek therapies that are not yet approved or available in their country. Novartis Gene Therapies “Managed Access Program” addresses this need by making certain investigational or unapproved therapies available to eligible patients.
Clinical Trials
Before an investigational therapy can be placed on the market, it must undergo well-controlled clinical trials to evaluate its safety and effectiveness, as well understand whether its potential benefit to patients outweighs the possible risks. Clinical trial results and related product information are then submitted to the relevant health authorities for review. Clinical trials result in the generation of evidence that may lead to the approval of an investigational therapy, which can make it more widely available to patients.