GENERAL OVERVIEW OF MANAGED ACCESS PROGRAM
Sarepta is committed to developing innovative therapies for patients with Duchenne muscular dystrophy (DMD). We take our responsibility to act as a true partner with patient communities seriously, and we are committed to working with physicians who have determined that their patient has an unmet medical need.
We believe that well-designed clinical trials are the best way to determine the safety and effectiveness of investigational therapies and for patients to gain access prior to regulatory approval. However, we understand that patients and families may be interested in accessing our investigational therapies prior to regulatory approval, and outside of the clinical trial setting, through managed access programs (also known as an early / expanded access, or named patient program).
A managed access program (MAP) is a mechanism through which physicians can prescribe, within their professional responsibility, an unapproved treatment for patients. Use of an unapproved treatment is based on physicians’ informed assessment of the patients under their care to determine whether there is a special need for the unapproved treatment because (a) there is no approved treatment available or (b) available treatments are not suitable for that patient. For this MAP, physicians will need to assess patient eligibility by determining whether the patient meets pre-specified medical and other criteria and also confirming funding can be secured.
MANAGED ACCESS PROGRAM ELIGIBILITY FOR ETEPLIRSEN
For certain patients in certain jurisdictions, as part of our commitment to facilitating patient access, we support a limited managed access program in selected geographies where eteplirsen is not currently approved. We plan to expand the program to include more countries over time.
COUNTRIES WHERE THE PROGRAM IS OFFERED
Argentina, Austria, Australia, Belgium, Brazil, Canada, China, Colombia, Cyprus, Denmark, France, Germany, Greece, Hong Kong, Iceland, India, Ireland, Italy, Japan, Mexico, Norway, Portugal, Russia, Singapore, South Korea, Spain, Sweden, Switzerland, Turkey, and United Kingdom.
All requests must be submitted by the treating physician on behalf of the patient.
HOW DO I SUBMIT A REQUEST FOR MANAGED ACCESS?
Healthcare providers can obtain details about submitting requests by calling a Clinigen representative at +44 1283 494 340, or emailing firstname.lastname@example.org.
HOW DO I SUBMIT A MEDICAL INFORMATION REQUEST FOR THE MANAGED ACCESS PROGRAM?
If you need additional medical information in connection with a request for medication through this program, Healthcare Providers and Health Authorities can submit requests to email@example.com.
CLINICAL DEVELOPMENT PROGRAMS
At this time, we are unable to provide managed access or compassionate use programs for our other investigational therapies or additional treatment modalities currently in clinical development. We will continue to evaluate the possibility of compassionate use and pre-approval access mechanisms in the future and will institute such programs should it be feasible to conduct such a program in a fair and sustainable way without compromising clinical development and / or potential regulatory approval of any of our investigational therapies.