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Compassionate use policy for orphan drugs

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“Compassionate use is a treatment option that allows the use of an unauthorised medicine. Compassionate use programmes are for patients who have a disease with no satisfactory authorised therapies or cannot enter a clinical trial. They are intended to facilitate the availability to patients of new treatment options under development”- EMA site

“Global access programmes, known by various names including compassionate use, named patient, expanded access and early access, provide a mechanism by which patients with rare diseases are able to access medicines that would otherwise be unavailable.

For rare and orphan diseases, these programmes can facilitate access to:

-medicines that are in clinical development,

-drugs unlicensed in a specific market but licensed elsewhere,

-drugs under assessment in the marketing authorisation application (MAA) process.

Importantly, through a global access programme, patients can access an orphan drug that may never be commercially available in their home country or can gain access to a medicine outside the structure of a formal clinical trial.” PMlive site

Early access to a drug for patients may be possible before its marketing authorisation is granted to the pharmaceutical industry that develops it, most often during the third phase of the clinical trial and when its safety and efficacy are strongly assumed. Two cases may arise :

  • an application for marketing approval has either been or is about to be submitted by the pharmaceutical company developing the drug in the country concerned. Then the company submits a temporary regulatory approval to the administrative authority for a group of patients (Temporary Use Autorization cohort in France and in Italy, or compassionate use authorization in the other European countries) that is valid for a limited time span in the country considered.
  • or the physician asks the administrative authorities for a nominative temporary regulatory approval that is valid for a specific patient and for a limited time span in the considered country.”- Orphanet site
Access to orphan drugs in Europe
Country Early access Access Comments
Sources: EMEA, London.

Abbreviations :

  • UC : Compassionate Use
  • NP : Nominative Base of Patients
  • TUA : Temporary Use Authorisation
GERMANY No Easy Nothing particular
AUSTRIA UC/NP Slow Nothing particular
BELGIUM UC/NP Slow ++ Nothing particular
DENMARK UC/NP Complex Nothing particular
FINLAND UC/NP Complex Nothing particular
FRANCE TUA Rapid Coordination at OMS level
SPAIN UC/NP Classic Nothing particular
GREECE UC/NP Classic Nothing particular
IRELAND UC/NP Classic Nothing particular
ITALY TUA Classic Nothing particular
LUXEMBOURG UC/NP Classic Nothing particular
THE NETHERLANDS UC/NP Classic Improvement to be discussed
PORTUGAL Depends on the case Depends on the case Special funds awarded
THE UNITED KINGDOM UC/NP Slow Considered as expensive
SWEDEN UC/NP Easy Nothing particular

In the USA:

“Compassionate use can be granted for experimental drugs when patients do not participate in a clinical trial and when the pharmaceutical company is willing to provide the drug. The expanded access programs for compassionate use administered by NORD (National Organization for Rare Disorders in the US) work differently from the European programs.

 The process is the following:

  • The doctor refers the patient in need of a drug to a Contract Research Organisation (CRO)
  • The CRO collects demands from doctors and sends them to NORD.
  • NORD checks that each demand is complete (e.g. it must include a patient consent form; the patients must comply with other rules of the protocol; and they must qualify under strict medical criteria).
  • NORD then enters the demands in a database and performs a “computerized random selection” to select the patients that will benefit from the program.
  • NORD notifies non-selected and selected patients, as well as their doctors and the pharmaceutical company.

These programs are sometimes referred to as lotteries, because the selection process is totally random and the expanded access programs (EAPs) have faced challenges: there is always a limited drug supply.” – Eurordis site

For BioMarin, requests for access to experimental therapies must be made by a qualified and licensed physician through BioMarin Medical Information and will be referred to a core team for evaluation.

I have colleted information from two regional directors in Europe, that BioMarin´s compassionate use shall be made as follows: BioMarin will ensure the administration of BMN-111 to every child included in phase 2 and phase 3 of the BMN-111 clinical trial even after the end of the trial, until the child´s country allows payment and administration of the BMN-111.

Updates of BMN-111 phase 2 results waited by the end of 2015.

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