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Ministry Status: Routine Monitoring and Engagement

How Drugs Are Considered: Funding Decisions

Drugs For Rare Diseases (DRD)

In the absence of a national strategy, the Ontario Ministry of Health has developed a framework for Drugs for Rare Diseases (DRDs).  This framework recognizes that an innovative approach is required for DRDs, which considers the level of available clinical evidence, patient need, and reimbursement.

In December 2007, the Ministry established a working group of clinical experts (including genetic medicine) and health economists to develop a new evaluation framework to review and evaluate DRDs for funding by the province.

The approach is based on the "best available evidence" to assist us in predicting the potential benefit or lack of benefit of a drug treatment in specific patients.  This new approach will help identify groups of individuals that may potentially benefit from treatment with a particular drug, and where we may consider funding.

The Ontario's Citizen Council met in January 2010 to discuss DRDs and provided recommendations to the Executive Officer on the values/approaches they feel are important in funding DRDs.  The Citizen's Council recommendations were provided to the Ministry and have been incorporated into the draft DRD review and evaluation framework.  Please see the following link for additional information on the Citizen's Council report related to DRDs and the Executive Officer's response.

The Ministry is using the draft DRD framework to assess funding requests for DRDs.  Ontario is leveraging its own work and experience to date to support a national (P/T) approach to evaluate drugs for rare diseases for public funding purposes.

DRD Submissions:

Drug products may be considered for reimbursement under the ODB program if the manufacturer makes a complete submission to the Ministry. The submission must comply with the requirements specified in O. Reg. 201/96 under the Ontario Drug Benefit Act and Regulation 935 under the Drug Interchangeability and Dispensing Fee Act.  Please see the following link for additional information on Ontario guidelines for drug submission and evaluation.

The Ministry may ask the manufacturer for additional information to assist in the DRD evaluation at anytime throughout the review process.

Due to the exhaustive nature of a DRD review, the Ministry will NOT entertain pre-NOC or post-NOC rapid review requests for products being reviewed through the DRD evaluation framework.

To assist manufacturers who wish to forward a submission for consideration through the DRD evaluation framework, the following additional information should also be provided with your submission:

Patient Evidence Submissions:

The Ontario government has now established a formal process for patients or caregivers, through an advocacy group, to submit evidence for new drugs undergoing funding review, including DRDs.  Please see the following link for additional information on the patient evidence submission process.

Tracking of Submissions:

Please find additional information on the drug submission status of first-review single source submissions.

Stakeholder Information:

Ontario Ministry of Health Presentation(s) at Canadian Organization for Rare Disorders (CORD) meeting(s)

Information on Ontario Ministry of Health DRD Stakeholder Sessions:

The Ministry sought input from patient groups, physicians, and manufacturer's in the development and further refinement of the DRD framework.  The first round of stakeholder sessions (patient/advocacy groups, prescriber, and manufacturer) were held in January - March 2009 to obtain feedback on the development of the DRD evaluation framework.

DRD Reviews:

Please see below for information related to products that have been evaluated through the DRD Evaluation Framework.

Aldurazyme (laronidase) for the treatment of Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I):

Elaprase (idursulfase) for the treatment of Hunter's Syndrome:

Ilaris (canakinumab) for the treatment of Cryopyrin-Associated Periodic Syndrome (CAPS):

Myozyme (alglucosidasae alfa) for both infantile and adult/late onset Pompe disease:

Zavesca (miglustat) for the treatment of Niemann Pick Type C (NPC):


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