Exceptional Funding of EDRDs

Expensive drugs for rare diseases (EDRDs) are drugs that treat rare diseases with a very high per-patient cost. EDRDs are considered non-benefits, but some drugs and patients may be eligible for coverage on a case-by-case, last-resort basis.

EDRDs are generally associated with limited clinical information about both the disease and treatment. Because of this, therapies must be carefully reviewed to ensure that the medication is effective for each person being treated. Manufacturers have set the costs for many EDRDs very high. The costs for these drugs range from $100,000 to more than $3,000,000 per patient per year. By comparison, in 2019/2020, the average PharmaCare beneficiary was reimbursed $1,534 for the year (see PharmaCare Trends 2019/2020 (PDF, 714KB)).

Before an EDRD can be considered for exceptional funding on a case-by-case basis, it must undergo the same thorough review as other PharmaCare drugs (see Drug Review), but with an additional EDRD patient review process (see below).

The B.C.-level review ensures that the PharmaCare program remains fair, effective and sustainable. At the end of the review, the Ministry decides which eligible drugs and indications are to be considered for case-by-case funding.

Physicians may apply on behalf of their patients for coverage.

Due to the complexity of many rare diseases and their treatments, the Ministry has an EDRD patient review process for exceptional requests, which includes advice from an arm’s‑length independent advisory committee and several clinical subcommittees. The EDRD patient review is administered by the Provincial Health Services Authority (PHSA).

Each physician request is reviewed by an appropriate disease-specific clinical subcommittee and the EDRD advisory committee. Clinical subcommittees are comprised of members with clinical experience with rare diseases and are responsible for making a recommendation to approve or deny the request. The advisory committee also includes clinical experts, as well as members with expertise in ethics, economics, and pharmaceuticals to provide a final recommendation to the Ministry. The Ministry is responsible for the final funding approval.

The process:

  1. Subspecialty subcommittees review new and renewal requests and make an independent patient-specific funding recommendation to the EDRD Advisory Committee.
  2. EDRD Advisory Committee reviews subcommittee decision and makes an arm's-length independent funding recommendation to the Ministry.
  3. Ministry of Health provides final exceptional, case-by-case funding approval.
  4. PHSA supports the Ministry and committees, and executes decisions made by the Ministry.
  1. To be eligible for an EDRD patient review, a patient must first complete and submit an EDRD Patient Eligibility form (obtained from their physician) to their physician. In determining eligibility for provincial coverage, a patient must:
    • Be a resident of B.C.; and
    • Be registered with MSP and Fair PharmaCare; and
    • Have no other means of funding (i.e. third-party plans and other coverage)
  2. If a patient meets the EDRD program patient eligibility requirements and their prescriber believes their medical circumstance warrant the use of a specific EDRD, the physician submits a request, in addition to the completed Eligibility Form, on behalf of the patient.
  3. If the EDRD review process results in a recommendation to fund a drug, it is funded at 100% coverage and there is no co-pay or patient deductible.

If you are a physician who would like to request an EDRD therapy for your patient, please email psp@phsa.ca with the name of the drug requested and the indication for use. Someone from PHSA will respond within 5 business days.

Please note that requests are reviewed by a clinical subcommittee and advisory committee (as detailed above) and the processing time may vary.

EDRD drugs
Brand Generic Condition  Approximate annual cost per patienta,b,c
(based on publicly available list prices rounded to the nearest $1,000)
Aldurazyme laronidase mucopolysaccharidosis I (MPS1) $761,000
Brineura cerliponase alfa neuronal ceroid lipofuscinosis type 2 (Batten disease) $844,000
Crysvita burosumab X-linked hypophosphatemia (XLH) $454,000
Elaprase idursulfase mucopolysaccharidosis 2 (MPS2) $1,315,000
Fabrazyme agalsidase beta Fabry disease $291,000
Galafold migalastat Fabry disease $310,000
Ilaris canakinumab cryopyrin-associated periodic syndrome (CAPS) $416,000
Kalydeco ivacaftor specific cystic fibrosis mutations $307,000
Myozyme alglucosidase alfa Pompe disease $612,000
Naglazyme galsulfase mucopolysaccharidosis VI (MPS6) $1,117,000
Onpattro patisiran hereditary transthyretin-mediated amyloidosis (hATTR) $677,000
Procysbi cysteamine nephropathic cystinosis $322,000
Replagal agalsidase alfa Fabry disease  $239,000
Soliris eculizumab atypical hemolytic uremic syndrome (aHUS)
paroxysmal nocturnal hemoglobinuria (PNH)
 
Initial: $728,000
Ongoing: $701,000
Initial: $539,000
Ongoing: $526,000
Spinraza nusinersen spinal muscular atrophy  Initial: $708,000
Ongoing: $354,000
Strensiq asfotase alfa hypophosphatasia $2,546,000
Takhzyro lanadelumab hereditary angioedema $400,000
Tegsedi inotersen hereditary transthyretin-mediated amyloidosis (hATTR) $420,000
Trikafta elexacaftor/tezacaftor/ivacaftor and ivacaftor specific cystic fibrosis mutations $307,000
Vimizim elosulfase alfa mucopolysaccharidosis IVA (Morquio A syndrome) N/A
VPRIV velaglucerase alfa Gaucher disease $559,000
Vyndaqel tafamidis transthyretin-mediated amyloidosis (cardiomyopathy) $195,000
Zavesca miglustat Niemann-Pick disease type C (NPC1) $119,000
Zolgensma onasemnogene abeparvovec spinal muscular atrophy $2,911,000d

Notes:

  1. Final pricing subject to agreement
  2. Based on an average weight of 70 kg, unless stated otherwise  
  3. Pharmacy mark-ups or dispensing fees not applicable
  4. One-time treatment

 

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