Exceptional funding of EDRDs
Last updated: May 23, 2023
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:
- Subspecialty subcommittees review new and renewal requests and make an independent patient-specific funding recommendation to the EDRD Advisory Committee.
- EDRD Advisory Committee reviews subcommittee decision and makes an arm's-length independent funding recommendation to the Ministry.
- Ministry of Health provides final exceptional, case-by-case funding approval.
- PHSA supports the Ministry and committees, and executes decisions made by the Ministry.
- 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)
- 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.
- 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.
| Brand | Generic | Condition | Approximate annual cost per patienta,b,c,d,e,f (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 |
| Dojolvi | triheptanoin | long-chain fatty acid oxidation disorders | $293,000f |
| Elaprase | idursulfase | mucopolysaccharidosis 2 (MPS2) | $1,315,000 |
| Evrysdi | risdiplam | spinal muscular atrophy | $354,000 |
| Fabrazyme | agalsidase beta | Fabry disease | $291,000 |
| Galafold | migalastat | Fabry disease | $310,000 |
| Ilaris | canakinumab | cryopyrin-associated periodic syndrome (CAPS) | $416,000 |
| Increlex | mecasermin | severe primary insulin-like growth factor-1 deficiency (SPIGFD) | $456,000e |
| Kalydeco | ivacaftor | specific cystic fibrosis mutations | $307,000 |
| Kanuma | sebelipase alfa | lysosomal acid lipase deficiency | $889,000 |
| Luxturna | voretigene neparvovec | inherited retinal dystrophy | $1,032,000 for both eyesd |
| 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 Vyndamax |
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:
- Final pricing subject to agreement
- Based on an average weight of 70 kg, unless stated otherwise
- Pharmacy mark-ups or dispensing fees not applicable
- One-time treatment
- Based on the maximum dose for a patient weight of 35 kg
- Average dosing based on 35% of patient’s total prescribed daily caloric intake
Coverage for EDRDs is limited to prescription dispenses of:
- 30 days' supply maximum, and
- A single dose for gene therapies and long-term maintenance drugs administered less frequently than every 30 days (i.e., nusinersen)
You must have 14 days or less of medications remaining from a previous fill when requesting a prescription refill.
Exemptions
Exemptions to the 30-day maximum supply are available if you:
- Reside in rural or remote areas and travel to a pharmacy is a significant barrier.
- What to do: Request a “rural supply exemption” from the Provincial Health Services Authority (PHSA) Provincial Specialized Programs (PSP) by emailing psp@phsa.ca:
- Are travelling. Request an early refill to “top up” the remaining supply of the drug to the 30-day maximum:
- What to do: If you are travelling 44 days or less, you must complete and sign a Travel Declaration on the date the travel supply is filled. A parent or guardian can complete the Travel Declaration for a child’s prescription. The pharmacy can provide the Travel Declaration form.
- What to do: If you are travelling more than 44 days, you must request email the the PHSA PSP at psp@phsa.ca to request an exemption. The Ministry of Health makes the final decision.
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