Exceptional funding of EDRDs

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,d,e,f,g (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
Empaveli	pegcetacoplan	paroxysmal nocturnal hemoglobinuria (PNH)	$517,000
Evrysdi	risdiplam	spinal muscular atrophy	$354,000
Fabrazyme	agalsidase beta	Fabry disease	$291,000
Galafold	migalastat	Fabry disease	$310,000
Givlaari	givosiran	acute hepatic porphyria	$773,000g
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

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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
5. Based on the maximum dose for a patient weight of 35 kg
6. Average dosing based on 35% of patient’s total prescribed daily caloric intake
7. Based on an average weight of 67 kg

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.