What  does  OHIP+  offer  children  and  young  adults  with  rare  disorders?

Nigel  S  B  Rawson,  PhD

Eastlake  Research  Group,  Oakville,  OntarioCanadian  Health  Policy  Institute,  Toronto,  Ontario

Affordability  of  rare  disorder  drugs

“We  had  to  turn  it  down  and  say  no  because  of  the  price  tag”

“There’s  that  feeling  of  helplessness:  I  may  lose  her  because  I  don’t  have  enough  money  to  save  her.”Globe  &  Mail,  May  19,  2017

Will  OHIP+  help  parents  like  this?

What  the  government  says  about  OHIP+?

§ Liberals  “are  making  sure  that  every  young  person  across  the  province  has  access  to  the  medications  they  need  to  stay  healthy,  feel  better  and  live  full  lives”  

§ They  “are  easing  parents’  worries,  while  making  life  more  affordable  for  them”

§ Access  to  medicines  will  be  improved  “by  eliminating  financial  barriers  to  prescribed  drugs”

OHIP+  will  only  cover  drugs  in  the  OPDP  formulary

§ General  Benefit  unrestricted  use  list:  almost  4,000  products  ―  OPDP  pays  <$1  for  73%;  <$5  for  87%;  >$100  for  1.6%

§ Limited  Use  list:  940  drugs  with  criteria  restricting  access  to  patients  with  specific  disease  characteristics  or  to  a  defined  treatment  period  ―  OPDP  pays  <$1  for  48%;  <$5  for  77%;  >$100  for  5.8%

§ Exceptional  Access  Program:  127  drugs  with  detailed,  frequently  complex,  access  criteria;  many  are  biotechnological  products  ―  often  expensive

Drugs  most  commonly  dispensed

Children  under  12 Older  children  &  young  adults

Antibiotics Antibiotics

Anti-­‐asthma  drugs Anti-­‐asthma  drugs

ADHD  drugs ADHD  drugs

Analgesics Antidepressants

Cough and  cold  remedies Contraceptives

Anti-­‐allergens

CDR  recommendations  for  rare  disorder  drugs

§ 55  CDR  recommendations  for  42  rare  disorder  drugs  submitted  between  2004  and  February  2016

§ 19  submissions  were  for  16  drugs  for  rare  genetic  conditions

§ 13  of  the  19  received  negative  reimbursement  recommendation  

§ Resubmissions  for  two  drugs  led  to  revised  CDR  assessments  later  in  2016

§ 8  of  the  submissions  (42%)  now  have  positive  recommendations

Brand  name Disorder  treated Reimbursement  recommendation

OPDP  listing

Replagal Fabry  disease Negative No

Fabrazyme Fabry  disease Negative No

Elelyso Gaucher  disease Negative No

Vpriv Gaucher  disease Positive No

Zavesca Gaucher  disease Negative No

Myozyme Pompe  disease Positive No

Aldurazyme Mucopolysaccharidosis  I Negative No

Elaprase Mucopolysaccharidosis  II   Negative No

Vimizim Mucopolysaccharidosis  IVA Positive* No

Brand  name

Disorder  treated Reimbursement  recommendation

OPDP  listing

Kalydeco CF  (G551D  mutation) Positive EAP

Kalydeco CF  (CFTR  gating  mutations) Positive NoKalydeco CF  (R117H  CFTR  gating  mutation) Positive NoAfinitor TSC-­‐associated  renal  angiomyo-­‐

lipoma  Negative EAP

Afinitor TSC-­‐associated  subependymal  giant  cell  astrocytoma  

Negative EAP

Diacomit Dravet  syndrome Positive EAP

Kuvan Phenylketonuria Positive* EAPIlaris Cryopyrin-­‐associated  periodic  

syndromesNegative No

Soliris Atypical  hemolytic  uremic  syndrome  

Negative EAP

Juxtapid Homozygous  familial  hyper-­‐cholesterolemia

Negative No

Listing  to  access

§ Kalydeco/Diacomit:  access  usually  reasonable

§ Afinitor:  access  only  after  surgery  tried

§ Kuvan:  harsh  access  criteria  has  led  to  no  patient  having  access

§ Soliris:  usually  reserved  for  acute  patients  and  those  who  have  had  a  kidney  transplant

BC AB SK MB ON QC NB NS PE NLReplagal

Fabrazyme

Elelyso

Vpriv

Zavesca

Myozyme EDS ES SA ESAldurazyme

Elaprase

Vimizim

Kalydeco G551D SA EDS EAP SA ES ESAfinitor TSC EAPDiacomit SA EDS EAP ES SA ES ESKuvan EDS EAP ESIlaris

Soliris EAPJuxtapid ES

Conclusions

§ Formulary  chiefly  limited  to  lower-­‐cost  generics  § Children  with  common  conditions  whose  

parents  have  no  current  provincial  or  private  coverage  will  benefit  most  from  OHIP+  

§ Children  with  rare  genetic  disorders  for  whom  new  innovative  drugs  are  becoming  available  are  unlikely  to  see  much  benefit  from  OHIP+  

§ For  those  that  do,  coverage  will  stop  at  age  25  § Advocacy  remains  essential