Access to Heme Treatment in Canada - Survey 2018

Transcription

1 Access t Heme Treatment in Canada - Survey 2018 The Canadian Assciatin fr Prphyria/Assciatin Canadienne de Prphyrie (CAP/ACP) asserts that patients with acute prphyria shuld have access t Hemin treatment, as needed, regardless f where they live in Canada. We supprt a system f medical care where this bld prduct is distributed efficiently and equitably, and by s ding, greatly imprve the lives f thse suffering frm acute hepatic prphyria. In rder t have a clearer understanding f Canadians with prphyria, Online Surveys in 2016 and 2018 were used t gather infrmatin abut patients experiences with Hemin treatment in Canada. These surveys were distributed t members and cntacts f CAP/ACP via Facebk, Website and s. Results were annymusly cllected and cmpiled t cllect the experiences f Canadians with acute prphyria (see Appendix). Overview: Acute Hepatic Prphyria Symptms and Treatment Prphyria is a rare, serius and life-threatening cnditin. The fllwing are a list f the 1 clinical features during an attack fr thse with an acute hepatic prphyria: Abdminal pain severe, prly lcalized. Pain can als affect back, legs and ther sites Nausea, vmiting, cnstipatin; Hypertensin, tachycardia, and rarely, arrhythmias; Agitatin, insmnia, cnfusin, psychsis with hallucinatins and unusual behaviur Cnvulsins frequently assciated with hypnatraemia Peripheral mtr neurpathy may prgress t flaccid paralysis, respiratry insufficiency, difficulty swallwing, urinary retentin r incntinence Dark urine clur darkens t range r red n expsure t light Hypnatraemia In additin t the excruciating pain experienced during an attack, many patients als experience chrnic pain as a result f neurpathic damage sustained during untreated attacks. 1 P Stein; Department f Medicine, Addenbrke s Hspital, Cambridge CB2 0QQ, Best practice guidelines n clinical management f acute attacks f prphyria and their cmplicatins This reprt was nt prepared by prfessinals and is fr infrmatinal purpses nly.

2 Hemin (a cncentrate fr slutin fr infusin and cnsisting primarily f prcessed human red bld cells) is the nly treatment available t patients with acute prphyria. Tw hemin prducts (Nrmsang and Panhematin) are distributed by Recrdati and are indicated fr treatment f acute attacks f hepatic prphyria (acute intermittent prphyria, prphyria variegata, and hereditary cprprphyria). 2,3 As f Nvember, 2018, Panhematin has been apprved by Health Canada and is the hemin treatment available fr treatment f acute prphyria symptms (unless there are medical circumstances that warrant Nrmsang). Access t Heme Treatment: 2018 Survey Results Current Treatment Availability in Canada Twenty-five respnses frm Canadians diagnsed with acute prphyria were received. Qutes frm survey respndents are included in italics thrughut this submissin. Patients in the survey spke, nce again, f the excruciating pain f attacks and the lng term effects fr themselves and/r their family members f nt receiving adequate treatment. Having t be in full attack befre I can access drugs that culd have prevented the attack in the first place is a terrible drain n myself and my family in every aspect imaginable frm physical, t mental, t mnetary... The inability t lead a nrmal life free f attacks due t nt having access t preventative therapy is cruel. I have n quality in my life. It is pain and illness cnstantly. Prphyria is treatable. Patients can be treated with Hemin and in the survey spke, nce again, f the benefits f receiving adequate treatment. With Nrmsang in preventive treatment ne dse every 10 days...n mre serius crisis needing t g t emergency and intensive care, Befre it was a crisis a mnth minimum Imprves my quality f life. Less neurlgical symptms after the attack. Less sick days. I had tw treatments f panhematin in It was amazing. It s the nly time I ve been symptm free in 40 yrs. 2 Nrmsang Prduct Infrmatin: SUMMARY OF PRODUCT CHARACTERISTICS, Orphan Eurpe 3 Panhematin Patient Infrmatin, Orphan Eurpe This reprt was nt prepared by prfessinals and is fr infrmatinal purpses nly.

3 Restricted Access t Hemin Treatment The Access t Heme Treatment, 2018 Survey results clearly demnstrates that there is unequal access t Hemin treatment acrss Canada. Fr thse diagnsed with an acute prphyria and tld by a physician that they shuld receive Hemin treatment nly 40% (4 f 10 patients) were able t access treatment as needed, 40% (4 f 10 patients) had limited access t Hemin treatment and 20% (2 f 10 patients) had n access. The majrity f Canadian prphyria patients (60%) are nt receiving timely Hemin treatment. Access t Apprpriate Hemin Treatment in Canada, 2016 Prvince Access Limited Access N Access AB 2 2 BC 2 2 MB 1 NS 1 QC 1 Ttal Of particular cncern is access t Hemin treatment in the prvinces f Manitba and Nva Sctia where the respndent in each prvince stated that they had n access t hemin treatment. The current system f delivering Hemin treatment t acute prphyria patients in Canada is failing and in sme cases increases suffering unnecessarily. Where yu live in Canada determines whether r nt yu will get treatment fr acute prphyria. The prvince yu live in will als determine hw cnsistent treatment will be and hw difficult it is t get apprval fr treatment. The utcme f nt adequately treating acute prphyria patients cncerns us all. In additin t dealing with the medical symptms, acute prphyria patients spke f the stress f advcating fr apprpriate care, lss f their capacity fr wrk and an inability t parent as 2 a direct result f their cnditin. Infrmatin t Patients abut Hemin Treatment One respndent stated that they were unaware f Hemin treatment r even mre alarmingly, several respndents mentined that the hspitals r physicians were refusing t prvide Hemin treatment because f the cst. These prphyria patients were nt able t access apprpriate Hemin treatment - the nly treatment fr their cnditin. Advcacy by CAP/ACP 2 W Sauvé, J Reimer, 2018 Survey: Access t Heme Treatment in Canada This reprt was nt prepared by prfessinals and is fr infrmatinal purpses nly.

4 The missin f CAP is t deliver evidence-based infrmatin and supprt t patients with prphyria, their families, health care prviders and the general public acrss Canada and t achieve standards and evidence-based cmprehensive care fr all peple with prphyria thrughut their lifespans. Based n the findings f the 2016 and 2018 Surveys: Access t Heme Treatment, ur gals fr advcacy will include: 1. Prpsing that Hemin treatment be distributed as ther bld prducts are in Canada - thrugh Canadian Bld Services (CBS). Please find the CAP/ACP presentatins t CBS here 2. Prviding accurate infrmatin regarding access t treatment fr prphyria patients 3. Advcating that Canadian acute prphyria patients, in general, receive hemin treatment, as needed. 4. Advcating with individual Canadian acute prphyria patients that they receive hemin treatment, as needed. The CAP/ACP view the 2016 and 2018 surveys as basis fr discussins arund hw best t ensure that thse with prphyria access treatment, as needed. We invite yu t cntact us at canadianasscfrprphyria.acp@gmail.cm. The 2018 Survey: Access t Heme Treatment was prepared by: Wendy Sauvé (President CAP/ACP) and Jeannie Reimer (Treasurer CAP/ACP) This reprt was nt prepared by prfessinals and is fr infrmatinal purpses nly.

5 APPENDIX SUMMARY Survey: Access t Hemin Treatment in Canada Questin 1: D yu live in Canada? All 25 respndents live in Canada. Questin 2: In what prvince r territry d yu live? Prvinces represented; AB (8), BC (6), MB (1), NS (2), ON (5), QC (3) Questin 3: Have yu been diagnsed with acute prphyria? 25 respndents indicated that they were diagnsed with an acute prphyria. Questin 4: If yu have received a diagnsis f prphyria, which type have yu been diagnsed with? Acute Intermittent Prphyria (AIP) 16 Variegate Prphyria (VP) 4 Hereditary Cprprphyria (HCP) 2 Acute Intermittent Prphyria (AIP) and Hereditary Cprprphyria (HCP) 2 Nt Indicated 1 The survey results are representative f the acute prphyria cmmunity where AIP is mst frequently diagnsed, fllwed by VP and, less frequently, HCP. Tw respndents indicated that they had been diagnsed with tw types f prphyria. Questin 5: Have yu ever been tld by a physician that yu shuld receive Heme treatment (Nrmsang and/r Panhematin)? 12 respndents were tld by their physician that they require Hemin treatment. Questin 6: Which f the fllwing treatments have yu received (if yu have been tld by a physician that yu shuld receive Hemin treatment Nrmsang and/r Panhematin)? Panhematin 2 Nrmsang 3 Panhematin & Nrmsang 5 N Treatment Available 4 This reprt was nt prepared by prfessinals and is fr infrmatinal purpses nly.

6 Questin 7: If yu have nt been able t receive Nrmsang r Panhematin, why can t yu get treatment? I was given a treatment f panhematin because I was in crisis (the treating dctrs didn t believe I was having a severe reactin t the medicatins). I asked the dctr if I culd receive a treatment if it was needed and the dctr answered n because it was t expensive fr the hspital. # f Respnses Reasn fr NOT being able t access Hemin Treatment 2 Physician wuld nt rder the treatment 2 Hspital stated that the treatment was t expensive 1 Patient did nt knw f the treatment ptin 1 Physician did nt rder the treatment 1 Gvernment wuld nt apprve Questin 8: If yu have nt been able t receive Nrmsang r Panhematin, please describe the impact t yu, yur family, and yur quality f life. Have been put n disability and n lnger wrking r able t take prper care f my family. I d what I can when I can and limit things as needed. # f Respnses Impact f NOT receiving Hemin treatment 3 Negatively impacts family life 2 Negatively impacts quality f life 2 Negatively impacts mental health 2 Patient is chrnically ill 1 Prer health 1 Lack f preventative treatment 1 Negatively impacts finances 1 Suffering intense pain 1 Negatively impacts emplyment This reprt was nt prepared by prfessinals and is fr infrmatinal purpses nly.

7 Questin 9: If yu have been able t receive Nrmsang r Panhematin, please describe the impact t yu, yur family, and yur quality f life. It saved my life! If I didn t receive it in August r 2002 I wuldn t be alive tday. Thankfully I gt it and since then I am able t live nrmal life. # f Respnses Impact f RECEIVING Hemin treatment 1 Less time in emergency 1 Less time in intensive care 1 Quality f life greatly imprved 1 N mre prphyria crises 1 Fewer sick days 1 Free f prphyria symptms 1 Saved patient s life 1 Allwed patient t life a nrmal life 1 Less neurlgical symptms Questin 10: Please use this area t share infrmatin that was nt included abve. With Nrmsang the life is nt nrmal but far better than befre, I wasted 400 days in hspital mainly emergency and intensive care befre the Nrmsang # f Respnses Additinal Cmments 3 Lack f knwledge by physicians 2 Lack f supprt frm physicians 2 Lack f hspital staff knwledge 2 Frustratin with lack f access t treatment 1 Benefit frm effective treatment 1 Lack f care fr elderly prphyria patients 1 Patient reassured if treatment was made available 1 Patient tld they were mental by nurses 1 Patient tld there was n such disease 1 Patient made t feel guilty fr the cst f treatment 1 Diagnsis (AIP) questined by physicians 1 Apprpriate Hemin treatment imprved cnditin This reprt was nt prepared by prfessinals and is fr infrmatinal purpses nly.

8 Name f the disrder Name f the bld prduct Name f the patient grup 2018 Access t Heme Treatment in Canada Survey Name f the primary cntact fr this submissin: Psitin r title with patient grup Acute hepatic prphyria ( Acute Intermittent Prphyria, Variegate Prphyria, and Hereditary Cprprphyria) (hereafter referred t as acute prphyria) Nrmsang and/r Panhematin (referred t as Hemin) Canadian Assciatin fr Prphyria/Assciatin Canadienne de Prphyrie (hereafter referred t as CAP/ACP) Wendy Sauvé President CanadianAsscFrPrphyria.acp@gmail.cm Telephne number (780) r (403) Ave NW Edmntn, AB Address T5M 2C8 Names f authrs Cnflict f Interest Declaratin Website CanadianAssciatinFrPrphyria.ca Wendy Sauvé and Jeannie Reimer Nne t declare This reprt was nt prepared by prfessinals and is fr infrmatinal purpses nly.