Cystic Fibrosis Panel Applications (Dornase Alfa) Contents

Transcription

1 Cystic Fibrosis Panel Applications (Dornase Alfa) Contents Page 2-4: Entry and Stopping Criteria for Treatment with Dornase Alfa Page 5-9: Application and consent forms for a one month trail and long term supply of dornase alfa Page 10: Entry criteria for treatment with dornase alfa for children under the age of 5 Page 11-13: Application and consent forms for supply of dornase alfa for children under the age of 5

2 Entry and Stopping Criteria for Treatment with Dornase Alfa The following conditions need to be met for subsidy. These conditions were established by paediatricians and physicians with expertise in cystic fibrosis, the New Zealand Cystic Fibrosis Association and the Respiratory Committee of the NZ Paediatric Society, in conjunction with the Pharmaceutical and Therapeutics Advisory Committee (PTAC). 1. Assessment at cystic fibrosis clinics Patients must be assessed at regional cystic fibrosis clinics or centres. These clinics and centres must be under the control of specialist respiratory physicians/paediatricians with experience and expertise in the management of cystic fibrosis. The prescribing of dornase alfa under the Pharmaceutical Schedule is limited to such physicians. 2. Measurement of lung function The measurement of lung function is to be conducted by experienced, qualified respiratory technicians. Allowance needs to be made for age/height related changes in children's lung function measurement. Spirometry should be the best of three measurements with repeat spirometry under the same circumstances. Adherence to American Thoracic Society (ATS) criteria for lung function testing is required, as referred to in: (a) American Journal of Respiratory and Critical Care Medicine 1995;152: ; and (b) The New England Journal of Medicine 1994; 331: Inclusion of patients in a national database All patients having treatment with dornase alfa should be included in the national cystic fibrosis database. Prescribing physicians/paediatricians are required to supply updated patient information on a six monthly basis. 4. Patients eligible for a one month trial of dornase alfa therapy Patients with cystic fibrosis who are eligible for an initial one month trial of dornase alfa are those who: (a) Are willing to undertake a trial of dornase alfa; and (b) Are aged five years or older; and (c) Are clinically stable as documented by 3 consistent (or stable) baseline measurements of FEV 1 and FVC within the six weeks prior to application; and (d) Have ongoing respiratory tract infections in keeping with cystic fibrosis; and (e) Have previously undergone a trial with, or are currently being treated with, hypertonic saline; and (f) Are provided with written acknowledgement of the nature of the trial of therapy and that continued treatment may not be recommended or made available; and (g) Are willing to continue with other standard treatments including, but not limited to; secretion removal techniques, pancreatic enzyme supplements and vitamin supplements. This phase of initial therapy is limited to one month s treatment with dornase alfa at a dose of 2.5 mg daily.

3 Immediately before starting dornase alfa therapy, a further baseline measurement of FEV 1 and FVC (best of three measurements) should be undertaken. Only if continuing stability is demonstrated should the trial be commenced. If previous bronchial hyper-reactivity has been demonstrated it is recommended that respiratory function tests should always be performed 10 to 15 minutes after treatment with a bronchodilator. 5. Application for a one month trial of dornase alfa Physicians/Paediatricians who have patients who seem to fulfil the eligibility criteria should apply to the Cystic Fibrosis Panel via the Coordinator: Mail: CF Advisory Panel Coordinator PHARMAC P O Box Wellington cfpanel@pharmac.govt.nz Fax: If a Panel member's patient is being considered, that Panel member may not partake in the decision making process. The Panel consists of at least three physicians/paediatricians experienced in the management of cystic fibrosis and appointed by PHARMAC. To be successful, an application must be approved by at least two Panel members. 6. Retesting Unsuccessful trials Patients whose FEV 1 measures do not met the criteria in 4. above may apply for a further one month trial of dornase alfa after a three month period. Up to three trials may be undertaken within a twelve month period. Abandoned trials If an infective exacerbation supervenes in a trial, which is severe enough to require intensive management (IV antibiotics and hospitalisation) and is associated with a deterioration of 10% or more in lung function, the trial should be abandoned and the Panel notified. To be eligible for a retrial the following criteria must be met: (a) a period of clinical stability following recovery from the infective exacerbations; and (b) lung function test demonstrating an FEV 1 consistent with pre-treatment values is submitted to the Panel. If FEV 1 post-exacerbation is not consistent with baseline values obtained pre-treatment, two further FEV 1 measurements taken within a six week period demonstrating clinical stability should be supplied to the Panel prior to a retrial being approved. Up to three trials may be undertaken within a twelve month period. 7. Patients eligible for long term dornase alfa therapy At or towards the end of the one month trial, patients must be reassessed and further lung function measurements undertaken (test under conditions as above). To be eligible for long term therapy, the following criteria must be met:

4 (a) The patient is willing to continue with treatment; and (b) A formal assessment by the physician that in their opinion, and in the opinion of the patient (or the patient's family, in the case of paediatric patients) dornase alfa is continuing to produce worthwhile benefits. Such an assessment should include a lung function test taken during the trial, impact on hospitalisation, infective exacerbations, antibiotic use, and another requirements as per the application form; and (c) Patients must demonstrate the following improvements in FEV 1 from baseline after a one month trial: for patients with a baseline FEV 1 greater than 90% predicted, a 3% or greater improvement in FEV 1 from baseline; or for patients with a baseline FEV 1 between 65-90% predicted, a 5% or greater improvement in FEV 1 from baseline; or for patients with a baseline FEV 1 less than 65% predicted, a 7.5% or greater improvement in FEV 1 from baseline. (d) Serial lung functions testing will be conducted at 3 monthly intervals with regular reviews by the physician; and It is important that other aspects of treatment such as physiotherapy be continued. Dornase alfa therapy should cease if there is not general agreement of benefit, as these is always the possibility of harm from unnecessary use. 8. Appeal procedure In the first instance, an appeal is to be referred directly to the CF Panel via the Coordinator. The Panel is to consider that appeal within a reasonable time and is to provide a summary of that appeal to PHARMAC with the Panel s report for the relative month. The Panel is to provide PHARMAC with such further details in relation to any such appeal as it may reasonably request. If after considering an appeal by an applicant, the Panel upholds its decision not to grant the applicant subsidised access to dornase alfa, then the Panel is, via the Panel Coordinator, to advise the applicant of his or her right of an appeal to the Director of Operations at PHARMAC against the determination of the Panel.

5 Application and consent forms for a one month trial and long term supply of dornase alfa (Pulmozyme) Please send applications to: CF Advisory Panel Coordinator PHARMAC P O Box WELLINGTON Phone: , option 6 Fax: cfpanel@pharmac.govt.nz Complete this section when applying for a one month assessment and, if details have changed, when applying for long term therapy. Patient Details - patient sticker is acceptable Surname: * First name/s:* NHI No:* Date of birth* Address: Phone No: Home: Work: Mobile: Applying physician:* Practice address:* NZMC #: Phone: Fax: * General Practitioner: Address: Phone: Fax: Designated pharmacy:* Address:* Phone: Fax: * Compulsory Field

6 Patient history Morbidity: Number of hospital admissions in the last 12 months for respiratory complications: Number of courses of IV antibiotics in the last 12 months: Outpatient visits: Number of outpatient visits in last 12 months: Management: Chest Physiotherapy Yes No Frequency: < once daily once-twice daily > twice daily Other therapies: Inhaled Antibiotic Yes No Long term macrolide Yes No Hypertonic saline - Current therapy Yes No - Previous Trial Yes No Other comments

7 Pulmonary function Measurement of lung function must be conducted by trained and experienced technicians and conform to the ATS criteria for lung function testing. Date of Application: / / Patient Details - patient sticker is acceptable Patient name:* NHI No:* D.O.B:* One month trial - Baseline lung function Baseline data: FEV1 measurements must be within 10% (mean FEV1 +/- 5%) and all measurements should be taken within a 6 week period. If reversible airway obstruction is present, results should be measured post bronchodilator. Visit date Age* Height* Weight* FEV1 (L) FEV1 (% predicted) FVC (L) FVC (% predicted) *Age, height and weight required in children each time; single age and height for adults. One month trial review data - please resubmit this form and any relevant correspondence Visit date Age* Height* Weight* FEV1 (L) FEV1 (% predicted) FVC (L) FVC (% predicted) *Age, height and weight required in children each time; single age and height for adults.

8 Applicant Consent form for access to dornase alfa Applying/prescribing physician/paediatrician to complete: Completed application form enclosed. Signed patient consent included. I confirm that I have the patient (or their guardian s) permission to make this application. I confirm that all information contained in this form is complete and accurate to the best of my knowledge and I acknowledge that I may be audited. I agree to ensure that dornase alfa will be administered according to the criteria established by PHARMAC via the Cystic Fibrosis Advisory Panel (the Panel), and that this administration may be subject to audit. I acknowledge that if there is insufficient response to therapy, subsidy for ongoing therapy will generally not be available, in accordance with the criteria. I acknowledge that this application for a trial of dornase alfa, if approved, will be valid for one month and that I will have to reapply after 3 weeks for ongoing therapy for this patient. I acknowledge that if approval for long term therapy is given, no further re-application is needed to PHARMAC, but that I will notify PHARMAC should the patient stop taking treatment. Signed: Date: / / Note: It is necessary to retest the patient at the appropriate time and promptly forward information to the Panel Coordinator, in order to avoid delays reassessing the application and to avoid any unnecessary interruption of treatment. Note: If the patient is un-well (acutely/sub-acutely) towards the end of the one or six month period and is not able to complete the required reassessment measurements, you must inform the Panel Coordinator immediately. Note: If treatment is suspended or discontinued for any reason, please notify the Panel Coordinator immediately.

9 Patient Consent form for access to dornase alfa Patient (or guardian) to complete: I acknowledge that, if approved for initial assessment, this is a one month trial after which the trial will end and I will be reassessed. I acknowledge that if there is not sufficient response to therapy after the one month trial, subsidy for ongoing therapy will generally not be available. I understand that information contained in my application is held securely by PHARMAC. This information will be provided to the CF Advisory Panel and, when necessary, to PHARMAC staff and the Director of Operations at PHARMAC for the purpose of administering and reviewing the application, and related purposes. This information may also be released to my specialist and my general practitioner on request. I acknowledge that I am not obliged to agree to this information being provided to PHARMAC, but that my application will not be able to be assessed without it. I understand that I have the right to access and correct any personal information contained in any of my application form/s for funding of dornase alfa, and that I can do so by contacting cfpanel@pharmac.govt.nz Signed: Date: / /

10 Entry criteria for treatment with dornase alfa for children under the age of 5 Initial approval criteria for children under the age of 5 Approvals valid for twelve months for applications meeting the following criteria: All of the following: 1. Patients must be assessed at regional cystic fibrosis clinics or centres which are under the control of specialist respiratory physicians/paediatricians with experience and expertise in the management of cystic fibrosis and the prescribing of dornase alfa under the Pharmaceutical Schedule is limited to such physicians; and 2. Any of the following: 2.1. Requiring more than two hospital respiratory admissions in a 12 month period; or 2.2. Requiring a hospital admission to have a PICC line inserted to manage exacerbations; or 2.3. A chest x-ray showing clear mucus plugging, focal consolidation or a Brasfield score <22/25 done at a time of stability despite currently approved treatment, including repeated physiotherapy assessment and education, and having had at least one admission to hospital; or 2.4. A bronchoscopy done as far as possible at a time of stability which shows significant mucus plugging despite currently approved treatment, including repeated physiotherapy assessment and education, and having had at least one admission to hospital; or 2.5. Diagnosis of allergic bronchopulmonary aspergillosis (ABPA); or 2.6. Undertaking eradication treatment is proposed; and 3. Patient has previously undergone a trial with, or are currently being treated with, hypertonic saline; and 4. All patients having treatment with dornase alfa should be included in the national cystic fibrosis database. Prescribing physicians/paediatricians are required to supply updated patient information on a six monthly basis. Renewal for children under the age of 5 Approvals valid for twelve months for applications meeting the following criteria: All of the following: 1. Patient is compliant with therapy; and 2. In the opinion of the patients treating clinician they believe dornase alfa to be of worthwhile benefit to the patient; and 3. Any of the following: 3.1. Reduction in hospital admissions in the previous 12 months; or 3.2. Reduction in the number of treatment courses of oral antibiotics in the previous 12 months; or 3.3. Significant improvement in radiological or bronchoscopy findings; or 3.4. An eradication of a pathological organism. Submitting an application Physicians/Paediatricians who have patients who seem to fulfil the eligibility criteria should apply to the Cystic Fibrosis Panel via the Coordinator: Mail: CF Advisory Panel Coordinator PHARMAC P O Box Wellington cfpanel@pharmac.govt.nz Fax: If a Panel member's patient is being considered, that Panel member may not partake in the decision making process. The Panel consists of at least three physicians/paediatricians experienced in the management of cystic fibrosis and appointed by PHARMAC. To be successful, an application must be approved by at least two Panel members. Appeal procedure In the first instance, an appeal is to be referred directly to the CF Panel via the Coordinator. The Panel is to consider that appeal within a reasonable time and is to provide a summary of that appeal to PHARMAC with the Panel s report for the relative month. The Panel is to provide PHARMAC with such further details in relation to any such appeal as it may reasonably request. If after considering an appeal by an applicant, the Panel upholds its decision not to grant the applicant subsidised access to dornase alfa, then the Panel is, via the Panel Coordinator, to advise the applicant of his or her right of an appeal to the Director of Operations at PHARMAC against the determination of the Panel. 1

11 Application and consent forms for supply of dornase alfa (Pulmozyme) for children under the age of 5 Please send applications to: CF Advisory Panel Co-ordinator PHARMAC P O Box WELLINGTON Phone: , option 6 Facsimile: cfpanel@pharmac.govt.nz Patient details Surname: First name/s: NHI: D.O.B: Address: Phone No: Home: Work: Mobile: Applying physician: Practice address: Date: Phone: Fax: General Practitioner: Address: Phone: Fax: Designated pharmacy Address: Phone: Fax: 2

12 Patient history and criteria Patient details Patient name: NHI: D.O.B: Current management: Chest Physiotherapy Yes No Frequency: < once daily once-twice daily > twice daily Other therapies: Inhaled Antibiotic Yes No Long term macrolide Yes No Hypertonic saline Initial approval criteria for children under the age of 5 - Current therapy Yes No - Previous Trial Yes No Patients has been assessed at regional cystic fibrosis clinics or centres is under Yes No the control of specialist respiratory physicians/paediatricians Any of the following*: 2.1. Patient has required more than two hospital respiratory admissions Number: in a 12 month period 2.2. Patient has required a hospital admission to have a PICC line Yes No inserted to manage exacerbations* 2.3. Patient has had a chest x-ray* showing: Clear mucus plugging, or Yes No OR Focal consolidation, or Yes No A Brasfield score <22/25 done at a time of stability, and Score: Has had at least one admission to hospital Yes No 2.4. Patient has had a bronchoscopy* done as far as possible, at a time Yes No of stability, which shows: Significant mucus plugging despite currently approved Yes No treatment, and Has had at least one admission to hospital Yes No 2.5. Diagnosis of allergic bronchopulmonary aspergillosis (ABPA) Yes No 2.6. Undertaking eradication treatment is proposed* Yes No Please confirm the number of treatment courses of oral antibiotics in the previous 12 months: *Please provide supporting clinical information Renewal approval criteria for children under the age of 5 Patient is compliant with therapy Yes No The treating clinician confirms dornase alfa is of worthwhile benefit to the patient Yes No 3.1. Reduction in hospital admissions in the previous 12 months Number: 3.2. Reduction in the number of treatment courses of oral antibiotics in Number: OR the previous 12 months 3.3. Significant improvement in radiological or bronchoscopy findings Yes No 3.4. An eradication of a pathological organism Yes No 3

13 Patient details Patient name: NHI: D.O.B: Additional supporting information Applicant Signature: Date of Application: / / 4