Patient Eligibility. The clinical community and patient organisations feel it is appropriate and right that all patients diagnosed with Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation who are aged 5 and over and who are ambulatory should have access to ataluren (TranslarnaTM) in England. Ataluren will be added to existing standard treatment, including use of corticosteroids. Patients must be made aware of the start and stop criteria for receiving ataluren treatment and are required to attend their clinics 2 times for assessment within a 14 month period. All patients will sign up to the ‘Managed Access Patient Agreement’.
Patient Eligibility. 4.1 Key patient eligibility criteria to start treatment within managed access include: • Patient meets one of the following criteria: o Clinical diagnosis of SMA type 1, 2, or 3. o Pre-symptomatic of SMA and has been confirmed to have SMA via genetic testing and has one to four SMN2 copies. • Risdiplam is used as a monotherapy. • Must not have had successful treatment with onasemnogene abeparvovec. Non- successful treatment is defined in appendix A. • No permanent ventilation (≥16 hours/day for 21 consecutive days in the absence of acute reversible infection)/ tracheostomy requirement at baseline. Patients who do not meet this criterion but otherwise meet the eligibility criteria should be discussed with the NHS England Clinical Panel. • Mandated data items have been collected prior to starting treatment within this MAA (see section 7, Outcome data). Patients who have started treatment for SMA prior to this MAA are not required to repeat an assessment if a previous assessment has captured all mandated data items (see table 2) within the last 6 months. • Patient/carer has signed the ‘Managed Access Patient Agreement’ and agreed to the associated monitoring, clinical assessments and sharing of data for the purpose of the MAA (see section 10, Patient consent). • Clinician confirms they: o will submit data to SMA REACH as set out in the DCA. o have made the patient/carer aware that there are other treatments for SMA, which may be more suitable for that patient. o confirm annually, via completion of an addition Blueteq form, that the patient continues to receive benefit from treatment. • Risdiplam will be otherwise used as set out in its Summary of Product Characteristics (SmPC).
Patient Eligibility a. Company and THPR will develop a system to verify patient eligibility. As used in the context of this Agreement, an individual is a "patient" of Company (with the exception of State-operated or funded AIDS drug purchasing assistance programs) only if:
Patient Eligibility. 3.1 Key patient eligibility criteria for the use of axicabtagene ciloleucel in the Cancer Drugs Fund include: The first part of the Blueteq form is for the approval of leucapheresis and manufacture of CAR-T cells. This includes the following eligibility criteria: • the application is being made by and that leucapheresis for and treatment with axicabtagene ciloleucel-modified CAR-T cells will be initiated by a consultant haematologist or medical oncologist specifically trained and accredited in the use of systemic anti-cancer therapy and working in an accredited CAR-T cell treatment centre and who is a member of the National CAR-T Clinical Panel for DLBCL and high-grade B-cell lymphoma (HGBCL) and a member of the treating Trust’s DLBCL and HGBCL CAR-T cell multidisciplinary team. • the patient is an adult (age 18 years or over) on the date of approval for axicabtagene ciloleucel by the National CAR-T Clinical Panel for DLBCL and HGBCL. • the patient has a confirmed histological diagnosis of DLBCL or HGBCL: o Diffuse large B-cell lymphoma (DLBCL) NOS (including ABC and GCB types) or o High grade B-cell lymphoma (HGBCL) with or without MYC and BCL2 (double hit) and BCL6 (triple hit) re-arrangements or o Transformed follicular lymphoma (TFL) to DLBCL or o T cell/histiocyte-rich large B-cell lymphoma or o Primary cutaneous DLBCL of leg type or o HHV8 positive DLBCL o DLBCL associated with chronic inflammation or o EB virus positive DLBCL Note: Patients with Burkitt lymphoma or primary mediastinal B cell lymphoma or primary CNS lymphoma or Xxxxxxx’x transformation to DLBCL are not eligible for treatment with axicabtagene ciloleucel in this indication. • the histological diagnosis of DLBCL or HGBCL or transformed lymphoma to DLBCL has been either made by or reviewed and confirmed by a designated lymphoma stem cell transplant centre. • prior to consideration of CAR-T cell therapy the patient’s disease has been re-biopsied unless either the patient had outright progressive disease on standard 1st line chemo-immunotherapy or a biopsy is unsafe in which case the patient must have progressive disease at previously known sites of active disease. In such situations the original diagnostic biopsy review is acceptable. o All patients with transformed follicular lymphoma to DLBCL who fulfil the criteria below must have a re-biopsy and have confirmation of DLBCL histology prior to consideration of CAR- T cell therapy, either: ▪ no biopsy necessary as the patient had outrigh...
Patient Eligibility. MANAGER shall: (a) obtain and maintain current eligibility lists from the Payors subject to this Agreement; (b) assist in determining eligibility of patients for coverage prior to the provision of medical or other services; and (c) reconcile retroactive denials of eligibility with the authorization processes established by IPA or the applicable Payor with the Payor Contract, and with applicable provisions of health plan benefit agreement(s) or other evidences of coverage.
Patient Eligibility. All admissions are subject to prior authorization by County. The following individuals shall be considered acceptable for admission:
Patient Eligibility. 3.1 Key patient eligibility criteria for the use of onasemnogene abeparvovec for pre-symptomatic SMA during the period covered by the MAA include: • patient has a confirmed genetic diagnosis of 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and up to 3 (three) copies of the SMN2 gene, • patient does not display any clinical manifestations that are strongly suggestive of SMA, • patient has not received any prior treatment with nusinersen or risdiplam, • patient has confirmed anti-adeno-associated virus 9 (anti-AAV9) antibody titres below 1:50, • onasemnogene abeparvovec will be otherwise used as set out in its Summary of Product Characteristics (SmPC).
Patient Eligibility. The clinical community and MPS Society feel it is appropriate and right that all patients have access to elosulfase alfa (Vimizim) in England. The only exception from starting elosulfase alfa in confirmed cases of MPSIVA will be where:- The patient is diagnosed with an additional progressive life limiting condition where treatment would not provide long term benefit e.g.; cancer or multiple sclerosis Patient / Parent are unwilling to comply with the associated monitoring criteria: The patient has a lung capacity (FVC) of less than 0.3 litres and require ventilator assistance; or The patient is unwilling to comply with the associated monitoring criteria: All patients are required to attend their clinics three times a year for assessment within a 14 month period. All patients will sign up to this ‘Managed Access Patient Agreement’.
Patient Eligibility. 3.1 Key patient eligibility criteria for the use of palbociclib in combination with fulvestrant in the Cancer Drugs Fund include: • The application for palbociclib in combination with fulvestrant is made by and the first cycle of palbociclib plus fulvestrant will be prescribed by a consultant specialist specifically trained and accredited in the use of systemic anti-cancer therapy • Patient has histologically or cytologically documented oestrogen receptor positive and HER-2 negative breast cancer • Patient has metastatic breast cancer or locally advanced breast cancer which is not amenable to curative treatment • Patient is male or is female and if female is either post-menopausal or if pre- or peri-menopausal has undergone ovarian ablation or suppression with LHRH agonist treatment • Patient has an ECOG performance status of 0 or 1 or 2 • Patient has received previous endocrine therapy according to one of the three populations as set out below as these are the groups on which the NICE Technology Appraisal for palbociclib plus fulvestrant focused. Please record which population the patient falls into: o Patient has progressive disease whilst still receiving adjuvant or neoadjuvant endocrine therapy for early breast cancer with no subsequent endocrine therapy received following disease progression or, o Patient has progressive disease within 12 or less months of completing adjuvant endocrine therapy for early breast cancer with no subsequent endocrine therapy received following disease progression or, o Patient has progressive disease on 1st line endocrine therapy for advanced/metastatic breast cancer with no subsequent endocrine therapy received following disease progression. • Patient has had no prior treatment with a CDK 4/6 inhibitor unless either abemaciclib (in combination with fulvestrant) or ribociclib (in combination with fulvestrant) has been stopped within 6 months of its start solely as a consequence of dose-limiting toxicity and in the clear absence of disease progression or palbociclib has been received as part of an early access scheme for the combination of palbociclib plus fulvestrant and the patient meets all the other criteria set out in this form. • Patient has had no prior treatment with fulvestrant • Patient has had no prior treatment with everolimus • Palbociclib will only be given in combination with a fulvestrant • Treatment will continue until there is progressive disease or excessive toxicity or until the patient chooses to discon...
Patient Eligibility. NICE, the HPP specialists and Metabolic Support UK agree that patients in England should be able to have asfotase alfa (Strensiq®) as long as they meet the starting rules that are described in the MAA and are being treated by a specialist in HPP in a specialist HPP centre. If a patient meets the starting rules, they will be required to go to their clinic appointments for tests at three months, six months and 12 months in the first year of treatment, and a then at least every 6 months after that for a check-up. • All patients (whether by themselves or through a parent or guardian) will need to sign up to this Managed Access Patient Agreement.
