Named patient use of Colistin Cyclops®
Please note: the information below applies to the Netherlands only. Colistin Cyclops® is not available for Named Patient use in other countries at this moment.
Colistin Cyclops® currently is an unregistered medicine in development, which means that its dosage guidelines, safety and efficacy have yet to be fully established. However, the European Medicines Agency (EMA) and the Dutch Health Inspectorate (IGJ) allow the use of unregistered medicines, such as Colistin Cyclops®, under strict conditions.
Conditions
The conditions for so-called ‘Named Patient use’ of Colistin Cyclops®, amongst others, include the following:
- there is no comparable and satisfactory registered medicine available for the intended indication. This means that other inhaled colistin products are burdensome or intolerable to the patient to such an extent that they cannot or will not be properly used to result in therapeutic efficacy. For example, the time consuming and cumbersome use of nebulizers, and the occurrence of severe cough with other colistin dry powder inhalers are often-used reasons for pulmonologists to prescribe Colistin Cyclops®;
- PureIMS manufactures Colistin Cyclops® following Good Manufacturing Practices (GMP).
Experiences and results
From January 2015 until July 2018 over 20,000 colistin dry powder inhalers (a predecessor of the Cyclops®) were prescribed to cystic fibrosis and bronchiectasis patients. In a retrospective study, lung physicians of the University Medical Center Groningen (UMCG) compared the therapeutic outcome and patient preference of Colistin Cyclops® with that of nebulized colistin. The researchers found that:
- there was no significant difference in annual decline in FEV1% predicted prior to and after switching from nebulization to our dry powder inhaler (median decline -1.56 [-5.57–5.31] and 1.35 [-8.45–6.36]), respectively, p = 0.45 (linear mixed effect model);
- no significant difference existed in the number of intravenous or combined total intravenous and oral antibiotic courses during use of our dry powder inhaler compared to nebulization (1.68 and 2.49 courses during dry powder inhalation therapy versus 1.51 and 2.94 courses during nebulization, p = 0.88 and p = 0.63, respectively);
- of all patients 71.4% preferred therapy with our dry powder inhaler over nebulization. Use of our dry powder inhaler resulted in high treatment adherence with an average adherence rate of 92.5%.
As a result, the researchers concluded that the predecessor of Colistin Cyclops® is a more patient friendly alternative to nebulization, and that its use does not result in significant differences in clinical outcome regarding lung function and exacerbation rate. Read the full study report here.
Based on the feedback from users we further improved the usability and reliability of our colistin dry powder inhaler and developed the Colistin Cyclops®. To date (October 2024) over 80,000 doses of Colistin Cyclops® have been prescribed, predominantly to cystic fibrosis and bronchiectasis patients.
Procedure
If lung physicians in the Netherlands wish to prescribe inhaled colistin, but cannot use one of the available registered options (e.g. nebulized colistin, Colobreathe®), they can fill out a form in which they describe the reasons for the medical need of Colistin Cyclops® use. This form should be sent along with a prescription to the public pharmacy that contacts patients and hands over or sends out the Colistin Cyclops® (i.e. pharmacy ‘De Vriendschap’ in Emmen). The required forms, a detailed information leaflet and instructions for use of the Colistin Cyclops® are available to healthcare providers upon request at info@pureims.com.
Reimbursement
Colistin Cyclops® is fully reimbursed by healthcare payers in the Netherlands if registered alternatives are inadequate.