Objectives: Australia relies on managed entry agreements (MEAs) for many medicines added to the national Pharmaceutical Benefits Scheme (PBS). Previous studies of Australian MEAs examined public domain documents and were not able to provide a comprehensive assessment of the types and operation of MEAs. This study used government documents approved for release to examine the implementation and administration of MEAs implemented January 2012 to May 2016.

Methods: We accessed documents for medicines with MEAs on the PBS between January 2012 and May 2016. Data were extracted on Anatomical Therapeutic Classification (ATC), type of MEA (financial, financial with outcomes, outcomes, and subcategories within each group), implementation and administration methods, source of MEA recommendation, and type of economic analysis.

Results: Of all medication indication pairs (MIPs) recommended for listing, one-third had MEAs implemented. Our study of eighty-seven MIPs had 170 MEAs in place. The Government's expert health technology assessment (HTA) committee recommended MEAs for 90 percent of the eighty-seven MIPs. A total of 81 percent of MEAs were simple financial agreements: the majority either discounts (32 percent) or reimbursement caps (43 percent). Outcome-based MEAs were least common (5 percent). Ninety-two percent of MEAs were implemented and operated through legal agreements. Approximately half of the MIPs were listed on the basis of accepted claims of cost-minimization. Forty-nine percent of medicines were in ATC L group.

Conclusion: Advice from HTA evaluations strongly influences the implementation of ways to manage uncertainties while providing access to medicines. The government relied primarily on simple financial agreements for the managed entry of medicines for which there were perceived risks.

Objectives: Ipilimumab is the first licensed immune checkpoint inhibitor for treatment of melanoma. The promising results of the registration clinical study need confirmation in real practice and its clinical success comes together with a relevant budget impact due to the high price of this drug. The aim of this work is to describe a new model of economical sustainability of ipilimumab developed in an Italian reference center for melanoma treatment.

Methods: This retrospective, observational, and monocentric study was carried out at the Veneto Institute of Oncology. Ipilimumab was administered to fifty-seven patients with advanced melanoma. Overall survival, progression free survival, and toxicity were evaluated. A local management procedure was evaluated together with the cost-saving strategies implemented by the Italian Medicines Agency (AIFA).

Results: We demonstrated that the use of ipilimumab for metastatic melanoma in real practice had an efficacy and toxicity similar to that reported in the literature. In this scenario, our management model (centralization of compounding + drug-day) permitted savings up to the 11.1 percent of the gross cost for the drug (calculated assuming that no cost saving procedures were applied) while the policy of cost containment designed by AIFA produced an additional 6.2 percent of savings.

Conclusions: In real practice conditions, the centralized administration of ipilimumab allows to replicate the results of clinical studies and in the meantime to contain the cost associated with this drug. The local strategy of management can be readily applied to most of the high cost drugs compounded in the hospital pharmacy. Impact of findings on practice: (i) We describe a new model of economic sustainability (drug-day, centralization of compounding, payback systems) of an expensive and innovative drug, ipilimumab, for treatment of melanoma within an Italian cancer center. (ii) This pivotal study demonstrated that a cost containment strategy is feasible and it needs the cooperation of all healthcare providers (oncologists, pharmacists, nurses, and technicians) to guarantee the full efficiency of the process.

Objectives: The AIFA (Agenzia Italiana del Farmaco—Italian Medicines Agency) Monitoring Registries track the eligibility of patients and the complete flow of treatments, guaranteeing appropriateness in use of pharmaceutical products, according to approved indications.

Methods: This study describes the Italian pharmaceutical context and the aims and functioning of AIFA Monitoring Registries, focusing on the applications to the Managed Entry Agreements (MEAs) and HTA approaches.

Results: The AIFA Monitoring Registries System has been operational in Italy since 2005. In 2012, the system became part of the NHS Information Technology system, aiming at enhancing appropriate use of pharmaceuticals and efficiency of the administrative activity. Currently, seventy-six medicines are monitored through the system, corresponding to fifty-eight therapeutic indications; individual treatments recorded are more than 515,000, for a population of approximately 505,000 patients. For each monitored product, patients eligible for treatment are registered in the specific therapeutic indication dynamic monitoring database to collect epidemiologic and clinical data, including data on the safety profile, and ex-post information missing at first evaluation stage.

Conclusions: AIFA Monitoring Registries allow the evaluation of the pharmaceuticals’ performance in clinical practice and may promote innovation and quicker access to medicines at affordable prices, for the benefit of patients.