Decision-making criteria for medicine reimbursement in Slovenia: an expert panel discussion.

BACKGROUND: Rational and transparent Health Technology Assessment and reimbursement decision-making are crucial for healthcare system sustainability. A part of the reimbursement process are decision-making criteria which should be clearly defined. METHODS: The study aimed to obtain an insight into understanding and relevance of potential criteria for the medicine reimbursement decision-making process in Slovenia. A semi-structured guided focus panel was performed in June 2017 with five Slovenian experts covering principal healthcare system sectors. First, criteria understanding and relevance for medicine reimbursement decision-making were discussed. Second, healthcare priorities and societal values affecting decision-making were debated. The analysis was carried out with NVivo 11 by two independent researchers who coded the verbatim transcript in three coding steps based on the experts' interpretations and original ideas. RESULTS: Seven decision-making criteria were derived. Among those, the impact a disease has on the lives of patient family and caregivers and the indirect medicine benefit for them were new aspects comparing to the existing criteria set in Slovenia. The experts expressed that the same decision-making criteria are relevant for evaluating any health technology, allowing for different criteria weights. They also suggested a system that would allow re-evaluation of reimbursement decisions once real-world clinical data are available. CONCLUSIONS: As proposed by the international frameworks and tools, the Slovenian healthcare experts consider including multiple aspects more ethical and comprehensive than considering a single criterion, e.g. cost-effectiveness, existing in some healthcare systems. They recognize that in the existing decision-making process, health perspectives of the public represent a largely missed aspect.

Patient Access to Medicines for Rare Diseases in European Countries.

BACKGROUND: The number of authorized orphan and non-orphan medicines for rare diseases has increased in Europe. Patient access to these medicines is affected by high costs, weak efficacy/safety evidence, and societal value. European health care systems must determine whether paying for expensive treatments for only a few patients is sustainable. OBJECTIVES: This study aimed to evaluate patient access to orphan and non-orphan medicines for rare diseases in 22 European countries during 2005 to 2014. METHODS: Medicines for rare diseases from the Orphanet list, authorized during 2005 to 2014, were searched for in the IMS MIDAS Quarterly Sales Data, January 2005 - December 2014 (IQVIA, Danbury, CT). The following three measures were determined for each country: number of available medicines, median time to continuous use, and medicine expenditure. A medicine was considered available if uninterrupted sales within a 1-year period were detected. RESULTS: From 2005 to 2014, 125 medicines were authorized and 112 were found in the search. Of those, between 70 (63%) and 102 (91%) were available in Germany, the United Kingdom, Italy, France, and the Scandinavian countries. These countries were also the fastest to enable continuous use (3-9 mo). Only 27% to 38% of authorized medicines were available in Greece, Ireland, Bulgaria, Romania, and Croatia, which took 1 to 2.6 years to begin continuous use. A country's expenditure on medicines for rare diseases in 2014 ranged between €0.2 and €31.9/inhabitant. CONCLUSIONS: Patient access to medicines for rare diseases varies largely across Europe. Patients in Germany, Scandinavian countries, Switzerland, France, and the United Kingdom can access larger numbers of medicines in shorter time.

Therapy modifications during hospitalization in patients with chronic heart failure.

BACKGROUND: Guidelines on suggested pharmacological treatments for heart failure (HF) are not optimally implemented in clinical practice and whether pharmacotherapy adjustment actually happens in daily practice is largely unknown. We aimed to investigate pharmacotherapy modifications during hospitalization. METHODS: This was a prospective observational survey where all admissions were screened for HF; 210 patients were included. The guideline adherence index (GAI) and modified GAI (mGAI, if ≥50% of target dose) were used to grade the pharmacotherapy. RESULTS: Among 198 patients discharged alive (mean age 77years, 51% male), 49% had preserved left ventricular ejection fraction (PLVEF) and 30% had left ventricular systolic dysfunction (LVSD); the echocardiography report was unavailable for 21%. Angiotensin-converting enzyme inhibitors/angiotensin receptor blockers, beta-blockers and mineralocorticoid receptor antagonists were prescribed to 78%, 58% and 20% of patients on admission and 72%, 65% and 23% at discharge, respectively. Overall, 14% of patients met GAI-3, but at discharge only 7% met mGAI-3. One of the key drugs was stopped or down-titrated in 27%. During follow-up, 21% of patients died (25% with LVSD). Patients with LVSD discharged with at least one HF drug had a lower risk of death than patients with none (HR=0.142, 95% CI=0.029-0.683, p=0.015). Patients with PLVEF had better prognosis than LVSD patients when no HF drugs were prescribed at discharge (HR=0.075, 95% CI=0.009-0.627, p=0.017). CONCLUSIONS: The pharmacotherapy of HF patients did not improve significantly during hospitalization, remaining suboptimal. Treatment with key drugs was terminated or reduced in a significant proportion of patients, mostly without specific written justification.

Clinical-pharmacist intervention reduces clinically relevant drug-drug interactions in patients with heart failure: A randomized, double-blind, controlled trial.

BACKGROUND: Incidence of drug-drug interactions (DDIs) increases with complexity of treatment and comorbidities, as in heart failure (HF). This randomized, double-blind study evaluated the intervention of the pharmacist on prevalence of clinically relevant DDIs (NCT01855165). METHODS: Patients admitted with HF were screened for clinically relevant DDIs, and randomized to control or intervention. All attending physicians received standard advice about pharmacological therapy; those in the intervention group also received alerts about clinically relevant DDIs. Primary endpoint was DDI at discharge and secondary were re-hospitalization or death during follow-up. RESULTS: Of 213 patients, 51 (mean age, 79 ± 6 years; male, 47%) showed 66 clinically relevant DDIs and were randomized. For intervention (n=26) versus control (n=25), the number of patients with and the number of DDIs were significantly lower at discharge: 8 vs. 18 and 10 vs. 31; p=0.003 and 0.0049, respectively. Over a 6 month follow-up period, 11 control and 9 intervention patients were re-hospitalized or died (p>0.2 for all). No significant differences were seen between control and intervention for patients with eGFR <60 mL/min/1.73 m(2) (78%) for re-hospitalization or death (10 vs. 7; p=0.74). CONCLUSIONS: Pharmacist intervention significantly reduces the number of patients with clinically relevant DDIs, but not clinical endpoints 6 months from discharge.