The use of economic evaluations produced by uvef for HTA report assesments in the Veneto Region

ORGANIZATION: The Pharmacy and Therapeutic Committee of the Veneto Region, Italy, is a regulating body responsible for the definition of the Regional Hospital Formulary in Veneto. Veneto comprised a territory of 4.800.000 inhabitants in the north-east part of Italy. The Unit for the Evaluation of Drug Efficacy (Unità di Valutazione dell'Efficacia del Farmaco, UVEF) is the technical group responsible for the evaluation reports of new drugs to be submitted to the Regional Committee (RC). The Committee evaluates the introduction of new technologies based on HTA reports that contain the following relevant sections: general information; alternative treatments and insights from existing guidelines; efficacy and safety data; other HTA reports; pharmacoeconomics data from the literature; comparative drug costs; budget impact analysis (BIA). PROBLEM OR ISSUE ADDRESSED: In august 2009 the regulation of the Committee was modified: the decisions are now taken based on HTA criteria. Since 1st January 2010 the stakeholders (manufacturers, clinicians, scientific societies or local drug & therapeutics committees) are involved in HTA process with the introduction of a new request form for the submission of new drugs to the RC. The stakeholders submitting a new drug application must present a BIA and other economic evidences supporting the introduction in the regional formulary. GOALS: We analyzed the HTA reports produced by UVEF from January 2009 to June 2010 in order to verify first of all the rate of assessments presenting economic evidences (published pharmacoeconomic studies, BIA, other HTA reports) and then to evaluate the usefulness of economic evidences for the appraisals. We also investigated if there is a significant difference between 2009 and 2010 in the number of BIA presented by the stakeholders. OUTCOMES ITEMS USED IN THE DECISION The presence of therapeutic alternatives for the same disease; efficacy data from main clinical trials (active controlled trials, placebo controlled trail or not controlled trials); considerations on safety, efficacy and cost by the main regulatory and HTA agencies in Europe, e.g. Scottish Medicine Consortium (SMC) and NICE; relevant organizational and cost issues arising from published pharmacoeconomic studies (cost-effectiveness, cost-utility etc), when applicable; comparative drug costs and BIA presented by the manufacturers or estimated by UVEF. IMPLEMENTARY STRATEGY We developed a Database to record all clinical and economic outcomes items for each drug evaluated from January 2009 to June 2010. We performed a statistical analysis using Fisher’s exact text (two tailed) to evaluate the variation on the number of BIA submitted by stakeholders before and after the introduction of the new regional regulation. RESULTS During the year 2009 and the first six months of 2010, UVEF produced 42 HTA reports: 30 new drugs and 12 new indications. Only 2 drugs had no alternative: an orphan drug for a rare disease (carglumic acid) and a last-line drug used in terminal patients (methylnaltrexone). For the other 40 drugs were available: in 22 cases RCTs with active-control, in 8 cases RCTs placebo-controlled, in 8 cases RCTs controlled versus no treatment or best supportive care and in 2 cases not-controlled trials. About the economic evidences were available: in 28 cases international HTA reports (20 from SMC and 8 from both SMC and NICE); in 14 cases pharmacoeconomic studies and in 11 cases BIA (5 produced by UVEF and 6 by both manufacturers and UVEF). Ten reports had no economic evidences. The Committee did not approve or suspended the appraisals for 8 drugs, in 6 cases for economic reasons: higher expenditures (67%), lack of manufacturers’ BIA/ pharmacoeconomic studies (33%). We verified a variation on the number of BIA produced by manufacturers before and after the introduction of the new regional regulation. Out of 28 submissions during 2009 only 1 had BIA, whereas out of 14 submission during 2010 5 had BIA; the difference performed using Fisher’s exact text was statistically significant (p=0.010). LESSON LEARNED: In our evaluation process, in the last two years the economical evaluations and BIA are considered increasingly. The new regulation of the D&T Committee of the Veneto Region increased significantly the number of BIA presented by the manufacturers. Most of negative appraisals were based also on economic reasons. The HTA criteria are now always applied in UVEF assessment and D&T Committee appraisal process