International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conference

Abstract

In Orlando, the 14th International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conference was held at Sea World, FL, USA. Despite the temptations of the environment, delegates from industry, academia and health policy convened, engaged in lively discussion and presented new and fascinating data on various topics. This year’s theme was ‘Healthcare reform revisited’. Researchers traveled from all over the world to present their research. A broad variation by region and topic could be seen, ranging from a cost analysis on hepatitis B vaccination in Iran and an analysis on Hoehn and Jahr staging of Parkinson’s disease in Singapore, to the costing of infertility in Turkey and posaconazole cost–effectiveness in the treatment of invasive aspergillosis in Korea.

Aside from the traditional focus of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) conferences on individual drug studies and integrative workshops, the continuing of some trends was again visible at this ISPOR Conference (2009). In particular, the ISPOR seemed to focus more on the recent topics of personalized medicine – for example, using genetic markers – and the subject of increasing healthcare costs owing to expensive in-hospital drugs, including monoclonal antibodies for oncology, hematology, rheumatology and Crohn’s disease. Furthermore, increasing attention was given to infectious diseases, inclusive mass vaccination programs for human papiloma virus, hepatitis B and (pandemic) influenza. In addition, the role of the Human Tissue Authority (HTA) in various countries was analyzed in detail, including the Scottish Medicines Consortium (SMC), NICE, the Canadian Agency for Drugs and Technologies in Health (CADTH), a range of developing countries and new EU-countries Slovenia and Slovakia (Tomek and Bielik).

Expensive in-hospital drugs & personalized medicine

Various new, expensive in-hospital drugs are emerging in hematology. Increasing requirements for cost–effectiveness in this area necessitate the development of quality-adjusted life-years (QALYs) measurements in these groups. For example, in mantle cell lymphoma the EuroQol EQ-5D was tested. It was concluded that the EQ-5D is a valid instrument in this disease.

Bevacizumab was investigated as an add-on to folinic acid, fluorouracil and irinotecan (FOLFIRI) in patients with metastatic colorectal cancer. Gyldmark et al. reported that the add-on would be a cost-effective treatment option compared with FOLFIRI alone. Various combinations of drugs were investigated in multicountry studies in Europe for non-small-cell lung cancer. In all countries, bevacizumab-based therapies were found to be economically favorable.

Two papers reported favorable cost–effectiveness for sunitinib and sorafenib in Brazil and Israel for the indication of renal cell carcinoma. This was found for the comparison with best supportive care and with other drug treatment options.

Studies on genetic testing prior to treatment initiation were directed at breast cancer and warfarin. Genotypes involved in these analyses were CYP2C9, VCORC1 and CYP2D6. One of the studies in breast cancer performed an extensive value-of-information analysis. The latter analysis suggested that primary research should preferably be diverted to genotype-specific tamoxifen efficacy.

Infectious diseases

In a GlaxoSmithKline coauthored study, Swedish researchers investigated the cost–effectiveness of the newly registered 10-valent pneumococcal non-typeable Haemophilus influenzae protein-D conjugate vaccine (PHiD-CV) for that country. They suggested that PHiD-CV might be cost effective when compared with no vaccination and with pneumococcal conjugated vaccination (PCV)-7 (Wyeth Pharmaceuticals) according to the criteria applied by the Swedish National Board of Health and Welfare. Health gains were primarily driven by cases of acute otitis media prevented. In another paper, GlaxoSmithKline reported results for lowered-dose schedules than the generally applied one of four doses. For Canada, slight decreases in cases prevented were estimated over the whole range of invasive and noninvasive disease if three instead of four doses were applied, for both PCV-7 and PHiD-CV. No results on noninvasive pneumonia were reported.

Some methodological work on different model approaches was presented. Snedecor et al. presented the first dynamic transmission model for PCV-7, enhancing the potentials for validly modeling the indirect herd protection effects in nonvaccinated populations. Chang et al. illustrated the use of frontier analysis in vaccination studies, focusing on the PCV in Taiwan. Demarteau and Standaert explicitly compared the features of a cross-sectional population model and a cohort model for PCV. It was concluded that if the indirect herd protection effects may be large the population model may have advantages.

Two studies analyzed the remaining potentials of the polysaccharide pneumococcal vaccine in the era of PCV-7, PHiD-CV and (yet to be registered) PCV-13. Studies for Canada (BioMedCom Consultants, Inc.) and Turkey (Haceteppe University, Ankara) were presented, both claiming that continued recommendation for elderly and at-risk groups is justified. Yet, updated cost–effectiveness analyses are warranted in the era of conjugated vaccines with potential herd protection effects of infant vaccination on these elderly and at-risk individuals.

Various studies on HIV/AIDS were presented. In a study from the University of South Carolina, USA, on Kenya, it was concluded that switching to second-line therapy with lopinavir/ritonavir would be cost effective if strict criteria for clinical and immunological failure were applied. Cost effective was defined according to the WHO-benchmarking at twice the country’s per capita gross domestic product (US$1700 for Kenya). Annemans et al. applied the efficiency frontier on 26 compared strategies in the treatment of resistant HIV/AIDS-patients in Germany. In Germany, the Institute for Quality and Efficiency in Health Care (IQWIG) has recently proposed the use of this frontier to support reimbursement decisions. Together with the John’s Hopkins University, MA, USA, Mexican researchers estimated that the use of Truvada^® in place of standard HIV therapy of combivir and kivexa would be cost effective for the Mexican healthcare system.

Garrison et al. showed favorable economic potentials for innovative measles vaccines, involving needle-free jet injection, aerolized delivery, inhalable dry-powder vaccine and DNA vaccine.

Individual drug studies

The first economic studies on aliskiren – the first marketed direct rennin inhibitor - were reported in Orlando, FL, USA. In particular, one study from Novartis and the Charles University, Prague, Czech Republic, suggests that once-daily aliskiren is highly cost effective as an add-on to losartan for hypertensive and diabetic patients with nephropathy in the Czech situation. Using different approaches, both low incremental cost–effectiveness ratio (ICERs) under 1000 per QALY and cost savings were reported. Generally, RAS-combination therapies provide conflicting results on their efficacy against monotherapies from that class. One further study by Medimpact Healthcare Systems CA USA, focused on utilization and persistence of aliskiren outside the clinical trial settings.

An exciting multidrug study was presented by the University of Houston, TX, USA, comparing comparative effectiveness of individual ACE-inhibitors in patients with chronic heart failure. In particular, enalapril, fosinopril and lisonopril were found to have a statistically significant additional protective effect on the risk of death within the 1-year study period. No cost–effectiveness implications were provided. Obviously, further comparative studies of this type are needed to draw definite conclusions.

Farkouh et al. analyzed the cost–effectiveness of rasagiline versus ropinirole and levadopa in US Parkinson’s disease patients. The analysis was based on one of recent studies to investigate pre-symptomatic treatments in Parkinson’s disease (the Early Monotherapy for Parkinson’s Disease Outpatients [TEMPO] study). Early rasagiline was associated with lower per patient costs (-US$1660) and higher QALYs (0.0608).

Based on noninferiority clinical trials, various studies reported that dabigatran etexilate is cost saving as thromboprophylaxis compared with enoxaparin, with comparable safety and efficacy. Cost savings are related to the oral form of dabigatran, enhancing patient ease of administration. Current studies focus on the application after hip and knee surgery; further studies in atrium fibrillation are underway. Other comparisons between antithrombotic drugs were also presented. A study in a Moscow hospital (Russia) compared enoxaparin and unfractionated heparin and concluded that enoxaparin was more beneficial, both in terms of costs and health compared with the unfractionated drug.

Mauskopf et al. presented a critical assessment of individual drug studies of dual antiplatelet therapy (generally, clopidogrel and aspirin). In particular, they noted that the sensitivity analyses that were performed were not fully adherent to existing HTA guidelines. For an overview of such guidelines, see the ISPOR website [1].

Workshops

During the ISPOR conferences, workshops generally tend to attempt a certain level of integration of various topics. For example, one panel session featured some of the UK researchers involved in the NICE assessments, including Mike Drummond and Mark Sculpher. NICE may certainly be considered as (one of) the leading institute(s) in applying economic evaluation in healthcare decision-making. The panel achieved its goal in further clarifying the approaches chosen by NICE, inclusive indirect comparison methodologies. A further workshop by the University of Groningen, the Netherlands described in detail in the different methods applied in indirect comparison if head-to-head trials are not available. Hundreds of participants visited the workshop. Both deterministic and Bayesian algorithms were presented. In particular, de Vries and Boersma presented application to recently registered antifungal drugs and antithrombotics. Next to value of information methods, indirect comparison may certainly be expected to be a key area for further methodology development in health economics in coming years. This was also stressed in one individual research paper by Heron Evidence Development Ltd UK, showing that indirect comparison is increasingly used in NICE submission and is now also reflected explicitly in NICE guidance.

Financial & competing interests disclosure

The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.