The emergence of new medical technologies and budget restrictions has led to a substantial increase in the use of hospital-based health technology assessment (HB-HTA). This qualitative study explores whether there is a possibility and interest to realize the collection and dissemination of HB-HTA reports on an international scale by exploring the opinions from HB-HTA experts.

A survey was designed and sent to an international group of experts knowledgeable in HB-HTA from eighteen different countries. The survey contained questions about their opinions on the collection and distribution of HB-HTA information, and about the meaningful dimensions, barriers and values about a database. The data obtained were analyzed through the method of content analysis.

A total of thirty-six experts (response rate of 18.3 percent) responded to the survey. The obtained data shows that all respondents agree that the collection of HB-HTA reports is useful. Moreover, 41.7 percent of respondents that are in the position of sharing HB-HTA reports (n = 24) mentioned that full HB-HTA reports can be shared. Many other respondents reported that confidentiality (45.7 percent) and investment into a database (40.0 percent) are important barriers for the dissemination of HB-HTA reports.

There seem to be enough demand and willingness to share all or most reports by a large community of HB-HTA producers. Therefore, there is a need for a well-designed database with regular maintenance and complete, comparable, and qualitative HB-HTA reports. The database of the AdHopHTA project could potentially facilitate this process.

The fifth Health Technology Assessment International (HTAi) Asia Policy Forum (APF) was held in Beijing, November 2017. The topic of the meeting was ‘Universal Health Care in the Asia Region: Overcoming the Barriers using HTA and Real World Data’. This presentation will focus on the use of real world data (RWD) as a mechanism of informing HTA in the Asia region – from the perspective of industry that were represented at the APF.

The 2017 HTAi APF had senior representatives from 11 pharmaceutical and medical device companies active in the region. A keynote presentation was delivered that explored the findings of the background paper and survey, and there were guided breakout group discussions.

RWD is needed in all stages of the life cycle of a product; however, of greatest importance to industry is RWD to develop value propositions, inform HTA assessments, gauge market need and demonstrate where a new technology sits within an established clinical pathway. Although the HTA agencies present at the APF stated that RWD is available in the Asia region, this was not reflected by industry members who had limited awareness and access. Key concerns raised included whether this RWD is available for public use (and what caveats will be placed around its release), the quality of the data, whether it is longitudinal, and if it includes costs.

There is a clear difference in the perspective of RWD between industry and HTA agencies in the Asia region. It is possible that this difference is driven by the apparent lack of publicly available data in the region and industry members comparing the use of RWD in Asia with the use in higher-income countries. This can be improved with clearer definitions, increased dialogue and multi-stakeholder collaboration in the region.

Colposcopes are expensive, heavy, and need specialized technical service, which may outreach the capacity of low-resource settings. Our aim was to assess the performance of smartphone-based digital images for the detection of cervical intraepithelial neoplasia grade 2 or worse (CIN2+).

Human papillomavirus (HPV)-positive women recruited through a cervical cancer screening campaign had VIA/VILI assessment (visual inspection after application of acetic acid/lugol's iodine). Cervical digital images were captured with a smartphone camera, randomly coded with no prior selection and distributed on an online database (Google Forms) for evaluation. Healthcare providers were invited to evaluate the images and identify CIN2+. The gold standard was the histopathological diagnosis. The sensitivity and specificity for the detection of CIN2+ was assessed for each reader and reported with the 95 percent confidence interval (Clopper-Pearson method).

One hundred twenty-five consecutive HPV-positive women were included, with 19 CIN2+ (15.2 percent). Forty-five gynecologists completed the assessment, one-third were considered as experts (>50 colposcopies) and two-thirds as novices (<50 colposcopies). The sensitivity and specificity for CIN 2+ detection was 71.3 percent (67.0–75.7 percent) and 62.4 percent (57.5–67.4 percent), respectively. The performance of novices and experts was similar. The readers assessed 73.1 percent of images as acceptable for diagnostic.

Smartphone-based digital images, with its high portability, have a great potential for the diagnosis of CIN2+ in low-resource context.

Universal health care (UHC) leads to better health, educational outcomes and productivity. However, Asian healthcare systems are experiencing huge pressures and are striving to achieve UHC. With this in mind, the Health Technology Assessment International (HTAi) Asia Policy Forum (APF) tackled, ‘Universal Health Care in the Asia Region: Overcoming the Barriers using HTA and Real World Data (RWD)’.

The HTAi APF convened forty-four senior representatives from HTA agencies and industry from Asia. Through a mixture of keynote presentations and guided group discussions, APF members spent two days grappling with the topic.

There were a number of key messages from the APF, plus actions that were identified by APF members. The actions included: (i) A standardized HTA methodology for the prioritization of technologies in the Asia region should be developed to support health care systems; (ii) HTAi need to define what real-world data means in the HTA glossary; (iii) Members of the APF should develop a catalogue of what public and private data is available across countries in the region; (iv) A policy statement that agencies can use with a common approach to the release of data needs to be developed.

HTA and access to RWD were identified as essential tools to be used in achieving the goal of UHC; this goal should indeed be pursued in the Asia region. However, issues such as transparency and accountability of HTA, and trust and collaboration between the public and private sectors are important concerns which need to be addressed in order to progress this goal. The recommendations for actions resulting from this successful APF update on progress towards these identified actions will be presented.

It is not yet established the advantages between amphotericin B lipid complex (ABLC) and liposomal (L-AmB) in patients with invasive fungal infections refractory to usual doses of conventional AmB (d-AmB), previous renal impairment, or unacceptable d-AmB renal toxicity. This systematic review aims to compare ABLC and L-AmB effectiveness and safety outcomes in these subgroups of patients.

The search was performed on Medline, Cochrane Library, EMBASE, and LILACS databases. Inclusion criteria: treatment comparing L-AmB with ABLC; patients who had (i) refractory infection after being treated with d-AmB, (ii) previous renal impairment, or (iii) unacceptable d-AmB toxicity. Two investigators independently screened the search results, assessed trial quality, and extracted data. A total of 1,054 articles were identified in the literature. Among those, eleven were selected for full-text reading and five met the inclusion criteria.

The five articles included reported on four separate observational studies. Overall, no significant difference was found in clinical relevant outcomes as new-onset dialysis, length of hospital stay, or mortality when comparing both lipid formulations. The studies reported a trend toward lower nephrotoxicity in patients treated with L-AmB. However, the results were imprecise and heterogeneous and the studies presented important methodological biases.

The studies included in this systematic review pointed toward less nephrotoxicity events in the L-AmB group. However, due to low quality of evidence and no statistically significant differences in other clinical relevant outcomes, there is no definitive evidence of overall superiority in effectiveness or safety outcomes regarding one lipid formulation or another in this population subgroup.

Managed Entry Agreements (MEAs) enable payers to subsidize access to new medicines while addressing uncertainties. Uncertainties may relate to the criteria for decision-making articulated in decision-making frameworks. The study's aim was to determine if there was any association between the type of MEA and criteria considered during decision-making.

All medicines with MEAs listed on the Australian national subsidy scheme between 2012-2016 were identified. Data were extracted on the types of MEA and information related to the criteria considered in decision-making for each medicine and its associated indication (i.e. a medicine-indication pair [MIP]). The criteria considered in decision-making included the comparator (therapy to which it was compared), type of economic analysis, accepted value, budget impact, financial cost of supply, cost of therapy per patient, access control (such as restrictions or prior authorization), and clinical need. Associations between types of MEA and the criteria were assessed using Chi Squared test.

There were 87 MIPs, of which 56 had only financial MEAs and 31 had performance-based MEAs. Coverage with evidence development MEAs had very high incremental cost-effectiveness ratio (ICER)/quality adjusted life year (QALY) (74 percent > AUD 50,000 [USD 37,822]). Financial MEAs where performance measures were linked to reimbursement had lower ICER/QALY (13 percent > AUD 50,000 [USD 37,822]) but greater budget impact (33% > AUD 80million [USD 60.5million]) compared to simple financial MEAs. A statistically significant association (Cramer's V = 0.5, p < 0.001) was only found between performance-based MEAs and the cost of unsubsidized therapy per patient.

The main influence on the choice of performance based MEA was the provision of access to clinically important medicines with a high treatment cost for patients.

Reimbursement decisions on orphan drugs carry significant uncertainty, and as the amount increases, so does the risk of making a wrong decision, where harms outweigh benefits. Consequently, patients often face limited access to orphan drugs. Managed access programs (MAPs) are a mechanism for managing risk while enabling access to potentially beneficial drugs. Patients and their caregivers have expressed support for these programs and see patient input as critical to successful implementation. However, they have yet to be systematically involved in their design. The objective of this study was to explore what a framework for MAPs might look like when designed by patients and caregivers.

Building upon established relationships with the Canadian Organization for Rare Disorders, the project team collaborated with patients and caregivers using the principles of participatory action research. Data were collected at two workshops and analyzed using a thematic network approach.

Patients and caregivers identified six aspects of an ideal MAP relating to accountability (program goals), governance (program-specific committee oversight; patient input; international collaboration), and evidence collection (outcome measures and stopping criteria; ongoing monitoring and registries). Additionally, patients and caregivers recognized that health care resources are finite and considered disease or drug eligibility criteria for deciding when to use a MAP (e.g. drugs treating diseases for which there are no other legitimate alternatives).

A patient and caregiver-designed framework was created, which emphasized patient involvement and transparency. Further research is needed to examine the feasibility of this framework and roles for other stakeholders.

In cases of high unmet clinical need, patients can access drugs prior to marketing authorization (MA) and Health Technology Assessment (HTA) through compassionate use programmes (CUP) or special access pathways (SAP). In theory, accelerated access is beneficial for patients with few therapeutic alternatives. In practice, it remains unclear if early access products actually deliver meaningful clinical benefit.

Seventy-five drug-indication pairs were identified that have proceeded through a CUP or SAP in one or more countries including Canada, Australia, France, Sweden, England, and Scotland. Data was collected from regulatory and HTA websites on length of CUP or SAP, time prior to MA, time prior to HTA decision, time between MA and HTA decision, French Transparency Commission added clinical benefit (ASMR), and HTA decision. Cohen kappa scores were calculated in order to assess inter-agency agreement.

Across the 75 drug-indication pairs, average time between CUP and marketing authorization was 243 days, and average time between MA and HTA decision was 252 days. No products were deemed to be of major added clinical benefit (ASMR I), only 2.7 percent of products had important added clinical benefit (ASMR II), 26.7 percent of products had moderate added clinical benefit (ASMR III), 40.0 percent of products had minor added clinical benefit (ASMR IV), and 22.7 percent of products had no added clinical benefit (ASMR V). There is little inter-agency agreement in HTA recommendations for products that have proceeded through a CUP. The highest amount of agreement was seen between Canada and Scotland (k = 0.24).

Preliminary results suggest that CUP and SAP products accelerate access, but often only provide only moderate or minor improvements in clinical benefit. Further, there is very little agreement across HTA agencies on the value of these products.