Conference Theme: Strengthening the Evidence-to-Action Connection
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- 03 January 2019, p. ix
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Oral Presentations
OP02 Real-World Experience For Health Technology Assessment In Hospitals
- Orna Tal, Inbal Tal
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- 03 January 2019, p. 1
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Introduction:
Health technology assessment (HTA) is scientifically incorporating the demand, effectiveness and expedience of adopting innovative technologies, supported by evidence-based medicine. The rapid development of innovations led to an accelerated need to comprehend their benefit and satisfaction through actual utilization. ‘Real World’ Experience (RWE) in action is a source for collecting information through practice that has not yet been formally published. The target is to identify challenges and barriers in adoption, considerations on significant investment and local adjustment. This study aimed to assess the benefit of RWE as a tool for gathering information understanding and evaluating new technologies as candidates for public funding.
Methods:Twelve new technologies were assessed by 36 technology-assessors from the Ministry of Health (MOH) and the hospital, and were ranked using two scales: (i) the contribution of using RWE to benefit the assessment mechanism, and (ii) the perceptual “value” of the technology.
Results:Analyzing the contribution of RWE to HTA, 67 percent of the technologies reviewed were recommended for adoption. Low RWE ranking correlated with a decision to reject technology adoption. The most important “value” among all assessors on RWE was “innovation”, and among hospital assessors, the parameter “local adjustment” was cardinal. MOH assessors highlighted RWE to increase knowledge of regulators to encourage adoption. The aspect relating to economic burden received only medium ranking.
Conclusions:RWE provides an essential contribution to HTA throughout all stages in the life of the technology, especially at early phases. It is an important methodology to gather evidence from the ongoing experience of clinicians, increasing awareness towards promising innovations and amplifying the confidence of decision-making. In this new era of HTA, new concepts arise on the horizon: multi-dimensional evaluations. Expert opinion and RWE are paving the way towards better understanding of beneficial technologies, even in the absence of formal published evidence.
OP03 Optimizing The Use Of RWE In HTA: Lessons From The ICER Summit
- Grace Hampson, Chris Henshall, Adrian Towse, Bill Dreitlein, Steven Pearson
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- 03 January 2019, pp. 1-2
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Introduction:
Real world evidence (RWE) is changing the overall data landscape and it has potential to advance the evaluation of real world performance (comparative effectiveness) of healthcare technologies by providing a greater quantity and quality of evidence. However, many are concerned that non-randomized RWE may be substituted for RCT data and thus increase uncertainty about effectiveness. This presentation sets out the opportunities and challenges for use of RWE by payers and HTA bodies to evaluate health care technologies.
Methods:Current uses, opportunities and challenges were identified via a literature review and interviews with nine experts. Interim results were discussed at the 2017 ICER Policy Summit, which brought together leaders from payer and life sciences organizations, to develop specific and actionable recommendations for the use of RWE in drug coverage and policy decision-making.
Results:RWE is utilized for multiple purposes in the US and globally, including: aiding design of drug development pathways; supporting regulatory approval decisions; monitoring safety; and informing HTA assessments and payer coverage decisions. Some stakeholders see great value in RWE and want to make greater use of these data sources, including for: drug effectiveness evaluations (including supplementing network meta-analyses); innovative study designs (including pragmatic trials); real time patient monitoring; and adaptive pathways or coverage with evidence development. However, others see numerous challenges, many of which are related to the quality and reliability of RWE sources. Acceptance of an expanded future role for RWE is not universal, and payers and developers must work together to find mutually beneficial strategies for progressing the development and use of RWE.
Conclusions:Specific and actionable recommendations will be presented which highlight the role that each stakeholder group can play in overcoming the challenges and realizing the potential for RWE.
OP04 Cardiac Implant Registries: Systematic Review Of Global Practices
- Peter Kolominsky-Rabas, Shixuan Zhang, Sebastian Gaiser
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- 03 January 2019, p. 2
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Introduction:
The importance of Cardiac Implant Registry (CIR) for ensuring a long-term follow-up in post-marked surveillance has been recognized and approved, but there is a lack of consensus standards on how to establish a CIR. The aim of this study is to investigate the structure and key elements of CIRs in the past decade (2006–2016) and to provide recommendations on “best practice” approaches.
Methods:A systematic search on CIR was employed in line with the PRISMA guidelines. The following databases were searched: the PubMed (Medline), ScienceDirect, EMBASE and the Scopus database. After identifying the existing CIR, an inductive approach was used to explore key elements emerging in the identified registries.
Results:The following eighty-two registries were identified: eighteen ICD registries, seven CRT registries, five pacemaker registries, and six Cardiovascular Implantable Electronic Device (CIED) registries which combined ICD, pacemaker and CRT implantation data; as well as twenty-two coronary stent registries and twenty-four TAVI registries. While seventy-one national or local registries are from a single country, forty-four are from European countries, and nine are located in USA. The following criteria have been summarized from the identified registries, including: registry working group, ethic issues, transparency, research objective, inclusion criteria, compulsory participation, endpoint, sample size, data collection basement, data collection methods, data entry, data validation and statistical analysis.
Conclusions:For HTA as well as regulatory decision making, medical device registries provide a “real-world” picture for patients, physicians, manufacturers, payers, decision-makers and other stakeholders. CIRs are important for regulatory decisions concerning the safety and approval issues of medical devices; for payers CIRs provide evidence on the medical device benefits and drive the decision as to whether the product should be reimbursed or not; for hospitals data from CIRs are important for sound procurement decisions, and CIRs also help patients and their physicians to reach a joint decision on which of the products is the most appropriate. However, many current CIRs are still lacking standards to inform on patient safety and ensure transparency.
OP07 Real World Evidence: How Can It Improve Health Technology Assessment?
- John Gillespie, Sebnem Erdol, Chris Foteff, Liesl Strachan
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- 03 January 2019, pp. 2-3
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Introduction:
Health Technology Assessment (HTA) considers the question of whether evaluated technologies are cost-effective in real world settings. As observed in HTA conducted by the Australian Medical Services Advisory Committee (MSAC), questions regarding the validity of data inputs to economic analyses that reflect real-world practice is a common reason for uncertainty on the cost effectiveness of new technologies. In addition to resource use and costs, there may be other uncertainties regarding the eligible patient population, patient management pathways and comparator selection. Our objective in this study was to present case studies from Australia where real world linked datasets could be better utilized to inform HTA conducted by the MSAC.
Methods:For selected therapy areas, assessment reports and public summary documents of HTA conducted by the MSAC published between January 2015 and November 2017 were reviewed. Our analysis identified HTAs where uncertainties around the inputs for health economic evaluations, as well as uncertainties in defining eligible patient numbers or current patient pathways of care were shown to exist. We then explored whether these uncertainties could have been addressed through real world linked datasets.
Results:Our preliminary investigations identified two assessments: MSAC assessment of capsule endoscopy and transcatheter aortic valve implantation - where availability of real world linked data could have addressed uncertainties around the inputs required for the health economic evaluations.
Conclusions:Australia has a range of real world datasets with the potential to be used to inform HTA conducted by the MSAC. This can only be achieved if the datasets could be better linked and accessible for use by key stakeholders in the MSAC HTA process (e.g. industry, clinician, patient societies). Use of these data sets in HTA will enable timelier patient access to cost-effective technologies and more effective implementation and review of technologies after adoption into clinical practice.
OP08 Using Real World Data To Support National Postmarketing Surveillance
- Yeqin Zuo, Bernie Mullen, Rachel Hayhurst, Karen Kaye, Renee Granger, Jonathan Dartnell
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- 03 January 2019, p. 3
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Introduction:
While medicines and medical tests are developed in a controlled clinical trial environment, postmarketing surveillance in the real world can be challenging. MedicineInsight—a database of longitudinal patient-level clinical information from primary care practices in Australia—is a novel program that collects primary care data to improve postmarketing surveillance at a national level.
Methods:MedicineInsight collects de-identified clinical information from primary care practice information systems using data extraction tools. MedicineInsight currently includes 3.6 million regular patients of 3,300 family physicians (general practitioners) from 650 primary care practices across Australia. MedicineInsight data include longitudinal clinical information on diagnosis and medicines (dose, strength, route of administration, medication switches over time, adverse events, and allergies), and pathology testing data. A series of observational studies was developed for postmarketing surveillance of management of a range of health priorities including type 2 diabetes mellitus (T2DM), chronic obstructive pulmonary disease (COPD), depression, and antibiotics use.
Results:Forty-four percent of patients with T2DM in the MedicineInsight database did not have a recorded hemoglobin A1c result and thirty-one percent did not have a recorded blood pressure reading in the previous 6 months. While guidelines recommend a stepwise approach to the initiation of COPD therapy, forty-nine percent of patients with COPD (with or without asthma) were prescribed dual therapy at initiation and a small number (4.5 percent) were prescribed triple therapy. Between 2011 and 2015, the annual rate of antidepressant prescribing per 1,000 family physician encounters increased by eight percent. High volumes of antibiotics were prescribed for respiratory tract infections in Australian primary care, notwithstanding guideline recommendations that antibiotics are not recommended in most cases.
Conclusions:Large scale, real-world clinical data from primary care practices can play an important role in postmarketing surveillance at a national level.
OP09 Summary Of The 2018 Health Technology Assessment International Latin America Policy Forum Background Paper
- Andres Pichon-Riviere, Rebecca Trowman
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- 03 January 2019, p. 4
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Introduction:
The Health Technology Assessment International (HTAi) Latin America Policy Forum (LAPF) will meet for the third time on 23 and 24 April 2018 in Montevideo, Uruguay. The topic chosen for this meeting is ‘defining the value of health technologies in Latin America’. Due to the differing levels of maturity in the HTA systems and approaches by countries in the region, this topic deserves specific analysis and consideration for the Latin America region.
Methods:A background paper is in development for the LAPF, and will be informed by a review of policy publications in scientific databases, google scholar plus other grey literature. The background paper will summarize the relevance of value frameworks around the world (building on the 2017 HTAi Global Policy Forum background paper and discussions) and will narratively synthesize key findings globally and draw out issues and experiences relevant to the Latin America region. This will be used to provoke the discussions and debates at the LAPF.
Results:This presentation will focus on summarizing the background paper that was used to inform the discussion over the two days of the LAPF, highlighting the key findings on value frameworks from the Latin America region. The presentation will also summarize the key questions for deliberation that were brought to the attention of the meeting attendees, plus the key questions that were posed for the guided breakout sessions will be presented.
Conclusions:The presentation will provide a great opportunity for HTAi members to hear about the ‘state of the art’ methodologies for valuing health technologies in the Latin America region.
OP10 Key Messages From The 2018 Health Technology Assessment Latin America Policy Forum
- Laura Sampietro-Colom, Rebecca Trowman
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- 03 January 2019, pp. 4-5
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Introduction:
The third Health Technology Assessment International (HTAi) Latin America Policy Forum (LAPF) will meet on 23 and 24 April 2018 in Montevideo, Uruguay. The format of the LAPF echoes that of the HTAi Global Policy Forum and provides a unique opportunity as a leadership meeting for senior people from public and private sector organizations using HTA. The aim of the LAPF is to provide an environment where senior people can engage in strategic discussions, informed by the perspectives of their different organizations, without the constraints associated with discussions of specific products or organizational policies.
Methods:The HTAi LPF will convene around 50 senior representatives from HTA agencies and industry members active in the Latin America region. The topic that has been chosen by the organizing committee is ‘defining the value of health technologies in Latin America’ and this will be discussed over two days by attendees.
Results:This presentation will focus on summarizing the key messages that resulted from the two days of discussions at the LAPF. The views reflected will balance those of the not-for-profit agencies and private industry sector attendees, while respecting the ‘Chatham House’ rule (whereby no quotes will be directly attributed to any attendee). It will draw out any conclusions from the plenary sessions and the guided breakout sessions from the event. Any actions that are resulting from the event will also be summarized.
Conclusions:The presentation will provide a great opportunity for the wider HTAi membership to hear reflections on the key messages resulting from the event. This could provide an opportunity for further questions and debate surrounding the key issues to occur, such us any difference between valuing technologies in the Latin America Region with the approaches made in the European Union and North America.
OP11 Findings From The 2017 Health Technology Assessment International Asia Policy Forum Survey
- Linda Mundy, Rebecca Trowman, Brendon Kearney
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- 03 January 2019, p. 5
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Introduction:
Health systems in the Asia region seek to achieve universal health care (UHC) by increasing access to essential healthcare services, while reducing health inequalities and out-of-pocket expenditure. With this in mind, the discussion for the 2017 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) centered around the topic of ‘Universal Healthcare in the Asia Region: Overcoming the Barriers using HTA and Real World Data’.
Methods:As part of the background paper prepared to inform discussions at the APF, attendees from public sector HTA agencies from nine countries, and industry attendees from eight companies, were surveyed to explore issues on the barriers and challenges of establishing UHC in the region and use of real world data (RWD).
Results:Most countries used evidence-based decision-making when considering which technologies to add to healthcare benefit packages; however, this was at times inconsistently applied. Prioritization criteria included burden of disease, unmet clinical need, clinical effectiveness, cost effectiveness and affordability. The political agenda and deference to expert opinion also played a role. Although all countries reported using RWD to inform decision-making, they also reported issues including lack of capacity, poor quality data and lack of collaboration between private and public sectors, as well as a conservative approach to data-linkage. It became apparent that a disconnect had been identified, as industry overwhelmingly identified access to RWD in the region as a major issue.
Conclusions:Discussing the differences in opinion between HTA agencies and industry led to a greater understanding of issues in the Asia region, and will lead to increased dialogue and opportunities to collaborate in the future. Building capacity in the region will assist countries to deliver on their goal of achieving UHC.
OP12 2017 Health Technology Assessment International Asia Policy Forum: The Importance Of Universal Health Care
- Andrew Rintoul, Rebecca Trowman
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- 03 January 2019, pp. 5-6
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Introduction:
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 goal of achieving universal health care (UHC) in the Asia region, and specifically the perspective of the World Health Organization (WHO).
Methods:The 2017 HTAi APF had senior representatives from HTA agencies, academia, industry active in the region plus representatives from the WHO Geneva office and the Western Pacific Regional Office. A keynote presentation was delivered by the WHO representative and there were guided breakout group discussions.
Results:UHC is a key component of the overall aims and objectives of the WHO; universal access to safe, effective, quality and affordable medicines and vaccines for all is at the heart of this. Pharmaceutical spending varies widely across the Asia region and all countries in the region share common problems in attaining UHC. These include inadequate financing, inefficiencies in procurement and supply chain management, limited use of effective pricing policies and negotiations, substandard quality of medicines and widespread inappropriate prescribing and use.
Conclusions:HTA can be used to help countries in the Asia region to achieve UHC; it is a tool to support good decision making and hence can help promote more efficient allocation of limited resources. Affordability, however, needs to be at the center of any decision to invest or disinvest, and incremental cost effectiveness ratios should not be used as the sole basis for decision making.
OP13 2017 Health Technology Assessment International Asia Policy Forum: The Perspective Of Not-For-Profit Members
- Raoh-Fang Pwu, Rebecca Trowman
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- 03 January 2019, p. 6
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Introduction:
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 the not-for-profit HTA agencies that were represented at the APF.
Methods:The 2017 HTAi APF had senior representatives from government and HTA agencies from 11 countries in the Asia region. This included first-time attendees from Iran, Indonesia and the Philippines. A keynote presentation on the lessons learnt from Taiwan on the use of RWD was delivered, and there were guided breakout group discussions.
Results:There are a number of registries and sources of RWD available to the HTA agencies across the Asia region. However it was noted that the databases are resource intensive to maintain, and there are noted time lags in the data collection and release. There are examples where the data from these sources have been used to evaluate local utilization of treatments and post-market surveillance, but it has not yet been used in pricing/reimbursement decisions.
Conclusions:Although RWD could be a good tool for healthcare decision-making in the Asia region, it is still unclear how to do so properly. Infrastructure and human capacity, as well as law, are key factors to the successful use of RWD. Standardized methodologies, increased transparency, quality control and capacity building are essential elements to better implementing RWD in HTA.
OP14 The 2017 Health Technology Assessment International Asia Policy Forum: The Perspective Of Industry Members
- Parashar Patel, Rebecca Trowman
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- 03 January 2019, pp. 6-7
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Introduction:
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.
Methods: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.
Results: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.
Conclusions: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.
OP15 Actions Arising From The 2017 Health Technology Assessment International Asia Policy Forum
- Brendon Kearney, Rebecca Trowman
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- 03 January 2019, p. 7
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Introduction:
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)’.
Methods: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.
Results: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.
Conclusions: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.
OP16 Decision Criteria That Influence Managed Entry Agreements
- Maxine Freeman-Robinson, Cathrine Mihalopoulos, Tracy Merlin, Elizabeth Roughead
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- 03 January 2019, pp. 7-8
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Introduction:
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.
Methods: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.
Results: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.
Conclusions: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.
OP18 A Patient And Caregiver-Designed Framework For Managed Access Programs
- Andrea Young, Devidas Menon, Jackie Street, Walla Al-Hertani, Tania Stafinski
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- 03 January 2019, p. 8
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Introduction:
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.
Methods: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.
Results: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).
Conclusions: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.
OP19 Are Compassionate Use Programmes Good Predictors of Clinical Benefit?
- Mackenzie Mills
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- 03 January 2019, pp. 8-9
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Introduction:
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.
Methods: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.
Results: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).
Conclusions: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.
OP20 When Are Nationally Available Discounts Introduced In NICE Appraisals
- Kathleen Noon, Christopher Painter, Stephen Montgomery
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- 03 January 2019, p. 9
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Introduction:
Offering a nationally available discount has become common to increase the chance of being recommended by the National Institute of Health and Care Excellence (NICE). This study reviewed all NICE technology appraisals (TAs) since October 2007 to determine whether a national available discount was submitted, and explore when these discounts were introduced.
Methods:All TAs between October 2007 and August 2017 were reviewed. The timing of the nationally available discount submission was allocated into one of four categories: initially submitted; initially submitted but changed; introduced after submission; or, other discount. An analysis was conducted to examine whether there was a temporal pattern in the introduction of nationally available discounts before or after January 2014, when the current Pharmaceutical Price Regulation Scheme (PPRS) came into effect.
Results:Before 1 January 2014, a nationally available discount was only used in the minority of cases across recommended (22 percent of cases) and not recommended (19 percent) technologies. In the period since 1 January 2014, use of a nationally available discount increased overall, but to a greater degree in technologies ultimately receiving a positive recommendation from NICE (not recommended: 19 percent to 39 percent; recommended: 22 percent to 59 percent). In the period since 1 January 2014, the proportion of technologies with a positive recommendation where implicit price flexibility during the appraisal was revealed increased (from 20/186) to 40/182.
Conclusions:With the current PPRS, the majority of technologies have offered a nationally available discount, most commonly at the time of submission; however, there is increasing evidence of implicit price flexibility during the appraisal process to achieve a positive recommendation.
OP23 Setting The Value Of New Technologies - A Survey
- Orna Tal, Yaron Connelly
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- 03 January 2019, pp. 9-10
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Introduction:
Technology assessment in hospital traditionally involves parameters of safety, effectiveness and costs. The prosperity of medical innovations in an era of scarce resources requires more precisely refined methodologies to measure ‘added value’. Our aim was to reveal the added values of technologies by asking professionals to prioritize their adoption into hospitals.
Methods:Twelve innovative technologies that were discussed for adoption over three years were controversial regarding their actual “added value”. Fifty-two managerial health professionals ranked these technologies on two scales: hierarchic importance (league scale) and comparative score rating (CSR), reflecting willingness-to-pay (WTP). The distribution of ranking indicates the internal agreement (IA) among the participants.
Results:There was only partial correlation between the two scales. For example, glucose-monitoring was ranked ‘highly important’ on the hierarchic (league) scale with high CSR/WTP, but with low IA. This can be interpreted as “a valuable technology but with disagreement on comprehensive adoption in the entire hospital”. The surgical robot was ranked ‘highly important’ on the hierarchic scale with low CSR/WTP, but with high IA, meaning “a valuable technology but with consensus to delay adoption in the hospital”. Overall, the participants raised thirty-two “values” that can be assorted into five clusters of significance: clinical effect (6 values), social/public dimension (8 values), patient-physician interaction (9 values), technological aspect (5 values) and policy-regulatory perception (4 values).
Conclusions:We identified different ‘;patterns’ for defining the ‘value’ of various technologies. Revealing these aspects can create a “set of values” of relative weights that may explain the added value considerations in prioritization of decision making. Interestingly, there were technologies that were ranked low, but achieved a high rating. This can be explained by individual personal-oriented added value perspectives. Using this innovative tool to incorporate social value-based scores can assist in understanding the determinants, beyond the current traditional rationing mechanism, that guide professionals while prioritizing medical technologies.
OP24 Sensitizing Researchers And Developers For Patient Needs And Value
- Anke-Peggy Holtorf, Gurmit Sandhu, Martina Dresler
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- 03 January 2019, pp. 10-11
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Introduction:
Valuable health technologies must improve health and well-being of patients. For sensitizing healthcare industry stakeholders for the unique perspective and values of patients we developed a workshop format including both knowledge transfer and experiential modules.
Methods:The one-day pilot workshop was attended by two patient representatives and multidisciplinary participants from the healthcare industry (n=12) who wanted to learn about patient involvement in health technology assessment (HTA) and healthcare decision making and the implications for product development. Three content sessions covered key aspects of HTA and patient engagement and each was followed by an session which aimed at discovering the values of participants as healthy individuals or when the diagnosis of a disease and the subsequent therapy decisions (including potential clinical trial participation) impact quality and length of life. The workshop concluded with the participants prioritizing their expectations for innovation and HTA as patients or as citizens.
Results:Overall, participants rated the workshop as excellent or good for knowledge and experiential sessions. Integration of both learning modalities was described as innovative, useful, and enjoyable. Participation in the clinical trial session triggered cognitive responses among the industry participants due to a strong focus on advancement of science for innovation. Otherwise, the responses of the industry participants matched those of the patient representatives well. Overall, patient perspectives were considered useful to enrich the value perceptions beyond those of industry. Emotions describing the personal experiences included despair, shock, anger, guilt, hope, and the will to live. As citizens, they emphasized expectations such as finding solutions, remaining independent, enjoying life and “giving back”.
Conclusions:Innovative learning structures integrating rational and emotional aspects can allow researchers, marketers, or other stakeholders from the life-science industry to better understand patient perspectives. The format may be well suited for team building and alignment of team values around patient-needs.
OP27 Patient Engagement At Scottish Medicines Consortium Committee Meetings
- Lindsay Lockhart, Jennifer Dickson, Anne Lee, Peter McGrath, Yvonne Hughes
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- Published online by Cambridge University Press:
- 03 January 2019, p. 11
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Introduction:
Since 2014 patient group representatives have been able to observe Scottish Medicines Consortium (SMC) committee meetings as members of the public. However, they have had no opportunity to participate in discussions on their submission on the patient experience of living with the condition under review. In 2017, to strengthen patient engagement, we revised our processes to enable representatives from all submitting patient groups to play a bigger part in the monthly meeting.
Methods:The SMC Public Involvement Network (PIN) Advisory Group consulted on potential issues around patient group participation in committee meetings. Recommendations approved for implementation included (i) provision of comprehensive information and support to participating patient group representatives, and (ii) holding an educational session for SMC members on ‘What matters to the patient’. The process change was introduced in June 2017. Patient group representatives are invited to complete an online survey on their experience of taking part in the meeting and working with the public involvement team. Implementation is being monitored and will be evaluated in a commitment to continuous improvement.
Results:Since June 2017, 14 patient group representatives have attended SMC meetings for the discussion of their submission. This has enabled them to answer questions from committee members and clarify points relating to their submission, if required. Early feedback has been positive with participants believing that patient engagement has been strengthened and that the patient voice was heard and valued. Patient groups expressed a willingness to participate again. The evaluation of their experience to date will be presented.
Conclusions:SMC now involves patient group participation at committee meetings, demonstrating commitment to listening and responding to stakeholders on patient engagement. Early feedback has been positive and suggests that discussions relating to quality of life impact on patients and carers better reflect the lived experience. This ensures we are meeting our commitment to openness and transparency and strengthens patient engagement in our process.