The Biotherapy Development Association convened a two-day workshop in January 2014 to assess access to innovative cancer medicines in Europe. This presentation by OHE's Adrian Towse covers the situation in England, examining challenges that are peculiar to England as well as the English experience with issues common across countries.
Personalised medicine holds great promised for both improving patients’ outcomes and enhancing the efficiency of treatment. Medicines paired with diagnostics are the backbone of personalised medicine, presenting new challenges in for health technology assessment. The situation in England, particularly how NICE might respond to this challenge, was the focus of the third networking event co-sponsored by the Association of the British Pharmaceutical Industry association (ABPI) and the British In Vitro Diagnostics Association. At this one-day event, speakers set the stage for discussion by presenting defining the context of this challenge for England.
OHE’s Adrian Towse presented on the economics. He discussed the elements of value of a diagnostics test (see our earlier blog post) and described the context necessary to produce useful assessments and to ensure subsequent use in the marketplace. His topics included issues of evidence generation, incentives for innovation, flexible approaches to access coincident with evidence development, and encouraging uptake and use.
Slides from the presentation on extrapolation from progression free survival to overall survival in oncology given at the 2017 HTAi Annual Meeting in Rome
The Value of Targeted Sequencing in Advanced Cancer: DCE to Elicit the Public...Office of Health Economics
This project seeks to elicit the public’s preferences for different features of a genomic test to sequence advanced solid cancer tumours. Understanding the relative preferences for various attributes of targeted testing are useful for determining the value of sequencing approaches, and informing technology adoption decisions. A discrete choice experiment (DCE) survey was designed to assess the preferences of members of the Australian general public for targeted sequencing in advanced cancer. The survey presented respondents with 12 questions in which they had to choose between two unlabelled tests (Test A and Test B). Tests were specified in terms of five attributes: time to receive the test result; cost of the test; likelihood that the test result will lead to a change in treatment; length of time health care professionals spend describing the test; and type of health care team who explains the test result. Respondents were sampled from an online panel and also completed questions related to demographic and socio-economic factors, experiences of cancer and familial history. We found that cost, timeliness, expertise/location and likeliness of changing treatment regimes were identified as attributes of genomic sequencing that are most valuable to a sample of the public. These results will ultimately be compared with the results of an ongoing DCE being conducted with patients with advanced cancer who are undergoing sequencing.
Author(s) and affiliation(s): Paula Lorgelly (OHE), Grace Hampson (OHE), James Buchanan (Oxford), Melissa Martyn (MGHA), Jayesh Desai (PeterMac), Clara Gaff (MGHA), and iPREDICT MGHA Flagship collaborators
Conference/meeting: EuHEA 2018
Location: Maastricht, the Netherlands
Date: 12/07/2018
Personalised medicine holds great promised for both improving patients’ outcomes and enhancing the efficiency of treatment. Medicines paired with diagnostics are the backbone of personalised medicine, presenting new challenges in for health technology assessment. The situation in England, particularly how NICE might respond to this challenge, was the focus of the third networking event co-sponsored by the Association of the British Pharmaceutical Industry association (ABPI) and the British In Vitro Diagnostics Association. At this one-day event, speakers set the stage for discussion by presenting defining the context of this challenge for England.
OHE’s Adrian Towse presented on the economics. He discussed the elements of value of a diagnostics test (see our earlier blog post) and described the context necessary to produce useful assessments and to ensure subsequent use in the marketplace. His topics included issues of evidence generation, incentives for innovation, flexible approaches to access coincident with evidence development, and encouraging uptake and use.
Slides from the presentation on extrapolation from progression free survival to overall survival in oncology given at the 2017 HTAi Annual Meeting in Rome
The Value of Targeted Sequencing in Advanced Cancer: DCE to Elicit the Public...Office of Health Economics
This project seeks to elicit the public’s preferences for different features of a genomic test to sequence advanced solid cancer tumours. Understanding the relative preferences for various attributes of targeted testing are useful for determining the value of sequencing approaches, and informing technology adoption decisions. A discrete choice experiment (DCE) survey was designed to assess the preferences of members of the Australian general public for targeted sequencing in advanced cancer. The survey presented respondents with 12 questions in which they had to choose between two unlabelled tests (Test A and Test B). Tests were specified in terms of five attributes: time to receive the test result; cost of the test; likelihood that the test result will lead to a change in treatment; length of time health care professionals spend describing the test; and type of health care team who explains the test result. Respondents were sampled from an online panel and also completed questions related to demographic and socio-economic factors, experiences of cancer and familial history. We found that cost, timeliness, expertise/location and likeliness of changing treatment regimes were identified as attributes of genomic sequencing that are most valuable to a sample of the public. These results will ultimately be compared with the results of an ongoing DCE being conducted with patients with advanced cancer who are undergoing sequencing.
Author(s) and affiliation(s): Paula Lorgelly (OHE), Grace Hampson (OHE), James Buchanan (Oxford), Melissa Martyn (MGHA), Jayesh Desai (PeterMac), Clara Gaff (MGHA), and iPREDICT MGHA Flagship collaborators
Conference/meeting: EuHEA 2018
Location: Maastricht, the Netherlands
Date: 12/07/2018
Eligibility for national screening programmes can be personalised according to individual risk in order to improve outcomes and reduce costs. Existing methods of economic evaluation can be adapted to identify risk thresholds and help optimise services. We describe the development of a decision model used to evaluate the cost-effectiveness of risk-based screening for diabetic retinopathy.
Author(s) and affiliation(s): Chris Sampson, Office of Health Economics Marilyn James, University of Nottingham David Whynes, University of Nottingham Antonio Eleuteri, University of Liverpool Simon Harding, University of Liverpool.
Conference/meeting: Health Technology Assessment International (HTAi) 2018
Location: Vancouver, Canada
Date: 03/06/2018
In a research report by Berdud, M., Drummond, M. and Towse, A. (2018), a reasonable price for an orphan drug was established based on the proposition that rates of returns from investments in developing orphan drugs should be no greater than the industry average (for all drugs). At the 2018 EuHEA conference held in Maastricht, The Netherlands, 11-14 July, Mikel showed (i) how the reasonable price should be established and (ii) how NICE's cost-effectiveness threshold should be adjusted to ensure a reasonable price for an orphan drug. In slides results are discussed and conclusions showed too.
Author(s) and affiliation(s): Mikel Berdud, PhD (OHE); Prof. Mike Drummond (University of York); Prof. Adrian Towse (OHE)
Conference/meeting: EuHEA 2018
Location: Maastricht, The Netherlands
Date: 12/07/2018
Unprecedented medical advances in cancer treatments are accompanied with huge financial challenges. Outcome-based payments have been proposed as a potential way to foster earlier access, tackle uncertainty, and address the financial challenge. But payments based on what outcomes? We conducted a literature review exploring what outcomes “matter most” in cancer.
Author(s) and affiliation(s): Amanda Cole, OHE Patricia Cubi-Molla, OHE Paula Lorgelly, OHE Jon Sussex, RAND Europe Jack Pollard, RAND Europe Miaoqing Yang, RAND Europe Richard Sullivan, King's College London
Conference/meeting: PROMS Research Conference 2018
Location: University of Birmingham
Date: 20/06/2018
Do EQ-5D-3L and EQ-5D-5L Capture the Same Changes in Quality of Life Over Tim...Office of Health Economics
The existence of important dissimilarities between EQ-5D-3L and EQ-5D-5L, both in terms of the health profiles and preference-based values, is a key topic in current research. This study compares the performance of the 3L and 5L versions of the EQ-5D in capturing changes in quality of life and the resulting impact on estimates of QALYs for a large cohort of cancer patients. Data were obtained from Cancer2015, a large-scale longitudinal cancer cohort study in Australia. Cancer 2015 enrols newly diagnosed, treatment-naïve cancer patients, who complete quality of life questionnaires at baseline, and at various follow-up points (approximately 3 and/or 6 months continuously). Genetic Matching techniques are used to construct a match comparison group of patients. Post-matching regression adjustment is also implemented to control for any remaining imbalances. For matched QLQ-C30 profiles, we compare 3L and 5L tariffs, as well as the magnitude of changes in quality of life at different points along the treatment trajectory of individuals. We pay special attention to the sensitivity of the measures, by exploring the impact of 3L vs 5L on larger changes in quality of life compared to smaller changes. Our analysis finds that improvements in HRQoL as measured by the QLU-C10D (which is derived from the condition specific EORTC QLQ-C30 instrument) appear to be associated with smaller changes in utility quantified by the 5L compared to the 3L. When HRQoL is deteriorating between observations then the 5L tariff is found to produce bigger utility losses. While the crosswalk (a) loses the increased sensitivity of the 5L (if it detects more change) but (b) it stretches out utility values across a larger range (the 3L range), and hence gains or losses are larger and more in line with the 3L tariffs.
Author(s) and affiliation(s): Paula Lorgelly (OHE), Patricia Cubi-Molla (OHE), Mark Pennington (King's College London), Richard Norman (Curtin)
Conference/meeting: EuHea 2018
Location: Maastricht, Netherlands
Date: 13/07/2018
This research explores the feasibility of introducing an Outcome-Based Payment approach for new cancer drugs in England. A literature review explored the current funding landscape in England, the available evidence on existing OBP schemes internationally, and
which outcomes cancer patients value most. Two focus groups and an online survey with patients and carers, as well as interviews with NHS and government stakeholders, healthcare
professionals, and pharmaceutical industry representatives, provided additional evidence on the feasibility and suitability of OBP schemes
The Cancer Drugs Fund in practice, under the new frameworkAlex Diamantopoulos
A review of the process and criteria used for consideration of treatments under the CDF framework. This work describes the extent of evidence collection while in the Fund.
Eligibility for national screening programmes can be personalised according to individual risk in order to improve outcomes and reduce costs. Existing methods of economic evaluation can be adapted to identify risk thresholds and help optimise services. We describe the development of a decision model used to evaluate the cost-effectiveness of risk-based screening for diabetic retinopathy.
Author(s) and affiliation(s): Chris Sampson, Office of Health Economics Marilyn James, University of Nottingham David Whynes, University of Nottingham Antonio Eleuteri, University of Liverpool Simon Harding, University of Liverpool.
Conference/meeting: Health Technology Assessment International (HTAi) 2018
Location: Vancouver, Canada
Date: 03/06/2018
In a research report by Berdud, M., Drummond, M. and Towse, A. (2018), a reasonable price for an orphan drug was established based on the proposition that rates of returns from investments in developing orphan drugs should be no greater than the industry average (for all drugs). At the 2018 EuHEA conference held in Maastricht, The Netherlands, 11-14 July, Mikel showed (i) how the reasonable price should be established and (ii) how NICE's cost-effectiveness threshold should be adjusted to ensure a reasonable price for an orphan drug. In slides results are discussed and conclusions showed too.
Author(s) and affiliation(s): Mikel Berdud, PhD (OHE); Prof. Mike Drummond (University of York); Prof. Adrian Towse (OHE)
Conference/meeting: EuHEA 2018
Location: Maastricht, The Netherlands
Date: 12/07/2018
Unprecedented medical advances in cancer treatments are accompanied with huge financial challenges. Outcome-based payments have been proposed as a potential way to foster earlier access, tackle uncertainty, and address the financial challenge. But payments based on what outcomes? We conducted a literature review exploring what outcomes “matter most” in cancer.
Author(s) and affiliation(s): Amanda Cole, OHE Patricia Cubi-Molla, OHE Paula Lorgelly, OHE Jon Sussex, RAND Europe Jack Pollard, RAND Europe Miaoqing Yang, RAND Europe Richard Sullivan, King's College London
Conference/meeting: PROMS Research Conference 2018
Location: University of Birmingham
Date: 20/06/2018
Do EQ-5D-3L and EQ-5D-5L Capture the Same Changes in Quality of Life Over Tim...Office of Health Economics
The existence of important dissimilarities between EQ-5D-3L and EQ-5D-5L, both in terms of the health profiles and preference-based values, is a key topic in current research. This study compares the performance of the 3L and 5L versions of the EQ-5D in capturing changes in quality of life and the resulting impact on estimates of QALYs for a large cohort of cancer patients. Data were obtained from Cancer2015, a large-scale longitudinal cancer cohort study in Australia. Cancer 2015 enrols newly diagnosed, treatment-naïve cancer patients, who complete quality of life questionnaires at baseline, and at various follow-up points (approximately 3 and/or 6 months continuously). Genetic Matching techniques are used to construct a match comparison group of patients. Post-matching regression adjustment is also implemented to control for any remaining imbalances. For matched QLQ-C30 profiles, we compare 3L and 5L tariffs, as well as the magnitude of changes in quality of life at different points along the treatment trajectory of individuals. We pay special attention to the sensitivity of the measures, by exploring the impact of 3L vs 5L on larger changes in quality of life compared to smaller changes. Our analysis finds that improvements in HRQoL as measured by the QLU-C10D (which is derived from the condition specific EORTC QLQ-C30 instrument) appear to be associated with smaller changes in utility quantified by the 5L compared to the 3L. When HRQoL is deteriorating between observations then the 5L tariff is found to produce bigger utility losses. While the crosswalk (a) loses the increased sensitivity of the 5L (if it detects more change) but (b) it stretches out utility values across a larger range (the 3L range), and hence gains or losses are larger and more in line with the 3L tariffs.
Author(s) and affiliation(s): Paula Lorgelly (OHE), Patricia Cubi-Molla (OHE), Mark Pennington (King's College London), Richard Norman (Curtin)
Conference/meeting: EuHea 2018
Location: Maastricht, Netherlands
Date: 13/07/2018
This research explores the feasibility of introducing an Outcome-Based Payment approach for new cancer drugs in England. A literature review explored the current funding landscape in England, the available evidence on existing OBP schemes internationally, and
which outcomes cancer patients value most. Two focus groups and an online survey with patients and carers, as well as interviews with NHS and government stakeholders, healthcare
professionals, and pharmaceutical industry representatives, provided additional evidence on the feasibility and suitability of OBP schemes
The Cancer Drugs Fund in practice, under the new frameworkAlex Diamantopoulos
A review of the process and criteria used for consideration of treatments under the CDF framework. This work describes the extent of evidence collection while in the Fund.
Outcome Measures in Cancer: Do disease specific instruments offer greater sen...Office of Health Economics
Paula's slides for her presentation on Outcomes Measures in Cancer given at the C2E2 Rounds Conference at the University of British Columbia on July 5th, 2017.
De castro _ Editorial guidelines for sex and gender equity in research (SAGER...Paola De Castro
How improved reporting of sex/gender can influence research outcomes and contribute to more equitable approach to research across disciplines. Presented at the 14th International COMET Conference
(Communication, Medicine, Ethics). University of Aalberg (Denmark). July 4-6, 2016
ICH Guidelines of Quality, Safety, Efficacy and Multidisciplinary guidelines that implemented by International Council for Harmonisation. ich stands for the harmonisation of Technical requirements of Pharmaceuticals for Human use.
WCRF International Continuous Update Project (CUP). Presentation given by Giota Mitrou PhD MSc, Head of Research Funding and Science Activities, World Cancer Research Fund International (WCRF International).
Paul Coplan, VP, Johnson & Johnson_mHealth IsraelLevi Shapiro
Pesentation, October 19th, 2021: What’s Next in RWE for Medical Devices: The Art of the Possible. Presented by Paul Coplan, ScD, MBA, FISPE, Vice President, Med Device Epidemiology and RWD Sciences, Johnson & Johnson; Adjunct Professor, Department of Biostatistics, Epidemiology and Informatics, University of Pennsylvania, Perelman School of Medicine; Fellow of the International Society of Pharmacoepidemiology
- Why RWE is Important for Medical Devices: Challenges with Clinical Trials of Medical Devices (Blinding, Surgeon skill/technique, Hospital process, Product modifications, Long term Follow up, Enrolment challenges)
- Types of Real-World Data Sources (Complaints like MAUDE, Eudramed and Company Databases, Hospital Databases, Electronic Health Records, Claims, Registries, Patient surveys, Surgeon surveys, PROs, Patient Preferences, wearables, sensors, social media, Surgical videos, device generated data, radiographic images)
- FDA CDRH Report on RWE Examples for Regulatory Decisions
- J&J Med Device Epidemiology & Real-World Data Sciences
- US National Evaluation System for Health Technology (NEST)
- RWE for Safety Assessments: Cobalt in Implants and at Work and Risk of Cancer
- Summary of Cobalt Exposure and All-Site Cancer Risk, by Study Type
- Comparative Effectiveness Studies Using RWE
- Summary
a. Use of RWE is important to benefit patients globally and enhance the safety and innovation of medical devices
b. Regulators are interested in using RWE for regulatory decisions but data quality and evidence needs to be regulatory grade
c. NEST has been a useful forum to advance the use of RWE for regulatory decisions in the US
d. RWE can be used for safety assessments, regulatory decisions, comparative effectiveness research, and R&D of products
On 31 October 2019, Adrian Towse and Chris Henshall from the Office of Health Economics (OHE) presented at the G20 meeting on antimicrobial drugs R&D in Paris organised by the Wellcome Trust. The topic of their presentation was HTA and payment mechanisms for new drugs to tackle antimicrobial resistance.
This presentation looks at ways in which governments can set prices, including “cost plus”, value, and the external referencing of prices elsewhere. It looks at the role that competition can play in keeping down prices. In that context it briefly discusses pricing proposals being considered in Malaysia. It makes the case for using HTA to inform pricing decisions.
Adrian Towse
% GDP spending in UK, G5 countries and OECD upper middle income countries. W...Office of Health Economics
This presentation looks at rates of GDP spend on health care, distinguishing between categories of country (i.e. levels of GDP pre capita). It looks at the relationship between rates of spending and moves to universal health coverage, and explores alternative ways of increasing expenditure and making decisions about which services to provide with the money available.
The role of real world data and evidence in building a sustainable & efficien...Office of Health Economics
This presentation defines RWD and RWE in the context of digital health, and looks at potential uses for RWD and RWE. It briefly sets out the current landscape in Malaysia and looks at the challenges in using RWE. In particular, the issues of access, governance and ensuring good quality are considered.
The aim of this educational symposium was to discuss why we should seek value across the health care system and how we can apply existing research methods to measure the value of services. While considerable political attention in developed countries continues to be focused on drug spending, there is also growing awareness of the significant contribution of non-drug components of health care (e.g., hospital services and inefficient care delivery) to overall spending growth and patient affordability. At the same time, there is growing interest in making greater use of value assessment and value-based payment to control spending and better align it with care quality. In order to promote greater value, and to do so in ways that respond to the needs of payers and patients, it is essential to assess value across both drug- and non-drug interventions and health care services. This panel will offer expert viewpoints to identify and discuss gaps in value information, rationale and approaches to track and reduce system-wide low value care, and research methods for how to measure health care services.
Role Substitution, Skill Mix, and Provider Efficiency and Effectiveness : Les...Office of Health Economics
Graham participated in an organised session on Monday July 15th 2019. In the session he presented his paper with his co-author Ioannis Laliotis from the London School of Economics. The paper revisits the relationship between workforce and maternity outcomes in the English NHS in an attempt to contribute knowledge to an important policy question for which there has been a paucity of research.
Understanding what aspects of health and quality of life are important to peopleOffice of Health Economics
Poster presentation from the EuroQol Plenary Meeting 2019, Brussels, Belgium. By Koonal Shah, Brendan Mulhern, Patricia Cubi-Molla, Bas Janssen, and David Mott.
Koonal presented as part of an organised session on ‘moving beyond conventional economic approaches in palliative and end of life care’. He summarised the empirical evidence on the extent of pubic support for an end of life premium, before discussing some novel approaches that have been used in recent studies. His presentation was discussed by Helen Mason of Glasgow Caledonian University.
Author(s) and affiliation(s): Koonal Shah, Office of Health Economics
Event: iHEA Congress
Date: 17/07/2019
Location: Basel, Switzerland
Assessing the Life-Cycle Value Added of Second Generation Antipsychotics in S...Office of Health Economics
This research presented in a poster at HTAi 2019, Cologne (Germany) by a team of OHE and IHE researchers, estimates the value added by second generation antipsychotics over their life-cycle in the UK and Sweden. It concludes that considering the entire life-cycle, the value added by SGAs to the system is higher than the expected value estimated at launch. P&R decisions should consider how to measure, capture and take into account the value added by medicines over the long-run.
Author(s) and affiliation(s): Mikel Berdud (Office of Health Economics, London), Niklas Wallin-Bernhardsson (Institute for Health Economics, Stockholm), Bernarda Zamora (Office of Health Economics, London), Peter Lindgren (Institute for Health Economics, Stockholm), Adrian Towse (Office of Health Economics, London)
Event: HTAi 2019 Annual Meeting
Date: 18/06/2019
Location: Cologne, Germany
There is growing recognition that HTA and contracting systems for antimicrobials need to be adapted to help fight the threat of antimicrobial resistance (AMR), but there is little agreement on how. This poster reports findings from a literature review, expert interviews and face-to-face discussions at a Forum on the current HTA and payment systems for antibiotics across Europe and a number of recommendations for adapting these systems to respond to the challenges of AMR.
Author(s) and affiliation(s): Margherita Neri (OHE) Grace Hampson (OHE) Christopher Henshall (OHE visiting fellow, independent consultant) Adrian Towse (OHE)
Event: HTAi annual conference 2019
Date: 18/06/2019
Location: Cologne, Germany
Assessing the Life-cycle Value Added of Second-Generation Antipsychotics in S...Office of Health Economics
This study aims to guide access decisions and drive the discussion on access and price, through recognition of the dynamic nature of value added by pharmaceutical innovation over the long-run. The analysis of the life-cycle value of risperidone estimates the value generated in the UK and Sweden. Results show that health systems were able to appropriate most of the life-cycle value generated, and this is larger than estimated at launch.
Author(s) and affiliation(s): Mikel Berdud(1), Niklas Wallin-Bernhardsson(2), Bernarda Zamora(1), Peter Lindgren(2), and Adrian Towse(1) (1) Office of Health Economics (2) The Swedish Institute for. Health Economics
Event: XXXIX JORNADAS DE ECONOMÍA DE LA SALUD
Date: 12/06/2019
Location: Albacete, Spain
Prescribed Specialised Services (PSS) Commissioning for Quality and Innovation (CQUIN) schemes were launched in 2013 in England with the aim of improving the quality of specialised care and achieving value for money. During this presentation, Marina Rodes Sanchez described the key features of the schemes and discussed its strengths and weaknesses based on international pay-for-performance literature.
Author(s) and affiliation(s): Yan Feng, Queen Mary University of London; Søren Rud Kristensen, Imperial College London; Paula Lorgelly, King’s College London; Rachel Meacock, University of Manchester; Marina Rodes Sanchez, Office of Health Economics; Luigi Siciliani, University of York; Matt Sutton, University of Manchester
Event: XXXIX Spanish Health Economics Association Conference
Date: 12/06/2019
Location: Albacete, Spain
In this session, Meng Li sets out estimates of real option value for drugs arguing that option value matters and can be calculated. Adrian Towse sets out likely payer concerns about incorporating real option value into decision making. Meng Li responds to these concerns. Jens Grueger sets out how industry considers investment opportunities, arguing that if patients (and society) have preferences these need to be reflected in P&R decisions.
Author(s) and affiliation(s): Meng Li, Postdoctoral Research Fellow, Leonard D Schaeffer Center, University of Southern California, Los Angeles, CA, USA. Adrian Towse, Emeritus Director, Office of Health Economics, London, UK Jens Grueger, formerly Head of Global Access, Senior Vice President at F. Hoffmann-La Roche
Event: ISPOR 2019
Location: New Orleans, USA
Date: 21/05/2019
MCDA OR WEIGHTED CEA BASED ON THE QALY? WHICH IS THE FUTURE FOR HTA DECISION ...Office of Health Economics
In this ISPOR session Chuck Phelps and Adrian Towse debated the case for and against using MCDA to support HTA decision making, as compared to weighting or augmenting a QALY based ICER approach. Chuck Phelps argued for use of MCDA, Adrian Towse for weighting the QALY. Nancy Devlin set the scene and moderated.
Author(s) and affiliation(s): Nancy Devlin, Director, Centre for Health Policy, University of Melbourne, Australia Adrian Towse, Emeritus Director, Office of Health Economics, London, UK Chuck Phelps, University of Rochester, Rochester, NY USA
Event: ISPOR 2019
Location: New Orleans, USA
Date: 21/05/2019
The issue of open-source models in the cost-effectiveness and disease-level (collaborative) models has been brewing for many years. There has been a marked growth in open science, and funding bodies and publishers increasingly require that research data be made available. As mentioned in our previous Issue Panel, “cost-effectiveness models synthesise a wide range of evidence to facilitate extrapolation over time and from intermediate to final decision endpoints. These models are often statistically sophisticated and require assumptions that are not directly testable. This can lead to decision-makers “discounting” the results of cost-effectiveness analyses, particularly if the developer is seen as partial.” Open-source models, then, would encourage greater transparency in pharmacoeconomic modeling and the reuse and updating of the best/most useful models; they are essential if cost-effectiveness analyses are to be widely accepted to reduce bias, increase transparency, improve model access, and allow for faster access to critical knowledge. The ISPOR-SMDM guidelines and the EUnetHTA joint action projects, are supportive of these views on collaboration, transparency, confidentiality, processes and consistency offered by the availability of open-source models to improve decision-making around health care and reimbursement. With openness and sharing, however, come issues of copyright and access and a need to define how model sharing can be achieved in a fair and equitable manner. There is, therefore, a need to develop an ongoing dialog on openness, especially where the research may be considered precompetitive and not worthy of IP investment. The pros and cons of open source models and the proposed mission of the Open Source Model SIG to curate an ongoing dialog regarding issues around creating, disseminating, sharing, evaluating, and updating open source cost-effectiveness and comparative effectiveness models will be debated amongst SIG members.
Author(s) and affiliation(s): Nancy Risebrough, Senior Principal, ICON plc, Toronto, Canada Jeroen P Jansen; Innovation & Value Initiative; Precision Medicine Group; and Stanford University Lotte Steuten, Vice President & Head of Consulting, Office of Health Economics, UK Renée JG Arnold, PharmD, RPh, ICON plc, New York, NY and Icahn School of Medicine at Mount Sinai, New York, NY, USA
Event: ISPOR 2019 Annual Meeting
Date: 20/05/2019
NVBDCP.pptx Nation vector borne disease control programSapna Thakur
NVBDCP was launched in 2003-2004 . Vector-Borne Disease: Disease that results from an infection transmitted to humans and other animals by blood-feeding arthropods, such as mosquitoes, ticks, and fleas. Examples of vector-borne diseases include Dengue fever, West Nile Virus, Lyme disease, and malaria.
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Report Back from SGO 2024: What’s the Latest in Cervical Cancer?bkling
Are you curious about what’s new in cervical cancer research or unsure what the findings mean? Join Dr. Emily Ko, a gynecologic oncologist at Penn Medicine, to learn about the latest updates from the Society of Gynecologic Oncology (SGO) 2024 Annual Meeting on Women’s Cancer. Dr. Ko will discuss what the research presented at the conference means for you and answer your questions about the new developments.
Prix Galien International 2024 Forum ProgramLevi Shapiro
June 20, 2024, Prix Galien International and Jerusalem Ethics Forum in ROME. Detailed agenda including panels:
- ADVANCES IN CARDIOLOGY: A NEW PARADIGM IS COMING
- WOMEN’S HEALTH: FERTILITY PRESERVATION
- WHAT’S NEW IN THE TREATMENT OF INFECTIOUS,
ONCOLOGICAL AND INFLAMMATORY SKIN DISEASES?
- ARTIFICIAL INTELLIGENCE AND ETHICS
- GENE THERAPY
- BEYOND BORDERS: GLOBAL INITIATIVES FOR DEMOCRATIZING LIFE SCIENCE TECHNOLOGIES AND PROMOTING ACCESS TO HEALTHCARE
- ETHICAL CHALLENGES IN LIFE SCIENCES
- Prix Galien International Awards Ceremony
Lung Cancer: Artificial Intelligence, Synergetics, Complex System Analysis, S...Oleg Kshivets
RESULTS: Overall life span (LS) was 2252.1±1742.5 days and cumulative 5-year survival (5YS) reached 73.2%, 10 years – 64.8%, 20 years – 42.5%. 513 LCP lived more than 5 years (LS=3124.6±1525.6 days), 148 LCP – more than 10 years (LS=5054.4±1504.1 days).199 LCP died because of LC (LS=562.7±374.5 days). 5YS of LCP after bi/lobectomies was significantly superior in comparison with LCP after pneumonectomies (78.1% vs.63.7%, P=0.00001 by log-rank test). AT significantly improved 5YS (66.3% vs. 34.8%) (P=0.00000 by log-rank test) only for LCP with N1-2. Cox modeling displayed that 5YS of LCP significantly depended on: phase transition (PT) early-invasive LC in terms of synergetics, PT N0—N12, cell ratio factors (ratio between cancer cells- CC and blood cells subpopulations), G1-3, histology, glucose, AT, blood cell circuit, prothrombin index, heparin tolerance, recalcification time (P=0.000-0.038). Neural networks, genetic algorithm selection and bootstrap simulation revealed relationships between 5YS and PT early-invasive LC (rank=1), PT N0—N12 (rank=2), thrombocytes/CC (3), erythrocytes/CC (4), eosinophils/CC (5), healthy cells/CC (6), lymphocytes/CC (7), segmented neutrophils/CC (8), stick neutrophils/CC (9), monocytes/CC (10); leucocytes/CC (11). Correct prediction of 5YS was 100% by neural networks computing (area under ROC curve=1.0; error=0.0).
CONCLUSIONS: 5YS of LCP after radical procedures significantly depended on: 1) PT early-invasive cancer; 2) PT N0--N12; 3) cell ratio factors; 4) blood cell circuit; 5) biochemical factors; 6) hemostasis system; 7) AT; 8) LC characteristics; 9) LC cell dynamics; 10) surgery type: lobectomy/pneumonectomy; 11) anthropometric data. Optimal diagnosis and treatment strategies for LC are: 1) screening and early detection of LC; 2) availability of experienced thoracic surgeons because of complexity of radical procedures; 3) aggressive en block surgery and adequate lymph node dissection for completeness; 4) precise prediction; 5) adjuvant chemoimmunoradiotherapy for LCP with unfavorable prognosis.
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...i3 Health
i3 Health is pleased to make the speaker slides from this activity available for use as a non-accredited self-study or teaching resource.
This slide deck presented by Dr. Kami Maddocks, Professor-Clinical in the Division of Hematology and
Associate Division Director for Ambulatory Operations
The Ohio State University Comprehensive Cancer Center, will provide insight into new directions in targeted therapeutic approaches for older adults with mantle cell lymphoma.
STATEMENT OF NEED
Mantle cell lymphoma (MCL) is a rare, aggressive B-cell non-Hodgkin lymphoma (NHL) accounting for 5% to 7% of all lymphomas. Its prognosis ranges from indolent disease that does not require treatment for years to very aggressive disease, which is associated with poor survival (Silkenstedt et al, 2021). Typically, MCL is diagnosed at advanced stage and in older patients who cannot tolerate intensive therapy (NCCN, 2022). Although recent advances have slightly increased remission rates, recurrence and relapse remain very common, leading to a median overall survival between 3 and 6 years (LLS, 2021). Though there are several effective options, progress is still needed towards establishing an accepted frontline approach for MCL (Castellino et al, 2022). Treatment selection and management of MCL are complicated by the heterogeneity of prognosis, advanced age and comorbidities of patients, and lack of an established standard approach for treatment, making it vital that clinicians be familiar with the latest research and advances in this area. In this activity chaired by Michael Wang, MD, Professor in the Department of Lymphoma & Myeloma at MD Anderson Cancer Center, expert faculty will discuss prognostic factors informing treatment, the promising results of recent trials in new therapeutic approaches, and the implications of treatment resistance in therapeutic selection for MCL.
Target Audience
Hematology/oncology fellows, attending faculty, and other health care professionals involved in the treatment of patients with mantle cell lymphoma (MCL).
Learning Objectives
1.) Identify clinical and biological prognostic factors that can guide treatment decision making for older adults with MCL
2.) Evaluate emerging data on targeted therapeutic approaches for treatment-naive and relapsed/refractory MCL and their applicability to older adults
3.) Assess mechanisms of resistance to targeted therapies for MCL and their implications for treatment selection
Knee anatomy and clinical tests 2024.pdfvimalpl1234
This includes all relevant anatomy and clinical tests compiled from standard textbooks, Campbell,netter etc..It is comprehensive and best suited for orthopaedicians and orthopaedic residents.
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Ve...kevinkariuki227
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
Acute scrotum is a general term referring to an emergency condition affecting the contents or the wall of the scrotum.
There are a number of conditions that present acutely, predominantly with pain and/or swelling
A careful and detailed history and examination, and in some cases, investigations allow differentiation between these diagnoses. A prompt diagnosis is essential as the patient may require urgent surgical intervention
Testicular torsion refers to twisting of the spermatic cord, causing ischaemia of the testicle.
Testicular torsion results from inadequate fixation of the testis to the tunica vaginalis producing ischemia from reduced arterial inflow and venous outflow obstruction.
The prevalence of testicular torsion in adult patients hospitalized with acute scrotal pain is approximately 25 to 50 percent
Explore natural remedies for syphilis treatment in Singapore. Discover alternative therapies, herbal remedies, and lifestyle changes that may complement conventional treatments. Learn about holistic approaches to managing syphilis symptoms and supporting overall health.
- Video recording of this lecture in English language: https://youtu.be/lK81BzxMqdo
- Video recording of this lecture in Arabic language: https://youtu.be/Ve4P0COk9OI
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New Drug Discovery and Development .....NEHA GUPTA
The "New Drug Discovery and Development" process involves the identification, design, testing, and manufacturing of novel pharmaceutical compounds with the aim of introducing new and improved treatments for various medical conditions. This comprehensive endeavor encompasses various stages, including target identification, preclinical studies, clinical trials, regulatory approval, and post-market surveillance. It involves multidisciplinary collaboration among scientists, researchers, clinicians, regulatory experts, and pharmaceutical companies to bring innovative therapies to market and address unmet medical needs.
Pulmonary Thromboembolism - etilogy, types, medical- Surgical and nursing man...VarunMahajani
Disruption of blood supply to lung alveoli due to blockage of one or more pulmonary blood vessels is called as Pulmonary thromboembolism. In this presentation we will discuss its causes, types and its management in depth.
The prostate is an exocrine gland of the male mammalian reproductive system
It is a walnut-sized gland that forms part of the male reproductive system and is located in front of the rectum and just below the urinary bladder
Function is to store and secrete a clear, slightly alkaline fluid that constitutes 10-30% of the volume of the seminal fluid that along with the spermatozoa, constitutes semen
A healthy human prostate measures (4cm-vertical, by 3cm-horizontal, 2cm ant-post ).
It surrounds the urethra just below the urinary bladder. It has anterior, median, posterior and two lateral lobes
It’s work is regulated by androgens which are responsible for male sex characteristics
Generalised disease of the prostate due to hormonal derangement which leads to non malignant enlargement of the gland (increase in the number of epithelial cells and stromal tissue)to cause compression of the urethra leading to symptoms (LUTS
Rationale and Procedure for Oncology Pricing and Reimbursement in England Towse 2014
1. The Rationale and Procedure for
Oncology Pricing and Reimbursement
Decisions in England
Adrian Towse
BDA1 Workshop:
Access to Innovative Oncology Medicines
Bonn • 16-17 January 2014
1Biotherapy
Development Association
2. Agenda
•
Pricing and reimbursement (P&R) context
•
Where are we now?
•
Issues for HTA in assessing oncology medicines
•
Value-based assessment and the end-of-life
(EoL) criteria
•
Emerging issues
– HTA for companion diagnostics
– differential pricing
P&R for oncology drugs in the UK
16th January 2014 2
3. UK P&R context
• In the UK, companies set prices, NHS decides
use
• England uses NICE to perform an HTA and
recommend on use; Scotland uses the Scottish
Medicines Consortium
• Hospitals use tenders when relevant
• Companies can offer discounts via patient
access schemes in England and the Scottish
equivalent
P&R for oncology drugs in the UK
16th January 2014 3
4. UK P&R context
Concerns 10 years ago: (1) poor uptake of new cancer
medicines in UK and (2) poor 5-year survival rates in the UK
Cancer a priority for the Government
NICE assesses all new cancer medicines
Introduction of EoL criteria
Introduction of Cancer Drugs Fund (CDF)
P&R for oncology drugs in the UK
16th January 2014 4
5. Improving UK cancer outcomes
•
•
•
•
•
Higher rates of curative
surgery
Increased drug use
Public health measures
Tackling late presentation
Increasing speed of
primary care referrals
P&R for oncology drugs in the UK
16th January 2014 5
6. Richards’ report on drug uptake
Brings together IMS data with
manufacturers’ own information.
Strong industry engagement in data
validation. Support and engagement
from industry trade bodies.
Data findings tested and potential
causes of variation explored with a
range of UK experts, including
clinicians, pharmacists, public health
doctors, academics, patients’
representatives and industry
Result: the most comprehensive
insight to date on international
variations in drug usage
P&R for oncology drugs in the UK
16th January 2014 6
7. International Variations in Drug Usage
Comparison of UK drugs use (by ranking and percentage of
14-country average)
Source: Presentation by Sir Mike Richards 2010
7
8. Richards’ international usage report country
ranking for cancer medicines classes
Number of years on UK market since launch in 2009
0-5 years
6-10 years
Rank
Country
1
France
2
Switzerland
3
Austria
4
Spain
5
Italy
6
Germany
7
USA
8
UK
10+ years
Rank
Country
1
France
2
Italy
3
Spain
4
Germany
5
Switzerland
6
Austria
7
USA
8
Sweden
Rank
1
2
3
4
5
6
7
8
Country
France
Austria
USA
Germany
Spain
Switzerland
Sweden
Italy
9
Norway
9
Australia
9
10
11
12
Australia
UK
Canada
10
11
12
Sweden
Canada
Norway
10
11
12
13
New Zealand
13 New Zealand
Hormonal cancer
treatments
Rank
1
2
3
4
5
6
7
8
Country
Italy
Spain
Germany
France
UK
Austria
Sweden
Norway
UK
9
Switzerland
Canada
Norway
Australia
10
11
12
Canada
Australia
USA
13
New Zealand
13 New Zealand
Source: Richards, 2010. Extent and causes of international variations in drug usage
P&R for oncology drugs in the UK
16th January 2014 8
9. Impact of patient access schemes
If all positive
decisions since 2009
where a PAS was
implemented were
assumed to be a
“not recommended”
decision in the
absence of a PAS
(bar labelled
“without PAS”) the
share of not
recommended
decisions increases
to 47%
Chart: share of decision outcome for all medicines
decisions from 2009 to Q3 2013, with and “without” PAS
Source: OHE analysis from data on NICE website
P&R for oncology drugs in the UK
16th January 2014 9
10. Trends in decision for cancer medicines
before and after establishment of cancer
drugs fund (Q4 2010- Q3 2013)
Source: OHE analysis from data on NICE website
P&R for oncology drugs in the UK
16th January 2014 10
11. Where are we now?
Uptake has hopefully improved (new report due out soon)
Inconsistency between use of NICE and existence of CDF
HTA in cancer has issues
Introduction of VBA will throw up further issues, e.g. future
of EoL criteria
Issues for the future:
• Small patient groups for rarer cancers – orphan and “ultra
orphan” drugs
• Use of companion diagnostics
• Different prices for different indications
P&R for oncology drugs in the UK
16th January 2014 11
12. Sources of uncertainty
•
•
•
Mapping from
progression-free survival
(PFS) to overall survival
(OS)
Correcting clinical trial
data to account for
treatment crossover
Complexity of treatment
sequencing options
Advances in Oncology Modelling: Recent
Results from POI Research
Posted on 18 September 2012 by OHE News
Editor
OHE and the Pharmaceutical Oncology
Initiative (POI) held a workshop to discuss and
debate several key issues in oncology
modelling. Participating were more than 50
experts from industry, academia and
government.
The focus of the workshop was three key
technical challenges associated with using
clinical trial data for anti-cancer medicines and
adapting it, in an unbiased fashion, to support
decisions about cost-effectiveness. These are:
extrapolating from progression-free
survival, correcting for patient crossover,
and efficient modelling of different
sequences of treatment.
http://news.ohe.org/2012/09/18/advances-inoncology-modelling-recent-results-from-poiresearch/
P&R for oncology drugs in the UK
16th January 2014 12
13. Limitations of using QALYs in cancer
Sensitivity of EQ-5D to
changes in health status of
cancer patients
Violation of constant
proportional trade-off
assumption at end of life
Using valuations from
members of the general
population
P&R for oncology drugs in the UK
16th January 2014 13
14. Eliciting social preferences: End-oflife findings highlight the challenges
Linley and Hughes (2012)
Shah, Tsuchiya and Wailoo (2013)
P&R for oncology drugs in the UK
16th January 2014 14
15. Authors.
journal
(date)
Shah,
Tsuchiya,
Hole,
Wailoo
DSU report
(2012)
Linley and
Hughes
Health Econ
(2012)
Olsen
Value in
Health
(2013)
Elicitation
Method(s)
Responden Mode of
t sample
admin
(n)
General
population
DCE
Online
survey
EoL comparison
Life expectancy without
treatment
3, 12 ,24 36, 60m
(3969)
Choice plus
trade off
questions
General
population
18 months vs 60 m
Online
survey
Life extension 6 months
Findings relevant to EoL
EoL was not a significant driving
factor (but longest LE 60 months)
No evidence of a value premium for
EoL
(4118)
Choice plus
benefit trade
off
General
population
(503)
Online
survey
Remaining lifetime
untreated;
Total lifetime untreated;
Relative loss in remaining
life expectancy (prop
shortfall)
Source: Rachel Baker, Glasgow Caledonian University
No evidence of EoL (proximity to
death) premium
Total lifetime inequality is most
important and ‘trumps EoL’
Not proportional shortfall but
absolute loss
16. Authors.
journal
(date)
Pinto Prades
et al
Working
paper
2013
Elicitation Responden
Method(s t sample
)
(n)
WTP
PTO
3 survey
versions:
(820)
General
population
Choice
tasks
EoL comparison
Probability of gains in
LE (EoL)
QoL (EoL)
QoL (non-terminal)
Findings relevant to EoL
EoL gains valued more highly than
gains for non-EoL conditions
Respondent split into 2 groups
Gains in QoL at Eol more valued than LE
gains.
Some support for prioritising patients
with shorter life expectancy.
Face to face
1 year vs 10 year LE
(50)
Respondents in 2 groups
QoL at EoL more valued than LE at EoL.
Preparation for death may be
important (suddenness).
Rowen,
Brazier et al
HESG (2014)
General
population
Face to face
Shah,
Tsuchiya,
Wailoo
Eur J Health
Econ (2013)
Mode of
admin
General
population
DCE
(3669)
Life expectancy without
condition (5, 20, 40, 80)
Online
survey
LE with condition w/out
treatment
(3m-5 years, 3m-10
yrs,3m-30 yrs, 3m-60
yrs)
Source: Rachel Baker, Glasgow Caledonian University
Size of the health gain important
Coefficient for EoL (as per NICE
definition) positive and significant.
Not clearly evident in attitudinal
questions
1422 respondents possible
misunderstood tasks.
17. Institutional processes for the value
assessment of new diagnostics
Source: Garau, M., Towse, A., Garrison, L., Housman, L. and Ossa, D. (2013) Can and should
value-based pricing be applied to molecular diagnostics? Personalized Medicine. 10(1), 61-72.
P&R for oncology drugs in the UK
16th January 2014 17
18. Need for flexible pricing and more
outcomes-based PAS
Garrison et al (2013)
Academy of Medical Sciences (2013)
P&R for oncology drugs in the UK
16th January 2014 18
19. A reordering of process?
Criteria: broader definition of value
(risks, benefits)
Affordability
(BIA)
Safety
Value for
money
(CE)
Efficacy,
effectiveness
Overall D-M Framework:
Other factors of value to D-M (ethical issues, social values, feasibility of Opportunity costs
implementation, unmet needs, innovation value, legal issues …)
(value-for-money)
Source: Professor Ron Goeree, Director PATH Research Institute, McMaster University
P&R for oncology drugs in the UK
16th January 2014 19
20. Conclusions
1.
England’s access to innovative oncology medicines has
(hopefully) improved as a result of a series of policy initiatives
2.
Inconsistencies in policy in England exist, e.g. role of CDF
3.
Issues for HTA in assessing oncology medicines
4.
Value-based assessment will have a big potential impact on
oncology medicines and the EoL criteria
5.
Social preferences as well as cost-effectiveness are now at the
core of value assessment in HTA, but they are not easy to
establish using empirical research
6.
Other issues for the future include addressing HTA for
companion diagnostics and differential pricing
7.
Price flexibility by indication / subgroup and outcomes-based
CED/ PBRSA schemes are important to effective use of drugs
P&R for oncology drugs in the UK
16th January 2014 20
21. References
Academy of Medical Sciences. (2013) Realising the potential of stratified medicines. London: Academy of Medical
Sciences.
Baker, R. (Glasgow Caledonian University). (2014) Plenary session presentation. Health Economists’ Study Group.
Sheffield. 8-10 January 2014.
Garau, M., Shah, K., Mason, A., Wang, Q., Towse, A. and Drummond, M. QALYs in cancer: A review of the methodological
limitations. PharmacoEconomics. 29(8), 673-685.
Garau, M., Towse, A., Garrison, L., Housman, L. and Ossa, D. (2013) Can and should value-based pricing be applied to
molecular diagnostics? Personalized Medicine. 10(1), 61-72.
Linley, W.G. and Hughes, D.A. (2012) Societal views of NICE, Cancer Drugs Fund, and value based pricing criteria for
prioritising medicines: A cross-sectional survey of 4118 adults in Great Britain. Health Economics. 22(8), 948-964.
OHE Editor. (2012) Advances in Oncology Modelling: Recent Results from POI Research. OHE News. [blog] 18 September.
Available at: http://news.ohe.org/2012/09/18/advances-in-oncology-modelling-recent-results-from-poi-research/
[Accessed 14 January 2014].
Richards, M. (2010) Extent and causes of international variations in drug usage. London: Department of Health. Available
at https://www.gov.uk/government/publications/extent-and-causes-of-international-variations-in-drug-usage.
Richards, M. (2011) Achieving world class outcomes in cancer treatment. London: Office of Health Economics/ Available at
http://www.ohe.org/publications/article/achieving-world-class-outcomes-in-cancer-treatment-3.cfm
Shah, K., Tsuchiya, A. and Wailoo, A. (2013) Valuing health at the end of life: An empirical study of public preferences.
European Journal of Health Economics. Epub ahead of print. doi: 10.1007/s10198-013-0482-3.
P&R for oncology drugs in the UK
16th January 2014 21
That is not to say that price flexibility is not important. The 2009 PPRS, negotiated shortly after the OFT Report was published, introduced a one-off ability for a price adjustment or for an outcomes based Patient Access Scheme to address uncertainty about the outcomes associated with a drug. The greatest number of these schemes are “simple discount” arrangements, designed to make a price offer to the NHS that is not revealed to other payers, to avoid changing the list price which is used for reference pricing. There are lots of questions about the sustainability of these schemes, but there is no doubt that they have substantially increased access to new drugs at prices that NICE deems offer value to the NHS.
This is an analysis of oncology decisions pre- and post- the CDF. It shows that the number of not-recommended decisions has more than doubled from 24% to 58% at the expense of restricted decisions. Straight approvals (green) have not changed. (Note – my colleagues tell me NICE does not agree with this analysis)One might expect this to happen if the existence of the CDF reduces the willingness of both parties to compromise. Companies have another route into the NHS. The Appraisal Committee might expect patients will still get access to the drug via the CDF if NICE turns it down. There is a global willingness to pay (i.e. outside of the UK) for oncology treatment that is substantially higher than that for health gain in other disease areas. This is the reason why the Government set up the CDF. We need a new process for ultra-orphans as well (HSTs – highly specialised treatments)