10 research outputs found
The evolving value assessment of cancer therapies: Results from a modified Delphi study
The move toward early detection and treatment of cancer presents challenges for value assessment using traditional endpoints. Current cancer management rarely considers the full economic and societal benefits of therapies. Our study used a modified Delphi process to develop principles for defining and assessing value of cancer therapies that aligns with the current trajectory of oncology research and reflects broader notions of value. 24 experts participated in consensus-building activities across 5 months (16 took part in structured interactions, including a survey, plenary sessions, interviews, and off-line discussions, while 8 participated in interviews). Discussion focused on: 1) which oncology-relevant endpoints should be used for assessing treatments for early-stage cancer and access decisions for early-stage treatments, and 2) the importance of additional value components and how these can be integrated in value assessments. The expert group reached consensus on 4 principles in relation to the first area (consider oncology-relevant endpoints other than overall survival; build evidence for endpoints that provide earlier indication of efficacy; develop evidence for the next generation of predictive measures; use managed entry agreements supported by ongoing evidence collection to address decision-maker evidence needs) and 3 principles in relation to the second (routinely use patient reported outcomes in value assessments; assess broad economic impact of new medicines; consider other value aspects of relevance to patients and society)
UK results from the myeloproliferative neoplasms (MPN) landmark survey on the symptom, emotional and economic burden of MPN
UK results from the myeloproliferative neoplasms (MPN) landmark survey on the symptom, emotional and economic burden of MPN
Comparisons of patient management in myeloproliferative neoplasm patients in the UK vs rest of the world: analysis from the international landmark survey
COMPARISONS OF SYMPTOM BURDEN IN MYELOPROLIFERATIVE NEOPLASM PATIENTS IN THE UK VS REST OF WORLD: ANALYSIS FROM THE INTERNATIONAL LANDMARK SURVEY
Comparisons of patient management in myeloproliferative neoplasm patients in the UK vs rest of the world: analysis from the international landmark survey
EHTA307 Evolving Principles for Defining and Assessing the Economic and Societal Value of Cancer Therapies
The evolving value assessment of cancer therapies: Principles from the cancer community.
Background: Oncology-relevant measures of disease and treatment support earlier detection, accelerated drug development, and inform treatment pathways. To advance access to transformative treatments for people with cancer, especially those for early-stage cancers where medical intervention has the greatest opportunity to impact the course of the disease, the cancer community identified seven principles that support the evolution of value assessment frameworks. Here we summarize the five key principles.
Methods: A group of 24 cancer community experts (9 oncologists, 6 patient advocacy leaders, 1 former regulator, 6 former payers, and 4 health economists) from 14 countries came together to discuss and build consensus on oncology-relevant value principles. Over five months, the expert group engaged via surveys, virtual discussion panels, and interviews.
Results: The expert group developed seven consensus principles covering two areas: oncology-relevant endpoints to assess the value of treatments; and additional important value components and how they can be integrated into value assessments. The key 5 principles were oncology-relevant endpoints assessing clinical effectiveness, including the following three items. 1) Consider oncology-relevant endpoints, other than Overall Survival (OS), with intrinsic value for decision-making e.g., Event-, Disease-, or Relapse-Free Survival. 2) Continue to build evidence for endpoints that indicate treatment efficacy earlier e.g., pathologic Complete Response. 3) Develop evidence for the next generation of predictive measures that detect and monitor disease, e.g., circulating tumor DNA and Minimal Residual Disease. Additional value components supporting patient access include the following two items. 1) Use managed entry (type of access) agreements supported by ongoing real world evidence collection to help address decision-maker (regulator and Health Technology Assessment body) evidence needs. 2) Routinely use Patient Reported Outcomes (PROs) in value assessments.
Conclusions: To achieve the common goal of all cancer community stakeholders—improved outcomes for people with cancer—collaboration across the cancer community is essential to define how value is assessed, especially for early-stage cancer treatments. This is particularly important since mature OS data is not always available during initial decision-making, and decision-makers need to evolve which are the most appropriate endpoints. This supports measuring clinical impact, but also broader social, financial and ethical impact for patients, caregivers, healthcare systems, society, and the economy (not considered in this abstract). To achieve these goals, the expert group recommends: 1) increasing awareness and systematically engaging people with cancer in value assessments, 2) leveraging PROs in those assessments, and 3) further validation in clinical trials of oncology-relevant endpoints that could provide an earlier indication of clinical outcomes
We need to talk about values: a proposed framework for the articulation of normative reasoning in health technology assessment
It is acknowledged that health technology assessment (HTA) is an inherently value-based activity that makes use of normative reasoning alongside empirical evidence. But the language used to conceptualise and articulate HTA's normative aspects is demonstrably unnuanced, imprecise, and inconsistently employed, undermining transparency and preventing proper scrutiny of the rationales on which decisions are based. This paper - developed through a cross-disciplinary collaboration of 24 researchers with expertise in healthcare priority-setting - seeks to address this problem by offering a clear definition of key terms and distinguishing between the types of normative commitment invoked during HTA, thus providing a novel conceptual framework for the articulation of reasoning. Through application to a hypothetical case, it is illustrated how this framework can operate as a practical tool through which HTA practitioners and policymakers can enhance the transparency and coherence of their decision-making, while enabling others to hold them more easily to account. The framework is offered as a starting point for further discussion amongst those with a desire to enhance the legitimacy and fairness of HTA by facilitating practical public reasoning, in which decisions are made on behalf of the public, in public view, through a chain of reasoning that withstands ethical scrutiny
Postoperative critical care and high-acuity care provision in the United Kingdom, Australia, and New Zealand
BACKGROUND: Decisions to admit high-risk postoperative patients to critical care may be affected by resource availability. We aimed to quantify adult ICU/high-dependency unit (ICU/HDU) capacity in hospitals from the UK, Australia, and New Zealand (NZ), and to identify and describe additional 'high-acuity' beds capable of managing high-risk patients outside the ICU/HDU environment. METHODS: We used a modified Delphi consensus method to design a survey that was disseminated via investigator networks in the UK, Australia, and NZ. Hospital- and ward-level data were collected, including bed numbers, tertiary services offered, presence of an emergency department, ward staffing levels, and the availability of critical care facilities. RESULTS: We received responses from 257 UK (response rate: 97.7%), 35 Australian (response rate: 32.7%), and 17 NZ (response rate: 94.4%) hospitals (total 309). Of these hospitals, 91.6% reported on-site ICU or HDU facilities. UK hospitals reported fewer critical care beds per 100 hospital beds (median=2.7) compared with Australia (median=3.7) and NZ (median=3.5). Additionally, 31.1% of hospitals reported having high-acuity beds to which high-risk patients were admitted for postoperative management, in addition to standard ICU/HDU facilities. The estimated numbers of critical care beds per 100 000 population were 9.3, 14.1, and 9.1 in the UK, Australia, and NZ, respectively. The estimated per capita high-acuity bed capacities per 100 000 population were 1.2, 3.8, and 6.4 in the UK, Australia, and NZ, respectively. CONCLUSIONS: Postoperative critical care resources differ in the UK, Australia, and NZ. High-acuity beds may have developed to augment the capacity to deliver postoperative critical care