Pcn490 Making Outcome-Based Payment a Reality in the National Health Service for England

Cole, A; Cubí‐Mollá, Patricia; Pollard, Jack; Sim, Duncan; Sullivan, Richard; Sussex, Jon; Lorgelly, Paula

doi:10.1016/j.jval.2019.09.682

Cited by 11 publications

(33 citation statements)

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“…Cancer Research UK and Greater Manchester Health and Social Care Partnership (GMHSCP) jointly commissioned the authors to explore the possibility of developing a new model of paying for some cancer drugs within the NHS, both in Greater Manchester and at a national level, explicitly on the basis of the outcomes they achieve, and specifically the outcomes that are most important to patients [8,9]. Outcome-based payment (OBP) aligns a medicine's cost to the NHS and the payment to its manufacturer with the benefits it delivers for patients in the real world.…”

Section: Introductionmentioning

confidence: 99%

Outcome-Based Payment Schemes: What Outcomes Do Patients with Cancer Value?

Lorgelly

Pollard

Cubí‐Mollá

et al. 2020

Patient

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Background Uncertainty about the benefits new cancer medicines will deliver in clinical practice risks delaying patient access to new treatment options in countries such as England, where the cost effectiveness of new medicines affects reimbursement decisions. Outcome-based payment (OBP) schemes, whereby the price paid for the drug is linked to patients' real-world treatment outcome(s) has been put forward as a mechanism to accelerate access. Although OBP schemes have generally focused on clinical outcomes to determine reimbursement, the degree to which these represent the outcomes that are important to patients is unclear. Objective To advance the application of OBP we ask, what outcomes do patients with cancer value (most) that might form a practical basis for OBP? Methods A review of the literature on outcomes in cancer produced a long list of candidates. These were evaluated in a focus group with patients with cancer and were then, in a second focus group, distilled to a shortlist of ten outcomes using a card sort method. The ten outcomes were included in an online survey of patients with cancer and carers, who were asked to rank the importance of each outcome. Results The focus groups identified a range of both clinical and functional outcomes that are important to patients. Analyses of the 164 survey responses suggested that the four most important outcomes to patients and carers are survival; progression, relapse or recurrence; post-treatment side effects; and return to normal activities of daily life. Conclusion Commissioners of cancer services wishing to instigate an OBP scheme should prioritise collecting data on these outcomes as they are important to patients. Of these, only mortality data are routinely collected within the national health service (NHS). Progression and some morbidity data exist but are not currently linked, creating a challenge for OBP. Key Points for Decision Makers Outcome-based payment schemes for new medicines should address the outcomes that patients and carers value most. The four most important outcomes to patients and carers are survival; progression, relapse or recurrence; posttreatment side effects; and return to normal activities of daily life. Only mortality data are routinely collected within the National Health Service; collecting data on the four core outcomes should be prioritised to realise outcome-based payment for some new cancer medicines in future.

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Section: Introductionmentioning

confidence: 99%

Outcome-Based Payment Schemes: What Outcomes Do Patients with Cancer Value?

Lorgelly

Pollard

Cubí‐Mollá

et al. 2020

Patient

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“…Whereas some of these additional data could potentially be collected through NCRAS, it increases complexity, especially when compared to EBMT, where all of these clinical outcomes are collected in one place. SACT in its current form could potentially facilitate OBR in certain cancers where the prognosis is particularly poor, e.g., where a meaningful outcome measure is a 30-day mortality; however, it does not seem equipped to support OBR schemes in cancers where patient survival is better (this is echoed also in CRUK's 2019 report [14]).…”

Section: Discussionmentioning

confidence: 99%

“…In terms of economic outcomes, this can potentially be sourced from Hospital Episode Statistics (HES), (Electronic) Health Records (EHR), SUS, and other sources using patients' NHS ID numbers; however, this requires investment in systems integration, as well as overcoming governance issues (e.g., EHR data are under the governance of individual trusts). PROMs and PREMs such as the ability to return to normal daily activities are increasingly regarded as important, perhaps especially for younger patients [14]; however, there are currently no national databases to collect these data. Nor is there currently an agreed standard or consensus on what PROMs and PREMs are most relevant in cancer, meaning that efforts would need to be made to identify these as well as facilitating an infrastructure to capture the data.…”

Section: Discussionmentioning

confidence: 99%

“…Against this backdrop, it is tempting to ask whether the particulars of the SACT and EBMT registries may (in part) help explain the lack of OBRs seen in practice. In February 2019, Cancer Research UK (CRUK) published a report (with an analysis undertaken by the Office for Health Economics), which highlighted shortcomings around SACT's outcomes data, and recommended that ' … a mapping exercise should be undertaken to ascertain the appropriate data sources, and identify "gaps" in the capacity to collect [outcomes] data … ' [14]. We support this view and believe that a similar gap analysis could be extended also to EBMT, to understand in more detail what would be required to implement OBR schemes for ATMPs in the cancer area.…”

Section: The European Society For Blood and Marrow Transplantation (Ementioning

confidence: 99%

“…While the idea of OBR is a fairly straightforward and has been covered extensively in the literature [7,[9][10][11][12][13][14][15], such reimbursement schemes have historically seen low levels of adoption in practice in Europe [16]. While it is not possible to point to one definitive reason for the low levels of adoption of such schemes, an often-cited reason is the administrative burden [14]. A central component of this administrative burden relates to the collection of outcomes data, which necessitates both having an appropriate data collection infrastructure (e.g., a database) and practice that provide the appropriate operational framework.…”

Section: Introductionmentioning

confidence: 99%

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Upgrading the SACT dataset and EBMT registry to enable outcomes-based reimbursement in oncology in England: a gap analysis and top-level cost estimate

Jørgensen

Kefalas

2019

Journal of Market Access & Health Policy

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Background: Outcomes-based reimbursement (OBR) can reduce decision uncertainty and accelerate patient access to cell and gene therapies, however, OBR is rarely applied in practice in England. Oncology is the therapy area with the most cell and gene therapies in late-stage development, and the Systemic Anti-Cancer Therapy (SACT) dataset and The European Society for Blood and Marrow Transplantation (EBMT) registry are two data collection infrastructures that could potentially act as conduits for implementing OBR in cancer in England. Objective: To perform a gap analysis to identify the key requirements for upgrading the SACT and EBMT databases for the purposes of enabling OBR, and a top-level estimation of how much this upgrade may cost, using either a manual (staff-heavy) workaround or part automation (technology-heavy) approach. Methodology: The analysis of current data capture and gaps is informed by secondary research, while the assumptions and data used to derive the top-level cost estimates were informed by consensus-based primary research with experts in healthcare information technology (IT) systems integration and platform development, as well as experts of SACT and EBMT. Findings: In its current form, the SACT dataset in isolation is largely unfit for enabling OBR in oncology, whether through clinical, economic or humanistic outcomes. The EBMT registry has a greater potential; however, this relates to key clinical outcomes only, not economic or humanistic outcomes. Part automation requires a higher upfront investment than the manual workaround (~£1.8 million vs.~£400k); however, lower annual costs (~£200 vs.~£260k-£850k) mean that part automation becomes a more cost-effective approach over time. Conclusions: An appropriately automated and scalable data collection infrastructure should be implemented, with the ability to integrate clinical, economic and humanistic outcomes with healthcare cost data and payment systems, to enable OBR not only in cancer but also in other therapy areas.

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Time to Review Authorisation and Funding for New Cancer Medicines in Europe? Inferences from the Case of Olaratumab

Pontes

Zara

Torrent-Farnell

et al. 2019

Appl Health Econ Health Policy

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The potential benefits of early patient access to new medicines in areas of high unmet medical need are recognised, but uncertainties concerning effectiveness, safety, and added value when new medicines are authorised and subsequently funded based on initial preliminary data only have important implications. In 2016, olaratumab received accelerated conditional approval from both the EMA and FDA for the treatment of soft tissue sarcoma based on the claims of a substantial reduction in the risk of death with an 11.8 month improvement in median overall survival in a phase II trial in combination with doxorubicin versus doxorubicin alone. The failure to confirm these benefits in the post authorisation pivotal trial has highlighted key concerns regarding early access and conditional approvals for new medicines. Concerns include potentially considerable opportunity clinical and economical costs, so that patients may have received suboptimal treatment and money spent has foregone the opportunity to improve access to effective treatments. As a result, it seems reasonable to reconsider current marketing authorisation models and approaches. Potential ways forward include closer collaboration between regulators, pharmaceutical companies and payers to enhance the generation of rapid and comparative confirmatory trials in a safe and fair way with minimal patient exposure to achieve robust evidence. Also, it may be time to review early access systems and to explore new avenues regarding who should pay or part pay for new treatments whilst information is being collected as part of any obligations for conditional marketing authorisation. Greater co-operation between countries regarding the collection of data in routine clinical care, and further research on post marketing data analysis and interpretation, may also contribute to improved appraisal and continued access to new innovative cancer treatments.

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Pcn490 Making Outcome-Based Payment a Reality in the National Health Service for England

Cited by 11 publications

References 0 publications

Outcome-Based Payment Schemes: What Outcomes Do Patients with Cancer Value?

Outcome-Based Payment Schemes: What Outcomes Do Patients with Cancer Value?

Upgrading the SACT dataset and EBMT registry to enable outcomes-based reimbursement in oncology in England: a gap analysis and top-level cost estimate

Time to Review Authorisation and Funding for New Cancer Medicines in Europe? Inferences from the Case of Olaratumab

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