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Displaying One Session

Channel 2 Proffered Paper session
Date
21.09.2020
Time
14:25 - 16:05
Room
Channel 2
Chairs
  • Rosa Giuliani (Liverpool, United Kingdom)
  • Jose M. Martin-Moreno (Valencia, Valencia, Spain)
Proffered Paper - Public Policy Proffered Paper session

1581O_PR - Estimation of European cancer burden for the year 2020

Presentation Number
1581O_PR
Lecture Time
14:25 - 14:37
Speakers
  • Tadeusz A. Dyba (Ispra, Italy)
Room
Channel 2
Date
21.09.2020
Time
14:25 - 16:05

Abstract

Background

Up-to-date cancer burden indicators provide an important source of information for supporting political decision making, as well as for epidemiological research and the general public. Nevertheless, observed cancer incidence and mortality suffer from an inherent registration delay in the data production workflow. To overcome this, the European Commission’s Joint Research Centre in collaboration with the WHO’s International Agency for Research on Cancer have computed estimates of cancer incidence and mortality, for the year 2020 and for European countries, in the framework of the European Cancer Information System (ECIS).

Methods

Predicted values for the year 2020 are based on the incidence data of more than 150 European population-based cancer registries included in ECIS, and on mortality data provided by WHO. Ad-hoc statistical models were developed on the basis of the most recent time trends of observed data to estimate cancer incidence and mortality rates in each EU country for the year 2020. Estimated rates were then applied to the projected population figures for 2020 from EUROSTAT in order to calculate the predicted number of new cases and deaths for 2020 in 40 European countries.

Results

The number of new cancer cases and deaths in 2020 has been estimated per country by sex and age group, for 25 major cancer sites. The results are included and disseminated through the European Cancer Information System (ECIS) web application.

Conclusions

The release of up-to-date cancer incidence and mortality estimates is of great importance to support EU evidence-based cancer policies. The homogeneity of the estimation methods applied throughout Europe guarantees the comparability of the estimated values between countries. Reliable and comparable estimates highlight differences between countries in cancer incidence and mortality, thus facilitating the identification of possible intervention areas. The applied methodology couldn’t take into account the possible impact of the COVID-19 pandemic on the projected rates. A future exercise to evaluate the discrepancy between projected and observed rates will allow quantification of this impact.

Legal entity responsible for the study

The authors.

Funding

European Commission.

Disclosure

All authors have declared no conflicts of interest.

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Proffered Paper - Public Policy Proffered Paper session

LBA66_PR - Disparities in access to oncology clinical trials in Europe in the period 2009-2019

Presentation Number
LBA66_PR
Lecture Time
14:37 - 14:49
Speakers
  • Ana Carneiro (Lund, Sweden)
Room
Channel 2
Date
21.09.2020
Time
14:25 - 16:05

Abstract

Background

Clinical trials are essential for advancing cancer treatment. Yet, there is limited data on their distribution and access in Europe. To ascertain the extent of potential inequalities in access to clinical trials in Europe, we compared their distribution among European countries.

Methods

The Clinicaltrials.gov database was searched for interventional clinical trials in adults with neoplasms. Available data from phase I-III trials between 06/2009 to 06/2019 in Europe were retrieved. We considered the number of clinical trials registered in each country and one “trial-entry” was defined as one trial/country.

Results

In total, 18454 trial-entries were identified, of which 12% were phase I, 10% phase I/II, 32% phase II, 2% phase II/III and 44% phase III; 74% were industry-sponsored, 15% were academic and 11% were an academic/industry partnership. The number of trials per country varied from 2.48 in Central/Eastern Europe to 5.33/100 000 inhabitants in Northern Europe. The proportion of phase I-II trials was higher in the Southern and Western regions (13-15%) compared to Central/Eastern and Northern regions (4-9%). The number of trial-entries/100 000 inhabitants/country ranged from 0.14 (Albania) to 10.7 (Belgium). Between 2010 and 2018, the total number of trials per country in Europe increased by 33%. The increase in early-phase trials was larger (phase I-II, 61%) than in late-phase trials (phase II-III, 7%). Portugal, Ireland, Finland, Greece and Norway registered the largest percentage increase in early-phase trials, while Ireland, Spain, Norway, Italy and Belgium led the largest percentage increase in late-phase trials. Five countries dominated in terms of an increase in the absolute number of total trial-entries in both early- and late-phase trials: Spain (90/40), France (45/16), UK (45/13), Italy (38/19) and Belgium (35/12). During this period there was no significant variation in the distribution of industry and academic sponsored trials but an increase in industry/academic partnerships was observed ( 8%).

Conclusions

The number of clinical trials varies greatly among European regions resulting in potential asymmetries in patients' access to clinical trials. The disparities in access to oncology trials need to be addressed by all the stakeholders.

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

T.M.S. Amaral: Honoraria (self), Travel/Accommodation/Expenses: Novartis; Honoraria (self), Travel/Accommodation/Expenses: BMS; Honoraria (self): Pierre Fabre; Honoraria (self): CeCaVa. M. Brandao: Speaker Bureau/Expert testimony, Travel/Accommodation/Expenses: Roche/GNE. M. Scheffler: Advisory/Consultancy, Speaker Bureau/Expert testimony: Boehringer Ingelheim, Amgen, AstraZeneca, BMS, Roche, Pfizer, Takeda; Research grant/Funding (self): Amgen, Dracen. R. Ferrara: Advisory/Consultancy: MSD. M. Jalving: Advisory/Consultancy: Merck, BMS, Novartis, Pierre Fabre, Tesaro, AstraZeneca; Research grant/Funding (self): BMS, AbbVie, Merck, Cristal Therapeutics. G. Lo Russo: Advisory/Consultancy: MSD, BMS, Astrazeneca; Travel/Accommodation/Expenses: BMS, Roche. I. Marquez-Rodas: Advisory/Consultancy: BMS, MSD, Novartis, Roche, Pierre Fabre, Amgen, AstraZeneca, Regeneron, Sanofi, Highlight Therapeutics, Incyte, GSK. L. Mezquita: Research grant/Funding (self): Bristol-Myers Squibb, Boehringer Ingelheim; Non-remunerated activity/ies, Lectures and educational activities: Bristol-Myers Squibb, Tecnofarma, AstraZeneca, Roche; Advisory/Consultancy: Roche Diagnostics, Roche; Travel/Accommodation/Expenses: Bristol-Myers Squibb, Roche; Research grant/Funding (self), International Mentorship Program : AstraZeneca. S. Pilotto: Advisory/Consultancy, Speaker Bureau/Expert testimony: AstraZeneca, Eli-Lilly, BMS, Boehringer Ingelheim, MSD and Roche. E. Saloustros: Advisory/Consultancy: Pfizer, AstraZeneca, Roche; Speaker Bureau/Expert testimony: Pfizer, AstraZeneca, Roche, Amgen, BMS. All other authors have declared no conflicts of interest.

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Proffered Paper - Public Policy Proffered Paper session

Invited Discussant 1581O_PR and LBA66_PR

Lecture Time
14:49 - 14:59
Speakers
  • Rosa Giuliani (Liverpool, United Kingdom)
Room
Channel 2
Date
21.09.2020
Time
14:25 - 16:05
Proffered Paper - Public Policy Proffered Paper session

Q&A and live discussion

Lecture Time
14:59 - 15:09
Speakers
  • Rosa Giuliani (Liverpool, United Kingdom)
Room
Channel 2
Date
21.09.2020
Time
14:25 - 16:05
Proffered Paper - Public Policy Proffered Paper session

1582O - Developing a real-world outcomes forecast model using matched oncology clinical trials and real world evidence to inform policy-making and reimbursement approaches

Presentation Number
1582O
Lecture Time
15:09 - 15:21
Speakers
  • Jennifer Hinkel (Oxford, United Kingdom)
Room
Channel 2
Date
21.09.2020
Time
14:25 - 16:05

Abstract

Background

Payers and pharmaceutical companies increasingly consider evidence-linked market access schemes or outcome-based pricing models, where access or payments depend on real-world outcomes and involve financial risk-sharing. A recognized barrier to implementation has been the uncertainty of basing policy or financial decisions on clinical trial results that may inadequately predict a product’s performance in real-world settings or populations. To inform such models and policy decisions, stakeholders would ideally be able to forecast financial impact prior to implementation. Doing so also protects potential down-side financial risk—a key consideration in health systems reliant on public funds with responsibility for prudent budget management. We therefore sought to develop a methodology for forecasting a range of reasonably expected real-world clinical outcomes (RWCOs) using published data from recent oncology clinical trials and post-market studies.

Methods

Using a database of 40 follow-on, longitudinal outcomes studies from 2008-2019 in a large US community oncology network, we identified and matched phase 3 trials and cohorts to real-world studies to compile a dataset. To approach forecasting a range of reasonably expected RWCOs for the identified clinical trials in this dataset, we used nonlinear regression analysis and Monte Carlo simulation to fit and stress-test a curve across the matched cohorts and outcomes.

Results

Through a series of comparisons between phase 3 trial results and corresponding, matched long-term follow-on study results, future RWCOs can potentially be simulated in order to set upper and lower bounds with utility for outcomes-based or risk-sharing pharmaceutical pricing market access models. Next steps include expanding and refining the scope of algorithms tested for their predictive utility and validating these models against other matched sets of clinical trial and post-market studies.

Conclusions

Forecasting and modeling tools that predict RWCOs would allow policy and pricing stakeholders to better understand financial impacts of RWE-based policy decisions and could mitigate stakeholders’ exposure to down-side financial risks.

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

T. Gorman: Full/Part-time employment: Lydion Research. A. Ray: Leadership role, Full/Part-time employment, Officer/Board of Directors: Lydion Research. S. Brar: Full/Part-time employment: Lydion Research. J. Hinkel: Officer/Board of Directors: Lydion Research; Full/Part-time employment: Caris Life Sciences; Officer/Board of Directors: Global Campaign for Cancer Survivorship.

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Proffered Paper - Public Policy Proffered Paper session

1583O - Clinical benefit of cancer drugs approved in Switzerland during the last decade

Presentation Number
1583O
Lecture Time
15:21 - 15:33
Speakers
  • Roman Adam (St. Gallen, Switzerland)
Room
Channel 2
Date
21.09.2020
Time
14:25 - 16:05

Abstract

Background

It is unknown to what extent cancer drugs approved in Switzerland by Swissmedic fulfil criteria of clinical benefit according to ESMO, ASCO and the Swiss OLUtool criteria.

Methods

An electronic search of studies that led to new marketing authorisations in Switzerland between 2010 and 2019 was performed. Studies were evaluated according ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) v1.1, ASCO-Value Framework v2 (ASCO-VF) and OLUtool v2. Substantial benefit for ESMO-MCBS, was defined as a grade A or B for (neo)adjuvant intent and 4 or 5 for palliative intent. For ASCO-VF and OLUtool clinical benefit was defined as score >45 and A or B, respectively. Correlation between the frameworks was calculated with Cohen’s Kappa (κ). Factors associated with clinical benefit were evaluated by logistic regression. All statistical tests were two-sided.

Results

In the study period, 48 cancer drugs were approved for 92 evaluable indications, based on 100 studies. Of all studies 93% were in the palliative setting and 81% were phase III studies. Ratings for ESMO-MCBS, ASCO-VF and OLUtool could be performed for 100, 86, and 97 studies, respectively. Overall, 39 (39%), 44 (51%), 45 (46%) of the studies showed substantial clinical benefit according to ESMO-MCBS, ASCO-VF, OLUtool criteria, respectively. There was fair concordance between ESMO-MCBS and ASCO-VF in the palliative setting (κ = 0.31, p=0.004) and moderate concordance between ESMO-MCBS and OLUtool (κ = 0.41, p<0.001). There was no concordance between ASCO-VF and OLUtool (κ = 0.18, p=0.12). Factors associated with clinical benefit in the palliative setting in multivariable analysis are shown in the table.

Factor OR (p-value)
ESMO-MCBS 1.1 Breast cancer (vs. lung cancer) Melanoma (vs. lung cancer) 0.21 (0.093) 0.21 (0.073)
ASCO-VF v2* CPI combination tx (vs. small molecule) Chemo mono tx (vs. small molecule) CDK4/6 inhibitor plus endocrine tx (vs. small molecule) 0.08 (0.028) 0.13 (0.093) 0.06 (0.017)
OLUtool v2.0 Blinded study (vs. open label) Phase III study (vs. phase I or II) 3.30 (0.012) 3.47 (0.080)

CPI, checkpoint inhibitor; OR, odds ratio (adjusted); tx, therapy.* no adjustments were made since no other covariates had a p-value <0.1 in univariable analysis.

Conclusions

Only around half of the trials supporting marketing authorisation of recently approved cancer drugs in Switzerland meet the criteria for substantial clinical benefit when evaluated with ESMO-MCBS, ASCO-VF or OLUtool. At best, there was only moderate concordance between the grading systems.

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.

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Proffered Paper - Public Policy Proffered Paper session

1584O - Health technology assessment (HTA) in England, France and Germany: What do we know about variations in cancer-related HTA outcomes?

Presentation Number
1584O
Lecture Time
15:33 - 15:45
Speakers
  • Ramon Schaefer (Heidelberg, Germany)
Room
Channel 2
Date
21.09.2020
Time
14:25 - 16:05

Abstract

Background

Variations in HTA outcomes can primarily be explained with reference to institutional context and administrative rules. Here we focus on the German Federal Joint Committee (Gemeinsamer Bundesausschuss, GBA), the French Haute Autorité de Santé (HAS) and the British National Institute for Health and Care Excellence (NICE) to identify matched drug pairs as a basis to compare HTA outcomes and to analyze cancer-related HTA results in detail.

Methods

Data were extracted from all published GBA resolutions from January 2011 – when the AMNOG legislation was introduced in Germany – to June 2018, as well as all publicly available HAS reports and NICE single technology appraisals during this period. We compared HTA outcomes of matched pairs overall, and separately for non-cancer and cancer drugs. Then, the potential role of additional attributes with regard to cancer drugs was explored, such as orphan drug designation in Germany, impact of reimbursement rates in France and consideration of end of life (EoL) criteria in England. The relationship between HTA outcomes and attributes was tested for statistical significance with a chi-square test or, if required, Fisher's exact test.

Results

By pairwise comparison, HTA outcomes (cancer, 58/102; non-cancer, 44/102) showed higher congruence for GBA/HAS (total, 67%; cancer, 72%; non-cancer, 59%) than for GBA/NICE and HAS/NICE (total, 54%; cancer, 57%; non-cancer, 50%). NICE recommended 85/102 (cancer, 42/58; non-cancer, 43/44) technologies, whereas GBA and HAS reported added benefit for 72/102 (cancer, 49/58; non-cancer, 23/44) and 60/102 (cancer, 37/58; non-cancer, 23/44) medicines, respectively. When we tested NICE cancer recommendations for the consideration of EoL criteria, no significant impact was found (p>.05, Fisher's test). In contrast, GBA cancer appraisals are associated with an orphan designation (p<.05, chi-square test), and cancer-related findings for the HAS indicate that the reimbursement rate correlated with the assessment of clinical added value (p<.05, chi-square test).

Conclusions

Our results confirm that variations in HTA outcomes frequently exist. However, cancer-related HTA results seem to be less divergent compared to non-cancer results.

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.

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Proffered Paper - Public Policy Proffered Paper session

Invited Discussant 1582O, 1583O and 1584O

Lecture Time
15:45 - 15:55
Speakers
  • Christophe Le Tourneau (Paris, France)
Room
Channel 2
Date
21.09.2020
Time
14:25 - 16:05
Proffered Paper - Public Policy Proffered Paper session

Q&A and live discussion

Lecture Time
15:55 - 16:05
Speakers
  • Rosa Giuliani (Liverpool, United Kingdom)
Room
Channel 2
Date
21.09.2020
Time
14:25 - 16:05