Displaying One Session

Salamanca Auditorium (Hall 3) Proffered Paper session
Date
30.09.2019
Time
16:30 - 18:00
Location
Salamanca Auditorium (Hall 3)
Chairs
  • E.G. Elisabeth De Vries (Groningen, Netherlands)
  • Peter M. Ellis (Hamilton, ON, Canada)
Proffered Paper - Public policy Proffered Paper session

1626O - ESMO-MCBS and health technology assessment (HTA): Does value for physicians correspond to value for payers? (ID 5276)

Presentation Number
1626O
Lecture Time
16:30 - 16:42
Speakers
  • Apoorva Ambavane (Dubai, United Arab Emirates)
Location
Salamanca Auditorium (Hall 3), Fira Gran Via, Barcelona, Spain
Date
30.09.2019
Time
16:30 - 18:00

Abstract

Background

Recognizing the important policy issue of the value of cancer therapies, the European Society of Medical Oncology (ESMO) has developed a validated and reproducible tool to standardize the grading of the relative clinical benefit of new therapies: the magnitude of clinical benefit scale (ESMO-MCBS). The final reimbursement decision about the value of a therapy from a payer’s perspective includes both clinical benefits and costs of new therapies, and relative clinical benefit is assessed with country-specific methods. This study examines the potential of the ESMO-MCBS score to approximate the clinical benefit assessment in HTA decisions in the United Kingdom.

Methods

The ESMO-MCBS scoring for 102 advanced oncology drugs, listed in Cherny 2019 and corresponding final HTA documents by UK’s National Institute for Clinical Excellence (NICE) were reviewed and extracted. The relationship between NICE’s final decision (recommended or not; yes, but with restricted indication; with a discount; or only temporarily using the UK’s Cancer Drugs Fund (CDF)), and the relationship between ESMO-MCBS and the main components of the NICE clinical assessment were investigated descriptively and using regression analyses.

Results

76 (74%) of the 102 drugs included in Cherny 2019 were assessed by NICE; 76% of which were recommended by NICE (57% with a discount and 10% with CDF). A mean ESMO-MCBS score > 3.5 was observed for all the different types of positive NICE recommendation. Significantly lower mean ESMO-MCBS score (2.28, SE 0.26) was associated with treatments not recommended. All (100%) of the medications which were recommended, with some condition (discount/CDF) had an ESMO-MCBS ≥ 3 while most (83.3%) of the treatments not recommended were associated with an ESMO-MCBS ≤ 3. The analysis suggests ESMO-MCBS <3 as the optimal threshold to predict positive/negative NICE decision achieving a positive predictive value of 87% and negative predictive value of 71.4%.

Conclusions

An association was found between clinical value for the UK payer and that for physicians using ESMO-MCBS despite the different perspective. Scoring therapies with the ESMO-MCBS can have use beyond clinical guidelines in health policy.

Legal entity responsible for the study

The authors.

Funding

Evidera Inc.

Disclosure

All authors have declared no conflicts of interest.

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Proffered Paper - Public policy Proffered Paper session

1627O - Change in magnitude of clinical benefit, overall survival (OS) and quality of life (QoL) between time of approval and post-marketing among cancer drugs approved by the US Food and Drug Administration (FDA) 2006-2015 (ID 2243)

Presentation Number
1627O
Lecture Time
16:42 - 16:54
Speakers
  • Aida Bujosa Rodríguez (Barcelona, Spain)
Location
Salamanca Auditorium (Hall 3), Fira Gran Via, Barcelona, Spain
Date
30.09.2019
Time
16:30 - 18:00

Abstract

Background

Clinical trials supporting approval for new drugs often evaluate surrogate endpoints, and data on meaningful outcomes like OS or QoL may not be available. Here, we evaluated changes in the magnitude of clinical benefit, OS and QoL after approval.

Methods

We examined data on pivotal trials supporting FDA accelerated (AA) and regular (RA) cancer drug approvals between January 2006, and December 2015. For AA drugs, if conversion to RA was granted, only the confirmatory trial was analysed. To determine any new evidence on OS and QoL in the postmarketing period (PMP) we performed a systematic search of Pubmed and ClinicalTrials.gov. European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) grades were applied for trials at approval and in the PMP. Substantial clinical benefit was defined as a grade of A or B for trials of curative intent and 4 or 5 for those of palliative intent.

Results

We identified 96 pivotal trials supporting the approval of 47 drugs for 94 solid tumour indications. Of these indications, 22 (23%) were granted AA and 21 (22%) were converted to RA. At time of approval, 45 (48%) trials showed improved OS, 15 (16%) improved QoL and 33 (34%) had substantial clinical benefit. With a median PMP of 3.7 years, 50 (52%) trials reported OS data and 27 (28%) on QoL. Of these, 48 could be graded by the ESMO-MCBS. Of the updated 51 trials approved based on surrogate endpoints, only 7 (14%) showed an improvement in OS. In advanced disease, out of 74 trials for which there was no evidence on QoL at the time of approval, 12 (16%) showed improved QoL subsequently. Updated results led to changes in clinical benefit in 11 trials (10 upgrades, 1 downgrade) with 30 (62%) showing substantial clinical benefit. Among all trials, 52 (54%) showed improved OS, 27 (28%) improved QoL and 42 (44%) met the threshold for substantial clinical benefit.

Conclusions

After 3.7 years of post-marketing time, 54% of FDA approved cancer drugs showed statistically significant improvement in OS and 28% in QoL. Less than a half of trials supporting FDA approval met the threshold for substantial clinical benefit using ESMO-MCBS.

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

E. Amir: Speaker Bureau / Expert testimony: Genentech/Roche; Advisory / Consultancy: Apobiologix; Advisory / Consultancy: Agendia; Advisory / Consultancy: Myriad Genetics. All other authors have declared no conflicts of interest.

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Proffered Paper - Public policy Proffered Paper session

1628O - Consistency of ESMO-MCBS scores with drug access recommendations in Catalonia (ID 5379)

Presentation Number
1628O
Lecture Time
16:54 - 17:06
Speakers
  • Mercè Obach (Barcelona, Spain)
Location
Salamanca Auditorium (Hall 3), Fira Gran Via, Barcelona, Spain
Date
30.09.2019
Time
16:30 - 18:00

Abstract

Background

The Catalan Healthcare System (CatSalut) runs a specific program for drug evaluation (PHF) aimed to guarantee equity in the access to innovative medications. PHF recommendations determine the degree of priority and mechanism for drug invoicing: clinical criteria with direct invoicing; individualised criteria requiring approval by an ad-hoc committee, and exceptional use with no budget allocation. In 2017, the program incorporated an EVIDEM based matrix for Multiple Criteria Decision Analysis a standard procedure. In 2018, the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) was included in the matrix for oncological drugs. This study retrospectively checked the consistency of recommendations issued by the PHF for oncological drugs with the ESMO-MCBS score.

Methods

The recommendations of the PHF between 2011 and 2018 for oncological drugs were retrieved from reports and meeting minutes. Pivotal studies were scored using ESMO-MCBS version 1.1 for each drug. Matchings and discrepancies were summarised, and the debate reflected in the minutes of the committees was retrieved.

Results

Throughout the period 2011 to 2018 a total of 47 decisions were made on 57 oncology drugs evaluated. All PHF recommendations for drugs with ESMO-MCBS scores ≥ 4 were positive. Discrepancies were seen for 2 drugs in the curative setting (A score and exceptional use), 8 drugs in the non-curative (1-2 score and clinical criteria) and 1 in the curative setting (C and clinical criteria). The main criteria supporting the PHF decisions for these products are summarised in the table.

Curative
Trial qualityMifamurtide
Safety concernsIpilimumab
Managed agreementPembrolizumab
Non curative
Consolidated useBevacizumab Aflibercept
SubgroupsNintedanib Eribuline
Managed agreementEverolimus Cabazitaxel
NoneTrastuzumab Abiraterone

Conclusions

Factors explaining discrepancies between PHF recommendations and ESMO-MCBS were mostly related to the context of the appraisal. ESMO-MCBS standardises efficacy but does not consider quality of trials, added value to other drugs or other factors. It may not be a single formula to reach decisions, but may be useful to ensure that the size of effect is systematically approached in appraisals, and to complement a multiple criteria based system.

Legal entity responsible for the study

CatSalut.

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 1626O, 1627O and 1628O (ID 7129)

Lecture Time
17:06 - 17:21
Speakers
  • E.G. Elisabeth De Vries (Groningen, Netherlands)
Location
Salamanca Auditorium (Hall 3), Fira Gran Via, Barcelona, Spain
Date
30.09.2019
Time
16:30 - 18:00
Proffered Paper - Public policy Proffered Paper session

1629O_PR - The price of added value for new anti-cancer drugs in France 2004-17 (ID 5174)

Presentation Number
1629O_PR
Lecture Time
17:21 - 17:33
Speakers
  • Patricia Marino (Marseille, France)
Location
Salamanca Auditorium (Hall 3), Fira Gran Via, Barcelona, Spain
Date
30.09.2019
Time
16:30 - 18:00

Abstract

Background

The cost of new cancer medications is increasing prompting clinicians and policymakers to ask if newer therapies are worth their cost. We evaluated the relation between the price of new cancer drugs and their added therapeutic benefit measured by France’s High Authority of Health (HAS) and the European Society for Medical Oncology (ESMO).

Methods

We studied drugs to treat solid tumors registered by the European Medicines Agency (EMA) from 2004 to 2017. Prices were obtained from the French Official Journal. Rating for added therapeutic value were obtained from the HAS Added Therapeutic Benefit ranking (ASMR 1 being the highest benefit, and 5 the lowest) and the v1.1 ESMO-Magnitude of Clinical Benefit Scale (MCBS, 5 being the highest and 2 being the lowest). We calculated monthly treatment costs for each new drug compared with existing treatment and the correlations between price increase and added therapeutic value using Spearman’s tests.

Results

Thirty-six drugs were approved for 68 indications (with 35 first registration). The median ESMO-MCBS and ASMR scores were 4 (range 1-5) and 4 (range 2-5), respectively. Forty-eight percent and 70% of drugs had low added value according to ESMO-MCBS and ASMR, respectively. The mean monthly price for new drugs and comparators were 4,616 and 2,314 euros, respectively and increased during the observation period. For all indications there were significant but weak correlations between ESMO-MCBS and ASMR (|rho| = 0.28, p = 0.019), ESMO-MCBS and price (|rho| = 0.33, p = 0.005) and between ASMR and price (|rho| = 0.35, p = 0.004). Correlation with price was higher when considering the first indication (ESMO-MCBS, |rho| = 0.48, p = 0.004; ASMR, |rho| = 0.37, p = 0.030). There was no correlation between price increases and ASMR or ESMO (|rho|<0.2, p > 0.1).

Conclusions

Most new drugs provided low added value. On average, new drug prices increased 2,525 euros over their comparator. Prices were weakly correlated with added value, but price increases were not.

Legal entity responsible for the study

The authors.

Funding

IMéRA Institute.

Disclosure

A. Gonçalves: Travel / Accommodation / Expenses: Novartis; Travel / Accommodation / Expenses: Roche; Travel / Accommodation / Expenses: Amgen Pfizer; Travel / Accommodation / Expenses: AstraZeneca; Travel / Accommodation / Expenses: Celgene. All other authors have declared no conflicts of interest.

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Proffered Paper - Public policy Proffered Paper session

1630O - Reimbursement reality for off-label use in cancer care: A systematic empirical investigation (ID 3815)

Presentation Number
1630O
Lecture Time
17:33 - 17:45
Speakers
  • Amanda K. Herbrand (Basel, Switzerland)
Location
Salamanca Auditorium (Hall 3), Fira Gran Via, Barcelona, Spain
Date
30.09.2019
Time
16:30 - 18:00

Abstract

Background

In situations of limited treatment options, off-label use (OLU) may be the most promising therapy for cancer patients. However, OLU is regulated by reimbursement restrictions. Little is known about the prevalence of OLU and factors that drive reimbursement decisions. We investigate the reimbursement reality of OLU in patients with solid or hematological malignancies in the Swiss healthcare system, which is characterized by a high diversity of jurisdictions and statutory health insurers.

Methods

We conduct an ongoing cross-sectional study using routinely collected health data. All patients with cancer who received drug treatment at three major hospitals in Switzerland between 01/2015 and 07/2018 are screened for OLU. For patients with at least one reimbursement request, we extract demographics, disease and treatment characteristics, and correspondence with the health insurer. We define OLU as intentional drug use outside of the Swissmedic (Swiss Agency for Therapeutic Products) approval label at the time of request. We use descriptive statistics to describe the frequency and characteristics of OLU requests and multivariable logistic regression to assess the association of pre-specified patient characteristics and the reimbursement decisions.

Results

So far, we screened medical records of 1561 eligible patients. For 276/1561 patients (18%), at least one reimbursement request for OLU was issued (319 requests in total, 1 - 3 per patient). The most frequent indications were adjuvant zoledronic acid in breast cancer (27; 8%), adjuvant nivolumab in melanoma (11; 3%) and atezolizumab in advanced urothelial cancer (10; 3%). Health insurers rejected the request in 93/319 cases (29%). Preliminary analyses showed no association of patient characteristics and reimbursement decisions (e.g. odds for disapproval for solid vs hematological malignancies, OR 1.43, 95% CI 0.70 to 2.91, p = 0.32).

Conclusions

Preliminary results indicate that access to cancer care with OLU in Switzerland is characterized by substantial inequity and lack of transparency of the underlying decision-making process. Further results and details on the relationship of reimbursement decisions and the underlying clinical evidence for OLU will be presented at the meeting.

Legal entity responsible for the study

The authors.

Funding

Swiss Cancer League.

Disclosure

All authors have declared no conflicts of interest.

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Proffered Paper - Public policy Proffered Paper session

Invited Discussant 1629O_PR and 1630O (ID 7130)

Lecture Time
17:45 - 18:00
Speakers
  • Bengt Jönsson (Stockholm, Sweden)
Location
Salamanca Auditorium (Hall 3), Fira Gran Via, Barcelona, Spain
Date
30.09.2019
Time
16:30 - 18:00