Time to reimbursement of novel anticancer drugs in Europe - a recent Case Study in DE, FR, UK, NL, NO, CH and BE

• A number of new #anticancer medicines are introduced each year and are subject to face delays and inequalities in patient access impacting the quality of life and life expectancy.
• Disparities exist in the time it takes for #reimbursement decisions at the national level after European Medicines Agency (#EMA) approval.
• In the European Union (#EU) and certain European Economic Area countries, access to medication involves centralized authorization decisions by the Committee for Medicinal Products for Human Use (CHMP) and subsequent approval by the European Commission .
• The study aims to evaluate the Time to Reimbursement (#TTR) for 35 novel #anticancer #medicines in seven high-income European countries (DE, FR, UK, NL, NO, CH, BE), examining factors such as medication-related aspects, country-specific factors, disease-related factors, and #pharmaceutical company-related factors that could influence the #reimbursement process.
• The selection of medicines involved #EMA approved new #anticancer medicines between January 2016 and January 2021.

"Time from approval to reimbursement for new anticancer medicines is long, even in most high-income European countries." - H. C. Post et. al., ESMO

This Newsletter is a summary of the research paper published by H. C. Post et. al. in ESMO Journal in April 2023 (for more detail, refer to the disclaimer section).        

The Case Study

The study employed a retrospective case study designed to investigate the TTR in the given countries (Germany DE, France FR, United Kingdom UK, Netherlands NL, Norway NO, Switzerland CH and Belgium BE), following EMA #Marketing Authorization (EU-MA). TTR is defined as the duration in days between EU-MA and the registered/announced date of #reimbursement, with a difference of >30 days considered #clinically relevant. Data collection involved using Health Technology Assessment (#HTA) and reimbursement websites for each #country to gather decision #information and dates related to inclusion in positive lists, #HTA recommendations, and #pricing and #reimbursement commissioning decisions.

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Descriptive statistics, including median, range, and interquartile range (IQR), were used to describe #TTR in different countries and sub-groups based on factors such as #ESMO-MCBS score, EMA-AA status, GDP, orphan status, conditional marketing authorization (#CMA), company size, and pre-assessment procedure.

The study describes the factors investigated which could influence the #TTR of new #anticancer #medicines in these countries. Medication-related factors included the level of evidence based on the #European #Society #Medical Oncology-Magnitude of #Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1), which assesses the relative benefit of anticancer #medicines using criteria such as overall survival, progression-free survival, response rate, #toxicity, and quality of life. High benefit was defined as a score of A-B or >3, while low benefit included other scores. The study also examined the level of innovation/therapeutic importance and compared TTR for medicines with Accelerated #Approval (AA) to those with regular assessment procedures.

Country-related factors considered were the presence or absence of a pre-assessment procedure and the Gross Domestic Product (#GDP). Indication-related factors included #orphan status, the presence of an unmet #medical need, and conditional marketing authorization for medicines addressing unmet medical needs. Rare #cancers were defined as those with an incidence of fewer than 5 in 100,000 persons per year. The size of the #pharmaceutical #company was based on the Big-12 pharmaceutical companies of the #world.

? Related read: PRIME: The PRIority MEdicines; an initiative from the European Medicine Agency

Out of 35, 16 medicines (46%) were reimbursed in all seven countries. The median TTR for these medicines was 234 days, meeting the EU Transparency Directive's 180-day limit in Germany (100%), France (51%), the UK and the Netherlands (29% each), Switzerland (14%), Norway (6%), and Belgium (3%).

The #TTR varied among countries, with Germany having the shortest median TTR (3 days). The pooled median TTR for all drugs in all countries was 407 days. Statistically significant differences existed between countries, with Germany differing significantly from all others, and France, the UK, and the Netherlands differing from Switzerland, Belgium, and Norway.

Medication-related factors, including #ESMO-MCBS score and EMA-AA status, showed no significant impact on TTR. Country-related factors, such as the absence of a pre-assessment procedure, significantly affected TTR, with Germany having unique regulations. A positive correlation between #GDP and TTR was found, indicating shorter TTR with higher GDP. Orphan status significantly increased #TTR, while conditional marketing authorization (CMA) had no significant impact. #Medicines from Big-12 #pharmaceutical #companies had shorter TTR than those from smaller #companies.

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Conclusions

1. The study highlights that the #time to #reimbursement (TTR) for innovative anticancer medicines is often prolonged and variable, leading to unequal access, even among high-income countries. Addressing this requires improvements in #anticancer #drug development and #healthcare system efficiency.
2. Findings indicate that TTR is generally too long, except in Germany, and varies significantly between countries. Factors contributing to shorter TTR include the absence of a pre-assessment procedure, higher GDP, and submission by Big-12 pharmaceutical companies.
3. The study observed no clinically relevant correlation between TTR and the level of clinical benefit (ESMO-MCBS v1.1). Country-related factors, such as Germany's unique system, significantly impacted TTR. A positive correlation between GDP and TTR was found, potentially linked to larger #markets and higher #HTA capacities in wealthier countries. #Orphan status and conditional marketing authorization (#CMA) were associated with slower #reimbursement, possibly due to pricing #negotiations and uncertain long-term #benefits.
4. Medicines from Big-12 pharmaceutical companies had a shorter TTR, possibly attributed to resource commitment and better infrastructure. Multivariate analysis identified pharmaceutical company size, country, and absence of a pre-assessment procedure as significant factors influencing TTR. Recommendations include international #collaboration for joint assessments and criteria development for #evidence and #reimbursement.
5. The study suggests that harmonizing health technology assessment (#HTA), #pricing and #reimbursement (P&R) negotiations, and national reimbursement authority (NRA) systems could be key measures to reduce TTR, especially among smaller countries with lower GDP. A united #price model promoting accessibility and innovation is also suggested, emphasizing the need for parallelized processes in #regulatory approval and #reimbursement.

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Avisek Ghose is a healthcare market researcher, driven by marketing passion. A seasoned consultant and trained bio- imaging specialist who shares his insights and outlook about recent trends, challenges and opportunities about early market access strategies, HEOR and HTA issues in Medtech and Pharma industry.

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Disclaimer:

• This #Newsletter is a summary of the #research paper titled "Time to reimbursement of novel anticancer drugs in Europe: a case study of seven European countries" published by H. C. Post et. al. in ESMO Journal, Vol. 8, Issue. 2 Apr. 2023 DOI: 10.1016/j.esmoop.2023.101208 on 06 Apr. 2023.
• Views expressed here is mirroring the aforesaid (open access) #research paper.
• For more details, readers must refer to the official publication available on the official webpage of #ESMO Journal or click here.

?? The author took extreme care for citing credits wherever applicable, however, in case of missing citation(s) and/or any other discrepancies, please inform Avisek Ghose immediately indicating the same with valid documentation(s).

ESMO - European Society for Medical Oncology #anticancermedicines, #drugaccess, #inequality, #regulatoryapproval #patientaccess #CHMP #casestudy #marketauthorization #healthtechnologyassessment #clinicalneed #timetoreimbursement #oncology #drugdevelopment #peicingstructure #drugpricing #HEOR #healtheconomicsoutcomeresearch #marketaccess #pharmaindustry #QALY