In July 2016, the new Cancer Drugs Fund was launched. Since then, each year, Remap Consulting has been conducting an analysis of oncology appraisals conducted in England. This year, we focused our analysis on comparing market access for oncology and non-oncology drugs. Specifically, our research compared the outcomes of draft and final NICE outcomes and in the case of positive outcomes, the use of patient population restrictions and financial agreements for oncology and non-oncology drugs.
Extremely high-cost CAR-T immunotherapies, specifically Yescarta (axicabtagene ciloleucel) and Kymriah (tisagenlecleucel), are becoming more widely available worldwide despite the limited clinical data available at launch.
This study investigates whether the evidence for Kymriah and Yescarta for the diffuse large B-cell lymphoma (DLBCL) indication was sufficient to secure a positive outcome from HTA bodies in Europe and the USA, and how gaps in the current evidence are linked to access restrictions and mitigation strategies.
In England, the National Institute of Health and Care Excellence (NICE) uses the cost of treatment per quality-adjusted life-year as the basis for its’ decision making. In general, technologies will only receive a positive NICE recommendation if their estimated cost per QALY is below the 20 000 to 30 000 incremental cost-effectiveness ratio threshold. In order to demonstrate cost-effectiveness, manufacturers must often agree financial agreements (such as confidential discounts) with NHS England. In this study, we compared the use of financial agreements for oncology and non-oncology drugs assessed by NICE between 2017 and 2019
The cost of innovative drugs for rare diseases has continually pushed the upper pricing thresholds of payers in recent years.
Recent drug launches have included acute use gene-therapies and life-saving treatments, which promise a ‘cure’ or at a minimum, a life-altering impact.
Previous publications have assessed annual costs only and group together both gene/acute therapies and chronic treatments which may not provide a complete picture and places a focus on acute treatments being ‘the most expensive drugs in the World’.
This study investigates costs of gene/acute therapies versus chronic treatments for rare diseases, on an annual and lifetime basis to provide a more balanced comparison.
Pharmaceutical companies are incorporating payer/HTA needs into their clinical and commercial launch planning. This is becoming increasingly relevant as growing healthcare expenditure means payers are more stringent regarding the evidence needed to achieve patient access at a price that is acceptable to both the pharmaceutical company and the payer. A lack of early pricing and market access input can lead to clinical and economic evidence challenges during HTA submissions.
This study investigates the industry perspective on drivers and challenges for a successful launch from a pricing and market access perspective, and the areas that can be improved in future.
Pharmaceutical pricing and market access is the critical challenge for successful product launch today. Amgen identified that country affiliates and global teams required pricing and market access training to help them make better business decisions. Amgen partnered with Remap Consulting due to their superior combination of pricing and market access training and instructional design capabilities. In partnership, Amgen and Remap Consulting designed and developed a bespoke eLearning training programme detailing the country pricing and market access challenges in order to improve commercial outcomes.
In April 2016, NICE modified the Cancer Drugs Fund (CDF) appraisal process, to review all new oncology indications and publish final guidance within 90 days of marketing authorisation. Additionally, 14 existing legacy treatments from the previous CDF had not undergone a NICE assessment and required review.
Treatments appraised under this process may be recommended for routine use by NHS England; retained in the CDF to gather additional evidence or not recommended. This study investigates the efficiency of the new CDF appraisal process for indications without prior NICE evaluations.
Since July 2016, NICE took over responsibility for the Cancer Drugs Fund (CDF) in England, which contained 25 drugs for 33 indications. Prior to 2016, the CDF in England provided funding for previously rejected oncology treatments and for those not yet assessed.
NICE is currently evaluating all existing treatments in the CDF to assess if they should be recommended for routine use by NHS England; retained in the CDF to gather additional evidence or not recommended.
This study investigates NICE’s evaluation of existing CDF treatments and its potential impact on funding and patient access.
Bemfola, an EMA approved biosimilar of Gonal-f, is a recombinant folliclestimulating hormone (rFSH) used for in-vitro fertilisation (IVF). Having demonstrated equivalent efficacy and safety to Gonal-f, Bemfola is approved for identical indications as Gonal-f and as such, is also a viable alternative to urinary derived FSH, Menopur.
NICE guidance (CG-156) concludes that there is no difference in clinical effectiveness between urinary and recombinant FSH products, and therefore recommends the use of either type of product. However, available products do differ in terms of their delivery device and available strengths; Gonal-f is provided as a multidose pen, Menopur in vials or multidose preparation, and Bemfola as a fixed dose pen.
The aim of this study was to determine the impact of delivery device on drug wastage and associated cost.
Approximately one in four prescription-only medicinal products are paid out of pocket by EU patients, whereas the rest is funded by the healthcare system or compulsory health insurances via reimbursement procedures. For reimbursed products, the price setting process for new pharmaceutical products are clearly defined across EU markets with information being readily accessible.
There is a lack of published information concerning the pricing processes for non-reimbursed prescription-only medicinal products, therefore, the objective of this research was to determine the national price submission procedures across Europe required for launch and patient access for non-reimbursed prescription-only medicinal products.
Pharmaceutical companies are increasingly seeking payer guidance to provide insights on how to build payer value into the clinical trial program. There are multiple ways to seek payer guidance throughout clinical development, either at an EU-wide or a country-specific level.
The objective of this research was to understand the processes and outputs from different European payer scientific advice procedures (PSAPs) to determine the most suitable approach for pharmaceutical companies.
There have been several recent pharma product launches for the treatment of metastatic castration resistant prostate cancer (mCRPC). The aim of this study was to assess the launch price of mCRPC treatments across the EU5 (France, Germany, Italy, Spain, UK) in relation to a standard clinical measure, overall survival (OS), to determine if payers equally value the cost per OS month for mCRPC treatments. Furthermore, the cost per month of additional OS benefit versus comparator was assessed. An analysis of cost/survival for prostate cancer treatments has been previously conducted in the US.
The introduction of biosimilars, as lower cost versions of branded biologics had the potential to generate cost savings over the coming years. The purpose of this study was to determine if EU pricing and reimbursement bodies have revised their pricing and reimbursement approval processes for biosimilar medicines to enable faster access and optimise the potential healthcare savings.
Since 29 July 2016, the NICE appraisal process for oncology indications in England has been modified to allow one of three outcomes; recommended for routine use, not recommended, or recommended within the Cancer Drugs Fund (CDF) managed access scheme. This study investigates the outcomes of NICE appraisals for new cancer drugs assessed via the modified process.
Setting the price and obtaining market access for a new pharmaceutical remains one of the biggest commercial challenges facing manufacturers. As the payer landscape becomes increasingly challenging, demonstrating product value and considering the payer perspective as early as possible in clinical development is increasingly important.
Since 29 July 2016, the National Institute for Health and Care Excellence (NICE) has employed a new fast-track appraisal process for oncology drugs. The aim of this process is to provide patients earlier access to new, more effective medicines. This study investigates the extent to which these targets have been met for cancer drugs receiving market access post-July 2016, in order to determine whether NICE’s new process is translating into faster patient access in practice.
Biosimilars are becoming more widely available across Europe, with many payers at each level involved in decision-making regarding their market access. Despite recognition of the benefits of biosimilars (reducing cost of treatment thus freeing up resources to treat more patients, maximising health care outcomes and enabling cost savings to support new innovation) decision-making and access processes are still finding their place within each country’s healthcare system. This study investigates the local market access needs of European countries in relation to biosimilars, in order to identify and understand the key criteria which currently drive payer decision making
Within the EU, there are multiple HTA scientific advice options pharmaceutical companies can take to understand payers’ clinical and economic HTA evidence requirements. The aim of this study is to compare the key aspects companies must consider when seeking HTA scientific advice within France, Germany, Italy and the UK.