World Cancer Day – How Can We Close the Care Gap When it Comes to Patient Access to Innovation? (Guest blog)
03.02.22
Each year, World Cancer Day is recognised on the 4th of February, providing an opportunity for the oncology community to come together to raise awareness and drive dialogue around the most pressing patient needs, and how we might work together to address them. While cancer is still the second leading cause of death worldwide, after cardiovascular disease,[i] we are also experiencing a time of great progress and hope for patients. Cutting-edge innovations in diagnostics, surgery and therapeutics are improving outcomes,[ii] and scientific advances continue to offer the potential to change what a cancer diagnosis means for patients. But to be truly transformative, these new therapeutic approaches must get to the patients who need them, when they need them, and that’s sadly often not the case.
The EFPIA Patients Waiting to Access Innovative Therapies (Patients W.A.I.T.) indicator highlighted the variations that exist between 34 European countries, both in terms of the range of new medicines available to patients and the delays in their availability. Overall, patients in Europe wait more than 18 months, on average, to access new cancer treatments following regulatory approval.[iii] Access varies greatly based on where patients live, with patients in Northern and Western Europe tending to gain access faster than those in Southern and Eastern Europe.[iv] In Germany, for example, the average time between marketing authorisation and access to new cancer treatments is 121 days (around four months) whilst in Estonia, the wait time is more than 1015 days (nearly three years).iii For patients with cancer, time is life, and many simply don’t have years or even months to wait for new treatment options. This is why the theme of this year’s World Cancer Day – closing the care gap - is incredibly relevant to one of the most urgent unmet patient needs of our time.
One of the key barriers to timely and equitable patient access to innovative cancer medicines relates to the value assessment framework – the way in which national health technology assessment (HTA) agencies evaluate the relative effectiveness and cost effectiveness of new medicines or other health technologies.[v] These agencies continue to rely heavily on mature overall survival data to secure favourable reimbursement outcomes.v However, as cancer outcomes continue to improve, thanks in large part to the efficacy of new therapies, demonstrating a significant difference in overall survival within the timeframe of a clinical trial is becoming increasingly unrealistic in some tumour types and in earlier lines of treatment.ii,v The evidence available at time of HTA appraisal can be perceived as immature and uncertain, which has negative ramifications for decision-making and often leads to lengthy negotiations, restrictions, rejections, or deferred decisions until more evidence is available.iv,v
The causes of delays in access to innovation include factors other than the value assessment framework too, and we know the present challenges will not be solved alone. As EFPIA have previously explained “Delays in access and uneven availability of medicines harm patients. We know that the root causes of these issues are multiple and multi-factorial, and in addition to value assessment, they include late start of market access assessment, duplicative evidence requirements, and national pricing and reimbursement policies. We support an evidence based dialogue across all stakeholders to find collaborative solutions.”iv
When it comes to solutions, I wholly agree that collaboration and flexibility are essential. Not all diseases and cancer therapies are the same and hence they should not be evaluated in the same way by HTA agencies and payers. We need to work together, HTA agencies, clinicians, patient groups and industry combined, to establish frameworks with clear guidance on when greater flexibility is warranted to ensure appropriate value recognition of innovative medicines.
A move towards more flexible, outcomes-based approaches will be pivotal in driving healthcare that is both sustainable and value-based. This will require a more holistic approach to medicines evaluation – one that considers a broader assessment of value that includes the wider benefits to patients, carers and society.
Collectively, we must also move away from static decision-making based on data observed at a single point in time. Instead, we should be looking towards alternative endpoints and iterative evidence generation that allows for the demonstration of value over time. Solutions like managed entry agreements and ongoing collection of real world data could enable us to make timely decisions with a commitment to provide more evidence to as it becomes available to demonstrate value to payers. A more flexible and pragmatic HTA process would help to limit reimbursement delays or restrictions for efficacious treatments.
While there has been recognition of the importance of the role of patients in medicines evaluations, there is still much work to be done for this involvement to be more meaningful, transparent, and systematically incorporated into HTA processes and decision-making. Integrating patient-relevant endpoints and improving the quality of real-world data from patient registries are routes towards elevating the patient voice in HTA processes. As part of this, stakeholders also need to consider how to make patient involvement in HTA processes more accessible. A great example of positive progress in accessibility is the HTAi International Summary of Information for Patients (SIP) - a key enabler to manufacturers being able to share relevant information with patients and patient groups, written using plain language that will be easily understood. The requirement to provide information in this way should help overcome any divergence in health literacy levels between different stakeholders – ensuring a common level of understanding of the disease and treatments, so all stakeholders can confidently and fully engage in important discussions.
In a world where innovations are taking us closer and closer to curing cancer every day, we cannot afford to keep patients waiting for the opportunity to access life-saving treatments. Closing this gap is a challenge that requires urgent and ongoing collaboration, and the co-creation of solutions that embrace innovation and place the patient at their core.
[i] Our World in data. Number of deaths by cause, World, 2019. Available at: https://ourworldindata.org/grapher/annual-number-of-deaths-by-cause?country=~OWID_WRL. Accessed January 2022.
[ii] Lynne W. et al. Blueprint for Cancer Research: Critical Gaps and Opportunities. Ca Cancer J Clin 2021; 71:107–139. Available at: https://acsjournals.onlinelibrary.wiley.com/doi/epdf/10.3322/caac.21652. Accessed January 2022.
[iii] EFPIA Patients W.A.I.T. Indicator 2020 Survey. April 2021. Available at: https://www.efpia.eu/media/602652/efpia-patient-wait-indicator-final-250521.pdf. Accessed January 2022.
[iv] CRA. EFPIA The Root Causes of Unavailability and Delay to Innovative Medicines. Available at: https://www.efpia.eu/media/602653/root-cause-unavailability-delays-cra-report-may-2021-final.pdf. Accessed January 2022.
[v] Every Day Counts – Improving Time to Patient Access to Innovative Oncology Therapies in Europe. Available at: https://www.efpia.eu/media/578013/every-day-counts.pdf. Accessed January 2022.
Date of preparation: January 2022
Job code: CP-290147
