Should we revise our definition of “patient access”? (Guest blog)
04.05.22
Access to medicines is probably one of the most discussed topics in health policy today, in Europe and elsewhere. It is also the main rationale for the forthcoming review of the European Union’s entire pharmaceutical legislative acquis. In its legislative proposals to come later this year, the European Commission explicitly envisages using its regulatory arsenal to achieve more harmonized access to innovative treatments for all European citizens, no matter where in Europe they live.
The European Commission’s legislative agenda also follows a flurry of forums, partnerships and initiatives all driven by the need to improve access to innovative medicines across EU countries. All these initiatives focus on the price of new treatments, linking closely the issue of access to medicines with the issue of affordability. This plethora of forums include well-established platforms such as EURIPID but also newly launched initiatives, such as the International Horizon Scanning Initiative (an offspring of the Beneluxa collaboration), the European Fair Pricing Network (focused on cancer treatments), and the Oslo Medicines Initiative.
More than a decade ago, EFPIA first addressed the issue of access equity in Europe with the launch of the first Patients W.A.I.T. Indicator. Since then, the Patients W.A.I.T. Indicator has consistently shown significant discrepancies among European patients regarding the time that it takes between a medicine being licensed by the European Commission and its availability to patients across the 27 EU countries.
Despite the EU’s deep and centralized regulatory approval mechanism for medicines, and a single market with free movement throughout from which we all draw the economic benefits, a patient in Portugal can still have to wait four times longer than a patient in Denmark to benefit from a new treatment even if they both suffer from the same disease.
Equally, not all medicines approved by the Commission are available across the EU27: patients in Italy can only access seven out of ten medicines approved by the Commission while in Romania it is less than two out of ten.[1]
The accepted wisdom is that access is conditioned by affordability, which itself is determined by the price charged by pharmaceutical companies for new treatments. But is price the fundamental issue?
The price of a medicine results from a different process from licensing decisions. It is the result (usually) of a HTA assessment, a comparison with other similar treatments available across different countries in Europe and, finally, negotiation between a company and a payer or a government. In Europe no company unilaterally determines the price which is paid. Indeed, as an industry, we have a strong interest in payers’ agreeing that the price we receive is fair.
I have worked on ensuring market access for new medicines and negotiated with national payers in Europe to find access solutions for more than 20 years.
First, I can attest that both sides want the negotiation to reach a successful outcome under fair terms. Both the company and the payer want to make the product accessible to patients.
Second, despite significant legal and practical constraints attached to healthcare administration and processes, we drive to find and co-create innovative solutions to overcome the challenges of affordability. Yes, creative solutions will be needed, sometimes specific access agreements, especially if there is uncertainty about the evidence of value, or the number of patients treated. Also, a transparent discussion between companies and payers will be needed.[2]
But in many cases we have found solutions which truly accelerated patient access at a fair price.
And that is my commitment as a market access professional.
Some examples are in order: patient access in Belgium and the Netherlands came four times faster[3] thanks to the adoption of multi-year-multi-indication (MYMI) agreements, which allow the structured introduction of new indications of new cancer medicines over time. In the UK, similarly, the Cancer Drug Fund has worked with companies to facilitate and accelerate patient access through new schemes such as “coverage with evidence development.”
In both these cases, the critical success factor was the ability of both sides, companies and payors, to engage into a meaningful dialogue in order to find creative ways to overcome previous constraints. Price might always be a factor (as of course it should as public money is involved) but these innovative procurement solutions show that it’s only one among many factors. Affordability has more dimensions than just price.
Third, and this is the point raised in the title, patient access doesn’t naturally flow from the successful conclusion of a price and reimbursement negotiation. The healthcare consultancy Vintura with the Swedish Institute of Health Economics, illustrated that this was too narrow a criteria.[4] In the cascade of decisions and processes that result in a patient receiving treatment, how long it takes for a drug to be reimbursed does not necessarily reflect how fast patients have access: Germany will reimburse a new treatment five times faster than France.[5] Can we assume that patients in Germany benefit from faster access? In reality, they don’t: studies show that 12 months after reimbursement only half of eligible patients will be treated with a new cancer therapy in Germany; while in France, it is almost eight out of ten patients eligible who receive the new treatment.[6] We can observe a similar situation in various European countries, where reimbursement doesn’t translate into access.
CONCLUSION
Patient access to new treatments is my personal goal. But it will always be joint effort.
So what is the way forward?
First, we need to be more creative regarding pricing and reimbursement. Every day, I see that the issue of “affordability” is not just about the unit price but also depends on how drugs are procured and how the drugs budget is organized. Arguably, it is time that we match therapeutic innovation with innovation in budget design and procurement. In too many countries, we will not solve the affordability problem if we don’t adapt national pricing and reimbursement processes to medical progress.
Second, we are only successful if patients are treated effectively. This is the definition of patient access under which our health systems should be measured.
Coming back to the initial question: we ultimately need a much broader concept of access. Improving the lives of patients – our main goal – can only be achieved when patients are being treated with the treatment they need. Against the backdrop of the broader concept of access, patient access is truly a shared responsibility.
[1] EFPIA (2021), EFPIA Patients W.A.I.T Indicator 2020 Survey; https://www.efpia.eu/media/602652/efpia-patient-wait-indicator-final-250521.pdf
[2] Lawlor R et al. (2021), Accelerating patient access to oncology medicines with multiple indications in Europe; J Market Access & Health Policy 9;1
[3] Lawlor R et al. (2021), Accelerating patient access to oncology medicines with multiple indications in Europe; J Market Access & Health Policy 9;1
[4] Jansen C, Amesz B (2020), Every Day Counts. Improving Time to Patient Access to Innovative Oncology Therapies in Europe; https://www.vintura.com/news/white-paper-every-day-counts/
[5] Jansen C, Amesz B (2020), Every Day Counts. Op. cit.
[6] Jansen C, Amesz B (2020), Every Day Counts. Op. cit.
