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All cancer patients deserve the best treatment. But what is the best treatment?

All cancer patients deserve the best treatment. But what is the best treatment?

Cancer patients want to have access to the best available treatment and they want it fast, as in many cases they expect it to be lifesaving.

Unfortunately, the current system to bring a new drug all the way to the patient is not fit for its purpose. Making cancer treatments available is a process that’s broken down into two levels: first, a supranational procedure with European Medicines Agency (EMA) for obtaining Market Authorization (MA), which focusses on a safety and efficacy assessment of the drug and second, a national Health Technology Assessment (HTA), which focusses on added therapeutic value and reimbursement.

Consequently, regulators (both national and European), HTA bodies and payers (be it governments or insurance companies) differ in their recommendations regarding all aspects of clinical trials. Needless to say, this complex web of stakeholders and watchdogs make for an immensely time and money consuming process.

What is he "best" treatment?

So what is the “best” available treatment then? You will get a different answer depending on who you’re asking in the cure-to-cancer chain. Does it imply longer survival or a better quality of life? A guaranteed chance of getting a little better or a small chance of getting a lot better, no matter the cost? Given the fact that a magical unicorn-esque cure for cancer has still not been found, the “best” treatment boils down to a treatment with a better overall survival (OS) and/or better quality-of-life, properly measured by “Patient Reported Outcome” (PRO), as compared to various relevant comparator(s).

For roughly half of the innovative cancer drugs offered to patients in this century, those data are either not available or the expectations were not met after follow-up. Nevertheless, these (mostly expensive) drugs are here to stay. Why? Because - from a regulation perspective - there is no single body authorized to consider all these factors for its approval. It is thus simply not within EMA’s power to approve the “best” treatment on the basis of our above standards.

Therefore, if the EU wants to live up to the “Europe’s Beating Cancer plan” – which stipulates “access to the best treatment for all” as one of its objectives – then the European Commission of Health will have to adjust the entire assessment process as we know it today.

In principle, it is feasible for EMA to expand its evidentiary requirements in the first phase in such a way that these already include the evidence needs of the HTAs for the second phase.

Is there a well-defined unmet need?

Robust endpoints like OS require long trials while end-of-treatment patients demand early access, especially to potential breakthrough therapies. In that context surrogate endpoints could be accepted to allow fast access if there is a well-defined and high unmet need, BUT this should always be linked to conditional approval and post-authorization effectiveness data collection (through confirmatory trials).

This fast access process should also imply that if those post-authorization data lack to demonstrate clinical benefit the drug has to be phased-out and license should be withdrawn.

To provide the best possible treatment to patients, European cooperation of HTAs and joint clinical assessments is an absolute necessity. Early dialogue between EMA and HTA organisations should lead to confirmatory trials with robust and meaningful clinical endpoints and comparators.

Confirmatory trials are key

In an ideal world, confirmatory trials should be designed by independent trialists in a patient-centred and efficient way, enabling reliable and timely access to evidence based treatment options. In our view, such an innovative approach could already be implemented for rare cancers (including paediatric cancers).

Platform trials managed by collaborative groups could be the preferred format since they will allow direct and efficient comparison of different treatment options. The infrastructure for these platform trials should be supported by public funds and each treatment arm could be financed by either a commercial or non-commercial applicant. This format is currently being explored in the assessment of COVID treatments on the different continents and has proven its value.

Furthermore, continuous proper registration of treatment outcomes of patients in actual clinical practice should be enforced by European legislation, as these data are crucial for cost-effectiveness calculations. Today treating physicians are not motivated to participate in post-approval studies or registries since it is time consuming and lacks reward. Therefore a compromise could be found in allowing them to prescribe innovative drugs under the condition that outcome data is tracked. Today registries are mostly on a voluntary base and quality and granularity is variable. If we want those crucial data to be fit for purpose, it will require public funds and also EMA support in the form of Scientific Advice.

So Europe, take ownership, if you really want to give the best treatment to the patient.

Lydie Meheus
Lydie Meheus

Lydie Meheus is the Managing Director of the Anticancer Fund. Lydie holds a PhD in Biochemistry.