The EU is launching a new strategy to improve and accelerate patients' access to safe and affordable medicines and to support innovation in the European pharmaceutical industry. The European Commission gave European citizens, organisations and businesses the opportunity to reflect on the roadmap it has defined. The Anticancer Fund gave feedback and sent its recommendations.

Clinical evidence

The Roadmap states that ‘new therapies need to be clinically better than existing alternatives’. We emphasise the importance of defining ‘clinically better’ and suggest that robust methodologies for obtaining evidence on efficacy and effectiveness of new treatments are of critical importance. In particular, we promote:

• Adequately designed trials (preferably multi-arm) allowing independent comparison between different treatments, using the correct comparator.
• Early involvement of Health Technology Assessment (HTA) bodies to ensure trials provide the necessary data for timely HTA.
• Use of consistent definition of patient-centred endpoints and inclusion of Patient Reported Outcome Measures (PROMs) in clinical trials.
• Fast-tracked conditional approval in the case of unmet need (this is important in oncology as timely treatments may potentially be life-saving).
• In oncology conditional approval based on surrogate endpoints can be acceptable in the case of unmet need, but post-authorisation studies or registry-based trials are an absolute requirement to prove effectiveness and value for patients and society.
• We emphasise the importance of evaluating clinical effectiveness (in oncology a drug is often only part of combined treatment administered to patients, and is rarely given without any other therapy and as such its clinical value in a real-world setting must be demonstrated). While safety and efficacy is an important first step in the evaluation process, it is not sufficient to demonstrate added clinical value for patients and society.

New therapies based on unmet need and aligned with public health requirements

The Roadmap cites the need for a ‘holistic, patient-centred, forward-looking EU Pharmaceutical Strategy’, we highlight:

• The importance of prioritising new therapies that serve unmet needs versus ‘me-too’ products.
• ‘Innovation’ should be defined as ‘new approaches to solve a challenge’ and should not be a synonym for high-tech solutions or new products. Finding new uses for ‘old’ drugs should also be considered innovative.

Health system preparedness

The Roadmap states that ‘health systems may need to be better equipped to ensure deployment and uptake of innovative solutions’.

We believe that health systems should independently ensure deployment and uptake of solutions with proven benefit for patients and society, with emphasis on ‘proven benefit’ rather than innovation.

Affordability of medicines

An overarching goal of the Pharmaceutical Strategy is to ensure Europe’s supply of safe and affordable medicines.

There are currently 280 generic drugs that are expected to be beneficial for cancer patients. These drugs have proven safety profiles and may be repurposed for use in new indications. Also in other disease areas, there are numerous repurposed drugs with exploratory evidence and especially for rare diseases these drugs could be a unique and accessible source of treatment options. Drug repurposing offers safe and affordable solutions, yet its potential remains untapped due to lack of funding, incentives and infrastructure. We recommend that these barriers are addressed urgently.

EU cooperation on Health Technology Assessment

A legislative proposal for strengthening EU cooperation on HTA is on the table and our comments are the following:

• Common clinical evaluation is an absolute requirement (as proven for COVID-19).
• The same clinical data from trials cannot be evaluated differently in different Member States.
• A European HTA body is needed to provide input on the design of clinical trials that provide data to answer both the research questions from EMA and HTA.

Consultation with stakeholders

The Roadmap states that ‘the Strategy will be supported by comprehensive consultation activities with a wide variety of stakeholders, including associations representing patients and consumers, healthcare professionals, the pharmaceutical industry, include SMEs, academia…’. Our reply:

Non-profit research organisations that do not fall under the definition of ‘academia’ (such as the Anticancer Fund) as they are not affiliated with universities, are not listed as stakeholders. This should be addressed urgently and a new category of stakeholders covering such organisations should be added.

Next step

The feedback period for the Roadmap of this European initiative is closed and will be followed by a public consultation. The Anticancer Fund also participates to this consultation. To find out more about of the European initiative, the Roadmap and the public consultation, please have a look here.

To discover more about how the Anticancer Fund connects with decision makers to contribute to the development of policy frameworks and regulations, click here.