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Drug innovations in recent years – and in the years to come – are bringing unprecedented hope for patients, such as the prospect of recovering from cancer or yet another disease for which there is still no known cure. We are witnessing a medical revolution the likes of which has not been seen for a very long time. However, these medicines come with numerous unresolved uncertainties, most notably in cases of short clinical development often seen for small patient sample sizes. So how do we safely allow early access to potentially life-saving treatments and still ensure these innovative medicines fulfil their promise? The HAS decided to tackle the issue with the development of an action plan for innovative medicines, adapted to a context that calls for greater agility of the overall assessment process.
Precision medicines, immunotherapy, gene or cell therapy : the advances made in the past few years have paved the way for the arrival of promising new therapies. Some help to prolong the life of patients whose prognosis was, until recently, uncertain (melanoma), while others aim to cure serious diseases, in particular certain types of cancer (leukaemia, lymphoma, etc.). Patients legitimately hope to be able to benefit from these treatments as quickly as possible. Rapid access to innovative treatments is a reality in France, thanks to the temporary authorisation for use early access scheme (ATU).
Innovative therapies offer hope for effective and curative treatments. Much like HAS, assessment agencies around the world are faced with the similar challenge of the keeping track and monitoring of these high-potential innovations but also in conducting rapid reviews that ensure long-term promises are fulfilled. In reality, these medicines are made available to patients early on. Their unconventional development – mainly based on non-comparative studies conducted on small numbers of patients – generates a number of uncertainties that need to be resolved during the first year of use : which patients respond to treatment ? What are their toxicities and how can they be anticipated ? What is their real-life effectiveness ? What is their role in the therapeutic strategy ? But also, what organisational healthcare changes are required ?
Wagering on early access means adapting our assessment processes
The number and nature of these medicines calls into question the assessment models used across organisations – including HAS – highlighting the need to concert our efforts to collectively address this point. Full transparency regarding the assessment of these products is imperative and of equal importance, especially given the very high – and constantly rising – prices asked by pharmaceutical companies, raising the issue of the financial sustainability of their reimbursement.
The HAS works and will continue to do so with all relevant stakeholders : the French National Agency for Medicines and Health Products Safety (ANSM), the Ministry, the pharmaceutical organisation, healthcare professionals, patients – central to our investigations – and international counterparts.
Assessing these medicines to promote access to innovations for all patients via their reimbursement also means making a distinction between key innovations and mere novelty. In this effort, the HAS is publishing its action plan to keep pace with innovation.
HAS action plan in keeping pace with innovation
- Issue conditional reviews until uncertainties are lifted
- Monitor medicines in real-life conditions to make sure they fulfil initial promises
- Reinforce HAS agility to better support innovation
- a. Focus on assessments offering high added value
- b. Improve early dialogues process to support clinical developments
- c. Promote accelerated assessment procedures (“fast-tracking”)
- d. Improve internal collective efficiency
- Systematically involve patients and consumers
- Improve transparency
- a. In terms of timeframes
- b. In terms of post-launch studies
- Increase European cooperation to pool knowledge
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Mohamed Bouhadda Head of service
Marina Malikité Press officer
Stéphanie Lecomte Press officer
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contact.presse[at]has-sante.fr
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