EARLY ACCESS TO MEDICINES THAT ADDRESS PUBLIC HEALTH NEEDS
In 2019, three medicines (Ervebo, Xospata and Zynteglo) were recommended for marketing authorisation following an accelerated assessment. This mechanism is reserved for medicines that address unmet medical needs. It allows for the assessment of eligible medicines by EMA’s scientific committees within a maximum of 150 days rather than the usual 210 days.
Eight medicines received a recommendation for a conditional marketing authorisation, one of the possibilities provided in the EU to give patients early access to new medicines: Ervebo, Libtayo, Lorviqua, Ondexxya, Polivy, Vitrakvi, Waylivra and Zynteglo.
As these medicines address unmet medical needs, the conditional authorisation allows for early approval on the basis of less complete clinical data than normally required (products for use in emergency situations may have less complete pharmaceutical or non-clinical data). These authorisations are subject to specific postauthorisation obligations to generate further data on the medicines.
One medicine (Dectova) was authorised under exceptional circumstances. This route allows for patients’ access to medicines that cannot be approved under a standard authorisation as comprehensive data cannot be obtained, either because very few patients have the disease, or the collection of complete information on the efficacy and safety of the medicine would be unethical, or there are gaps in the scientific knowledge.
Dectova is an antiviral medicine used to treat complicated and potentially life-threatening influenza (flu) caused by either the influenza A or B virus in adults and children from 6 months of age. Medicines authorised under exceptional circumstances are subject to specific postauthorisation obligations and monitoring.
MEDICINES FOR RARE DISEASES
The EU framework for orphan medicines provides incentives for developers to encourage the development and marketing of medicines for patients with rare diseases. A medicine that was granted an orphan designation during its development can benefit from ten years of market exclusivity after its marketing authorisation, provided EMA’s Committee for Orphan Medicinal Products (COMP) confirms its orphan status at the time of approval.
Among the medicines recommended for marketing authorisation, seven had had their orphan designation confirmed by the end of the year: Epidyolex, Isturisa, Palynziq, Polivy, Waylivra, Xospata and Zynteglo.
In 2019, the following applications lost their orphan status before receiving marketing authorisation, which means they were still authorised as medicinal products but not as orphan medicinal products: Trecondi, Cufence, Esperoct, Ultomiris, Vitrakvi and Revlimid. More information can be found in the COMP monthly reports.