Treatments for rare diseases

Access to treatment

Understanding rare diseases Access to treatment

Meeting unmet medical needs

For many rare diseases, treatment options remain limited or even non-existent.

Patients and their families then face a double challenge: living with an often disabling disease and coping with the lack of a suitable solution. Bringing new treatment options to these patients is now a major challenge, involving researchers, clinicians, industry and health authorities. At KÔL, our commitment was born of this reality: enabling patients with no therapeutic option to access innovative treatments that meet unmet medical needs.

A long and complex development process

Developing a medicine is a long process that can span several years.

This is even more true for rare diseases, where the small number of patients often makes clinical trials more complex. Before it can be made available, every treatment must pass through many stages designed to demonstrate its quality, safety and efficacy. These requirements are essential for patients, but they can also delay access to innovation.

Enabling rapid access to innovation

A marketing authorisation (MA) is required before a medicinal product can be made available.

Obtaining it can sometimes take a long time and, in the meantime, patients cannot benefit from the treatment. When the medical situation justifies it and no satisfactory therapeutic alternative is available, health authorities can grant early access to a medicine.

Named-Patient Program or Early Access Authorisation

The Named-Patient Program or Early Access Authorisation is granted on a named-patient basis, under the responsibility of the prescribing physician, when the medicine is likely to provide a benefit. Each request is validated by the health authorities. A Named-Patient Program or Early Access Authorisation may be granted:

To treat, prevent or diagnose serious or rare diseases.
When no appropriate treatment is available on the market.
If the product’s efficacy and safety are presumed and treatment cannot be postponed.

AAP

Autorisation d’accès précoce

L’AAP est délivrée pour un groupe de patients, toujours sous la responsabilité du médecin prescripteur lorsque le médicament est susceptible de présenter un bénéfice. Elle permet d’utiliser un médicament destiné à être commercialisé avant l’obtention de l’AMM. L’AAP peut être délivrée :

To treat, prevent or diagnose serious or rare diseases.
When no appropriate treatment is available on the market.
Si l’efficacité de la spécialité et sa sécurité d’emploi sont fortement présumées et si la mise en œuvre du traitement ne peut pas être différée.
Si le médicament est présumé innovant.