Our client was a French biotech developing its first drug for neurological rare disease. The drug was granted an orphan drug designation a year before we met.Their international headquarters are in France. When they first came to Rare Insight, their pivotal Phase III study was ongoing. They wanted to open a subsidiary in Italy to start prelaunch activities.


First of all, Rare Insight conducted an early access program assessment with a specific focus on the disease at stake. We presented our local early access system. We made an assessment and scheduled a timeline for a potential early access program (or compassionate use) in our client’s orphan indication.Following this first step, Rare Insight proposed more cost-effective way to meet customers’ needs. Considering the size of the market and the future indication of their product, opening affiliates would have been very costly. Few days per month to start the activities was the appropriate level of resources needed for prelaunch activities.


Since 2017, Rare Insight has been working with this client on:

  • Local market access activities:
     Early access program assessment and implementation
     Regulatory support
  • Local Medico-marketing activities:
     Mapping reference centers in Italy
     Building relationship with patient advocacy groups
     Supporting in the selection of centers for their pediatric pivotal study
     Better understanding of disease management through physicians’ interviews
     Supporting QoL observational study and patient pathway communication
     Writing technical documents
     Participating in local congresses for disease awareness
     Advisory board with clinicians

To coordinate these activities, Rare Insight appointed a Project Director who interacted with the costumers in systematic interactions (monthly reporting and teleconferences).


  • A cost effective approach:
     Invest only needed time
     Budget control adapting the number of days worked by Rare Insight according to our client’s needs
  • Highly flexible attitude:
     No internal employees to hire and manage, substitution of Affiliates before Marketing Authorization to prepare the market locally
     Dedicated team bringing speed and momentum
  • Time and coordination of effective process:
     Plug and play: mission started within a month
     One centralized point of contact to gather local information
     Monthly updates for the client