Involve the Patient Early in Drug Development

The Challenge

A rare disease focused biopharma was pareparing for IND. The team had all the pertinent clinical data and had to be convinced of the value of patient input at this early stage.

The Solution

Due to stong relationships we had built with advocacy groups globally we were able to co-ordinate conversations with the Head of R&D and Clinical Operations team leadershp to help them see this could be a good opportunity, though a non-traditional opportunity. In the end, there was patient input pre-IND that helped to ensure that once the investment of years and money were made the final drug could actually be used by the target population.

We Brought Value to Patients and Their Families

We Averted Developing a Formulation that Would Have Had Limited Use

Number of Patients/Caregiver Input Prior to Our Intervention

Number or Patients Effected by This Rare Disease Globally

Number of Advocate Input


Probability of Use by Patients Post-Intervention

There is Value in Getting Patient Input Early in Drug Development


Opportunity to Understand the Real World

In this case, had there not been patient input the formulations being developed could not have been used by the targeted patients. Especially as there is no viable drug alternative it is critical that this drug can be useable, otherwise this could have been an expensive drug with no audience. The result could have been a great drug but formulations that would not have met the patient’s lifestyle.

Building Relationships with Patients/Caregivers Early Will Help Throughout the Product Lifecycle

Patients and caregiveers want drug comapnies to know their lives/how they live. They know that the more you know the greater the chance you can help them. There ARE regulatory hurdles but these hurdles can be managed successfully.

If you want to know the lives of patients/caregivers you have to ask them

The industry continues to think that market research will provide the answers. Market research is good and can be better supported with ongoing relationships built with patients/caregivers.

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