Once you have a clear idea of the type of drug you are repurposing, you need to consider how you plan to deliver that drug to patients. There are two major routes to consider:
- On-label – the drug is prescribed for a condition for which it has received marketing approval
- Off-label – the drug is prescribed for a condition for which it does not have marketing approval
Each route has its own additional benefits and challenges:
- Securing marketing authorisation gives a route to reimbursement, uniform patient access nationwide, and likely suggests that there is robust evidence for the efficacy and safety of the drug.
- Obtaining regulatory approval requires a much higher level of evidence and therefore longer and more costly clinical trials.
- Off-label drugs tend to be less costly with shorter studies.
- Less industry input is therefore needed, providing the opportunity to distribute at a low-cost generic drug price.
- These drugs tend to have less evidence of efficacy and monitoring of safety without marketing authorisation.
- Therefore, their patient access is likely to be less uniform (depending on how many clinicians serve the population.)
- Off-label drugs could also be subject to predatory price hikes.
- It is important to evaluate the above considerations when making strategic decisions about your repurposing candidate.
Bringing the strategy together
It is important to evaluate the above considerations when making strategic decisions about your repurposing candidate. The likely opportunities based on each option are summarised in the figure below.