When considering starting a dug repurposing project, it is not enough to simply identify a candidate you must also think about how you will deliver that to patients successfully therefore you need to build an effective strategy. There are two major things that are always worth considering at the outset: firstly, how doe the status of you repurposing candidate affect your project plan? And, secondly what is the most appropriate way to deliver a drug successfully to patients.
The sections below outline some of these strategic considerations, you can also watch Rick Thompsons video outlining strategy considerations.
- Understanding your repurposing candidate
- Delivering your drugs to patients
Understanding your repurposing candidate
In any repurposing project, the status of the candidate and the route to delivering a treatment to the patient make a huge impact on your strategy for selecting a drug. As described in the “What is drug repurposing?” section of this course, it is possible to target existing generic drugs for repurposing (those that are no longer under patent and widely available), current branded compounds (sold under licence and protected by intellectual property) or shelved compounds.There are a number of benefits and challenges associated with both generic drugs and those under patent/benefiting from marketing exclusivity, which should be carefully considered when selecting a candidate drug:
- Patented drugs have an obvious route to approaching the patent-holding industry partner to develop a drug for your condition, with a clear business benefit for the patent holder to repurpose
- Robust clinical evidence is required to convince the patent holder to work on the repurposing project
- There is likely to be limited understanding of safety in a wider range of patients
- These drugs are likely to have lots of data available on human use and are usually at a low cost
- This means that they are readily producible and have good access for off-label use
- They are therefore ideal for an academic-led clinical trial
- Due to an absence of a patent, a repurposed generic drug is likely to have less options for its intellectual property to be defended
- Consequently, there will be a less obvious business opportunity for industry partners to repurpose this drug
- The drug’s current formulation/delivery method may not be appropriate for the patient population in question
- It is possible that the drug could be taken by patients outside of clinical trials if they hear about potential benefits of the treatment. This would make recruitment for the clinical trials more challenging, and may put patients at risk since they are not being monitored in the context of the trial
Delivering your drug to patients
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.