#apaperaday: Repurposing of Medicines in the EU: Launch of a Pilot Framework
In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Repurposing of Medicines in the EU: Launch of a Pilot Framework.
A paper by Asker-Hagelberg et al from Frontiers In Medicine on the launch of the pilot framework for a medicine repurposing project. I’ll summarize but recommend the paper to all interested in this topic. Easy & nice read. Doi 10.3389/fmed.2021.817663
Drug repurposing means using an existing drug for a new purpose or indication. This can involve a new formulation of the existing drug. Repurposed drugs can be 1)compounds not yet approved for any indication, 2)compounds approved and still under IP 3)approved compounds out of IP
When compounds are no longer IP protected, pharmaceutical companies generally are less keen in investing in repurposing (as they cannot recoup the investment). Academics often are interested in repurposing these type of compounds but lack regulatory experience
This means that often these repurposed drugs do not get marketing authorization for the new indication (as academic groups do not have the expertise and infrastructure to obtain and hold a marketing authorization). Thus the drugs are used off-label for the new indication.
Off-label use is not optimal for several reasons: 1) There is no formal information about the drug for that disease (e.g. in the summary of product characteristics from EMA or patient information leaflet). Thus patients have no information of risks & side effects and optimal use.
2) Access & reimbursement for off-label drug use is suboptimal. In some countries off-label use is reimbursed while in others it is not (as there is no formal data on efficacy). Not all clinicians will prescribe the off-label drug.
3) If the drug is withdrawn from the market for the indication it was approved for, it will no longer be available for the off-label use. There needs to be a better mechanism to register drugs for new indications – also taking into account that collecting extra data costs money.
A framework was proposed by STAMP (a multistakeholder subgroup of European Commission’s Expert Group on Safe & Timely Access to Medicines for Patients, with representatives from EMA, nat. competent authorities, patient organizations, research institutes & pharmaceutical industry)
The framework aimed to facilitate drug repurposing and marketing authorization by a champion (academic organisation, patient organisation etc). The idea would be that the champion would liaise with the marketing authorization holder so they could apply the use for new indication
A pilot to test the framework was planned to launch in March 2020. Sadly, it had to be postponed as the COVID-19 pandemic then launched. However, due to the pandemic some repurposing initiatives still happened – mainly for COVID-19 which had not been an indication before 2019
Authors note that many of these initiatives were led by academic groups rather than pharmaceutical companies. They also mention that often pharmaceutical companies facilitated efforts by donating drugs.
After a major delay the framework has now been launched as of October 28. The authors recommend us to stay tuned. I surely will and am very keen to see what this will bring. Repurposing is of course something that is especially appealing for rare diseases.
Pictures by Annemieke, used with permission.