For patients with an untreated serious illness, clinical trials offer a ray of hope.
Should that hope be fulfilled and the investigational therapy prove successful, patients naturally wish to remain on the therapy. There is a gap, however, between the close of a trial and the availability of the investigational product on the market – a gap that could extend for months or even years. What then, should patients who wish to continue on therapy do?
Sponsors have two compassionate options: a long-term follow-up (LTFU) study, which runs parallel to the regulatory approval process and negotiations over pricing and reimbursement with payers and health technology assessment bodies, or a managed access program (MAP), which is a regulatory mechanism for providing patients with access to the investigational product while it is still under review for marketing approval and reimbursement.
There are compelling reasons for sponsors to opt for a MAP over a LTFU study. Depending upon the sponsor’s evidence goals, MAPs may serve as a cost-effective alternative to expensive LTFU studies, however, they require early planning and expertise that differs from that needed in clinical trials and must be run based on a different set of goals, procedures, and skill sets.
Clinical teams are, therefore, often unfamiliar with how best to manage MAPs since they are not, in any sense, clinical trials.
Assembling the correct project team with the expertise and skills specific to a MAP is critical to ensuring a successful programme. Navigating the regulatory and logistical intricacies requires individuals capable of flexible and strategic critical thinking. Every MAP has a distinct personality based on the disease type, sponsor philosophy, patient population, development stage, etc. The existing guidance in both the US and EU does not provide details to address each potential scenario, so sponsors must ensure that they fully understand the requirements and allowable processes from the outset.
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This blog is an edited version of “Mind the gap: Managed access programmes as an alternative to long-term follow up studies” which appeared in the March 2021 issue of Pharmafocus. To view the full article, please visit https://en.calameo.com/read/0061133858eb13eaeff04?page=16.