Are Accelerated Approval Mechanisms a Predictor to Early Access?


Panos Kanavos, Associate Professor in International Health Policy, Deputy Director, LSE Health, Department of Health Policy, London School of Economics and Political Science (LSE)

There are significant advances being made in the treatment of cancer, as drugs with improved efficacy and tolerability profiles are reaching the market in record time. This is, in part, due to regulatory bodies responding to the rapid pace of innovation and unmet patient need.

Regulatory bodies including the US Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) have implemented initiatives that grant conditional approval to innovative medicines based on single-arm Phase II data, until full data from Phase III randomised controlled trials are available.

These accelerated, conditional, approvals are granted to help ensure that therapies that hold a significant promise to deliver a significant benefit over existing treatments reach the patients that need them as soon as possible. Theoretically, these initiatives could bring therapies to patients months, if not years, earlier than an approval based on Phase III data. However, an accelerated regulatory approval does not always guarantee accelerated access as Health Technology Assessment (HTA) agencies have often been much slower to adapt their processes and evidentiary requirements to this new approach. This can delay patients from being able to access therapies designated with accelerated regulatory approval.

To learn more about the impact of accelerated regulatory pathways, LSE is conducting research into the interrelationship between accelerated approval schemes for cancer drugs and national HTA processes across thirteen jurisdictions globally.

At the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 20th Annual European Congress in Glasgow this week, we are presenting the initial findings of this research.

First, we identified 15 pairs of drugs, indicated across melanoma, lung cancer and haematology that have been granted an accelerated approval in the US, EU, or both. We subsequently analysed what, if any, impact the accelerated approval pathway had on coverage and funding pathways in 13 countries.

Preliminary analysis indicates that access to drugs authorised under an early access mechanism continues to be dictated on whether data satisfies the requirements of the HTA review. Early conclusions also suggest that accelerated approval pathways do not guarantee coverage or early market access at population level and may result in delays in access.

Second, we will present the results of a global survey which was was undertaken to identify, map, and differentiate early access pathways in 17 countries. Results from this survey suggest that early access schemes can be grouped into four different pathways: marketing authorisations with a faster review time, marketing authorisations with lower evidence thresholds, marketing authorisations based on approval in other settings and access to medicines prior to marketing authorisation.

Whilst there were many noticeable differences in the eligibility criteria and conditions attached to early access, we noticed that all pathways required there to be a significant unmet clinical need in the relevant indication, or evidence of significant improvement over existing therapies.

The findings we are presenting at the ISPOR Annual European Congress are preliminary, but give good insight into how academia can work with industry, regulatory bodies and reimbursement authorities, to bring access to medicines, quickly and safely, to patients.

These findings are part of a London School of Economics research project supported by AstraZeneca. The findings will be published in summer 2018.