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Company Profile Report

ACCELERATED APPROVAL

Editor's letter

STAGE 1

Gaining approval

STAGE 2

Confirming benefit

STAGE 3

Modifying decisions

THE PATH AHEAD

Stretching the vision

0903 BtS18 Stretching the vision
BioCentury

Back to School 

Stretching the vision of accelerated approval

A learning system across pre and postmarket studies, bringing more drugs to more patients in more diseases

A learning system across pre and postmarket studies, bringing more drugs to more patients in more diseases.

Sep 3, 2021 | 1:00 PM GMT

There are two co-existing views of FDA’s accelerated approval pathway. It has been a grand success for cancer treatment, an area it has revolutionized. It has also been an underperformer, doing little for almost any other disease, and has been tainted by controversy.

The future is more optimistic. 

Alongside global efforts to address the pathway’s problems, technologies are emerging and ideas evolving that could stretch the vision of expedited approvals around the world to benefit all patients with severe unmet diseases and no therapeutic options — the constituents for whom the pathways were created.

That vision would see expedited approvals commonplace in many diseases — from complex, prevalent diseases where progression is slow and trials are long, such as neurodegeneration, autoimmunity and liver disease, to rare genetic diseases where new therapeutic modalities promise functional cures and surrogate endpoints are built in.

Moreover, it would see accelerated approvals serve as a springboard for innovation, spurring uptake of molecular and digital biomarkers throughout premarket and postmarket studies to create a seamless learning system that can better characterize treatment effects and identify the patients likely to benefit. 

It would also serve as a platform for innovative pricing models that allow reimbursement to be tied to the level of certainty around an outcome, with adjustments based on the value provided to patients, as well as for forms of regulatory flexibility where regulators have the tools to enable postmarket evidence to shape access to the right drugs for the right patients.

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