It can also be called as benefit-risk ratio, benefit-risk analysis, or benefit-risk balance. Ideally the concept is same which involves weighing the positive – therapeutic effect of a medicine (benefit) against the negative – undesirable side effects of a medicine (risks).

Benefit-Risk (BR) analysis is crucial for all the medicinal products in any stage of their life cycle from Pre‑clinical development to marketed phase.

Preclinical trail to Clinical entry

Possibility – 1: Positive BR ratio

Acceptable toxicity level (no non-clinical evidence of genotoxicity, hepatotoxicity, nephrotoxicity etc.,) weighed against the anticipated therapeutic benefit (from in vivo or in silico or in vitro studies)

The sponsor can request approval from regulatory authority for the conduct of clinical trial in human subjects.

Possibility – 2: Negative BR ratio

Investigational molecule exhibiting more toxicity than the anticipated benefit. Sponsor would performing terminate the further studies of development or would re-design the molecular structure for optimising the toxicity/safety profile etc.,

Clinical trial to Market entry

Possibility – 1: Positive BR ratio

Investigational medicinal product has demonstrated the anticipated efficacy and shown acceptable safety profile in human subjects from the clinical trial results.

Proposed benefit-risk ratio: Based on the therapeutic benefit from the clinical trial results and expected safety concerns (risks), the marketing authorization holder or a sponsor or a pharmaceutical company proposes the positive benefit risk profile to regulatory authority for the release of medicinal product into the market.

Possibility – 2: Negative BR ratio

The investigational medicinal product has not shown anticipated therapeutic benefit (lack of therapeutic effect) or it has shown some serious (fatal or life-threatening) adverse drug reactions which are of major concern to the safety of patient.

Sponsor might terminate/suspend the clinical trial study for change in the protocol, or investigation medicinal products dosage, formulation or can permanently discontinue the study owing to the negative benefit risk balance.

Medicinal product’s sustainability in the market:

Possibility – 1: Consistent and favourable BR profile

The approved medicinal product from regulatory authority showing therapeutic benefit and acceptable risk profile with no major impact on the proposed benefit risk profile by sponsor at the time of marketing authorisation application to regulatory/national health authority.

In this scenario, the marketing status of the medicinal product in the market would remain unchanged.

Possibility – 2: Inconsistent and unfavourable BR profile

Emerging safety findings from the post-marketing exposure which are significant with potential hazard to public safety or to the specific group of special population.

In this scenario, regulatory authority/marketing authorization holder would restrict/suspend/permanently withdraw the medicinal product from market.


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