Table 3 MPLC Scenarios for Q-methodology and AHP
MPLC Scenario | COLUMN A Q-Methodology Description | COLUMN B AHP Description |
|---|---|---|
A: Early Development (mechanism of action well understood) | Phase 2a results are complete and phase 2b is being designed. The indication and population are well-defined. The clinical and commercial teams are discussing the criteria and requirements for a target product profile (TPP), including which benefits, risks and tolerability issues to include and what levels of each are the target. The TPP decision is an in-house activity for now, with information being shared with commercial and clinical development teams. The mechanism of action is well-understood. This is a novel indication of a treatment that has been on the market for years. | A drug is being developed for a certain population. The mechanism of action, meaning the specific biochemical interaction by which a drug produces an effect, is well-understood. The drug has been on the market for years for a different condition and its benefit-risk profile is well-understood in that population. However, this is a novel indication of the treatment, and the benefits, risks, and dosing strategy are still being identified in the new population and condition. Phase 2a studies have been conducted to demonstrate clinical efficacy. Phase 2b studies are being designed to find the optimum dose that has the greatest efficacy with minimal side-effects. The internal clinical and commercial teams are discussing the criteria and requirements for a successful treatment. The preference study would be conducted for internal decision-making on whether or not the medication should advance further in development. |
B: Early Development (mechanism of action is not well understood) | Phase 2a results are complete and phase 2b is being designed. The indication and population are well-defined. The clinical and commercial teams are discussing the criteria and requirements for a target product profile (TPP), including which benefits, risks and tolerability issues to include and what levels of each are the target. The TPP decision is an in-house activity for now, with information being shared with commercial and clinical development teams. The mechanism of action is not understood. This is novel indication. | A drug is being developed for a certain population. The mechanism of action, meaning the specific biochemical interaction by which a drug produces an effect, is not understood. This is a novel indication of the treatment, and the benefits, risks, and dosing strategy are still being identified. Phase 2a studies have been conducted to demonstrate clinical efficacy. Phase 2b studies are being designed to find the optimum dose that has the greatest efficacy with minimal side-effects. The clinical and commercial teams are discussing the criteria and requirements for a target product profile (TPP), including which benefits, risks and tolerability issues to include and what levels of each are the target. The TPP decision is an in-house activity for now, with information being shared with commercial and clinical development teams. The preference study would be conducted for internal decision-making on whether or not the medication should advance further in development. |
C: Late Phase III | Clinical data available for pivotal trials. Mechanism of action is understood. Advisory committee/scientific advisory group meeting is scheduled. The goal is to provide data to support benefit-risk assessment to health authorities for regulatory dossier submission. | The benefits and risks dosing strategy of a medical product are reasonably well-characterized, as clinical trials in patients have been completed to assess efficacy, effectiveness, and safety. Mechanism of action is understood, (meaning the specific biochemical interaction by which a drug produces an effect). There is an advisory committee/scientific advisory group meeting scheduled. The goal is to provide patient preference data to support benefit-risk assessment when submitting dossiers to regulators and HTA bodies. |
D: Post-Marketing | The treatment approved a year ago is now discovered from a registry or observational data to have a clinical significant side effect. Currently, the discussion is all in-house, but the signal is likely to lead to a discussion with health authorities. | A medical product approved a year ago is now discovered from a registry or observational data to have a clinical significant side effect. Currently, the discussion is all in-house, but the signal is likely to lead to a discussion with health authorities. The preference study would be used to complement the clinical data by providing the patient’s perspective on benefit-risks. |