CREATE Bio Example: Target Product Profile (TPP)

A Target Product Profile (TPP) is a planning tool for therapeutic candidates based on FDA guidelines.

The CBER Office of Cellular, Tissue and Gene Therapies (OCTGT) web page for industry education also has a Webinar on TPP 

Below are example worksheets that define the minimal/ideal profile of the final marketed product and shows the ultimate goals of the proposed therapy development effort such as disease indication, patient population (with details such as symptomatic or pre-symptomatic patients for some genetic diseases), delivery mode, treatment duration, treatment regimen, and standards for clinical efficacy. The details of the TPP should be stage appropriate.

1.  Example for ischemic stroke- revascularization

Product Targets

Minimum Acceptable Result

Ideal Results

Primary Product Indication Emergency medicine for acute stroke patients immediately on hospital arrival Emergency medicine for acute stroke patients in the community even before arrival to a hospital
Patient Population Adults of all ages with moderate to severe stroke, with potential concurrent use with tPA Adults of all ages with moderate to severe stroke, with potential concurrent use with tPA or replacement of tPA
Treatment Duration Acute Acute
Delivery Mode IV IV
Dosage Form Solution in pre-filled syringes Solution in autoinjectors
Regimen Bolus Bolus
Efficacy

20% or more favorable in comparison to placebo on minimal or no disability 30 days after treatment in patients using

Modified Rankin Scale

(score≤1) and NIHSS (score≤1).

Exploratory endpoint: imaging evidence of revascularization

30% or more favorable in comparison to placebo on minimal or no disability 30 days after treatment using

Modified Rankin Scale

(score≤1) and NIHSS (score≤1).

Exploratory endpoint: imaging evidence of revascularization

Risk/Side Effect Devoid of symptomatic intracranial hemorrhage and significant mechanism related adverse effects Devoid of any symptomatic intracranial hemorrhage and any mechanism related adverse effects
Therapeutic modality Protein

 

2. Example for pain associated with diabetic neuropathy

Product Properties

Minimum Acceptable Result

Ideal Results

Primary Product Indication Relief of pain symptoms in diabetic neuropathy Relief of symptoms in neuropathic pain syndromes
Patient Population Adults with diabetes who experience moderate to severe pain Adults with diabetes who experience moderate to severe pain
Treatment Duration Chronic Chronic
Delivery Mode Subcutaneous injections Subcutaneous injections
Dosage Form Prefilled vials with liquid Prefilled vials with liquid
Regimen Once every month Once every 2 months
Efficacy A 40% decrease in pain score in 30% of patients A 70% decrease in pain score in 50% of patients
Risk/Side Effect Devoid of local injection effect and clinically significant CNS side effect Devoid of local injection effect and any CNS side effect
Therapeutic modality Antibody