A Target Product Profile (TPP) is a strategic planning tool that guides the development of safe and effective medical products. It outlines the desired product characteristics, including its intended use, target population, and key performance features, ensuring that efforts in research and development align with specific clinical needs and regulatory requirements. By defining these attributes early on, a TPP fosters stakeholders’ alignment, facilitates efficient resource allocation, and increases the likelihood of developing a successful product. This success translates to addressing unmet medical needs, improving patient outcomes, and enhancing the potential for commercial success. Furthermore, to remain effective, a TPP should be a dynamic document, continuously evolving with emerging data and insights, supporting informed decision-making at every stage of development.
Key Components of a TPP
A TPP outlines essential product characteristics. Table 1 presents a TPP worksheet for a new pharmacotherapeutic, informed by the FDA's Guidance for Industry and Review Staff: Target Product Profile — A Strategic Development Process Tool (Draft Guidance, 2007). It maps key attributes from the drug labeling section (leftmost column), product properties, minimum acceptable results, and ideal results for the proposed product. Notably, Affordability and Accessibility are highlighted as key product properties in addressing substance use disorders. Additional product properties can be included to refine and differentiate the pharmacotherapy.
| Drug Label Attributes | Product Properties | Minimum Acceptable Results | Ideal Results |
|---|---|---|---|
| Indications and Usage | Primary Indication | ||
| Indications and Usage; Clinical Studies | Target Population | ||
| Dosage and Administration | Treatment Duration | ||
| Dosage and Administration | Delivery Mode | ||
| Dosage and Administration | Dose Regimen | ||
| Dosage Forms and Strengths | Dose Form | ||
| Clinical Studies | Clinical Efficacy | ||
| Adverse Reactions | Risk/Side Effect | ||
| How Supplied, Storage and Handling | Product Stability and Storage | ||
| Clinical Pharmacology | Mechanism of Action, Pharmacokinetics, Pharmacodynamics | ||
| Drug Interactions | Drug Interactions | ||
| Affordability (Price) | |||
| Accessibility |
Evaluating the Treatment Landscape for Effective TPP Development
A TPP is informed by the current treatment landscape, requiring a comprehensive analysis of the existing and emerging treatments. For example, when developing a medication to mitigate opioid withdrawal symptoms, a thorough appraisal of existing options, for instance, FDA-approved Lucemyra (lofexidine), is critical. As illustrated in Table 2, a TPP can be constructed using Lucemyra as a benchmark, with the key product properties for Lucemyra extracted from its drug label. This comparative approach helps ensure the new proposed therapy meets or surpasses current standards, maximizing its potential for patients, providers, and payors while addressing unmet medical needs. By carefully analyzing the properties of similar products, innovators can pinpoint areas for improvement and develop a superior treatment.
| Drug Label Attributes | Product Properties | FDA-approved medication: Lucemyra (lofexidine) | Minimum Acceptable Results | Ideal Results |
|---|---|---|---|---|
| Indications and Usage | Primary Indication | Mitigation of opioid withdrawal symptoms to facilitate abrupt opioid discontinuation in adults | ||
| Indications and Usage; Clinical Studies | Target Population | Adults meeting the criteria for opioid dependence who were physically dependent on short-acting opioids | ||
| Dosage and Administration | Treatment Duration | Up to 14 days | ||
| Dosage and Administration | Delivery Mode | Oral | ||
| Dosage and Administration | Dose Regimen | Three 0.18 mg tablets taken orally 4 times daily at 5- to 6-hour intervals. Treatment may be continued for up to 14 days with dosing guided by symptoms. | ||
| Dosage Forms and Strengths | Dose Form | 0.18mg tablet | ||
| Clinical Studies | Clinical Efficacy | Primary endpoints: Mean SOWS-Gossop total score on Days 1 – 5: 7.0 (placebo, 8.9); Proportion of patients that completed 5 days of treatment: 49% (placebo, 33%) | ||
| Adverse Reactions | Risk/Side Effect | Low blood pressure (hypotension), slow heart rate (bradycardia) and fainting | ||
| How Supplied, Storage and Handling | Product Stability and Storage | Store at 25°C (77°F) | ||
| Clinical Pharmacology | Mechanism of Action, Pharmacokinetics, Pharmacodynamics | Central alpha-2 adrenergic agonist. A half-life of approximately 12 hours and a bioavailability of 72% | ||
| Drug Interactions | Drug Interactions | Oral Naltrexone: Concomitant use may reduce the efficacy of oral naltrexone. Methadone: Methadone and Lucemyra both prolong the QT interval. ECG monitoring is recommended when used concomitantly. | ||
| Affordability (Price) | Prescription use. $900 for Lucemyra 0.18 mg (36 tablet bottle) and $300 for generic lofexidine 0.18 mg (36 tablet bottle) on GoodRx | |||
| Accessibility | Prescription |
How TPPs Are Used in Different Product Types
TPPs serve as a valuable tool across various medical product categories. Whether for drugs, medical devices, or diagnostic tests, TPPs provide a structured approach to ensuring safety, efficacy, and usability. TPPs should be created for medical device development. Table 3 provides an example of an analogous TPP for a device that is intended for opioid withdrawal using a device from NET Recovery as the benchmark. The NET Device is a non-invasive, battery-powered, portable, reusable, prescription device designed to provide bilateral, transcranial, transcutaneous, alternating current stimulation (tACS) to be used in patients experiencing opioid withdrawal under the supervision of trained clinical personnel. The system is comprised of one component (the NET Device), accessories (patient leads, electrodes, USB cable), and software (the clinician application). The key product properties of NET Recovery were derived from its FDA Premarket Notification (510(k)).
| Product Properties | NET Recovery (K233166) | Minimum Acceptable Results | Ideal Results |
|---|---|---|---|
| Intended Use / Indications for Use | The NET Device is a transcutaneous alternating current stimulator (tACS) that is intended to be used in patients experiencing opioid withdrawal in conjunction with standard symptomatic medications and other therapies for opioid withdrawal symptoms under the supervision of trained clinical personnel. | ||
| Patient Population | Patients experiencing opioid withdrawal, aged 18 and older, diagnosed with opioid use disorder, who have a Clinical Opiate Withdrawal Scale (COWS) score of moderate for opioid withdrawal symptoms | ||
| Treatment Duration | 1 hour per day, not to exceed 7 days | ||
| Summary of Technological Characteristics | Electrical stimulation at the auricular region | ||
| Summary of Clinical Testing | Primary endpoints: The Clinical Opiate Withdrawal Scale (COWS) score decreased from an average of 18.1 points at baseline to an average of 7.0 points at 60 minutes, demonstrating a mean reduction in the COWS score of 61.3% at 60 minutes. | ||
| Safety Analysis/Risk for Health | The risks are consistent with those of the predicate device, Sparrow (Spark Biomedical, Inc.), and non-clinical testing of Sparrow did not identify any safety concerns. | ||
| Affordability (Price) | Prescription Use. $600 - $1,500 | ||
| Accessibility | Prescription |
Example TPP for In Vitro Tests
Table 4 presents a practical example of a TPP for an in vitro test using a fentanyl urine test as a case study. This profile was developed based on the framework provided in Cocco P. et al., “Target Product Profiles for Medical Tests: A Systematic Review of Current Methods.” The framework outlines key product properties and minimum acceptable and ideal results. The table provides an example of the product properties of the InstraStrip Fentanyl Rapid Test (K240295). For Stability during Transport and Time to Results, both minimum acceptable and ideal results are shown to illustrate the potential characteristics of a new product.
| Product Property | InstaStrip Fentanyl Rapid Test (K240295) | Minimum Acceptable Results | Ideal Results | |
|---|---|---|---|---|
| Unmet Clinical Need | Medical need | Detection of fentanyl in urine | ||
| Intended use | Detection of fentanyl concentrations in urine >1 ng/ml | |||
| Target user | Adults 18 years old and older | |||
| Target population | People with OUD and healthcare professionals involved in OUD treatment | |||
| Stability during transport | +10°C - +30°C | +10°C - +30°C | +4°C - +40°C | |
| Analytical Performance | Target molecule | Fentanyl | ||
| Assay design/ Format | Lateral flow immunoassay | |||
| Sample Type | Urine | |||
| Time to Result | 5 minutes | 10 minutes | 3 min. | |
| Comparative reference method | Laboratory analyzer - ELISA | |||
| Sample Volume | 0.5 ml | |||
| Calibration | None | |||
| Assay type | Competitive immunoassay | |||
| Manual sample preparation | Yes | |||
| Reproducibility | 0.98 | |||
| Internal quality control | Yes | |||
| Diagnostic specificity | See the table for cross-reactivity (K240295) | |||
| Test type | Qualitative | |||
| Diagnostic sensitivity | 0.5 ng/ml | |||
| Clinical Validity | Training and Education | Box insert | ||
| Result documentation and display | Visual detection can be photographed. | |||
| Human Factors | Ease of test interpretation | Easy | ||
| Test outcome | Presence/absence of fentanyl | |||
| Service and support | Not required | |||
| Test size and portability | 0.5'x 0.3'x 0.2' (box), 2"x 0.25"x 0.001' (test strip) | |||
| Tool format and complexity | Test strip | |||
| Temperature and humidity | Room temperature and humidity during testing | |||
| Storage conditions and shelf life | +10°C - +30°C, 1 year | |||
| Affordability (Price) | $2 per test strip | |||
| Accessibility | Directly from company | |||
Conclusion
A well-structured TPP provides a clear vision for product development, guiding regulatory strategy and commercial planning. Whether for pharmaceuticals, medical devices, or diagnostic tests, a TPP enhances decision-making, minimizes risks, and increases the likelihood of successful product approval and adoption.