The first step for every wise drug developer beginning a drug development program is to determine the feasibility of a proposed product by asking several high-level questions:

• Is the product scientifically, medically, and commercially viable?
• What are the regulatory requirements and potential hurdles?
• What nonclinical and/or clinical studies will be required for approval?
• What is the estimated timeline and cost of the development program?
• Can the time and/or cost of the proposed drug development program be reduced?

A Strategic Assessment can answer these questions and provide guidance on the most cost- and time-efficient drug development program. In particular, for a 505(b)(2) New Drug Application (NDA), a Strategic Assessment is strategically important to efficiently assess and establish the optimal developmental program before the first step is taken.

Strategic Assessments are clearly advantageous at early stages of development, however can also be valuable when development does not go according to plan and companies want to reassess and adjust their proposed development plan, as discussed in the case study below.

How Strategic Assessment Makes a Difference: A Case Study

Prior to working with Premier Consulting, a Biotech company worked with another regulatory consultant who utilized a 505(b)(1) approach to develop its drug development program. After discussion with the FDA at an initial Pre-IND meeting, the Biotech company received Agency agreement on a drug development strategy which proposed extensive clinical trials, estimated to cost $25 million and with a timeline of more than 3 years to completion. The frustrated Biotech company could not afford this agreed-upon plan and contacted Premier Consulting to see if an alternative approach could be developed.

Premier Consulting conducted a Strategic Assessment of the scientific and medical viability and regulatory requirements of the proposed product. Utilizing a 505(b)(2) regulatory strategy, Premier Consulting identified a previously unrecognized resource in available published studies which could be leveraged to develop an alternative 505(b)(2) drug development program with minimal clinical trials required. The proposed 505(b)(2) program included reducing the need for Sponsor-conducted studies through reliance upon published studies via a demonstrated scientific bridge to the published literature through a bioequivalence study, eliminating the costly and time-consuming extensive clinical program initially suggested by the original regulatory consultant.

The results: a dramatic reduction in development time and cost.

Following Premier Consulting’s recommendations, the Biotech company contracted with Premier Consulting to request and conduct a second Pre-IND meeting with FDA to discuss this new alternative 505(b)(2) drug development program. After Premier Consulting presented the 505(b)(2) drug development program, the Agency agreed to require only a bioequivalence study for approval, minimizing the previous 3-year clinical program to only months and significantly reducing the development costs to a fraction of the initial estimated $25 million cost. In addition, Premier Consulting’s proposed 505(b)(2) development plan also reduced the time to market by more than 2 years, a significant advantage for the company.

What Is Included in a Strategic Assessment?

The purpose of a Strategic Assessment is to outline a clear drug development plan for the proposed drug product by evaluating the aspects of drug development. A Strategic Assessment is scalable for each Sponsor’s specific needs, based upon the following four aspects of drug development:

• Medical
• Scientific
• Regulatory
• Commercial

A full Premier Consulting Strategic Assessment provides information on commercial and medical viability, as well as regulatory, clinical, nonclinical, and Chemistry, Manufacturing, and Controls (CMC) requirements, with competitive assessment available, as well. The Strategic Assessment evaluates available information and based on an assessment of required scientific and regulatory components, provides a recommended development plan for drug product approval, including an estimated timeline and cost.

Premier Consulting’s Strategic Assessment considers all regulatory requirements for the proposed drug product, including those needed to gain approval as well as an assessment of other applicable regulatory rules, such as exclusivity or a request for orphan drug status. A Strategic Assessment outlines a specific plan and strategy to ensure that the proposed drug development program meets these regulatory requirements as well as providing recommendations on the value of a Pre-Investigational Drug (Pre-IND) Type B meeting or Pre-NDA Type B meeting, which provide opportunity for discussion with the Agency to get their buy-in to the proposed program.

Benefits of a Strategic Assessment

As 505(b)(2) applications can rely upon studies that were not conducted by or for the Sponsor, a reduced development program may be possible for drug approval. A Strategic Assessment for a 505(b)(2) application can be invaluable in identifying areas where the need for clinical or nonclinical studies can be reduced or eliminated based upon the identification of available published data that can be relied upon through the establishment of a scientific bridge. This may include reliance upon the Agency’s previous findings of efficacy and safety for an approved drug product (listed drugs [LDs]) or relevant data available in the published literature. Premier Consulting’s Strategic Assessment identifies and provides recommendations on the selection of the appropriate LD(s), as well as relevant published studies, and develops a strategy for establishing an efficient 505(b)(2) drug development program.

If a Sponsor provides a proposed development program, a Premier Consulting Strategic Assessment can be used to assess the proposed nonclinical, clinical, and CMC program, identifying where Sponsor-conducted studies can be streamlined, reduced, or eliminated based upon the identification of and reliance upon publicly available data.

As discussed above, Premier Consulting’s Strategic Assessment identifies and provides recommendations on the selection of LD(s) as well as relevant publicly available studies. In addition, the Strategic Assessment can identify any gaps that may require additional Sponsor-conducted nonclinical or clinical studies to support the efficacy and safety of the proposed product to meet regulatory requirements for approval.

A Premier Consulting Strategic Assessment provides Sponsors with a clear and informed picture of the full proposed drug development program, allowing them to make critical decisions on the viability of the proposed program. This can save both time and money, as adjustments in the development program such as elimination of a previously proposed nonclinical and/or clinical study(ies), can be made early in development. In addition, if desired, an informed go/no go decision can be made early in development versus later when time and money have already been invested in a program.

How to Begin with a Strategic Assessment

A Strategic Assessment is a valuable tool which provides the Sponsor with a clear vision of the requirements of the full drug development program for the proposed product. This allows informed critical decisions to be made by providing a full understanding of the likely development program, timeline, and cost. Premier Consulting has extensive experience in providing both high-level (i.e., focused only on a few key questions or concerns) and full Strategic Assessments. To learn more about how to engage our experts in launching your drug development program in the most cost- and time-efficient manner possible, contact us.