One of Premier Consulting’s differentiating offerings is the portfolio analysis, a high-level, integrated evaluation of the scientific, medical, regulatory, and commercial viability of each product within a sponsor’s portfolio. Informed by today’s competitive drug development landscape, as well as by the sponsor’s strategic goals and capabilities, a portfolio analysis involves assessing product concept and differentiation, strategic planning, factoring in unmet patient needs, and estimating timelines and expenses. Gaining a thorough understanding of these factors can significantly impact development strategy and increase the likelihood of marketplace success.

Why conduct a portfolio analysis?

Each product is unique, and so is the associated development strategy. Sponsors must consider many variables when building a strategic plan. Each path forward, which is tailored to target client goals and patient needs, has positive and potentially negative implications. In addition, the size and complexity of a development program can vary significantly depending on product characteristics, market dynamics, and regulatory pathway. A thorough portfolio analysis helps identify the most cost-effective and time-saving strategy by outlining development timelines and expected costs and identifying potential risks, such as lack of product differentiation.

Therefore, a portfolio analysis may prioritize programs for product candidates with greater differentiation from approved versions, even if those programs are more complex and expensive. For example, streamlined programs can be limited to literature searches, nonclinical studies, demonstration of bioequivalence to an approved product, or a combination of nonclinical and bioequivalence studies, and their relatively low cost may be appealing. However, they may generate undifferentiated labels that are not conducive to commercial success.

In addition to prioritizing programs most closely aligned with sponsor objectives, a portfolio analysis can help prevent a program from failing due to misalignment of the various factors that affect drug development, including clinical, nonclinical, regulatory, scientific, manufacturing, and market dynamics. It can also help identify which of these factors are most critical to commercial success.

Fine-tuning strategic assessment

Premier Consulting’s team leverages its deep and nuanced understanding of regulatory requirements to align sponsors’ business goals with those requirements. We use FDA guidance documents, information from approved labels, sponsor-conducted studies, and relevant published literature to conduct portfolio analyses and product prioritizations to help clients make decisions regarding the following parameters, among others:

Regulatory pathway: Premier Consulting is adept at helping sponsors determine the most appropriate regulatory pathway (e.g., ANDA, 505(b)(1) NDA, 505(b)(2) NDA, or over-the-counter [OTC] monograph), based on whether the product is a new chemical entity or has an approved US reference listed drug (RLD) counterpart. Premier Consulting’s expertise can also help clients avoid an FDA Refuse-to-File letter and other regulatory hang-ups.

Incentive programs: Treatments for serious or rare diseases and treatments with marked advantages over existing therapies may qualify for Fast-Track, Breakthrough Therapy, or Orphan Drug Designation and receive corresponding market exclusivity, potentially benefiting the sponsor and patient communities alike.

Pediatric Research Equity Act (PREA) requirements: Sponsors of NDAs for a new active ingredient, new indication, new dosage form or regimen, or new route of administration may be required to conduct pediatric assessments, even if the indication is not intended for pediatric populations.

Clinical pharmacology: This is a key factor in designing Phase 2 and 3 trials. Usually performed in healthy volunteers, these studies assess dosage safety and the pharmacokinetic (PK) profile of a proposed drug product. Clinical pharmacology may also involve comparative bioavailability analyses, which are generally required for drug formulation bridging studies, to demonstrate bioequivalence.

Biowaiver eligibility: For products that have a U.S. RLD counterpart, the FDA may waive in vivo study requirements depending on the drug formulation, proposed indication, active ingredients, solubility, permeability, and other factors.

Clinical safety and efficacy: This information is obtained in Phase 2 and 3 trials, which are usually performed in the patient population that is targeted for the treatment. These studies can be a critical inflection point for strategy, value, and investment and typically form the basis for market approval.

Nonclinical requirements: A nonclinical safety evaluation program typically includes pharmacology, general toxicity, toxicokinetics, nonclinical PK, reproductive toxicity, genotoxicity, and carcinogenesis. Nonclinical studies are required mostly to evaluate the safety of a product and/or its excipients in animal models before administration to humans.

Chemistry, manufacturing, and controls (CMC) strategies: These are put in place to ensure product quality in compliance with FDA requirements. They can be particularly important for streamlined programs in which the proposed active pharmaceutical ingredient (API) has an approved RLD counterpart, in which case the proposed formulation should be as close as possible to the approved formulation.

Based on the above inputs, a portfolio analysis outlines the anticipated drug development program for each product in a sponsor’s portfolio. It also ranks products based on complexity, differentiation, timing, and cost of overall development, weighing those factors in accordance with a sponsor’s goals and patient needs.

Beginning with the end in mind

Premier Consulting views portfolio analysis as a vital exercise conducted in partnership with sponsors. In each case, Premier Consulting works with the sponsor to understand its product development goals and designs a portfolio assessment with those objectives in mind. Informed by lessons learned from previous development initiatives, Premier Consulting’s portfolio analysis and product prioritization capabilities epitomize our principle of “beginning with the end in mind” — an approach that helps sponsors start their programs on the right foot, with a clear roadmap for each step of the development process. These capabilities help sponsors operate within the relevant regulatory framework to meet their goals.

For additional information or specific input on your product portfolio, contact us.

Author:

Vanessa Atayde, PhD
Scientist, Regulatory Strategy