FirstInClass versus BestInClass Need Novelty Competitive Advantage

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r Unmet medical need (Stand alone vs Add-on)

r Market (# addressable patients; 5 year revenue)

r Competition (Products, Company, Tactics)

Category & MOA (Novelty)

k Efficacy (Competitive advantage)

k Dose/Schedule (Practical)

k Formulation (Convenience)

k Safety (Less AEs)

k Pharmacokinetics (Disease fit)/Drug Interactions (Less)

k Economics/reimbursement climate (Favorable)

Fig. 2.19. The Key Factors in a Product Profile indication, and competitive product issues. This profile is the target for research to try to achieve in formulation, indications, efficacy, dosing, and adverse effects. As studies are done and more information is known about the company's product, the profile incorporates these data, and then the product profile is for the product being developed including its good and bad properties.

Three goals serve as overriding considerations for all the factors in the product profile; need, novelty, and competition; that is, is such a product needed to treat this disease? are we a unique and innovative product? and do we have advantages over competitive products? Positive answers on these three questions will increase the scientific acceptance in the medical community and health systems and substantially expand the market and possible revenues over the life of the product. Also, a particular philosophy regarding a new family of products being developed by a company must be addressed, that is, do we want to be first-in-class to market or later and best-in-class. First-in-class historically has the prime marketing position, with more rapid and complete formulary acceptance and then loyalty of prescribers and health systems. In some cases, a breakthrough product changes the treatment paradigm, such that the medical community has to be educated to facilitate the uptake of the new approach and put the new benefits in perspective. However, a best-in-class product has the benefit of knowing the product weaknesses of the first product that can be exploited. The second or third product can have significant advantages in formulation, dosing, efficacy, or safety, such that prescribers switch to the best product. The benefit of being first-in-class has decreased also because the time for market approval for the first to the second product was reduced from 8.2 years in the 1970s to 5.5 years in 1980s to 1.8 years in 1995-1998 time period [7, 17, 22].

Project or product planning in R&D is a very detailed oriented process, certainly covering all the critical pathway studies. The project plans must be quite comprehensive incorporating the work of all departments, worldwide efforts, all staff involved, any budget issues, and timelines for all projects (Fig. 2.20). Actually, every work activity that significantly r PreClinical (Tox - P'col - Pkin) r Formulations/Pharmacokinetics r Manufacturing:

o Clinical trial & Marketed product p Process engineering & scale-up o Packaging engineering p Quality control (analyses, stability)

p 5 year plan, inventory, SKUs o Distribution channels r Clinical Trials:

p Indications, dosing p PIs, sites, patients o Data management, stats, writing p Phase 1, 2, 3 & 4, QOL, economic r Regulatory: P IND, NDA P FDA meetings, Audits

Fig. 2.20. Project Plans (WW, All groups, Timing, All Resources)

impacts any section of the package insert, the regulatory package, NDA/CTD, and any marketing launch program must be integrated and tracked. The apparently minor change in the diluents of an injectable product could cause an NDA to be not approved because their potential side effects or their impact on stability of the active drug was not sufficiently addressed. In marketing, launch of the product will require often extra analyses of clinical data to assist in educational or promotional material preparation, and market research will assist R&D in the types and amount of research to be done. One small study in one country anywhere in the world may surface a serious adverse experience that may require further evaluation and could hold up a regulatory submission. Figure 2.20 lists for us 10 major areas in bullet points and more than 50 work areas or items to be done and tracked by project planners for the product teams, some of which will be included in portfolio analyses. The timing of the different projects in pre-clinical, manufacturing, clinical, marketing, and other areas need to be integrated and especially sequenced because one project may depend on information from another in a completely different division of the company.

Research contributes the mechanism of action, assessment of adverse experiences in animal models, the preliminary pharmacology, toxicology, and pharmacokinetics in animals or in vitro, formulation workup, and stability studies. Manufacturing provides the manufacturing process and of course the product during clinical trials. Process engineering and product packaging needs to be addressed early during the clinical development period, because minor product changes and stability issues can create breakdown products (ingredients) and impact clinical and regulatory requirements. Later, manufacturing and marketing of the product with product scale-up is addressed further by process engineering and product packaging, along with needs for inventory of product and its control, an sku (shelf keeping unit), and a distribution system. Clinical development work is r Safety r Market research: P Disease & Product P Pricing & Sales p Competition r Business plans: P Publications P Thought-leaders o Reimbursement c Launch [market programs, medical, staffing/sales] p Partnerships p Life cycle r Patent status & Trademarks r Financial:

p COGS, Budgets, Costs o P&Ls, Revenues, NPV

substantial as expected: all the necessary studies (investigators, sites, forms, and data) and reports for approval, labeling, and marketing (publications, education, and promotion) and safety labeling and reports. Drug performance may deviate from the original hypothesis and necessitate new plans and studies to be done. The critical role of safety data in an NDA or BLA warrants special attention through a separate medical unit for safety in clinical development focusing on recording, analyzing, and reporting on adverse experiences with all pipeline and marketed products. Internal audits are done by a quality assurance group for both all processes (procedures and operational guidelines) and outcomes of R&D for an NDA. Regulatory manages the applications to and interactions with regulatory authorities (meetings, letters, calls, and audits). Regulatory also performs an oversight role for compliance with government regulations.

Marketing performs all the market research analyses and generates related reports for disease, product profile, competition, pricing, and potential sales, identifies thought leaders and investigators, and produces for launch all the plans for publications, educational programs, promotion and advertising, and reimbursement. The product team is the recipient for the analyses from marketing and the provider of data for product to be marketed. The team also addresses the first and subsequent indications, integrated into their goals and timing as necessary. Law department is responsible for the product patent with research and manufacturing providing the patent data; they assesses competitive patents; and they ensure compliance with laws. Finance group tracks and examines budgets, expenditures, cost of goods, profit and loss (P&Ls), and revenues for a team. Resources of staffing are evaluated by departments and/or human resources to provide enough of the best possible people with training as needed. A company faces a big challenge making sure everyone is up to date on both the clinical and regulatory science to enable them to contribute to optimal performance. External resources are used most often to conduct many of the development projects by clinical research organizations, and also market research is done by outside companies with appropriate access to data and expertise. Life cycle plans are done by the team and PPM from discovery to approval and throughout marketing to maximize the franchise, which will be discussed later. in global organizations, both clinical researchers and medical affairs practitioners provide critical input and feedback about the unique features and practices in their individual countries.

PPM depends on the availability of systems and databases to gather and process the data regarding product status. "Enabling technologies" are the systems, equipment, or processes that are intended to improve operational effectiveness of department functions, such that more and better products can be developed faster and at less cost (Fig. 2.21). Many of them are needed for PPM, and actually, PPM is an enabling technology for the planning, tracking, and analysis functions. For example, e-clinical is electronic data capture for the data in clinical trials to facilitate ongoing analyses and decision making by the R&D management team. PPM is central to coordinating the collection of the information and the data for progress reports in clinical trial progress. Currently, research is going to develop spp. tools that will directly both identify and collect patient data (e.g., blood pressure readings) and send it to the company's study data center without handling by the site. This technology will facilitate decision making and save valuable time. informatics can be structured to collect, store, and analyze data from any part of the organization. informatics certainly can incorporate the global product plans with milestones for and progress on study plans, regulatory milestones, timelines, safety reports, costs, and future projected sales.

One of the major challenges faced by the pharmaceutical industry is how to optimally handle the massive amount of information collected by all levels of R&D. A global standard for the collection, organization, storage, and analysis opens the door for substantial enhancement in organizational performance. With global standards in place, a data warehouse can be established, which modelers can draw on to conduct simulations designed to assist in evaluating program potential and greatly improve the design efficiency of individual studies. The building of these data warehouses requires collaboration and communication across the R&D organization. Informatics in the research arena can store, manipulate, and analyze varied databases for genomics, pharmacology, receptors, ligands, proteomics, structure-activity relationships, to help identify targets and leads for further research. High-throughput screening (HTS) in lead analyses is estimated to have increased discovery rate of molecules by 6% from 1994 to 1998 and by 11% from 1998 to 2002 [20].

Other enabling technologies are considered to improve efficiency in establishing effectiveness and safety in clinical trials by using biomarkers and surrogate markers of efficacy early in development, even in the preclinical stage, to kill the poor performing product candidates sooner and advance the more likely winners faster. Conducting clinical trials by having more practical study protocols and with better use of outsourcing are enabling technologies for efficiency, too, which can reduce costs of operations for trials by getting only needed data (less) collected faster that still meets NDA/CTD requirements for a disease [8, 9, 11, 17, 19, 22, 44, 46, 47].

PPM monitors decision points in research and development, also called decision gates, related to milestones and timelines in a product's advance toward approval. A paramount area of governance is decision making, including decision authority, decision criteria, time frames, communication of decisions (informing and consulting), performance management, and incentives, all of which needs to be engaged in PPM. A product team presents and must show the data,

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