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The benefit to patients for R&D outcomes is first and foremost the new products that will improve their disease status with the least amount of side effects, but it is not just any product (Fig. 3.1). The 2004 Health Report for the United States from the Centers for Disease Control and Prevention documents the major reductions in death rates for heart diseases, cerebrovascular disease, and cancer and states that new drugs and more use of existing drugs were primary contributors to this improvement in health in America [27]. The optimal product should meet an unmet medical need of patients,

significantly advancing the care of patients (e.g., statins in hypercholesterolemia in the 1990s). More untreatable diseases are finding amelioration or improvement through product innovation over the past 20 years (e.g., HIV infections with new classes of antiviral drugs, anemia of kidney disease and cancer with epoietin alfa, and enzyme deficiency diseases such as Gaucher disease with enzyme replacement). A novel product choice has been created because of its unique mechanism of action different from existing products, altering a key newly identified pathophysiologic process for a disease (e.g., aromatase inhibitor [Arimidex®] or oncogene inhibitor [Herceptin®] for breast cancer), or a better side effect profile has been achieved (e.g., Nonsteroidal anti-inflammatory drugs for arthritis versus aspirin). The new product achieves patient care improvement with higher efficacy over prior therapy, becoming a clinically superior or even best-in-class product (e.g., Crestor as a statin for high cholesterol vs. Pravachol; or Gliadel wafers [BCNU drug] for glioblastoma with local cranial placement after brain surgery and higher tumor resolution with less systemic adverse effects). Convenience for the patient and/or health care provider and/or health care system is created (e.g., pegylation of proteins substantially extends the half-life of biological products, such that injectible interferon for hepatitis C can be given weekly, Peg-Intron® or Pegasys®, instead of thrice weekly as Intron®; or filgrastim can be given weekly as Neulasta® for neutropenia correction with cancer chemotherapy, instead of daily as Neupogen®; or, insulin can be given by inhalation vs. injection, which is in clinical trials in 2005; or oral migraine products such as Imitrex® vs. its prior injectable form; or long-acting oral forms with daily vs. multiday dosing, Cardizem LA®).

A second outcome for R&D is relationships of the company with various constituencies that a company needs to be working with or serving. Favorable relationships with these varied audiences will assist not just the reputation of the company, but also good working relationships will enhance the process of product development (Fig. 3.2). The manage ment and research staff at the company will work with their investigators at study sites (institutions), who at some point in time down the road after product approval and marketing will be both product experts and customers as well. Adherence to protocols, flexibility with study changes, and later product usage all may be improved with such good working relationships. Patients and the public favor companies with good reputations as well as good products, for example, in their product choices, in a willingness to be study subjects, by giving them the benefit of the doubt when serious adverse effects arise, and even in stock investments, which is part of the profitability and cash a company needs for its research. Good working and ethical relationships with regulatory authorities help in their receptivity in negotiations with the company for product approvals (even speed of approvals), audits by government, possibly labeling changes, and advertising approvals. Such good working relationships are based on good regulatory practices; for example, following FDA guidances in all operations, not having warning letters regarding adverse effects or advertising, experiencing audits without compliance problems, and having complete NDA or BLA applications in data or manufacturing. Payer relationships foster favorable payment policies and procedures, when a company tries and meets payer needs as much as possible, such as indigent care programs (free drug), or payment assistance programs for providers, or applicable cost-benefit and related data being provided. Providers are the prescribers and gatekeepers for product usage. With most products, multiple choices exist to treat a disease. A provider or their institution will work with a company and prescribe their product that of course is safe and effective, but also meets their needs for information, or education, or reimbursement assistance. These providers and the investigators who are experts for a disease are considered thought leaders, who advise the company about the diseases and products. Most companies (about 88%) have interactive programs in place with thought leaders early in research during at least phase 2 research. The legislative and executive branches of the government write the laws governing research investments, product approvals, and access to products (Medicare, Medicaid, VA, DOD), for example. A company with good relationships will have the opportunity to be consulted and influence outcomes in these and other areas, which assist in product development. Finally, the investment community needs as much information as possible about a company to make the best decisions for recommendations for stock purchases by their clients. A robust product pipeline of a company and progress of it over time are closely followed by investors as measures of corporate success. Furthermore, companies that are cooperative without compromising confidentiality will receive the benefit of the doubt if problems arise down the road in their R&D.

Relationships between a company and the university are core to the successful function of an R&D operation and carry benefits and risks for both sides (Fig. 3.3). The company receives direct R&D benefits and potential future benefits as well from the university in their collaboration. For their clinical trials, access to investigators to conduct the study and access to patients as subjects for the trials are obvious absolute needs for a company. Besides these clinicians having both therapeutic/disease expertise, their research expertise can help get the work done more effectively. Even before the trial starts, the university experts, often called thought leaders, provide consultation to R&D for the optimal product profile in a particular disease and study designs. Ultimately, the NDA completion requires this collaboration to create the data, reports, and possibly even FDA testimony. While a study is being conducted, the institutional staff of health care professionals (HCPs) is being given an education about this new product in a well-controlled situation. The pharmacy often group as the investigational distributor and quality control person in the institution. This working relationship can give the pharmacy, a gatekeeper for product usage, knowledge about and experience with the product, which may help future discussions and deliberations toward formulary review and approvals after the product's marketing. Data can be shared between the university and the company, including the data required for the study and possibly other research data, product usage, or patient care information. Data sharing may assist the investigator in furthering their research and the company with better knowledge of their product's fit in the university setting. A pivotal study in phase 2 or 3 for an NDA usually involves a highly selective group of patients, based on a strict study design, which may not be fully representative of typical patients. The university can additionally provide health outcome information beyond such clinical data, that is, practical health care information to be used in pharmacoeco-nomic assessments about the care of these patients (e.g., charges for tests and procedures, or quality of life).

On the other side of the ledger, risks or added demands exist for a company. Research costs in pivotal studies include grants to the university and investigator for patient accrual at costs of $5,000 to $15,000 per patient, plus institutional overhead of 25% to 75%. The study's patient accrual may go too slowly or there just may be an inadequate number of patients at one institution, straining their relationship. Study data is owned by the company as is the norm in study contracts, especially for investigational products. With major new findings for a product, especially unexpected outcomes beyond the study design, universities desire to patent their discoveries and share the downstream revenues, creating legal battles and even lost revenue for a company. Confidentiality of the product and study data is important for the NDA, patent issues, and competition issues with other companies. Timing of release and placement of new data are major issues with the study data for companies, which needs to be negotiated with investigators. Publications and their reception by the medical community can have significant impact on the success of an NDA, future prescription potential, patenting especially of new indications, and even stock investment impact. Publication rights (independence in content, publishability, and placement) are concerns of the research faculty, but most companies are flexible. They may require company preparation of the draft

Company " Benefits:

o Access to patients o Access to investigators c Access to thought leaders c NDA trial completion c Data sharing from university c Health outcome data c Work with pharmacy c Educate practitioners c Formulary opportunity « Risks: c Cost c Slow patient accrual c Patents c Confidentiality of NDA data

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