Delivering drugs to the patients who need them can be an increasingly complex, time-consuming, and costly undertaking, particularly for smaller companies with limited funding. Development time ranges from 10-15 years, and costs to bring a product to market are estimated at over $700 million to $2.7 billion, depending on therapeutic area and other factors.
Other considerations impacting a drug’s development and ultimate commercial success include regulatory and clinical trial complexity, comparative effectiveness, pricing, and reimbursement pressures, among others. These challenges are significant for any company large or small, but may be particularly acute for small biotech firms.
Adding to the complexity of the external environment is the multifaceted interplay of functional activities that must be coordinated and carefully managed during the development process. Once a lead candidate has been identified, the focus for many companies is on starting the first clinical trial. A multitude of other factors and activities that need to be considered are often overlooked. These include, but are not limited to: drug substance and product process and scale up, a multitude of pharmacology and other IND-enabling non-clinical studies, regulatory interactions and submissions, clinical pharmacology and clinical study design and execution, clinical supply manufacturing, packaging, and labeling, commercial product scale-up, real world evidence studies and many others.
PRA Health Sciences offers unparalleled resources, experience, and support through comprehensive drug development services throughout the drug lifecycle, from concept to compound to cure.
Before the first patient is dosed, there are many questions to consider that impact the design of the trial, the overall development program, and ultimately, the success of the product. These include:
- What is the optimal route of administration (for patients and in the marketplace)?
- Does preclinical data support the route of administration or are other studies needed?
- What animal safety and pharmacology studies are needed before exposing subjects?
- Is the current formulation acceptable for animal and eventually human studies?
- Are there any manufacturing or drug supply constraints that impact potential clinical studies?
- What regulatory path is best for my development plan?
- Is my compound eligible for special designation (Orphan Drug, Fast Track, Breakthrough, and Priority Review)?
- Is regulatory agency advice a pre-IND or scientific advice needed or potentially helpful?
Successfully bringing new drugs to market in today’s complex environment requires a roadmap and integrated development plan to ensure efficient and timely execution. A target product profile (TPP) serves as the roadmap and should be developed prior to undertaking design of the clinical development program (yes, even before the first patient is dosed). The content and context of a TPP can range from a few critical components used by the company to craft a development strategy to a comprehensive tool as a format for discussions with regulatory agencies as development progresses (cite FDA guidance). In its simplest form, a TPP needs to contain a few key elements that will drive the overall drug development strategy and supporting clinical program, including, but not limited to:
- Product vision - Describes the long-term goals for the program, such as first-in-class, best-in-class, transforming treatment paradigms, and more. It is a guide and reminder to the development team and key stakeholders why the product is being developed and the goals the company hopes to achieve.
- Indication and patient population - Selecting the indication and patient population involves researching unmet needs in the target disease/therapy area and understanding the patient journey, current treatment guidelines, and the value proposition for both patients and the company.
- Route of administration - While the route of administration may seem straightforward based on the current formulation of the drug candidate, it is important to consider patient perspectives and current therapies. For example, a new drug requiring intravenous infusion at a specialized center in a market where all of the competition is self-administered subcutaneous injections in the patient’s home will have significant market challenges.
- Key safety and efficacy claims - Clearly defining the key efficacy and safety claims is critical to all aspects of trial design, including endpoints, duration, sample size, etc.
- Differentiation from competitor products - Among the most important aspects of the TPP is to clearly describe how the drug being developed will differentiate from the competition. Differentiation can take many forms, including enhanced efficacy, improved safety, price, convenience, compliance, and other considerations.
- Potential economic value - While data and science will drive much of the development program, understanding the potential economic value is important. Items such as competitor pricing, reimbursement, cost of goods, and potential market share, among others, will impact company decision-making and ultimately the development program.
- Key unanswered scientific or clinical questions - Once the above have been agreed upon and documented, the most critical aspect of the TPP can be tackled. Specifically, what are the key unanswered scientific questions the development program must address? These may include questions like:
- Is efficacy greater than the competition?
- Can the drug be formulated and be bioavailable for oral administration?
- Does the candidate drug improve clinical outcomes?Are there any effects of diet, time of day, or other factors which impact the drug’s pharmacokinetics?
Developing a TPP is a cross-functional exercise including research and development, regulatory, preclinical, clinical, marketing, operations, and other key stakeholders within the company.
Successful drug development requires much more than design and execution of a single clinical trial. In an upcoming post, we will explore the importance of an integrated development plan and development of global clinical and regulatory strategies that support the TPP.
Traditionally, pharmaceutical companies have viewed Contract Research Organizations as service providers for clinical trial delivery. In addition to clinical trial support, PRA provides numerous additional services supporting many aspects of drug development.
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