The late phase research environment has changed dramatically in recent years. Regulators in the US and abroad are demanding a more proactive approach to safety and risk management. In light of these changes, sponsors must begin working with their strategic research partners early in the investigative process to anticipate and plan for late phase studies.
2. FOCUSED. TRUSTED. GLOBAL.
Drug Development Life Cycle
MAA /NDA
submitted
Phase I
Phase II/III
Marketing
Authorisation
Phase IIIb
Product Development
Data to address:
Long-term safety
Quality of Life
Cost-Effectiveness
Phase IV
Commercialization
Maximize product
potential
3.
4. FOCUSED. TRUSTED. GLOBAL.
Regulatory Framework
Food and Drug Administration Amendments Act (FDAAA)
o Pre FDAAA-Before 2007
Post-marketing studies or
clinical trials to demonstrate
clinical benefit for drugs
approved under accelerated
approval requirements
Deferred Pediatric studies
where required under the
Pediatric Research Equity Act
Studies of clinical trials to
demonstrate safety and
efficacy in humans to be
conducted at the time of use
of products approved under
the Animal Efficacy Rule.
o Post FDAAA
Assess a known serious risk
related to the use of the drug
Assess signals of serious risk
related to the drug
Identify an unexpected serious
risk when available data
indicates the potential for a
serious risk.
Start early
developing risk
mitigation strategies
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Study types:
Interventional/Observational
o Randomized clinical trials
o Observational epidemiologic studies
o Expanded Access Programs
o Post-Authorization Safety/Efficacy studies
o Health Economics and Outcomes Research
o Registries
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Key Objectives For Successful
Commercialization
o Long-term real-world safety data and risk management
FDA/EMA mandated
Adverse claims
New endpoints, health outcomes, effectiveness
o Maximize market penetration
New comparators and dosage forms
Label extension
o Provide access to yet unregistered drugs
EAP/Named Patient/Compassionate use studies
o Build relationships with prescribers and payers
Brand loyalty
o Health economics and Patient Reported Outcomes
Reimbursement
7. FOCUSED. TRUSTED. GLOBAL.
Outsourcing for Efficiency
o
o
o
o
o
o
Facilitate early planning
Leverage previous learning
Ensure process clarity
Utilize innovative technologies
Streamline project execution
Maintain organizational focus
The result?
Saving
8. FOCUSED. TRUSTED. GLOBAL.
Find the right team
o Therapeutic
expertise
o Local knowledge
and global reach
o Operational
efficiencies
o Market access
expertise
o Greater flexibility
The late phase research environment has changed dramatically in recent years. Regulators in the US and abroad are demanding a more proactive approach to safety and risk management. (click) In light of these changes, sponsors must begin working with their strategic research partners early in the investigative process to anticipate and plan for late phase studies.
Early planning for late phase should optimally begin well before the MAA/NDA is submitted. (Click) The early collection of data for long-term safety, quality of life, and compound efficacy is recommended – (click) to maximize product potential in the commercialization stage of the product.
The regulatory landscape has changed over the past ten years with new regulations and study requirements. Mandates put into place in response to massive drug safety concerns and recalls make for a challenging environment. While the picture can be daunting, early consultation taking into consideration (click) required safety considerations, additional data requirements, and health economics, can make the path smooth.
In the last few years, the industry has seen an increase in focus and guidance from the regulatory bodies on post-marketing safety surveillance and risk management. Compliance with these regulatory rules impacts the race to commercial success. (Click) Before 2007 mostlate phase studies were used strictly to demonstrate clinical benefit. (Click) In 2007 the FDA enacted a set of mandates to assess known safety signals associated with late phase drugs. These mandates have contributed to longer, more complex studies. In 2008 a similar set of mandates was adopted by the European Medicines Agency (EMA). (Click) Developing an early strategy for risk mitigation will enable companies to conduct late phase studies efficiently.
Post marketing studies may take on different forms (Click) but the unique characteristics of both interventional and observational studies should be considered early on - as a basis for designing effective late phase studies.
(Click) To be successful in the current marketplace, the product should have the long term real world safety and effectiveness data addressing questions from the regulators, payers and patients alike. (Click) Embedding post-marketing considerations into earlier phases facilitates further approval of label extensions and new dosage forms. (click) Although not contributing directly to marketing applications, expanded access programs serve an important role in improving access to investigational drugs for patients with serious or immediately life-threatening diseases or conditions who do not have comparable or satisfactory alternative therapies. (click) One key issue for developers is an attention to the many stakeholders involved regarding late phase considerations. (click) Building relationships with these groups helps to establish brand loyalty later on. (click) In turn, a greater understanding of the health economics of a product and its impact not only on clinical but also on economical and humanistic outcomes is critical for a clear pathway to reimbursement.
Outsourcing with study partners drives study efficiency! Teams can start planning for late phase activity years before filing for registration. Embedding late phase considerations into the process before key trial protocols are established ensures optimal research design.(Click)Every study has a learning curve. Engaging a strategic partner at the beginning of the process and working with them throughout means the research team climbs the learning curve only once. What’s more, the team can integrate its growing body of knowledge into each successive phase of the research driving innovation. A long-term strategic relationship ensures consistent communication, process clarity, and research continuity translating to faster, more economical research. (Click) The result? (click)Saving of time and money.
Selecting the right team to produce total quality is critical for long term success. Teams that offer, (click) therapeutic expertise, (click) local knowledge and global reach, (click) operational efficiencies,(click) market access and (click) expertise, and (click) greater flexibility will allow sponsors to maximize commercialization efforts.
New approaches to Late Phase Research yield new views for research and development. (click) Multiple objectives arising from both R&D and Marketing influence the study design. Choosing a flexible, strategically focused CRO will deliver a Win-Win result for all stakeholders: the Sponsor, the CRO and the Patients.