1) The document provides an overview and approach to assessing life science technologies, with an emphasis on quantitative and qualitative assessments of potential product leads and their path to market.
2) It discusses conducting thorough due diligence on technologies, including evaluating the technology itself, intellectual property, developmental stage, path to market, competition, and assessing the technology's value.
3) Key areas of focus for technology assessment include the scientific basis of the technology, intellectual property protection, required clinical and regulatory paths, competition, market potential and dynamics, and difficulties estimating early-stage technology value. The document provides resources for evaluation.
About UCI Applied Innovation:
UCI Applied Innovation is a dynamic, innovative central platform for the UCI campus, entrepreneurs, inventors, the business community and investors to collaborate and move UCI research from lab to market.
About the Cove @ UCI:
To accelerate collaboration by better connecting innovation partners in Orange County, UCI Applied Innovation created the Cove, a physical, state-of-the-art hub for entrepreneurs to gather and navigate the resources available both on and off campus. The Cove is headquarters for UCI Applied Innovation, as well as houses several ecosystem partners including incubators, accelerators, angel investors, venture capitalists, mentors and legal experts.
Follow us on social media:
Facebook: @UCICove
Twitter: @UCICove
Instagram: @UCICove
LinkedIn: @UCIAppliedInnovation
For more information:
cove@uci.edu
http://innovation.uci.edu/
This document discusses strategies for pharmaceutical research and development (R&D) and drug development. Some key points:
- Developing a new drug takes 15-16 years on average and costs around $800 million, with 75% of costs attributed to high failure rates. Only 1 in 5-10 drugs that enter development make it to market.
- Patent protection is important for pharmaceutical innovation given the high costs and risks of drug development. Without patent exclusivity periods, companies would have no way to recoup their investments.
- Competition in the pharmaceutical industry drives improvements to existing drugs like increased efficacy, reduced side effects, better dosing, and new delivery methods. This benefits patients.
How early is too early for pharmaceutical market insights and product forecasts. A discussion on pipeline product research during early product development
The document discusses the FDA's Critical Path Initiative, which aims to improve the process of drug development and evaluation. It notes that the current drug development process is becoming a bottleneck. The initiative seeks to identify specific projects through a science-driven, shared effort between stakeholders to improve efficiency. The FDA must lead efforts to question assumptions that slow new product development and identify more efficient alternatives. The document also discusses two proposed projects - to create a "current state" process map to identify opportunities for improvement, and to develop a knowledge management system using ICH Q8 to ensure appropriate connectivity between all regulatory disciplines involved in drug development and review.
Regulatory strategy for medical device start-upsRina Nir
The document discusses regulatory considerations for medical device startups. It notes that the regulatory environment will significantly impact business plans by influencing budgets, timelines, staffing needs and more. Startups must thoroughly assess their regulatory situation and strategy to navigate the complex approval processes in both the US and EU. Later slides provide more details on specific regulatory pathways, standards, and trends to consider for medical device development.
IBM Watson: Create the Future Today | Neil Sahota | Lunch & LearnUCICove
About UCI Applied Innovation:
UCI Applied Innovation is a dynamic, innovative central platform for the UCI campus, entrepreneurs, inventors, the business community and investors to collaborate and move UCI research from lab to market.
About the Cove @ UCI:
To accelerate collaboration by better connecting innovation partners in Orange County, UCI Applied Innovation created the Cove, a physical, state-of-the-art hub for entrepreneurs to gather and navigate the resources available both on and off campus. The Cove is headquarters for UCI Applied Innovation, as well as houses several ecosystem partners including incubators, accelerators, angel investors, venture capitalists, mentors and legal experts.
Follow us on social media:
Facebook: @UCICove
Twitter: @UCICove
Instagram: @UCICove
LinkedIn: @UCIAppliedInnovation
For more information:
cove@uci.edu
http://innovation.uci.edu/
FDA Regulatory Considerations for Life Sciences CompaniesMichael Swit
April 2008 presentation to Swedish-American Chamber of Commerce Entrepreneurial Days on FDA issues for product development, including focus on:
Planning, approval process, barriers to entry
How to Make Postmarket Surveillance More Cost EffectiveApril Bright
When it comes to postmarket surveillance (PMS), it’s common for the costs to outweigh the value. But, by working with the right team, you’ll be able to execute a study that maximizes return on investment and minimizes the financial impact of conducting further observational research. Postmarket study challenges that must be addressed include enrollment delays, patient attrition, long-term follow-up, resourcing demands and global payor requirements. This session will provide a case study of one orthopaedic company’s seamless transition between postmarket approval and post-approval studies.
About UCI Applied Innovation:
UCI Applied Innovation is a dynamic, innovative central platform for the UCI campus, entrepreneurs, inventors, the business community and investors to collaborate and move UCI research from lab to market.
About the Cove @ UCI:
To accelerate collaboration by better connecting innovation partners in Orange County, UCI Applied Innovation created the Cove, a physical, state-of-the-art hub for entrepreneurs to gather and navigate the resources available both on and off campus. The Cove is headquarters for UCI Applied Innovation, as well as houses several ecosystem partners including incubators, accelerators, angel investors, venture capitalists, mentors and legal experts.
Follow us on social media:
Facebook: @UCICove
Twitter: @UCICove
Instagram: @UCICove
LinkedIn: @UCIAppliedInnovation
For more information:
cove@uci.edu
http://innovation.uci.edu/
This document discusses strategies for pharmaceutical research and development (R&D) and drug development. Some key points:
- Developing a new drug takes 15-16 years on average and costs around $800 million, with 75% of costs attributed to high failure rates. Only 1 in 5-10 drugs that enter development make it to market.
- Patent protection is important for pharmaceutical innovation given the high costs and risks of drug development. Without patent exclusivity periods, companies would have no way to recoup their investments.
- Competition in the pharmaceutical industry drives improvements to existing drugs like increased efficacy, reduced side effects, better dosing, and new delivery methods. This benefits patients.
How early is too early for pharmaceutical market insights and product forecasts. A discussion on pipeline product research during early product development
The document discusses the FDA's Critical Path Initiative, which aims to improve the process of drug development and evaluation. It notes that the current drug development process is becoming a bottleneck. The initiative seeks to identify specific projects through a science-driven, shared effort between stakeholders to improve efficiency. The FDA must lead efforts to question assumptions that slow new product development and identify more efficient alternatives. The document also discusses two proposed projects - to create a "current state" process map to identify opportunities for improvement, and to develop a knowledge management system using ICH Q8 to ensure appropriate connectivity between all regulatory disciplines involved in drug development and review.
Regulatory strategy for medical device start-upsRina Nir
The document discusses regulatory considerations for medical device startups. It notes that the regulatory environment will significantly impact business plans by influencing budgets, timelines, staffing needs and more. Startups must thoroughly assess their regulatory situation and strategy to navigate the complex approval processes in both the US and EU. Later slides provide more details on specific regulatory pathways, standards, and trends to consider for medical device development.
IBM Watson: Create the Future Today | Neil Sahota | Lunch & LearnUCICove
About UCI Applied Innovation:
UCI Applied Innovation is a dynamic, innovative central platform for the UCI campus, entrepreneurs, inventors, the business community and investors to collaborate and move UCI research from lab to market.
About the Cove @ UCI:
To accelerate collaboration by better connecting innovation partners in Orange County, UCI Applied Innovation created the Cove, a physical, state-of-the-art hub for entrepreneurs to gather and navigate the resources available both on and off campus. The Cove is headquarters for UCI Applied Innovation, as well as houses several ecosystem partners including incubators, accelerators, angel investors, venture capitalists, mentors and legal experts.
Follow us on social media:
Facebook: @UCICove
Twitter: @UCICove
Instagram: @UCICove
LinkedIn: @UCIAppliedInnovation
For more information:
cove@uci.edu
http://innovation.uci.edu/
FDA Regulatory Considerations for Life Sciences CompaniesMichael Swit
April 2008 presentation to Swedish-American Chamber of Commerce Entrepreneurial Days on FDA issues for product development, including focus on:
Planning, approval process, barriers to entry
How to Make Postmarket Surveillance More Cost EffectiveApril Bright
When it comes to postmarket surveillance (PMS), it’s common for the costs to outweigh the value. But, by working with the right team, you’ll be able to execute a study that maximizes return on investment and minimizes the financial impact of conducting further observational research. Postmarket study challenges that must be addressed include enrollment delays, patient attrition, long-term follow-up, resourcing demands and global payor requirements. This session will provide a case study of one orthopaedic company’s seamless transition between postmarket approval and post-approval studies.
Orphanetics aims to create new biotechnology companies focused on rare and ultra-rare diseases by evaluating therapeutic opportunities, conducting de-risking experiments through collaborations, and establishing new venture-backed companies upon demonstrating proof of concept in man for priority rare disease projects.
How to Work Effectively with Research Teams in New Product PlanningAnthony Russell
Presented at the 3rd New Product Planning Summit. The presentation was designed to help professionals in New Product Planning to present a case for why commercial strategy input is needed early in the process of developing new therapeutics. The presentation also includes suggested approaches and tools to help with effective engagement with Research teams.
Orphanetics aims to create new biotechnology companies focused on rare and ultra-rare diseases by evaluating therapeutic opportunities, conducting de-risking experiments, and seeking candidates compatible with forming new ventures; it utilizes a collaborative model working with various organizations to identify assets, de-risk programs, and accelerate the formation of new companies developing therapeutics for diseases with unmet medical needs.
FDA 2013 Clinical Investigator Training Course: Issues in Clinical Trials Des...MedicReS
FDA 2013 Clinical Investigator Training Course: Issues in Clinical Trials Designs for Devices
Owen Faris, Ph.D.,Deputy Director, Division of Cardiovascular Devices, Office of Device Evaluation, CDRH, FDA
The document discusses a new technology for guiding cryoablation of prostate cancer using optoacoustic tomography (OAT) to provide high resolution temperature monitoring and visualization of the ice ball, aiming to reduce rectal damage, impotence, and cancer recurrence. It provides estimates of the total available market, served available market, and target market for the technology, projecting sales of 600 units for $84 million. Customer interviews conducted during an I-Corps program helped shift the business model from direct sales to physicians to partnerships with cryoablation providers.
Competitive intelligence in the pharmaceutical industry involves systematically collecting and analyzing information about competitors' objectives, strategies, capabilities, and assumptions. It benefits pharmaceutical companies by incorporating external market forces into strategic planning, enhancing various business functions, and minimizing decision risk. Competitive intelligence for pharma focuses on monitoring competitors' product development, pricing strategies, and commercial priorities throughout a product's lifecycle from research to marketing. It is important for pharmaceutical companies to establish strategic competitive intelligence functions that reach across departments to gather and share critical competitor information.
Il processo di innovazione in atto: dalle strategie di brevettazione al fundr...Sardegna Ricerche
L'intervento di Rachele Berria (Global Vice President Primary Care Medical Unit Sanofi) in occasione dell'evento "Il valore della ricerca biomedica" che si è tenuto a Pula (CA) il 3 aprile 2019.
Cybele is developing a Huntington's disease cell model using induced pluripotent stem cells to enable drug discovery. The team aims to create affected medium spiny neurons in vitro that can be used by academic researchers and pharmaceutical companies to identify potential drug targets and test compounds. Cybele is exploring commercializing purified cell populations for research or providing screening services using their cell model with partners. The company's goal is to accelerate Huntington's disease drug development and reduce costs by enabling earlier testing and failure of non-viable compounds.
The Cell Therapy Catapult was established to help grow the UK cell therapy industry by providing facilities, expertise, and funding for collaboration between researchers and industry. It has laboratories and teams working on process development, clinical trials, and business development. In its first year it has over 60 staff and is engaged in both small and large collaborative projects. The goal is to deliver life-changing cell therapies and investible projects that help establish UK leadership in this industry.
New Product Planning Strategies to Ensure Organizational Robustness for LaunchAnthony Russell
- New product planning strategies are important for small to medium sized biopharma companies to ensure organizational robustness for product launches.
- Early commercial planning can impact a product's launch success by clearly defining its value proposition and positioning relative to competitors.
- A framework for evaluating new targets and indications involves assessing factors like clinical development feasibility, regulatory pathways, market potential, and risks. Strategic segmentation of patient populations also guides efficient planning.
- Lifecycle management strategies like pursuing new indications can extend revenue and provide competitive barriers to improve return on investment.
How and When to Kill a Program in New Product PlanningAnthony Russell
Presented at the 4th New Product Planning Summit in Boston (Dec 2 -3 , 2019). Presentation covers why weak programs should be cut from pharmaceutical and biotech pipelines, what defines a "weak" program, and describes objective methods to evaluate programs to help prioritize assets.
The document describes the evolution of a biotech startup's efforts to develop a new drug to treat spinal cord injury (SCI). Initially, the team was optimistic about validating the need and market potential. However, through customer interviews, they learned big pharma is not interested in early-stage SCI drugs due to previous failures. The team then developed a new 36-month plan to generate more rigorous preclinical data for FDA approval and attract partners. They also identified new financing options and pivoted to explore opportunities in multiple sclerosis based on stronger customer interest.
This document discusses Contract Research Organizations (CROs) and their role in supporting pharmaceutical clinical trials. It provides the following key points:
- CROs are service organizations that conduct clinical trials and other drug development work on behalf of pharmaceutical companies. They help companies that lack sufficient resources or expertise to conduct all required trials themselves.
- Selecting the right CRO is important for a successful clinical trial. Sponsors should clearly define study specifications, evaluate a CRO's capabilities and compatibility, and consider cost. Ongoing management of the sponsor-CRO relationship also impacts success.
- Common issues like selecting an inappropriate CRO, unclear study specifications from the sponsor, or poor management can lead
Regulatory Challenges In Executing Clinical Trials GloballyMichael Swit
This document discusses regulatory challenges for conducting successful global clinical trials. It covers keys to regulatory success such as standards of care, ethical standards, and following essential sponsor procedures. It also provides case studies on key regulatory issues and considerations for specific countries and regions, including Europe, India, China, Indonesia, and more. The document emphasizes the importance of properly designing global clinical trials to account for cultural, infrastructure, and other local factors in different countries and regions. It also focuses on the risks of non-compliance and fraud at clinical sites that can undermine trial integrity.
Harrison Hayes Identifying Innovation OpportunitiesBrand Acumen
Harrison Hayes utilizes various proprietary and non-proprietary tools like the Transforium database of 3 million technologies and the PIPV analysis to identify innovation opportunities, assess their value, and make strategic recommendations. They option promising early-stage assets, analyze their potential over different time periods, and sell or license the options to generate returns for investors. Harrison Hayes was founded by Bill Smith to provide strategic research and advisory services to life science clients through an extensive network and aggressive research team.
Lifecycle Management in the Pharmaceutical IndustryAnthony Russell
This document discusses lifecycle management strategies in the pharmaceutical industry. It begins by outlining the key drivers for effective lifecycle management, including the high costs of drug development and need to extend patent protection. Several lifecycle management strategies are then described, such as developing new indications, formulations, delivery methods, or integrating digital health tools. The document emphasizes that lifecycle management planning should begin early and consider factors such as clinical feasibility, regulatory pathways, intellectual property and commercial impacts when selecting strategies.
This document discusses outsourcing bioanalytical work and provides guidance on contracting with external laboratories. It notes that 50% of R&D expenditures are external and clinical bioanalysis is outsourced at 90%. Reasons to outsource include flexible workforces, expertise, and GLP compliance. Aspects that can be outsourced include methods, sample analysis, and troubleshooting. Care must be taken in selecting qualified laboratories based on location, technical capabilities, people, culture, costs and quality systems. Managing outsourced work requires clear communication, site visits, and building relationships. Outsourcing can succeed by knowing why and what to outsource, selecting the right partners, and maintaining communication.
Making Key Decisions in New Product Planning When “Perfect” Information is No...Anthony Russell
Presentation given at New Product Planning Summit 2021.
Learning Objectives:
* Review the types of decisions typically made in New Product Planning
* Discuss the nature of information available to support decision-making in New Product Planning
* Review the impact and context of decision-making in New Product Planning
* Review potential approaches to assist in New Product Planning decision-making
Haapalinna antti 2nd annual r&d process excellence summit 22nd october15...Antti Haapalinna
The document discusses performance management at Orion Pharma R&D. It provides background on Orion Corporation and describes Orion Pharma R&D's pipeline and effectiveness before 2007, which was thin. It then discusses how Orion Pharma R&D underwent a comprehensive change process from 2007-2015 that led to improved performance. This included conducting a self-diagnosis, developing a new strategic direction, focusing on culture change, reorganizing structures, and defining a desired culture. As a result, Orion Pharma R&D's research and clinical pipelines significantly expanded and strengthened between 2007-2015.
Moore Expedited Devel & Rev Of New Drugs 2010 (L)jmoore89
This document summarizes strategic regulatory options for expediting drug development and approval through the FDA. It discusses expedited review pathways including Subpart E for life-threatening diseases and Subpart H (accelerated approval) for serious or life-threatening conditions. Key elements covered are early consultation, use of surrogate endpoints in phase 2 trials, post-approval studies, and consideration of disease severity and treatment alternatives in the risk-benefit assessment. The goal is to provide patients sooner access to promising new therapies while maintaining adequate evidence of safety and efficacy.
This document describes the design and development of medical devices including hip and knee prostheses and related instruments. It discusses the 3D modeling and technical documentation created for the prostheses and instruments. It also details the complete development and manufacturing of total hip uncemented and cemented systems, including femoral stems, acetabular cups, liners, and various surgical instruments. Additionally, it outlines the concept and redesign of shark pliers, including sketches, ergonomic and functional optimization, and the final manufactured prototype.
Orphanetics aims to create new biotechnology companies focused on rare and ultra-rare diseases by evaluating therapeutic opportunities, conducting de-risking experiments through collaborations, and establishing new venture-backed companies upon demonstrating proof of concept in man for priority rare disease projects.
How to Work Effectively with Research Teams in New Product PlanningAnthony Russell
Presented at the 3rd New Product Planning Summit. The presentation was designed to help professionals in New Product Planning to present a case for why commercial strategy input is needed early in the process of developing new therapeutics. The presentation also includes suggested approaches and tools to help with effective engagement with Research teams.
Orphanetics aims to create new biotechnology companies focused on rare and ultra-rare diseases by evaluating therapeutic opportunities, conducting de-risking experiments, and seeking candidates compatible with forming new ventures; it utilizes a collaborative model working with various organizations to identify assets, de-risk programs, and accelerate the formation of new companies developing therapeutics for diseases with unmet medical needs.
FDA 2013 Clinical Investigator Training Course: Issues in Clinical Trials Des...MedicReS
FDA 2013 Clinical Investigator Training Course: Issues in Clinical Trials Designs for Devices
Owen Faris, Ph.D.,Deputy Director, Division of Cardiovascular Devices, Office of Device Evaluation, CDRH, FDA
The document discusses a new technology for guiding cryoablation of prostate cancer using optoacoustic tomography (OAT) to provide high resolution temperature monitoring and visualization of the ice ball, aiming to reduce rectal damage, impotence, and cancer recurrence. It provides estimates of the total available market, served available market, and target market for the technology, projecting sales of 600 units for $84 million. Customer interviews conducted during an I-Corps program helped shift the business model from direct sales to physicians to partnerships with cryoablation providers.
Competitive intelligence in the pharmaceutical industry involves systematically collecting and analyzing information about competitors' objectives, strategies, capabilities, and assumptions. It benefits pharmaceutical companies by incorporating external market forces into strategic planning, enhancing various business functions, and minimizing decision risk. Competitive intelligence for pharma focuses on monitoring competitors' product development, pricing strategies, and commercial priorities throughout a product's lifecycle from research to marketing. It is important for pharmaceutical companies to establish strategic competitive intelligence functions that reach across departments to gather and share critical competitor information.
Il processo di innovazione in atto: dalle strategie di brevettazione al fundr...Sardegna Ricerche
L'intervento di Rachele Berria (Global Vice President Primary Care Medical Unit Sanofi) in occasione dell'evento "Il valore della ricerca biomedica" che si è tenuto a Pula (CA) il 3 aprile 2019.
Cybele is developing a Huntington's disease cell model using induced pluripotent stem cells to enable drug discovery. The team aims to create affected medium spiny neurons in vitro that can be used by academic researchers and pharmaceutical companies to identify potential drug targets and test compounds. Cybele is exploring commercializing purified cell populations for research or providing screening services using their cell model with partners. The company's goal is to accelerate Huntington's disease drug development and reduce costs by enabling earlier testing and failure of non-viable compounds.
The Cell Therapy Catapult was established to help grow the UK cell therapy industry by providing facilities, expertise, and funding for collaboration between researchers and industry. It has laboratories and teams working on process development, clinical trials, and business development. In its first year it has over 60 staff and is engaged in both small and large collaborative projects. The goal is to deliver life-changing cell therapies and investible projects that help establish UK leadership in this industry.
New Product Planning Strategies to Ensure Organizational Robustness for LaunchAnthony Russell
- New product planning strategies are important for small to medium sized biopharma companies to ensure organizational robustness for product launches.
- Early commercial planning can impact a product's launch success by clearly defining its value proposition and positioning relative to competitors.
- A framework for evaluating new targets and indications involves assessing factors like clinical development feasibility, regulatory pathways, market potential, and risks. Strategic segmentation of patient populations also guides efficient planning.
- Lifecycle management strategies like pursuing new indications can extend revenue and provide competitive barriers to improve return on investment.
How and When to Kill a Program in New Product PlanningAnthony Russell
Presented at the 4th New Product Planning Summit in Boston (Dec 2 -3 , 2019). Presentation covers why weak programs should be cut from pharmaceutical and biotech pipelines, what defines a "weak" program, and describes objective methods to evaluate programs to help prioritize assets.
The document describes the evolution of a biotech startup's efforts to develop a new drug to treat spinal cord injury (SCI). Initially, the team was optimistic about validating the need and market potential. However, through customer interviews, they learned big pharma is not interested in early-stage SCI drugs due to previous failures. The team then developed a new 36-month plan to generate more rigorous preclinical data for FDA approval and attract partners. They also identified new financing options and pivoted to explore opportunities in multiple sclerosis based on stronger customer interest.
This document discusses Contract Research Organizations (CROs) and their role in supporting pharmaceutical clinical trials. It provides the following key points:
- CROs are service organizations that conduct clinical trials and other drug development work on behalf of pharmaceutical companies. They help companies that lack sufficient resources or expertise to conduct all required trials themselves.
- Selecting the right CRO is important for a successful clinical trial. Sponsors should clearly define study specifications, evaluate a CRO's capabilities and compatibility, and consider cost. Ongoing management of the sponsor-CRO relationship also impacts success.
- Common issues like selecting an inappropriate CRO, unclear study specifications from the sponsor, or poor management can lead
Regulatory Challenges In Executing Clinical Trials GloballyMichael Swit
This document discusses regulatory challenges for conducting successful global clinical trials. It covers keys to regulatory success such as standards of care, ethical standards, and following essential sponsor procedures. It also provides case studies on key regulatory issues and considerations for specific countries and regions, including Europe, India, China, Indonesia, and more. The document emphasizes the importance of properly designing global clinical trials to account for cultural, infrastructure, and other local factors in different countries and regions. It also focuses on the risks of non-compliance and fraud at clinical sites that can undermine trial integrity.
Harrison Hayes Identifying Innovation OpportunitiesBrand Acumen
Harrison Hayes utilizes various proprietary and non-proprietary tools like the Transforium database of 3 million technologies and the PIPV analysis to identify innovation opportunities, assess their value, and make strategic recommendations. They option promising early-stage assets, analyze their potential over different time periods, and sell or license the options to generate returns for investors. Harrison Hayes was founded by Bill Smith to provide strategic research and advisory services to life science clients through an extensive network and aggressive research team.
Lifecycle Management in the Pharmaceutical IndustryAnthony Russell
This document discusses lifecycle management strategies in the pharmaceutical industry. It begins by outlining the key drivers for effective lifecycle management, including the high costs of drug development and need to extend patent protection. Several lifecycle management strategies are then described, such as developing new indications, formulations, delivery methods, or integrating digital health tools. The document emphasizes that lifecycle management planning should begin early and consider factors such as clinical feasibility, regulatory pathways, intellectual property and commercial impacts when selecting strategies.
This document discusses outsourcing bioanalytical work and provides guidance on contracting with external laboratories. It notes that 50% of R&D expenditures are external and clinical bioanalysis is outsourced at 90%. Reasons to outsource include flexible workforces, expertise, and GLP compliance. Aspects that can be outsourced include methods, sample analysis, and troubleshooting. Care must be taken in selecting qualified laboratories based on location, technical capabilities, people, culture, costs and quality systems. Managing outsourced work requires clear communication, site visits, and building relationships. Outsourcing can succeed by knowing why and what to outsource, selecting the right partners, and maintaining communication.
Making Key Decisions in New Product Planning When “Perfect” Information is No...Anthony Russell
Presentation given at New Product Planning Summit 2021.
Learning Objectives:
* Review the types of decisions typically made in New Product Planning
* Discuss the nature of information available to support decision-making in New Product Planning
* Review the impact and context of decision-making in New Product Planning
* Review potential approaches to assist in New Product Planning decision-making
Haapalinna antti 2nd annual r&d process excellence summit 22nd october15...Antti Haapalinna
The document discusses performance management at Orion Pharma R&D. It provides background on Orion Corporation and describes Orion Pharma R&D's pipeline and effectiveness before 2007, which was thin. It then discusses how Orion Pharma R&D underwent a comprehensive change process from 2007-2015 that led to improved performance. This included conducting a self-diagnosis, developing a new strategic direction, focusing on culture change, reorganizing structures, and defining a desired culture. As a result, Orion Pharma R&D's research and clinical pipelines significantly expanded and strengthened between 2007-2015.
Moore Expedited Devel & Rev Of New Drugs 2010 (L)jmoore89
This document summarizes strategic regulatory options for expediting drug development and approval through the FDA. It discusses expedited review pathways including Subpart E for life-threatening diseases and Subpart H (accelerated approval) for serious or life-threatening conditions. Key elements covered are early consultation, use of surrogate endpoints in phase 2 trials, post-approval studies, and consideration of disease severity and treatment alternatives in the risk-benefit assessment. The goal is to provide patients sooner access to promising new therapies while maintaining adequate evidence of safety and efficacy.
This document describes the design and development of medical devices including hip and knee prostheses and related instruments. It discusses the 3D modeling and technical documentation created for the prostheses and instruments. It also details the complete development and manufacturing of total hip uncemented and cemented systems, including femoral stems, acetabular cups, liners, and various surgical instruments. Additionally, it outlines the concept and redesign of shark pliers, including sketches, ergonomic and functional optimization, and the final manufactured prototype.
5 Trends to Watch in the Medical Device Industry in 2016Mercer Capital
Demographic shifts underlie the long-term market opportunity for medical device manufacturers. While efforts to control costs on the part of the government insurer in the U.S. may limit future pricing growth for incumbent products, a growing global market provides domestic device manufacturers with an opportunity to broaden and diversify their geographic revenue base. Developing new products and procedures is risky and usually more resource intensive compared to some other growth sectors of the economy. However, barriers to entry in the form of existing regulations provide a measure of relief from competition, especially for newly developed products.
Hyperthermia, defined as elevating temperature above normal physiological levels, can be used to directly kill cancer cells or sensitize them to radiation and chemotherapy. Key points:
- Temperatures of 40-45°C can directly kill cells in a time and temperature dependent manner.
- Lower temperatures of 40-43°C do not directly kill cells but can sensitize them to radiation by improving oxygenation and inhibiting DNA damage repair.
- Hyperthermia can also sensitize cells to chemotherapy by increasing drug uptake and oxygen radical production.
- The combination of hyperthermia with radiation and chemotherapy has shown improved local tumor control compared to these treatments alone.
Shedding Some Light on the Insights Lurking in the PMA DatabaseRevital (Tali) Hirsch
May 28, 2016 marked forty years of modern day federal authority over medical devices in the U.S. Not only is this period brief in and of itself, but it’s also shorter by half compared to the duration of federal authority over pharmaceuticals, which began with the Food, Drug and Cosmetics Act of 1938. In the past several years the FDA has been the target of much criticism with regards to the approval of high-risk medical devices. Some of that critique is likely merited, but it is important to keep in mind that the medical device arm of the FDA is a work-in-progress that has had considerably less time to invent itself in the larger context of history.
This paper compares and contrasts different stakeholders’ perspectives and takes a deeper dive into the data. In doing so, this paper outlines practical changes and enhancements to the PMA database that can be carried out in the immediate present to increase transparency between the FDA and Industry.
The deeper dive also identifies several potential avenues for follow-on research, including PMAs that do not reach a positive conclusion and PMAs that are sponsored by early-stage and/or inexperienced filers. Insights from such research may hold the key to longer-term regulatory process improvements within the existing framework to promote high-risk medical device innovation and shorten these devices’ time-to-market without compromising the higher standards of the domestic regulatory system or the safety of patients.
Mercer Capital's Value Focus: Medical Device Manufacturers | Q4 2016 | Pfizer...Mercer Capital
Mercer Capital provides medical device manufacturers, related start-up enterprises, and private equity funds with valuation services, including purchase price allocation, 409a compliance, goodwill impairment testing, and other transaction and valuation advisory services.
Each issue includes a segment focus, market overview, mergers and acquisitions review, and more.
Hypothermia occurs when a body's temperature drops below 36.5 degrees Celsius. The document discusses risk factors for hypothermia in newborns such as prematurity, low birth weight, and improper care immediately after birth. It outlines signs of hypothermia like cold feet and weak crying. If left untreated, hypothermia can cause complications including death. The nurse's role is to prevent heat loss and maintain normal body temperature through immediate drying, skin-to-skin contact with the mother, and covering with blankets.
Hyperthermia is an elevated body temperature due to failed thermoregulation where the body produces or absorbs more heat than it can dissipate. When body temperatures become too high, it is a medical emergency requiring immediate treatment. The most common causes are heat stroke from prolonged heat exposure and adverse drug reactions. Treatment involves cooling measures like rest in shade, drinking water, and even immersion in cool water or medical cooling for severe cases. Prevention focuses on limiting heat exposure, staying hydrated, and using personal cooling systems for those at high risk.
2015 trends in global medical device strategy and issues for the supply chain...Tony Freeman
This presentation reviews critical business trends shared by major medical device companies and the implications for their manufacturing supply chain. Consolidation of device OEMs, product line and marketshare changes brought on by accountable care, and OEM desire for larger outsource partners are the dominant themes.
Device Sponsor Information Day: Session 2: Clinical evidence - pre-market and...TGA Australia
This presentation provided an insight into the clinical evidence requirements for medical devices. It also gave information about the level of clinical evidence required for conformity assessment procedures and during application audits. Lastly, it outlined requirements to keep contemperaneous clinical evidence once a device is included in the ARTG.
Many emerging companies make the mistake of putting all of their resources into immediate needs, and often neglect longterm regulatory strategy concerns when it comes to submissions and approvals. Don’t neglect the strategy piece in your planning! This lunch will provide a deep-dive foundation of how to develop a regulatory strategy. Topics to be addressed include:
What are different types of regulatory submissions for devices?
What are current trends in regulatory agencies?
What regulations around devices affect your organization?
Attendees will have the opportunity to ask questions with their company’s needs in mind.
Join us and Halloran Consulting at M2D2 for this expert lunch. Food will be served.
Importance Of Patents In The Life Sciences Industrybatane
This document discusses the importance of patent protection in the life sciences industry. It notes that developing new drugs and medical devices is a long, expensive process requiring huge investments. Patents are critical because they provide exclusivity rights for companies to recoup their investments and protect their products from competition during the regulatory review period and post-approval commercialization. The document outlines the drug and device development pathways and associated costs, emphasizing the need for strong intellectual property strategies to build competitive moats around new technologies and innovations.
Medical Device Clinical Studies and Protocol DesignMichael Swit
August 17, 2006 presentation to the IVT Medical Device Conference, focusing on the following relative to medical devices:
* Standards of Approval – What the Protocol Targets
* Key Considerations in Designing Clinical Studies
* Practical Lessons in Clinical Trial Design & Execution
Identifying Safety Signals by Data Mining the FDA Adverse Event Reporting Sys...Perficient, Inc.
Ever since the European Union (EU) introduced new legislation that requires life sciences companies to proactively detect, prioritize, and evaluate safety signals, there has been an increased interest, not only from sponsors and CROs in the EU, but globally, in pharmacovigilance systems that can assist with the signal management process.
Perficient's Chris Wocosky, an expert in signal detection and management, shows how your organization can use Empirica Signal, Oracle's state-of-the-art signal detection system to data mine the existing FDA Adverse Event Reporting System (FAERS) to determine safety signals. This presentation and demonstration willhelp you bettter understand how this solution can be used in daily pharmacovigilance activities.
Wallace H. Coulter c3i - Addressable Market PresentationHeidjer Staecker
This document provides guidance on how to properly assess the market potential for a new technology. It emphasizes that the focus should be on solving customer needs rather than just the product itself. Key steps include understanding who the target customers are, what problems they currently face and how they address those problems now, who the major competitors are, and determining if the new technology clearly provides benefits over existing solutions. The document also stresses understanding market trends, size, regulatory hurdles, intellectual property landscape, and identifying the appropriate business model to successfully commercialize the technology. Product-market fit is an important first step, but more work is needed to navigate risks, determine the path to market, and establish a sustainable business.
Elizabeth Mansfield, PhD, discusses the FDA’s approach to regulation
of in vitro diagnostic tests.
Part of Dx2010, a workshop at MaRS focused on best practices and regulatory considerations for developing gene-based diagnostic and prognostic tests.
Therapeutic Goods Evaluation Panel Roadshow - Overview of Clinical EvaluationsTGA Australia
The document provides an overview of external evaluations of prescription medicines conducted by the Therapeutic Goods Administration (TGA) in Australia. It discusses the role of the TGA in regulating medicines and evaluating clinical data in applications. Clinical evaluators within the TGA are responsible for independently assessing application dossiers, summarizing the clinical data, and writing Clinical Evaluation Reports which make recommendations regarding approval and product information. The reports follow a template and guidelines to ensure a high-quality and rigorous evaluation is conducted.
The document discusses the complex and unpredictable nature of the FDA drug approval process. While the steps of drug development may seem formulaic, including discovery, preclinical testing, and clinical trials, success is not guaranteed as programs face many risks and intangible factors. Understanding these challenges is important for mitigating risks and strategizing development approaches. The FDA approval process aims to ensure new drugs are safe and effective for patients.
Get Your Development Program Started on the Right FootBrook White, PMP
You think you have a potential pharmaceutical or biotechnology product based on animal or in vitro data—what is the next step? Two documents you need at an early stage are the Target Product Profile (TPP) which defines expectations for your potential medicine and an Integrated Product Development Plan (IPDP) which describes the activities required through approval of your marketing application.
The document discusses pharmacovigilance, which involves monitoring the safety of medical products. It outlines various methods for collecting and analyzing adverse event reports, including spontaneous reports from healthcare professionals and patients, literature reviews, and solicited reports from clinical trials. It also discusses prioritizing cases, signal detection methods, and actions that may be taken in response to potential safety issues. The main objectives are to minimize risks for patients and the company while meeting regulatory requirements.
The document outlines an agenda for a webinar on best practices for promotional content review. The webinar will cover what good promotional practices are, the importance of implementing them now, a four-step PACE process for content review, standard operating procedures for review, and a case study on tradeshow preparation. It includes slides on the key topics that will be discussed, such as establishing a cross-functional review committee and using categorization to ensure consistent application of review standards.
The webinar aims to (1) attract entrepreneurs to license technologies to start companies, (2) attract corporations to license technologies for new products, and (3) solicit input on commercial viability. The agenda includes a schedule, two 10-minute technology presentations on medical devices to diagnose swallowing disorders, and a question period. The first device, Down the Hatch, provides objective data from endoscopy videos to diagnose and prevent aspiration pneumonia. The second, Tongue Twister, is a mobile app that provides objective data on speech and swallowing tasks to assess patients. Both technologies are patent-pending and seeking start-up partners for further development and commercialization.
ICH Guidelines Effective for Regulating Quality of Medicines?Ajaz Hussain
ICH Guidelines: Effective tools for regulating the quality of medicines? Enabling regulatory considerations – the ICH Q8 – 12 are such considerations. Effective implementation of enabling regulatory considerations is a challenge. The challenge is what we know and what we can implement are two different things. Education, training, and experience linked to measures of professional development, as it relates to PQS, should bridge what we know and what we implement. What are such measures? Some corporations are already focused on making their PQS effective (e.g., Amgen) but most are not. In a global supply chain (with ~ 90% of Rx being generics) this can pose a major challenge to deliver assurance patients need. Why? #education #medicine #assurance #regulations #globalization #corporations #bridging #measurements
The document discusses conducting effective due diligence for business deals. It outlines three categories of due diligence: technical, commercial, and legal. Pre-deal due diligence involves initial checks like opportunity size and fit. Confirmatory due diligence aims to confirm information and find reasons not to proceed. An effective due diligence team represents key disciplines and has experience evaluating information within tight timeframes. Information requests cover areas like discovery, clinical trials, manufacturing, and compliance. Critical questions examine clinical trial design and whether data will support desired labeling. Potential problems include lack of access to data, staff, or incomplete information.
Genable Technologies is developing RhoNova, a gene therapy using two AAV vectors, for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP), a genetic disorder causing progressive vision loss. RhoNova aims to overcome the diversity of over 200 RHO mutations by using RNA interference to destroy mutant RHO mRNA and replacing RHO through a gene resistant to mutations. Proof of concept has been shown in animal models. Orphan drug status has been granted and GMP manufacturing and preclinical toxicology studies are underway to enable clinical trials in 2017.
PLx Pharma is developing next generation NSAIDs to improve gastrointestinal (GI) safety. Jason Moore, Vice President of PLx Pharma, gave a presentation about positioning oneself for a leadership career in biotech and about the challenges faced by PLx Pharma, including developing drugs with improved GI safety profiles to address the large market for osteoarthritis pain relief while managing various risks in the drug development process such as technical, regulatory, financial and commercial risks.
BullFrog AI is a technology enabled drug development company using machine learning to usher in a new era of precision medicine. Through its collaborations with leading research institutions, including Johns Hopkins University and J. Craig Venter Institute, BullFrog AI is at the forefront of AI-driven drug development. Using its proprietary bfLEAP™ artificial intelligence platform, BullFrog AI aims to enable the successful development of pharmaceuticals and biologics by predicting which patients will respond to therapies in development. BullFrog AI is deploying bfLEAP™ for use at several critical stages of development with the intention of streamlining data analytics in therapeutics development, decreasing the overall development costs by decreasing failure rates for new therapeutics, and impacting the lives of countless patients that may have otherwise not received the therapies they need.
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Adani Group Requests For Additional Land For Its Dharavi Redevelopment Projec...Adani case
It will bring about growth and development not only in Maharashtra but also in our country as a whole, which will experience prosperity. The project will also give the Adani Group an opportunity to rise above the controversies that have been ongoing since the Adani CBI Investigation.
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Moore Tech Assess April 2009 (L)
1. Framework for TechnologyFramework for Technology
AssessmentAssessment
Life Science Entrepreneurship Certificate Program
Rice University
Jason E. Moore, M.S., M.B.A.
Vice President, PLx Pharma Inc.
PLx Pharma Inc.
2. Overview & ApproachOverview & Approach
• Quantitative and qualitative assessments of potential product leads
– The technology itself
– Path-to-marketPath to market
– Intellectual property
– Regulatory issues
– Competition and market
– Product pricing
– Inventor and institution
• Perspectives
– Entrepreneur or start-up company licensing professional
– Development-stage company product portfolio managers
– Big company technology assessment
• Emphasis on life science company key areas• Emphasis on life science company key areas
• Technology focused, with an emphasis on therapeutic products…
• Not “How to Evaluate a Business Plan”
PLx Pharma Inc.
3. FundamentalsFundamentals
Stating the ObviousStating the Obvious
• The technology should lead to a useful, commercially valuable
product
• The technology/product should be adequately protected and other IP• The technology/product should be adequately protected and other IP
must not block the path to commercialization
• There must be a clear business model/strategy that can be
formulated for generating a profit from the technology’sformulated for generating a profit from the technology s
commercialization
• The technology should address a need in a large and growing market
Th h ld b ffi i t l it i th th t k t d• There should be sufficient clarity concerning the path to market and
the regulatory environment
• Every technology is unique
• Life science product development is development research, is very
complex, and is defined by its many uncertainties and assumptions
• Evaluating early-stage technologies is difficult
PLx Pharma Inc.
4. Due DiligenceDue Diligence
Establishing an ApproachEstablishing an Approach
• Conducting Due Diligence
– Have and use a thorough framework for
technology assessment
"There is an increasing
concern that in modern
h f l fi di
– Be thorough and systematic
– Ask for, and expect, access to all data
(with CDA)
Look at source data when possible
research, false findings may
be the …vast majority of
published research claims. A
new claim about a research
finding is more likely to be
false than true."
– Look at source data, when possible
– Critically evaluate all claims and data,
including published articles
– Use experts routinely; don’t rely only on your own
intellect or scientific knowledge
• Caveat emptor
– “I have the cure for cancer.”
“Thi ill b th k t i [l th 10] !”– “This will be on the market in [less than 10] years!”
– “There is no competition.”
– Institution has no interest in the technology/inventor is the only owner
PLx Pharma Inc.
5. Due DiligenceDue Diligence
Establishing an ApproachEstablishing an Approach
• Assess
– The technology itself (the scientific basis and its applications)
– Intellectual propertyte ectua p ope ty
– Path-to-market
– Commercial opportunity/valuation
– Licensing deal structure/economics
– Feasibility and “other”
• Complete assessment within a global framework (eg, SWOT)
– Strengths – Opportunities
W k Th t /Ri k– Weaknesses – Threats/Risks
• Understand the psychology of inventor and investor
– Group think – Wishful thinking – Need to Believe!
• Define licensing criteria prospectively* (aka know your strategy)• Define licensing criteria prospectively (aka, know your strategy)
– Time-to-market – Deal terms (up-fronts, royalty rates, etc.)
– Therapeutic area – Relative aggregate risk
– Valuation or annual sales target
PLx Pharma Inc.
g
6. Technology AssessmentTechnology Assessment
What is it?What is it?
• What is it?
• How well characterized is it/can it be?
C l bi l i l– Complex biologicals…
• How are its effects measured (in vitro, in vivo)?
• Are there useful animal models to evaluate early efficacy?
– The ability to translate basic findings into disease cures often relies on
animal models that serve as surrogates of the human condition
– Many diseases lack validated animal models, hampering translational
progressprogress
• What is the amount and quality of the discovery science?
• How novel is the technology?
– Patentable
– Regulatory and commercial analogies
– Regulatory and market risks
PLx Pharma Inc.
7. Technology AssessmentTechnology Assessment
How is it to be used?How is it to be used?
• How will it be used in the marketplace?
– Who are the real users of (market for) the technology (patients,
physicians caregivers)?physicians, caregivers)?
– What indications, therapeutic areas are addressed with the technology
– On what basis will the product compete?
• What is the standard of care for the given indication/use?• What is the standard of care for the given indication/use?
– Critical to understand the SOC…
• Degree of unmet medical need
• Market size and dynamicsMarket size and dynamics
• Likely comparator in pivotal clinical trials
• Implications for a marketed product (displaces others, used in combination?)
• What scientific evidence indicates the technology could actually begy y
used as asserted?
– Let data provide the primary voice
PLx Pharma Inc.
8. Technology AssessmentTechnology Assessment
Intellectual PropertyIntellectual Property
• Most often, the real product is the intellectual property (and not the pill,
injectable, or device)
– The IP is typically the heart of any major transaction in the pharmaceutical, biotechnology,
and medical device industries
– The strength of the IP can significantly affect the terms and value of any transactions (or
whether it will occur at all)
• What is the nature and extent of the intellectual property protection? Whatp p y p
jurisdictions filed?
– Composition of matter
– Utility patent
• The most useful patents in biopharma are those that cover
the composition of an effective drug and its application
to treating particular diseases
• Exclusivity• Exclusivity
– Ownership: Assignments and licenses, encumbrances;
you want to see a clear chain of ownership
– Claims analysis: Coverage and validity (eg, will the patent
PLx Pharma Inc.
claims cover the product for which FDA approval is sought?)
9. Technology AssessmentTechnology Assessment
Intellectual PropertyIntellectual Property
• Patent analyses
– Freedom to operate (FTO) : Formal legal analysis to answer the key question:
“Will any aspect of development or commercialization infringe the patent of ay p p g p
third party?”
– “Opinions of Counsel”
• Note Patent (20 years +/- PTO adjustments) and Non-Patent (FDA) exclusivity
N P t t l i it• Non-Patent exclusivity
• 7 years – orphan indications
• 5 years – new chemical entity (NCE)
• 3 years – new indications/formulations3 years new indications/formulations
• 6 months – pediatric indications
• What effective patent life remains for key patents?
• Hatch-Waxman: “Drug Price Competition and Patent Term Restoration Act”
• Beware provisional patents
• What non-patent IP exists, and is it effectively transferrable?
PLx Pharma Inc.
10. Technology AssessmentTechnology Assessment
What developmental stage is it?What developmental stage is it?
Innovation/ Validation/ Pre-Clinical Clinical Market
Discovery Prototypes Models Trials
• What evidence is there that there is a “product”?
• Has an attempt been made to “product-ize” the technology?
– Formulated?Formulated?
– Prototype exist?
– Tested in animal models or in
clinical trials?
– “Proof of concept”
• Regulatory status and
communications
Drug Substance (API) Drug Product
PLx Pharma Inc.
Drug Substance (API) Drug Product
11. Technology AssessmentTechnology Assessment
What is the path to market?What is the path to market?
• What non-clinical studies will be
required (GLP)
– Deviations from the “standard
k ”?package”?
– Validated animal models?
– What do non-clinical studies
reveal about safety issues thaty
may be present in humans
– Law of combinatorial risk: safety
issues rarely “go away”
CMC Issues
• Knowing the general framework is
• CMC Issues
– Drug substance vs. drug product
– CMC Framework
• Identity
only the beginning
• Critical to understand the nature and
size of both nonclinical and clinical
studies that will be required
y
• Strength
• Quality
• Purity
• Potency
studies that will be required
• CMC is the iceberg beneath the surface
• What special challenges does this
technology present?
PLx Pharma Inc.
– GMP Standards
– Continued…
technology present?
12. Technology AssessmentTechnology Assessment
What is the path to market?What is the path to market?
• CMC Issues (cont)
– What are the key technical
issues?
• Applicability of regulatory
mechanisms for accelerated
development
– Is the product “difficult to
formulate”?
– What evidence for product stability
is there? What conditions?
development
– Subpart E
– Subpart H/Accelerated Approval
– Fast-track Designation
is there? What conditions?
– What do you know about COG?
– Can (must) the API be formulated
for the desired route of
– Orphan Drug Designation
– Special Protocol Assessments
– Priority Review
administration?
– Myriad technology-specific issues
• Clinical studies
N b f t i l
• Beware of overly aggressive/
optimistic assertions of
accelerated development
– Number of trials
– Size of trials (target enrollment)
– Understanding “pivotal” clinical
trials
• Substantial evidence and the
two-trial rule (AWCCT)
• A note about biomarkers
PLx Pharma Inc.
13. Additional InformationAdditional Information
D d Bi l i A l ThDrug and Biologic Approvals: The
Complete Guide for Small Businesses —
FDA Financial Assistance and Incentives
B F k J S i ki d K t R K tBy Frank J. Sasinowski, and Kurt R. Karst,
Hyman, Phelps & McNamara, P.C.
2000/160 pages/soft cover
ISBN: 1 885259 68 9ISBN: 1-885259-68-9
Available at FDLI: http://www.fdli.org/pubs
Regularly updated
information about federally
and privately supported
• Finding labels for approved drug products
finding generic drug products for a brand
name drug product
NLM resource for published
literature
clinical research in human
volunteers
• Finding all drugs with a specific active
ingredient
• Viewing the approval history of a drug
• More…
PLx Pharma Inc.
14. Assessing the CompetitionAssessing the Competition
• Therapeutic products compete on a number of basic dimensions,
which combine to dictate pricing, reimbursement and market
positionposition
– *Safety/Efficacy profile
– Ease of use (eg, route of administration, dose regimen)
– Some measure of “Value” (eg selling price reductions in hospital days– Some measure of Value (eg, selling price, reductions in hospital days,
lost work days) and/or “Quality of Life”
– A note about CMS…
• Three areas of focus What would constitute aThree areas of focus
– Currently marketed competitive products
• Standard of care
• First-line treatment
clinically meaningful
improvement over the
standard of care?
• Second and subsequent treatment
– Pipeline competition
– Market and market dynamics
PLx Pharma Inc.
y
15. Resources for AssessingResources for Assessing
Competition and MarketsCompetition and Marketspp
• Marketed Product Competition
– Safety and efficacy; PDR®,
package insert
• Market Dynamics
– Key products and companies
– (Molecular) Targets andpackage insert
– Pricing: Thompson Red
BookTM
• Pipeline Competition
(Molecular) Targets and
technologies
– Total market size and key brands
– Share changes by quarter
• Pipeline Competition
– News
• MedAd News
• BioCentury
– Key patent expirations
– Unmet needs of patients,
physicians, caregivers
– Payor and FDA dynamicsy
• BIO SmartBrief
• Venture Wire
– Information Portals
K l d E
Payor and FDA dynamics
• Information Sources
– *Clinical thought leaders
– IMS Health and others’ reports
• KnowledgeExpress
• Nerac
– Scientific Publications and
Meetings
p
– Conference proceedings (industry,
scientific)
– FDA
PLx Pharma Inc.
g
16. Assessing Technology ValueAssessing Technology Value
• Estimating the value of early-stage technologies is notoriously difficult
• Approaches
– Top downTop down
• Estimates of market size, market share of key products, penetration/share of your
product for a given time period (year)
• By Analogy: comparison of sales for similar product in similar clinical setting
Bottom up: build a financial (NPV) model which estimates future cash flows for a– Bottom up: build a financial (NPV) model, which estimates future cash flows for a
given product (by indication); includes
• Manufacturing costs Sales, general and administrative costs
• Selling price per unit Penetration/uptake rate
S l l Di t d ( t l ) f f t• Sales volume Discounted (present value) of future revenues
• Pharma uses
– Basic NPV models
– Real Option Analyses
Real Options Analysis…
Real Option Analyses
– Monte Carlo simulations with NPV models
– Decision Tree Analyses
…captures the effects of uncertainty and
change inherent in many projects. Unlike
discounted cash flow analysis, it recognizes
active project management and the learning
that occurs during development research.
PLx Pharma Inc.
that occurs during development research.
17. • Decision Tree Analysis
– Accounts for development risk
– Maps development path schematically,
Decision Tree Analysis for a
Drug Development Program
p p p y
assigns probabilities to categorical
outcomes
– Allows discounting of NPV, based on
development risk
– Valuable complement to commercial
revenue discounting
– Excellent for comparison of relative
risks/value between products (portfolio
management
• Monte Carlo Simulation with
(NPV) Revenue Model
– Accounts for commercial risk (eg,
selling price COGS uptake rates)
management
Value
selling price, COGS, uptake rates)
– Allows one to describe the
“probabilistic input variables”
– Provides descriptive statistics for
possible outcomes
Assumptions
Utility
possible outcomes
2001 2002 2003 2004 2005 2006 2007 2008 2009 2010
PL 2,389,696$ 13,857,855$ 22,476,229$ 32,410,723$ 35,052,197$ 37,908,951$ 40,998,530$ 35,471,928$ 30,690,312$ 26,553,258$
NHL 21,182,784$ 90,327,628$ 144,440,652$ 205,307,942$ 218,868,532$ 233,324,798$ 248,735,901$ 212,131,926$
HRMPC 54,751,049$ 107,147,803$ 157,266,188$ 205,179,953$ 200,768,584$ 196,452,060$ 192,228,340$
AML 1,759,672$ 15,224,564$ 24,698,527$ 35,615,577$ 38,722,059$ 42,087,834$ 45,734,217$ 39,747,414$ 34,536,274$
RCC 682,668$ 1,476,611$ 2,395,433$ 3,454,214$ 3,735,733$ 4,040,195$ 4,369,471$ 3,780,466$
H&N 3,813,594$ 7,934,564$ 12,381,490$ 17,173,952$ 17,866,062$ 18,586,064$ 19,335,083$
Total 2,389,696$ 15,617,527$ 59,566,245$ 207,478,132$ 332,586,226$ 455,040,844$ 528,044,534$ 537,205,784$ 538,581,222$ 488,565,347$
PV (Inflation) 2,320,093$ 14,721,017$ 57,831,306$ 195,568,038$ 322,899,249$ 428,919,638$ 512,664,596$ 506,367,974$ 522,894,390$ 460,519,697$
– Allows sensitivity analysis
and fuller scenario analysis
– Better than point estimates
PLx Pharma Inc.
( )
PV (million) 2.32$ 14.72$ 57.83$ 195.57$ 322.90$ 428.92$ 512.66$ 506.37$ 522.89$ 460.52$
p
18. Additional ReadingAdditional Reading
Introduction to Simulation and The New Math for Drug Licensing
Risk Analysis
(2nd Edition)
by James R. Evans, David L. Olson
2001/392 pages/hard cover
g g
by James Kalamas, Gary S. Pinkus,
and Kevin Sachs
In: The McKinsey Quarterly
2002 No 4 pp 9 122001/392 pages/hard cover
ISBN: 0130329282
The Real Power of Real Options
2002, No. 4, pp 9-12.
Scientific Management at Merck: An
by Keith J. Leslie and Max P. Michaels
In: The McKinsey Quarterly
1993, No. 3, pp 97-108.
Available at: www.mckinseyquarterly.com
Interview with CFO Judy Lewent
by Nancy A. Nichols
In: Harvard Business Review
Pub date: January 1, 1994yq y Pub date: January 1, 1994
Available at: www.hbsp.harvard.edu
Real Options Analysis: Tools and Techniques for
Valuing Strategic Investment and Decisions
2 d Editi2nd Edition
by Johnathan Mun
2006/667 pages/hard cover
ISBN: 0471747483
PLx Pharma Inc.
ISBN: 0471747483
19. A Note on LicensingA Note on Licensing
• A key to evaluating technology value is
analyzing the “deal”
• Framework for understanding the deal
Early-Stage Technologies:
Valuation and Pricing
by Richard Razgaitss
– Technology (nature, scope/”field of use”)
– Term/duration
– Exclusivity
– Rights conferred, in what geographies
Deal economics (up fronts milestones
by Richard Razgaitss
1999/291 pages/hard cover
ISBN: 0471328561
– Deal economics (up-fronts, milestones,
royalties, maintenance fees, etc.)
– Milestones
– Reversionary rights
– Transferability/change of control
Licensing Executives Society
URL: www.usa-canada.les.org/
– Principal technology-specific risks
– “Intangibles”
• What-if scenario modeling to clarify the
implications of tradeoffs recommended
g
Les Nouvelles
Journal of Licensing Executives
Society International
URL: www lesi org
• Find and use key attorneys/
consultants for early deals
URL: www.lesi.org
PLx Pharma Inc.
20. “Other”“Other”
• Inventor and institution
– Company proximity to inventor considered by some to be the “single
most important factor” for (early-stage) technology successmost important factor for (early stage) technology success
– Evaluate the inventor and the institution
• Bureaucracy
• PersonalitiesPersonalities
• Potential for access to facilities/sponsored research
• Reputation, grant funding success
• Aggregate risks• Aggregate risks
• Pass the smell test?
• Unconventional approaches needed to “make it work” (eg, new surrogate
endpoint)endpoint)
• “50% of the time…”
PLx Pharma Inc.