This course provides an overview of biomedical innovation and its relation to healthcare. It introduces medical technology innovations by identifying clinical needs, inventing new devices and instruments, and implementing devices in patient care. Students will learn the needs finding, inventing, and implementation phases of biodesign. They will validate medical needs, assess markets, define the invention to implementation process, write business plans, and think critically. The course uses readings, projects, presentations and discussions to achieve these goals.
This document provides a summary of Yasser Jasser's personal and professional experience. It includes his contact information, education history, work history in project management and marketing roles for F. Hoffman-La Roche Ltd from 2000 to present, descriptions of his roles and responsibilities, and a list of his training courses and workshops. He has over 18 years of experience in pharmaceutical sales and marketing.
Vedic Lifesciences is a contract research organization founded in 2000 that provides drug development services to pharmaceutical companies. It has managed over 34 clinical trials and 248 preclinical studies across multiple therapeutic areas. Services include regulatory submissions, clinical project management, data management, biostatistics, and medical writing. Vedic has experience in areas such as oncology, diabetes, orthopedics, andrology, and inflammation. It employs a team of experts including clinical researchers, medical writers, and regulatory specialists to support its clients.
Vedic Lifesciences is a contract research organization founded in 2000 that provides drug development services to pharmaceutical companies. It has managed over 34 clinical trials and 248 preclinical studies across multiple therapeutic areas. Services include regulatory submissions, clinical project management, data management, biostatistics, and medical writing. Vedic has experience in areas such as oncology, diabetes, orthopedics, andrology, and inflammation. It employs a team of experts including clinical researchers, medical writers, and regulatory specialists to support its clients.
Clinical development, contract & outsourcing in mena & asia pac webinar-l aju...Larry Ajuwon
This document provides a summary of a webinar on clinical development, contracting, and outsourcing in the MENA and Asia-Pacific regions. The webinar covered the clinical development process and landscape in these regions, opportunities and challenges, notable industry partnerships and transactions, and key growth areas. It found that clinical development accounts for a large share of pharmaceutical R&D spending. Both regions are growing markets and R&D is shifting there due to lower costs and other advantages. Outsourcing of clinical activities is increasing to access these markets and improve productivity. The CRO market in Asia-Pacific is more developed with strategic partnerships, while MENA's market is smaller and local.
This presentation gives the details about career opportunities in clinical research, clinical research courses available in India and clinical research training.
This course provides an overview of biomedical innovation and its relation to healthcare. It introduces medical technology innovations by identifying clinical needs, inventing new devices and instruments, and implementing devices in patient care. Students will learn the needs finding, inventing, and implementation phases of biodesign. They will validate medical needs, assess markets, define the invention to implementation process, write business plans, and think critically. The course uses readings, projects, presentations and discussions to achieve these goals.
This document provides a summary of Yasser Jasser's personal and professional experience. It includes his contact information, education history, work history in project management and marketing roles for F. Hoffman-La Roche Ltd from 2000 to present, descriptions of his roles and responsibilities, and a list of his training courses and workshops. He has over 18 years of experience in pharmaceutical sales and marketing.
Vedic Lifesciences is a contract research organization founded in 2000 that provides drug development services to pharmaceutical companies. It has managed over 34 clinical trials and 248 preclinical studies across multiple therapeutic areas. Services include regulatory submissions, clinical project management, data management, biostatistics, and medical writing. Vedic has experience in areas such as oncology, diabetes, orthopedics, andrology, and inflammation. It employs a team of experts including clinical researchers, medical writers, and regulatory specialists to support its clients.
Vedic Lifesciences is a contract research organization founded in 2000 that provides drug development services to pharmaceutical companies. It has managed over 34 clinical trials and 248 preclinical studies across multiple therapeutic areas. Services include regulatory submissions, clinical project management, data management, biostatistics, and medical writing. Vedic has experience in areas such as oncology, diabetes, orthopedics, andrology, and inflammation. It employs a team of experts including clinical researchers, medical writers, and regulatory specialists to support its clients.
Clinical development, contract & outsourcing in mena & asia pac webinar-l aju...Larry Ajuwon
This document provides a summary of a webinar on clinical development, contracting, and outsourcing in the MENA and Asia-Pacific regions. The webinar covered the clinical development process and landscape in these regions, opportunities and challenges, notable industry partnerships and transactions, and key growth areas. It found that clinical development accounts for a large share of pharmaceutical R&D spending. Both regions are growing markets and R&D is shifting there due to lower costs and other advantages. Outsourcing of clinical activities is increasing to access these markets and improve productivity. The CRO market in Asia-Pacific is more developed with strategic partnerships, while MENA's market is smaller and local.
This presentation gives the details about career opportunities in clinical research, clinical research courses available in India and clinical research training.
A guide to become clinical research associatepptxRidhimaPatel2
An integral part of modules in clinical research courses focus on the ethical standards that have to be implemented without compromising the quality of trial results.
Adel G. Said is seeking a position as a drug safety and pharmacovigilance associate. He has over 7 years of experience in the pharmaceutical industry, including roles in pharmacovigilance and drug safety at Servier Pharmaceuticals in Egypt. He has a bachelor's degree in pharmaceutical sciences and is skilled in pharmacovigilance tasks like adverse event reporting and coding, clinical trial monitoring, and ensuring drug safety guidelines are followed. Currently he works as a pharmacy intern at Rite Aid pharmacy, where he counsels patients and gains experience in retail pharmacy procedures.
This document provides an overview of biosimilars and their development process according to international guidance. It begins with definitions of biosimilars and their differences compared to generic small molecule drugs. It then describes the two key steps in biosimilar development: 1) Developing a highly similar product through extensive analytical comparison to the reference product, and 2) Confirming biosimilarity through a targeted clinical program. It reviews guidelines from the WHO, EMA, and FDA on evaluating biosimilarity and the need for analytical, nonclinical and clinical comparisons. The document aims to explain the rigorous process of demonstrating biosimilarity that biosimilar developers must undergo to obtain regulatory approval.
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
Clinical research and its importance career in clinical researchRidhimaPatel2
This document is expressing the carrier opportunities in clinical research and how it important to learn clinical research for our society and the best institute providing absolute best training for the clinical research
- Dr. Meena K. Thakkar has over 14 years of experience in the medical industry including 10 years in drug safety and pharmacovigilance. She currently works as an Assistant Manager for Tata Consultancy Services providing oversight on CAPA management for a Japanese client.
- She has experience developing strategies to improve pharmacovigilance processes using tools like structured problem solving, FMEA, and Lean Six Sigma.
- Prior to her current role, she held positions with increasing responsibility in drug safety and medical affairs at various pharmaceutical companies.
How early is too early for pharmaceutical market insights and product forecasts. A discussion on pipeline product research during early product development
Most reliable diagnostic and pathology center to watch..Merry D'souza
This edition of Most Reliable Diagnostic and Pathology Centres to Watch places the spotlight on healthcare service providers that are leveraging such revolutionary technologies to deliver the best to those seeking their services.
Setting up of new pharmacovigilance centresPriti Gupta
Cure & Care Pharma proposes establishing a Pharmacovigilance Centre to monitor drug safety. The centre would be approved by regulatory authorities and function to collect, analyze and communicate information about adverse drug reactions and risks. The proposal outlines the need for pharmacovigilance due to increasing drug availability and irrational practices. It describes the centre's scope, reporting processes, infrastructure needs and budget to effectively oversee post-marketing drug safety.
Regulatory documents are required by health authorities before approval of new drugs, devices, or biologics. These documents include protocols, clinical study reports, investigator brochures, common technical documents, informed consent forms, and risk management plans. Protocols describe the study procedures and analysis plans. Clinical study reports integrate full study reports according to ICH guidelines. Investigator brochures provide safety information to investigators. Common technical documents assemble quality, safety, and efficacy data for regulatory submissions. Informed consent forms provide study information for participants. Risk management plans describe safety profiles and risk minimization plans.
The document outlines the establishment of the Hospital Accreditation Commission (HAC) in the Philippines. It discusses the timeline of events leading to the creation of HAC through a DOH Administrative Order in 2013. The order established HAC as the national accrediting body for hospitals, using PhilHealth standards to improve quality. It describes HAC's activities in 2013 like orientations and its strategic planning workshop. Finally, it presents HAC's proposed calendar of activities for 2014, including advocacy campaigns, training surveyors, and piloting hospital surveys.
This document outlines various clinical research activities and provides descriptions for each. It includes activities such as drug accountability, patient care, obtaining informed consent, patient education, scheduling, data management including query resolution, research chart management, case report form completion, safety reporting, screening and enrollment log management, source document development, subject registration, protocol training, meetings, and general protocol review.
The document provides information about opportunities in the clinical research industry. It discusses what clinical research is and the drug development process. It outlines the growing industry opportunity, with increasing demand for clinical research professionals and rapid investment growth in the field. Various career opportunities and paths in clinical research are also presented, along with salary ranges and eligibility requirements to work in clinical research.
This document provides a summary of Paul Rohricht's professional experience and qualifications. It outlines his experience in business development, strategic planning, transaction structuring, and executive management roles in the biotechnology industry, with over $450 million in transactions completed. It details positions held at various biotech companies, including founding several startups, and highlights accomplishments such as negotiating licensing deals, securing funding, and growing revenues.
The clinical research industry in India is growing rapidly due to the booming pharmaceutical sector. The Indian pharmaceutical market is expected to triple in size by 2015. This growth has fueled a rise in clinical research opportunities in India. However, the clinical research industry faces high attrition rates. There is a high demand for trained clinical research professionals to work for the growing number of contract research organizations. Focus Edu Care has launched a postgraduate program in clinical research to train professionals to meet this demand and fill various roles in areas like clinical research, data management, business development, and medical writing.
The document discusses the mission and function of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee (ODAC). The subcommittee evaluates and prioritizes new cancer therapeutics for children and provides recommendations to ensure timely access. It also advises on improving consistency in availability. Membership includes experts from relevant fields. The Office of Oncology Drug Products at the FDA's Center for Drug Evaluation and Research oversees oncology product review and policy. It coordinates activities within the FDA and with external groups. The document also outlines the roles of the Radioactive Drug Research Committee and the FDA's divisions of oncology products.
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. This outsourcing has grown as drug development has become more complex.
- When deciding whether and how to use a CRO, companies consider tactical, maximal, or strategic outsourcing based on their internal resources. Selecting the right CRO requires evaluating their capabilities, compatibility with the sponsor's needs, and costs.
- Managing the sponsor-CRO relationship is critical to ensure success. This involves clearly defining roles and responsibilities, establishing performance metrics,
Frank Spinelli, New York City's Cabrini Medical Center - Speaker at the marcus evans PharmaMarketing Summit 2012, held in Wheeling, IL, April 30 - May 2, 2012, delivered his presentation entitled A Doctor’s Perspective on the Future Role of Pharmaceutical-Doctor Relationships and How They Are Evolving through Technology
Changing times ask for solid relationships.
Times are changing. A renewed interest from big pharma in the rare disease landscape
has awakened due to large-scale patent expirations, competition from generics &
biosimilars, anemic pipelines, escalating clinical trial costs and a global health-care
reform. This means that the traditional blockbuster model has become less viable while
the revenue-generating potential of orphan drugs has shown to be huge with a greater
return on investment than non-orphan drugs. According to EvaluatePharma, the orphan
drugs sales will grow at an annual rate of 11% and constitute 19% of the total share of
prescription drugs by 2020, totalling 176 billion dollars.
The rare disease landscape is very complex due to the large amount of stakeholders
involved. Despite their different interests they have one goal in common: getting an
orphan drug approval that will help save or improve lives. But there are many challenges
on the road to orphan drug designation:
• Complex and changing national and regional regulations
• Clinical trial design and finding & keeping patients
• The lack of a central database designed specifically to list patient registries, which
asks for close stakeholder engagement
• Partnering and establishing financing for future development
• Establishing a foundation for price that is balanced and sustainable
• Achieving an efficient and timely access to market with equal access for patients
around the world
• Achieving timely and correct diagnosis to enable higher quality of life and more time
and information for developers
More information: http://www.orphandrugssummit.com/
Rare Diseases: A Report on Orphan Drugs in the PipelinePhRMA
Rare diseases, when taken together, are not that rare at all. In fact, according to the National Institutes of Health (NIH), 30 million Americans have one of the nearly 7,000 diseases that are officially deemed “rare” because alone they each affect fewer than 200,000 people in the United States.
A guide to become clinical research associatepptxRidhimaPatel2
An integral part of modules in clinical research courses focus on the ethical standards that have to be implemented without compromising the quality of trial results.
Adel G. Said is seeking a position as a drug safety and pharmacovigilance associate. He has over 7 years of experience in the pharmaceutical industry, including roles in pharmacovigilance and drug safety at Servier Pharmaceuticals in Egypt. He has a bachelor's degree in pharmaceutical sciences and is skilled in pharmacovigilance tasks like adverse event reporting and coding, clinical trial monitoring, and ensuring drug safety guidelines are followed. Currently he works as a pharmacy intern at Rite Aid pharmacy, where he counsels patients and gains experience in retail pharmacy procedures.
This document provides an overview of biosimilars and their development process according to international guidance. It begins with definitions of biosimilars and their differences compared to generic small molecule drugs. It then describes the two key steps in biosimilar development: 1) Developing a highly similar product through extensive analytical comparison to the reference product, and 2) Confirming biosimilarity through a targeted clinical program. It reviews guidelines from the WHO, EMA, and FDA on evaluating biosimilarity and the need for analytical, nonclinical and clinical comparisons. The document aims to explain the rigorous process of demonstrating biosimilarity that biosimilar developers must undergo to obtain regulatory approval.
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
Clinical research and its importance career in clinical researchRidhimaPatel2
This document is expressing the carrier opportunities in clinical research and how it important to learn clinical research for our society and the best institute providing absolute best training for the clinical research
- Dr. Meena K. Thakkar has over 14 years of experience in the medical industry including 10 years in drug safety and pharmacovigilance. She currently works as an Assistant Manager for Tata Consultancy Services providing oversight on CAPA management for a Japanese client.
- She has experience developing strategies to improve pharmacovigilance processes using tools like structured problem solving, FMEA, and Lean Six Sigma.
- Prior to her current role, she held positions with increasing responsibility in drug safety and medical affairs at various pharmaceutical companies.
How early is too early for pharmaceutical market insights and product forecasts. A discussion on pipeline product research during early product development
Most reliable diagnostic and pathology center to watch..Merry D'souza
This edition of Most Reliable Diagnostic and Pathology Centres to Watch places the spotlight on healthcare service providers that are leveraging such revolutionary technologies to deliver the best to those seeking their services.
Setting up of new pharmacovigilance centresPriti Gupta
Cure & Care Pharma proposes establishing a Pharmacovigilance Centre to monitor drug safety. The centre would be approved by regulatory authorities and function to collect, analyze and communicate information about adverse drug reactions and risks. The proposal outlines the need for pharmacovigilance due to increasing drug availability and irrational practices. It describes the centre's scope, reporting processes, infrastructure needs and budget to effectively oversee post-marketing drug safety.
Regulatory documents are required by health authorities before approval of new drugs, devices, or biologics. These documents include protocols, clinical study reports, investigator brochures, common technical documents, informed consent forms, and risk management plans. Protocols describe the study procedures and analysis plans. Clinical study reports integrate full study reports according to ICH guidelines. Investigator brochures provide safety information to investigators. Common technical documents assemble quality, safety, and efficacy data for regulatory submissions. Informed consent forms provide study information for participants. Risk management plans describe safety profiles and risk minimization plans.
The document outlines the establishment of the Hospital Accreditation Commission (HAC) in the Philippines. It discusses the timeline of events leading to the creation of HAC through a DOH Administrative Order in 2013. The order established HAC as the national accrediting body for hospitals, using PhilHealth standards to improve quality. It describes HAC's activities in 2013 like orientations and its strategic planning workshop. Finally, it presents HAC's proposed calendar of activities for 2014, including advocacy campaigns, training surveyors, and piloting hospital surveys.
This document outlines various clinical research activities and provides descriptions for each. It includes activities such as drug accountability, patient care, obtaining informed consent, patient education, scheduling, data management including query resolution, research chart management, case report form completion, safety reporting, screening and enrollment log management, source document development, subject registration, protocol training, meetings, and general protocol review.
The document provides information about opportunities in the clinical research industry. It discusses what clinical research is and the drug development process. It outlines the growing industry opportunity, with increasing demand for clinical research professionals and rapid investment growth in the field. Various career opportunities and paths in clinical research are also presented, along with salary ranges and eligibility requirements to work in clinical research.
This document provides a summary of Paul Rohricht's professional experience and qualifications. It outlines his experience in business development, strategic planning, transaction structuring, and executive management roles in the biotechnology industry, with over $450 million in transactions completed. It details positions held at various biotech companies, including founding several startups, and highlights accomplishments such as negotiating licensing deals, securing funding, and growing revenues.
The clinical research industry in India is growing rapidly due to the booming pharmaceutical sector. The Indian pharmaceutical market is expected to triple in size by 2015. This growth has fueled a rise in clinical research opportunities in India. However, the clinical research industry faces high attrition rates. There is a high demand for trained clinical research professionals to work for the growing number of contract research organizations. Focus Edu Care has launched a postgraduate program in clinical research to train professionals to meet this demand and fill various roles in areas like clinical research, data management, business development, and medical writing.
The document discusses the mission and function of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee (ODAC). The subcommittee evaluates and prioritizes new cancer therapeutics for children and provides recommendations to ensure timely access. It also advises on improving consistency in availability. Membership includes experts from relevant fields. The Office of Oncology Drug Products at the FDA's Center for Drug Evaluation and Research oversees oncology product review and policy. It coordinates activities within the FDA and with external groups. The document also outlines the roles of the Radioactive Drug Research Committee and the FDA's divisions of oncology products.
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. This outsourcing has grown as drug development has become more complex.
- When deciding whether and how to use a CRO, companies consider tactical, maximal, or strategic outsourcing based on their internal resources. Selecting the right CRO requires evaluating their capabilities, compatibility with the sponsor's needs, and costs.
- Managing the sponsor-CRO relationship is critical to ensure success. This involves clearly defining roles and responsibilities, establishing performance metrics,
Frank Spinelli, New York City's Cabrini Medical Center - Speaker at the marcus evans PharmaMarketing Summit 2012, held in Wheeling, IL, April 30 - May 2, 2012, delivered his presentation entitled A Doctor’s Perspective on the Future Role of Pharmaceutical-Doctor Relationships and How They Are Evolving through Technology
Changing times ask for solid relationships.
Times are changing. A renewed interest from big pharma in the rare disease landscape
has awakened due to large-scale patent expirations, competition from generics &
biosimilars, anemic pipelines, escalating clinical trial costs and a global health-care
reform. This means that the traditional blockbuster model has become less viable while
the revenue-generating potential of orphan drugs has shown to be huge with a greater
return on investment than non-orphan drugs. According to EvaluatePharma, the orphan
drugs sales will grow at an annual rate of 11% and constitute 19% of the total share of
prescription drugs by 2020, totalling 176 billion dollars.
The rare disease landscape is very complex due to the large amount of stakeholders
involved. Despite their different interests they have one goal in common: getting an
orphan drug approval that will help save or improve lives. But there are many challenges
on the road to orphan drug designation:
• Complex and changing national and regional regulations
• Clinical trial design and finding & keeping patients
• The lack of a central database designed specifically to list patient registries, which
asks for close stakeholder engagement
• Partnering and establishing financing for future development
• Establishing a foundation for price that is balanced and sustainable
• Achieving an efficient and timely access to market with equal access for patients
around the world
• Achieving timely and correct diagnosis to enable higher quality of life and more time
and information for developers
More information: http://www.orphandrugssummit.com/
Rare Diseases: A Report on Orphan Drugs in the PipelinePhRMA
Rare diseases, when taken together, are not that rare at all. In fact, according to the National Institutes of Health (NIH), 30 million Americans have one of the nearly 7,000 diseases that are officially deemed “rare” because alone they each affect fewer than 200,000 people in the United States.
El documento describe las dos fases del desarrollo de medicamentos, incluyendo ensayos preclínicos y cuatro fases de ensayos clínicos para probar la seguridad y eficacia. Explica que las patentes permiten a las compañías recuperar costos de investigación y desarrollo, y que los medicamentos genéricos ofrecen opciones más baratas una vez que expiran las patentes originales.
‘Orphan drugs’ future growth potential for indian pharmaceutical marketNitin Patel
This document discusses orphan drugs and their potential in the Indian pharmaceutical market. It defines orphan drugs as those developed for rare diseases affecting less than 8% of the population. The US was the first to pass orphan drug legislation in 1983 to incentivize development of these drugs. Similar laws now exist in other countries and provide benefits like 7 years of market exclusivity. While initially developed for small populations, some orphan drugs like Rituxan have become very profitable blockbusters. The global orphan drug market was worth $50 billion in 2011 and is growing faster than other drug markets. Several Indian companies are now developing or manufacturing orphan drugs for rare diseases.
This document discusses the Orphan Drug Act and rare disease policies. It provides background on the US Orphan Drug Act of 1983, which aims to facilitate rare disease drug development by providing incentives. Key points include that the Act defines rare diseases as affecting fewer than 200,000 people, and has led to over 450 approved orphan drugs. However, the document notes that rare disease policies and the Orphan Drug Act are not the same. It advocates for India to enact its own rare disease policies and definitions to improve diagnosis, treatment and research. The Organization for Rare Diseases India is working towards this goal.
This document discusses orphan drugs, which are medications developed to treat rare diseases affecting small patient populations. It provides background on orphan drug regulations in the US and other countries, including incentives established by the Orphan Drug Act of 1983 in the US to encourage development of these drugs. The document also summarizes recent orphan drug approvals by the FDA, and provides market data showing the orphan drug market is growing significantly and expected to reach $355 billion by 2030. It concludes that developing orphan drugs faces unique challenges but is important for treating rare disease patients.
VAC3 Clinical Services is an Indian clinical research organization established in 2010 that provides global standard clinical and medical services including medical writing, clinical research, investigator selection and training, and regulatory consultation, focusing on therapeutic areas like oncology, cardiology, and neurology. Their team of experts have decades of experience in areas like clinical research, drug development, and medical marketing. They have conducted seminars on topics like drug development processes and ethics in clinical research.
This document summarizes a presentation on personalized medicine and companion diagnostics. It discusses:
1. How the FDA defines companion diagnostics and regulates them along with their corresponding drugs or therapies. This includes different types of biomarkers and validation requirements.
2. Key questions to consider regarding how a technology platform is used for infectious disease or oncology applications and in clinical decision making.
3. Examples of companion diagnostic technologies like tumor vaccines and an FDA approved test for Herceptin.
4. Potential regulatory pathways for companion diagnostic tests including clinical trials and clearance through 510(k) or PMA.
5. Conclusions that statistical analysis differs for co-developed drugs and diagnostics, and positioning a
The Central Drugs Standard Control Organisation (CDSCO) is India's national regulatory authority for pharmaceuticals and medical devices. It is responsible for approving new drugs, licensing manufacturing facilities, and monitoring drug quality. CDSCO's vision is to protect and promote public health in India. It has headquarters in New Delhi and offices across India. CDSCO regulates clinical trials, biosimilars, medical devices, and investigational new drugs. It also oversees state licensing authorities and provides guidance on drug regulation.
This document summarizes regulatory perspectives on approving drugs for rare diseases from a presentation given by an FDA official. It provides an overview of drug development for rare diseases before and after the 1983 Orphan Drug Act. While the Act increased incentives for rare disease drug development, approval challenges remain due to small patient populations and lack of understanding of rare diseases. The FDA aims to apply flexibility in effectiveness standards and innovative trial designs and analyses. Ongoing collaborations between the FDA, industry, academia, and patient groups are needed to continue advancing treatments for rare diseases.
Orphan Drugs – the Challenges and Benefits of Navigating FDA’s Regime Governi...Michael Swit
Webinar sponsored by The Weinberg Group on Orphan Drugs, covering these topics:
The Basics of the Orphan Drug Act
Benefits of Orphan Drug status
Exclusivity
Protocol assistance, tax credits, and research grants
When is an indication is “rare”?
Orphan Drug Designation Requests – ensuring yours
robust and persuasive
Approval criteria for orphan products – how they
compare to non-orphan products
Challenges in the Orphan Drug Process
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to
developing and commercializing novel therapeutics to tackle unmet medical needs.
Aptorum's current drug pipeline includes indications in orphan diseases, infectious
diseases, and metabolic diseases. In 2021, the company plans to bring two candidates to
clinical trials. Aptorum’s Smart-ACT™ platform is designed to bring an average of three
drug candidates for orphan diseases to clinical trials every 12-18 months. The company is
now preparing to launch a dietary supplement for women undergoing menopause and
experiencing related symptoms, including osteoporosis. Targeting a global woman’s health
supplement market that is expected to reach $17 billion in 2025, Aptorum is expected to
generate near-term revenue with significant long-term growth potential.
Clinical Pharmacology: Leveraging Science to Provide AccessE. Dennis Bashaw
1) The document discusses the paradigm shift in biomarker development from a "learn and confirm" model to an "identify, confirm, refine, and learn" model to continuously learn from clinical trials.
2) It outlines policies used by the FDA to incentivize orphan drug development, including expedited programs, marketing exclusivity, and fee reductions. International collaboration is important for developing policies tailored to individual countries.
3) Successful partnerships between the FDA, NIH, patient groups, and industry have helped advance rare disease drug development, including through biospecimen repositories and training programs.
The document discusses strategies for successful global drug development. It focuses on navigating FDA accelerated approval programs, new frontiers in personalized medicine, and evolving regulatory paradigms for digital health. The presentation provides an overview of key FDA expedited programs like fast track designation and breakthrough therapy designation. It also examines how regulators are adapting approaches to personalized medicine and digital health technologies.
The document summarizes FDA's drug review process. Key points:
1. FDA's Center for Drug Evaluation and Research (CDER) ensures drugs marketed in the US are safe and effective. CDER reviews new drug applications but does not test drugs itself.
2. Developing a new drug involves preclinical testing, clinical trials in 3 phases with increasing number of participants, and submitting a New Drug Application for FDA review.
3. The FDA review process evaluates whether clinical trials demonstrate a drug's safety and effectiveness for its intended use. If approved, the FDA continues monitoring the drug for safety after market.
ProRelix offer several professional, educational and informative services and resources for drug development and analysis on the industry to support clinical research
This document discusses approved regulatory bodies and agencies that regulate drugs. It provides information on regulatory authorities in countries like the US, EU, India, Japan, and their functions. The key regulatory bodies mentioned are the US FDA, EU, India's CDSCO under the Ministry of Health and Family Welfare. The functions of regulatory bodies include drug evaluation, monitoring safety and efficacy, licensing, and adverse reaction monitoring. They aim to ensure drug safety, efficacy, and quality. Harmonization of regulatory standards globally and strengthening authorities are needed to regulate the pharmaceutical industry effectively.
The document provides an overview of the Investigational New Drug Application (IND) and New Drug Application (NDA) processes required by the FDA to develop and approve new drugs. It describes how developing a new drug takes 15 years and over $900 million on average. The IND allows testing of new drugs in humans and provides safety data, while the NDA provides all clinical trial data for the FDA to determine if the drug is safe and effective for approval. Both the IND and NDA are lengthy applications that require extensive non-clinical and clinical data to gain FDA approval to market a new prescription drug.
The document introduces four students who are members of the team "Pakistan Pharma Career Door". It provides brief descriptions of each student, noting their educational achievements and strengths. Sameeta Malik is an energetic student engaged in scientific activities at Dow College of Pharmacy. Iffrah Naushad is a meritorious student with experience attending national events. Abira Khalid is an associate of Dow College of Pharmacy who is talented and one of the genius students. Qaisara Boota is one of the most active students who is highly courageous and ready to take on difficult tasks. The document was prepared by these four students from Dow College of Pharmacy.
The Role of RWE in Drug Development_4Jun2015_finalBilly Franks
This document discusses how real-world evidence (RWE) can be incorporated earlier and more broadly in the drug development process. It proposes a revised model where RWE is used from early research through commercialization to inform target profiles, messaging on unmet needs, and clinical trial design. Specifically, it suggests using RWE to "stress test" enrollment projections and identify referral sources. The document also advocates for moving beyond observational research to conduct cluster-randomized real-world experiments addressing outcomes important to payers and patients like treatment patterns and costs.
Bay View Advisory Services is a subsidiary of Bay View Clinic that provides hospital management consulting, research and development consultancy, and hospital project consulting. It has a team of experienced professionals with expertise in various areas of healthcare, including clinical research, hospital administration, and biomedical engineering. The company aims to help hospitals and healthcare organizations with services spanning concept development, operations management, research, and regulatory compliance. It also runs online platforms for medical equipment sales and education institution listings.
Rare Disease Series Slide Deck Part 1.pptxMMS Holdings
This document summarizes an MMS Expert Insights webinar on rare disease research in the United States. The webinar discusses challenges in rare disease research such as limited patient populations and understanding of diseases. It provides an overview of the regulatory history around orphan drugs and incentives in the US. It also discusses how the FDA flexibly applies regulatory standards for rare disease drug development, including allowing single efficacy studies, modified clinical trial designs, and use of existing data in some cases.
Regulatory aspects of orphan drugs devolpmentsJITHIN K JOY
This document discusses regulatory aspects of orphan drugs and developments. It begins by defining orphan diseases and the need for orphan drug regulation to incentivize development of treatments for rare conditions. It describes orphan drug regulations in various countries like the US, Japan, Australia and challenges in developing orphan drugs. In India, around 6000-8000 rare diseases have been identified but many lack treatments. The document calls for India to introduce its own orphan drug act to define rare diseases, provide incentives for research and improve access to existing orphan drugs.
Anny Serafina Love - Letter of Recommendation by Kellen Harkins, MS.AnnySerafinaLove
This letter, written by Kellen Harkins, Course Director at Full Sail University, commends Anny Love's exemplary performance in the Video Sharing Platforms class. It highlights her dedication, willingness to challenge herself, and exceptional skills in production, editing, and marketing across various video platforms like YouTube, TikTok, and Instagram.
4 Benefits of Partnering with an OnlyFans Agency for Content Creators.pdfonlyfansmanagedau
In the competitive world of content creation, standing out and maximising revenue on platforms like OnlyFans can be challenging. This is where partnering with an OnlyFans agency can make a significant difference. Here are five key benefits for content creators considering this option:
Starting a business is like embarking on an unpredictable adventure. It’s a journey filled with highs and lows, victories and defeats. But what if I told you that those setbacks and failures could be the very stepping stones that lead you to fortune? Let’s explore how resilience, adaptability, and strategic thinking can transform adversity into opportunity.
[To download this presentation, visit:
https://www.oeconsulting.com.sg/training-presentations]
This presentation is a curated compilation of PowerPoint diagrams and templates designed to illustrate 20 different digital transformation frameworks and models. These frameworks are based on recent industry trends and best practices, ensuring that the content remains relevant and up-to-date.
Key highlights include Microsoft's Digital Transformation Framework, which focuses on driving innovation and efficiency, and McKinsey's Ten Guiding Principles, which provide strategic insights for successful digital transformation. Additionally, Forrester's framework emphasizes enhancing customer experiences and modernizing IT infrastructure, while IDC's MaturityScape helps assess and develop organizational digital maturity. MIT's framework explores cutting-edge strategies for achieving digital success.
These materials are perfect for enhancing your business or classroom presentations, offering visual aids to supplement your insights. Please note that while comprehensive, these slides are intended as supplementary resources and may not be complete for standalone instructional purposes.
Frameworks/Models included:
Microsoft’s Digital Transformation Framework
McKinsey’s Ten Guiding Principles of Digital Transformation
Forrester’s Digital Transformation Framework
IDC’s Digital Transformation MaturityScape
MIT’s Digital Transformation Framework
Gartner’s Digital Transformation Framework
Accenture’s Digital Strategy & Enterprise Frameworks
Deloitte’s Digital Industrial Transformation Framework
Capgemini’s Digital Transformation Framework
PwC’s Digital Transformation Framework
Cisco’s Digital Transformation Framework
Cognizant’s Digital Transformation Framework
DXC Technology’s Digital Transformation Framework
The BCG Strategy Palette
McKinsey’s Digital Transformation Framework
Digital Transformation Compass
Four Levels of Digital Maturity
Design Thinking Framework
Business Model Canvas
Customer Journey Map
[To download this presentation, visit:
https://www.oeconsulting.com.sg/training-presentations]
This PowerPoint compilation offers a comprehensive overview of 20 leading innovation management frameworks and methodologies, selected for their broad applicability across various industries and organizational contexts. These frameworks are valuable resources for a wide range of users, including business professionals, educators, and consultants.
Each framework is presented with visually engaging diagrams and templates, ensuring the content is both informative and appealing. While this compilation is thorough, please note that the slides are intended as supplementary resources and may not be sufficient for standalone instructional purposes.
This compilation is ideal for anyone looking to enhance their understanding of innovation management and drive meaningful change within their organization. Whether you aim to improve product development processes, enhance customer experiences, or drive digital transformation, these frameworks offer valuable insights and tools to help you achieve your goals.
INCLUDED FRAMEWORKS/MODELS:
1. Stanford’s Design Thinking
2. IDEO’s Human-Centered Design
3. Strategyzer’s Business Model Innovation
4. Lean Startup Methodology
5. Agile Innovation Framework
6. Doblin’s Ten Types of Innovation
7. McKinsey’s Three Horizons of Growth
8. Customer Journey Map
9. Christensen’s Disruptive Innovation Theory
10. Blue Ocean Strategy
11. Strategyn’s Jobs-To-Be-Done (JTBD) Framework with Job Map
12. Design Sprint Framework
13. The Double Diamond
14. Lean Six Sigma DMAIC
15. TRIZ Problem-Solving Framework
16. Edward de Bono’s Six Thinking Hats
17. Stage-Gate Model
18. Toyota’s Six Steps of Kaizen
19. Microsoft’s Digital Transformation Framework
20. Design for Six Sigma (DFSS)
To download this presentation, visit:
https://www.oeconsulting.com.sg/training-presentations
Zodiac Signs and Food Preferences_ What Your Sign Says About Your Tastemy Pandit
Know what your zodiac sign says about your taste in food! Explore how the 12 zodiac signs influence your culinary preferences with insights from MyPandit. Dive into astrology and flavors!
Brian Fitzsimmons on the Business Strategy and Content Flywheel of Barstool S...Neil Horowitz
On episode 272 of the Digital and Social Media Sports Podcast, Neil chatted with Brian Fitzsimmons, Director of Licensing and Business Development for Barstool Sports.
What follows is a collection of snippets from the podcast. To hear the full interview and more, check out the podcast on all podcast platforms and at www.dsmsports.net
Profiles of Iconic Fashion Personalities.pdfTTop Threads
The fashion industry is dynamic and ever-changing, continuously sculpted by trailblazing visionaries who challenge norms and redefine beauty. This document delves into the profiles of some of the most iconic fashion personalities whose impact has left a lasting impression on the industry. From timeless designers to modern-day influencers, each individual has uniquely woven their thread into the rich fabric of fashion history, contributing to its ongoing evolution.
IMPACT Silver is a pure silver zinc producer with over $260 million in revenue since 2008 and a large 100% owned 210km Mexico land package - 2024 catalysts includes new 14% grade zinc Plomosas mine and 20,000m of fully funded exploration drilling.
Digital Marketing with a Focus on Sustainabilitysssourabhsharma
Digital Marketing best practices including influencer marketing, content creators, and omnichannel marketing for Sustainable Brands at the Sustainable Cosmetics Summit 2024 in New York
The Most Inspiring Entrepreneurs to Follow in 2024.pdfthesiliconleaders
In a world where the potential of youth innovation remains vastly untouched, there emerges a guiding light in the form of Norm Goldstein, the Founder and CEO of EduNetwork Partners. His dedication to this cause has earned him recognition as a Congressional Leadership Award recipient.
Best practices for project execution and deliveryCLIVE MINCHIN
A select set of project management best practices to keep your project on-track, on-cost and aligned to scope. Many firms have don't have the necessary skills, diligence, methods and oversight of their projects; this leads to slippage, higher costs and longer timeframes. Often firms have a history of projects that simply failed to move the needle. These best practices will help your firm avoid these pitfalls but they require fortitude to apply.
Garments ERP Software in Bangladesh _ Pridesys IT Ltd.pdfPridesys IT Ltd.
Pridesys Garments ERP is one of the leading ERP solution provider, especially for Garments industries which is integrated with
different modules that cover all the aspects of your Garments Business. This solution supports multi-currency and multi-location
based operations. It aims at keeping track of all the activities including receiving an order from buyer, costing of order, resource
planning, procurement of raw materials, production management, inventory management, import-export process, order
reconciliation process etc. It’s also integrated with other modules of Pridesys ERP including finance, accounts, HR, supply-chain etc.
With this automated solution you can easily track your business activities and entire operations of your garments manufacturing
proces
The Genesis of BriansClub.cm Famous Dark WEb PlatformSabaaSudozai
BriansClub.cm, a famous platform on the dark web, has become one of the most infamous carding marketplaces, specializing in the sale of stolen credit card data.
Part 2 Deep Dive: Navigating the 2024 Slowdownjeffkluth1
Introduction
The global retail industry has weathered numerous storms, with the financial crisis of 2008 serving as a poignant reminder of the sector's resilience and adaptability. However, as we navigate the complex landscape of 2024, retailers face a unique set of challenges that demand innovative strategies and a fundamental shift in mindset. This white paper contrasts the impact of the 2008 recession on the retail sector with the current headwinds retailers are grappling with, while offering a comprehensive roadmap for success in this new paradigm.
1. Orphan Medication Status in Japan (Orphan Item Approvals)
Key offerings: Create Technique and Certification to Acquire Orphan Medication Status in Japan
Identify regulating path
Literature Look for the Industry (the variety of individual and healthcare practice)
Identify Unmet Medical Needs for Orphan Products
Consultation with KOLs in Japan
Negotiate with MHLW (Ministry of Wellness, Work and Welfare)
Manage Medical Trials
File NDA for Orphan Products
Support Integrating Initiatives for Orphan Products
Apr 14, 2010
PLC Appointed as JETRO US Medical Business Advisor
Pacific-Link Consulting (PLC) ..
2. Our consultants have particular expertise with obtaining Orphan Drug Designations in Japan and the United
States for novel biologics and drugs with a successful track record of approves through the PMDA and FDA. We
also specialize in Japanese Bridging Strategies and clinical trial applications in Japan forOrphan Drugproducts.
Pacific-Link is committed to support clients in meeting their business objectives, project milestones and
customizes our approach to the needs of the client company.
Orphan Drug
3. Pacific-Link Consulting can help the clinical improvement needs for our customer's starting clinical trials.
Key offerings: Regulatory Strategy Development and Consultation
JapaneseOrphan DrugDesignation and Bridging Strategies
Medical and Technical writing for reg. submissions (e.g. CSRs, CMC Sections, Module 2 Summaries)
Preparation of electronic Common Technical Dossier (eCTD) applications for submission to Health Authorities
Investigational New Drug Applications (INDs) and Investigational Medicinal Product Dossiers (IMPDs) in the Unites States, Japan and Europe
4. Our expert Regulatory staff can develop strategic regulatory plans, facilitate Agency in the United States and Japan.
Key offerings:Orphan Drug& Designation prepare documents
Request and hold meetings
5. Dr. Tanimoto is a pharmaceutical professional with over 15 years of experience in clinical research and development of biotechnology products. She is the
Managing Director for Pacific-Link Consulting providing a range of development services to biotechnology and pharmaceutical companies.
Orphan Drug
6. Ms. Joyce Reyes is an executive with over 15 years of Regulatory Affairs and Quality Assurance experience in various roles at both small and large
biotechnology or pharmaceutical companies. Ms. Reyes has extensive experience in cell and viral production processes and has been in both
manufacturing and quality assurance roles prior to joining regulatory groups at various companies.
7. Orphan Drug Designation in Japan (Orphan Product Approvals)
·Develop Strategy and Documentation to ObtainOrphan DrugDesignation in Japan
·Identify regulatory path
·Literature Search for the Market (the number of patient and medical practice)
·Identify Unmet Medical Needs for Orphan Products
·Consultation with KOLs in Japan
·Negotiate with MHLW (Ministry of Health, Labor and Welfare)
·Manage Clinical Trials
·File NDA for Orphan Products
8. Full Project Management
Manage Manufacturing site and prepare study drug for clinical trial
Manage GLP Toxicology Studies
Work with Academia on Preclinical Program
FDA Interactions and Consultation
Pre-Pre IND Consultation
Pre-IND Meeting
Multiple informal discussions, consultations and negotiation
Early FDA Interactions and PreIND Meeting
File NIH RAC
File IND
Clear IND
Prepare and conduct Phase I/II Stud
9. An overview on Japan Orphan Designation
In Japan, drugs and medical devices could be designated asorphan drugs
or medical devices based on the Article 77-2 of the Pharmaceutical
Affairs Law. This was in the event that they are expected for use
in fewer than 50,000 patients in Japan. For which there is a high
medical necessity. They are designated by the Minister of Health,
Labor and Welfare based on the idea of the Pharmaceutical Affairs
and Food Sanitation Council (PAFSC).
•Japan Orphan Designationdoes not immediately prompt promoting approbation.
•The objectives and diagram of the system are described beneath.
10. The basis of the designation system
TheMHLWOrphandrug/medical apparatus system had established
drugs and medical devices to be used for the treatment of troublesome
to-treat diseases and (AIDS) had not been sufficiently created.
This was despite the high medical needs because the amount of
patients was small. With the diversification of open healthcare needs,
safe and quality medical products were obliged to be supplied to
patients as soon as possible. As needs be, there is a huge increase in rising
open expectations and the changing circumstances of drug and medical-
mechanism research and improvement. It had been chosen to take special
measures to support and push research activities for the advancement
of orphan drugs/medical devices.
OrphanDrug
11. Criteria for designation
The Minister of Health, Labor and Welfare may designate drugs
and medical devices satisfying the accompanying criteria as orphan drugs
medical devices. This was in the wake of getting applications for
OrphanDrugfrom the applicants. The amount of
patients who may use the drug or medical mechanism should be less
than 50,000 in Japan. The amount of patients could be estimated
based on the report of Health and Labor Science Research or the
information published by solid scientific societies. The amount of patients
with a challenging to-treat disease is sometimes troublesome to estimate
correctly because of absence of research on the patient populace.