Global Medical Cures™ | Novel New Drugs- 2013 Summary CDER FDA
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Dr Dev Kambhampati | FDA CDER Novel New Drugs Approved, 2013Dr Dev Kambhampati
CDER approved 27 novel new drugs in 2013, similar to the average of previous years. Many of these drugs treat rare diseases or cancers, and one-third were first-in-class treatments. CDER used expedited review pathways for nearly half of the drugs to help bring important new treatments to patients faster.
Endometrial Cancer Global Clinical Trials Review, H2, 2012ReportsnReports
The document provides a 107-page report on endometrial cancer clinical trials in the second half of 2012. It details the number of clinical trials conducted by region, country, phase, status, subjects recruited, and leading sponsors. The report aims to give an overview of the global clinical trial landscape for endometrial cancer and benchmark trial activity and trends over time. It is available for individual purchase at a price of $2,500.
Gastric Cancer Global Clinical Trials Review, H2, 2012ReportsnReports
The document provides a summary of a report on gastric cancer clinical trials in the second half of 2012. It includes 123 pages of data on over 1,000 clinical trials conducted globally, including information on the number and location of trials, participating companies and institutions, trial status and recruitment numbers. The report is available for purchase for $2,500 and provides detailed insight into ongoing and discontinued gastric cancer trials worldwide.
Fast track drug development is a process designed to expedite FDA review of drugs for serious conditions with unmet medical needs. Drugs approved through this process treat life-threatening diseases like AIDS, cancer, and Alzheimer's. The process provides sponsors with more frequent guidance from the FDA, accelerated approval if certain criteria are met, and priority review to receive approval more quickly if all requirements are satisfied.
This document discusses clinical practice guidelines for oncology treatment. It defines what guidelines are and their purpose in helping clinicians make complex treatment decisions. Guidelines should be based on systematic reviews of evidence, developed by multidisciplinary experts, and provide recommendations along with ratings of evidence quality. The document notes limitations of guidelines and the need for them to be relevant to the local healthcare system and population. It advocates for guidelines developed through a transparent process with management of conflicts of interest.
Investigational new drug application must be submitted after discovering a new drug and before beginning of clinical trials. Here given a brief note on the topic.The topics included are types of IND, criteria for application, Information in IND application, resources for IND application, laws.regulations, policies and procedures, IND forms and instructions, IND content requirements and review of IND
IND (Investigational New Drug) industrial perspectiveAYESHA NAZEER
Describing the Industry's/sponsor's/drug manufacturers' perspective of the Investigational New Drug Application (IND) program based on the survey conducted by the Office Of Inspector General (OIG).
AN OVERVIEW ON FIXED DOSE COMBINATIONS AND ITS REGULATIONS IN INDIA JAYA PRAKASH VELUCHURI
This document provides an overview of fixed dose combination drugs and regulatory requirements for FDCs in India. It discusses the classification of FDCs, clinical trial requirements, bioavailability and bioequivalence data requirements. It also addresses the reasons why 328 FDCs were banned in India, including that they were found to have no therapeutic justification and safer alternatives were available. Success factors for FDCs include addressing formulation challenges, patent feasibility, and physician considerations.
Dr Dev Kambhampati | FDA CDER Novel New Drugs Approved, 2013Dr Dev Kambhampati
CDER approved 27 novel new drugs in 2013, similar to the average of previous years. Many of these drugs treat rare diseases or cancers, and one-third were first-in-class treatments. CDER used expedited review pathways for nearly half of the drugs to help bring important new treatments to patients faster.
Endometrial Cancer Global Clinical Trials Review, H2, 2012ReportsnReports
The document provides a 107-page report on endometrial cancer clinical trials in the second half of 2012. It details the number of clinical trials conducted by region, country, phase, status, subjects recruited, and leading sponsors. The report aims to give an overview of the global clinical trial landscape for endometrial cancer and benchmark trial activity and trends over time. It is available for individual purchase at a price of $2,500.
Gastric Cancer Global Clinical Trials Review, H2, 2012ReportsnReports
The document provides a summary of a report on gastric cancer clinical trials in the second half of 2012. It includes 123 pages of data on over 1,000 clinical trials conducted globally, including information on the number and location of trials, participating companies and institutions, trial status and recruitment numbers. The report is available for purchase for $2,500 and provides detailed insight into ongoing and discontinued gastric cancer trials worldwide.
Fast track drug development is a process designed to expedite FDA review of drugs for serious conditions with unmet medical needs. Drugs approved through this process treat life-threatening diseases like AIDS, cancer, and Alzheimer's. The process provides sponsors with more frequent guidance from the FDA, accelerated approval if certain criteria are met, and priority review to receive approval more quickly if all requirements are satisfied.
This document discusses clinical practice guidelines for oncology treatment. It defines what guidelines are and their purpose in helping clinicians make complex treatment decisions. Guidelines should be based on systematic reviews of evidence, developed by multidisciplinary experts, and provide recommendations along with ratings of evidence quality. The document notes limitations of guidelines and the need for them to be relevant to the local healthcare system and population. It advocates for guidelines developed through a transparent process with management of conflicts of interest.
Investigational new drug application must be submitted after discovering a new drug and before beginning of clinical trials. Here given a brief note on the topic.The topics included are types of IND, criteria for application, Information in IND application, resources for IND application, laws.regulations, policies and procedures, IND forms and instructions, IND content requirements and review of IND
IND (Investigational New Drug) industrial perspectiveAYESHA NAZEER
Describing the Industry's/sponsor's/drug manufacturers' perspective of the Investigational New Drug Application (IND) program based on the survey conducted by the Office Of Inspector General (OIG).
AN OVERVIEW ON FIXED DOSE COMBINATIONS AND ITS REGULATIONS IN INDIA JAYA PRAKASH VELUCHURI
This document provides an overview of fixed dose combination drugs and regulatory requirements for FDCs in India. It discusses the classification of FDCs, clinical trial requirements, bioavailability and bioequivalence data requirements. It also addresses the reasons why 328 FDCs were banned in India, including that they were found to have no therapeutic justification and safer alternatives were available. Success factors for FDCs include addressing formulation challenges, patent feasibility, and physician considerations.
An IND application is submitted to the FDA to request permission to conduct clinical trials on an investigational new drug. It contains preclinical animal and toxicity data, manufacturing information, and clinical trial protocols and investigator information. The FDA reviews the IND over 30 days to ensure the protection of human subjects and that the investigational plan allows for evaluation of safety and effectiveness. Clinical trials cannot begin until the IND is approved or the 30-day review period has ended without FDA objection. Sponsors must submit annual reports updating the FDA on the progress of investigations under the IND.
This document provides guidance on IND (Investigational New Drug) regulations for initial Phase 1 clinical trials. It clarifies that Phase 1 IND submissions require basic information to ensure safety rather than detailed development plans. For protocols, manufacturing information, and other sections, flexibility is allowed to adapt to early study results. The focus should be on safety aspects like monitoring and dose adjustments rather than predetermined experimental designs. This guidance aims to help sponsors provide sufficient information without being overly burdensome for early phase trials.
The document provides an overview of the regulatory process for bringing a new drug to market, beginning with pre-clinical studies and submission of an Investigational New Drug (IND) application to the FDA. If approved, the IND allows clinical trials to be conducted in three phases to evaluate safety and efficacy. If phase 3 trials demonstrate a drug is safe and effective, a New Drug Application or Biologics License Application can be submitted for approval to market the drug. Ongoing monitoring of safety continues even after approval.
This document discusses expedited drug development and review pathways at the FDA, including Subpart E and Subpart H. Subpart E allows for an expedited approval based on early consultation and compressed clinical trials for drugs treating serious diseases lacking alternatives. Subpart H, called accelerated approval, allows for earlier approval based on surrogate endpoints, with required post-approval studies and restrictions until clinical benefit is verified. Both aim to get drugs to patients faster while maintaining safety standards.
This document provides guidance on the Investigator's Brochure (IB), which compiles clinical and nonclinical data on investigational products relevant for human subject studies. The IB aims to inform investigators and others about the product's dose, administration, and safety monitoring. It also helps clinicians independently assess the risks and benefits of a proposed trial. The IB includes a summary of nonclinical pharmacology, toxicology, and human effects studies. It provides guidance to investigators on understanding the potential risks, adverse reactions, and precautions needed for safe clinical trial conduct.
This document summarizes findings from a survey of 50 primary care physicians on their knowledge and perceptions related to treating type 2 diabetes. The survey found that many physicians were unfamiliar with current guidelines regarding appropriate A1c targets and misunderstood results from the ACCORD trial. Physicians were also more likely to recall claims made in marketing materials compared to evidence from clinical studies. Overall, the survey suggests that disseminating evidence-based practices to physicians remains challenging and that new strategies are needed to accelerate adoption of optimal treatment guidelines.
This article summarizes a large healthcare system's comprehensive approach to reducing inappropriate opioid prescribing. The healthcare system implemented policies restricting opioid prescriptions, monitoring processes for patients on long-term opioids, and integrated these changes into their electronic health records. An evaluation found reductions in high dose opioid prescriptions, large opioid prescriptions, combination opioid prescriptions, and brand name opioid prescriptions after implementing these interventions between 2010-2015. The article concludes the interventions were effective in positively affecting opioid prescribing practices in this healthcare system.
This document discusses the rise of contract research organizations (CROs) that conduct clinical trials and other services on behalf of pharmaceutical companies. It notes that CROs have grown dramatically since the 1980s as drug companies increasingly outsource research to cut costs. While CROs claim to provide independent research, some experts argue they still face conflicts of interest since they rely on industry funding. The document also examines how CROs are expanding globally and investing in new technologies to increase their service capacity and distinguish themselves from competitors in a growing market.
Application for New Drug Discovery (NDD) according to USFDA guidelines Bharathiar university
The document discusses the US FDA's process for approving new drug applications (NDAs). The FDA is responsible for ensuring drugs are safe, effective and quality-controlled. Developers must submit clinical data to FDA's Center for Drug Evaluation and Research which reviews the data. If benefits outweigh risks, the drug receives approval. For serious conditions, accelerated approval is possible where post-market studies confirm initial findings. Designations like Fast Track can expedite review of innovative drugs that address unmet needs. A complete NDA includes manufacturing and clinical information. Advisory committees provide recommendations to FDA on approval.
Fast track Designation is a designation for accelerated approval of drugs and medicines in US. Presentation contains brief view of this expedite program.
The document discusses access to drugs for rare diseases in Canada. It notes that Canada currently has no orphan drug policy or definition of rare diseases, unlike the US and EU. As a result, Canadian patients have access to only about half of the orphan drugs approved in the US and EU. A new proposed Canadian orphan drug regulatory framework aims to align with international standards to promote drug development and patient access. It also discusses challenges for drug reimbursement in Canada given the high costs of rare disease drugs and limitations of current review processes. Lifecycle approaches and managed entry programs are proposed to help improve sustainable access.
Global Medical Cures™ | Diabetic Retinopathy
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | Prescription Painkiller Overdoses (Growing Epidemic A...Global Medical Cures™
The document summarizes key findings from the CDC about the growing problem of prescription painkiller overdoses among women:
- Prescription painkiller overdoses are a leading cause of death among women, killing over 18 women per day in 2010. The rate of increase in overdose deaths has been greater among women than men since 1999.
- Women between ages 25-54 are most at risk of emergency department visits related to prescription painkiller misuse or abuse. Non-Hispanic white and Native American women have the highest risk of overdose death.
- The prescription painkiller epidemic affects women differently than men due to factors like higher rates of chronic pain and prescription rates among women.
Global Medical Cures™ | Cholesterol Medicines
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Cirrhosis is scarring of the liver caused by long-term liver damage and disease. It can be asymptomatic in early stages but progresses to cause fatigue, loss of appetite, nausea, weight loss, easy bruising, fluid buildup, and jaundice. Cirrhosis is most often caused by alcohol abuse, hepatitis B/C, fatty liver disease, and genetic disorders. It is diagnosed through blood tests, imaging, and liver biopsy showing scar tissue buildup. While cirrhosis cannot be reversed, treatment focuses on managing the underlying cause to prevent further damage, and may include medications, lifestyle changes, and liver transplant for severe cases.
The document summarizes new features in the next version of Hyper-V including improved Hyper-V Manager, ability to dynamically resize VM memory and add/remove network adapters while running, support for Linux secure boot, production checkpoints, cluster upgrades without downtime, PowerShell direct on VMs, and other resiliency and container improvements. It also discusses VM versioning and manual upgrades being required when moving to new versions.
Global Medical Cures™ | USA Chartbook on HealthCare for Blacks
IMPORTANT NOTE TO USERS OF WEBSITE & DOCUMENTS POSTED ON SLIDESHARE- Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
www.globalmedicalcures.com
Global Medical Cures™ | Is my Child at risk for KIDNEY DISEASE?
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | Health Risks of Being OVERWEIGHT
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Skeletal muscles help the body move and are connected to bones by tendons. There are over 600 muscles in the human body that perform different jobs like movement, breathing, and pumping blood. Healthy muscles are important for physical activities and preventing injuries. To keep muscles healthy, one should engage in regular physical activity, eat a nutritious diet, stay hydrated, gradually increase activity levels, and rest injured muscles. Proper warmups, cooldowns, stretching and protective gear can also help prevent muscle strains or injuries.
Global Medical Cures™ | Physical Activity Guidelines for Americans
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
An IND application is submitted to the FDA to request permission to conduct clinical trials on an investigational new drug. It contains preclinical animal and toxicity data, manufacturing information, and clinical trial protocols and investigator information. The FDA reviews the IND over 30 days to ensure the protection of human subjects and that the investigational plan allows for evaluation of safety and effectiveness. Clinical trials cannot begin until the IND is approved or the 30-day review period has ended without FDA objection. Sponsors must submit annual reports updating the FDA on the progress of investigations under the IND.
This document provides guidance on IND (Investigational New Drug) regulations for initial Phase 1 clinical trials. It clarifies that Phase 1 IND submissions require basic information to ensure safety rather than detailed development plans. For protocols, manufacturing information, and other sections, flexibility is allowed to adapt to early study results. The focus should be on safety aspects like monitoring and dose adjustments rather than predetermined experimental designs. This guidance aims to help sponsors provide sufficient information without being overly burdensome for early phase trials.
The document provides an overview of the regulatory process for bringing a new drug to market, beginning with pre-clinical studies and submission of an Investigational New Drug (IND) application to the FDA. If approved, the IND allows clinical trials to be conducted in three phases to evaluate safety and efficacy. If phase 3 trials demonstrate a drug is safe and effective, a New Drug Application or Biologics License Application can be submitted for approval to market the drug. Ongoing monitoring of safety continues even after approval.
This document discusses expedited drug development and review pathways at the FDA, including Subpart E and Subpart H. Subpart E allows for an expedited approval based on early consultation and compressed clinical trials for drugs treating serious diseases lacking alternatives. Subpart H, called accelerated approval, allows for earlier approval based on surrogate endpoints, with required post-approval studies and restrictions until clinical benefit is verified. Both aim to get drugs to patients faster while maintaining safety standards.
This document provides guidance on the Investigator's Brochure (IB), which compiles clinical and nonclinical data on investigational products relevant for human subject studies. The IB aims to inform investigators and others about the product's dose, administration, and safety monitoring. It also helps clinicians independently assess the risks and benefits of a proposed trial. The IB includes a summary of nonclinical pharmacology, toxicology, and human effects studies. It provides guidance to investigators on understanding the potential risks, adverse reactions, and precautions needed for safe clinical trial conduct.
This document summarizes findings from a survey of 50 primary care physicians on their knowledge and perceptions related to treating type 2 diabetes. The survey found that many physicians were unfamiliar with current guidelines regarding appropriate A1c targets and misunderstood results from the ACCORD trial. Physicians were also more likely to recall claims made in marketing materials compared to evidence from clinical studies. Overall, the survey suggests that disseminating evidence-based practices to physicians remains challenging and that new strategies are needed to accelerate adoption of optimal treatment guidelines.
This article summarizes a large healthcare system's comprehensive approach to reducing inappropriate opioid prescribing. The healthcare system implemented policies restricting opioid prescriptions, monitoring processes for patients on long-term opioids, and integrated these changes into their electronic health records. An evaluation found reductions in high dose opioid prescriptions, large opioid prescriptions, combination opioid prescriptions, and brand name opioid prescriptions after implementing these interventions between 2010-2015. The article concludes the interventions were effective in positively affecting opioid prescribing practices in this healthcare system.
This document discusses the rise of contract research organizations (CROs) that conduct clinical trials and other services on behalf of pharmaceutical companies. It notes that CROs have grown dramatically since the 1980s as drug companies increasingly outsource research to cut costs. While CROs claim to provide independent research, some experts argue they still face conflicts of interest since they rely on industry funding. The document also examines how CROs are expanding globally and investing in new technologies to increase their service capacity and distinguish themselves from competitors in a growing market.
Application for New Drug Discovery (NDD) according to USFDA guidelines Bharathiar university
The document discusses the US FDA's process for approving new drug applications (NDAs). The FDA is responsible for ensuring drugs are safe, effective and quality-controlled. Developers must submit clinical data to FDA's Center for Drug Evaluation and Research which reviews the data. If benefits outweigh risks, the drug receives approval. For serious conditions, accelerated approval is possible where post-market studies confirm initial findings. Designations like Fast Track can expedite review of innovative drugs that address unmet needs. A complete NDA includes manufacturing and clinical information. Advisory committees provide recommendations to FDA on approval.
Fast track Designation is a designation for accelerated approval of drugs and medicines in US. Presentation contains brief view of this expedite program.
The document discusses access to drugs for rare diseases in Canada. It notes that Canada currently has no orphan drug policy or definition of rare diseases, unlike the US and EU. As a result, Canadian patients have access to only about half of the orphan drugs approved in the US and EU. A new proposed Canadian orphan drug regulatory framework aims to align with international standards to promote drug development and patient access. It also discusses challenges for drug reimbursement in Canada given the high costs of rare disease drugs and limitations of current review processes. Lifecycle approaches and managed entry programs are proposed to help improve sustainable access.
Global Medical Cures™ | Diabetic Retinopathy
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | Prescription Painkiller Overdoses (Growing Epidemic A...Global Medical Cures™
The document summarizes key findings from the CDC about the growing problem of prescription painkiller overdoses among women:
- Prescription painkiller overdoses are a leading cause of death among women, killing over 18 women per day in 2010. The rate of increase in overdose deaths has been greater among women than men since 1999.
- Women between ages 25-54 are most at risk of emergency department visits related to prescription painkiller misuse or abuse. Non-Hispanic white and Native American women have the highest risk of overdose death.
- The prescription painkiller epidemic affects women differently than men due to factors like higher rates of chronic pain and prescription rates among women.
Global Medical Cures™ | Cholesterol Medicines
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Cirrhosis is scarring of the liver caused by long-term liver damage and disease. It can be asymptomatic in early stages but progresses to cause fatigue, loss of appetite, nausea, weight loss, easy bruising, fluid buildup, and jaundice. Cirrhosis is most often caused by alcohol abuse, hepatitis B/C, fatty liver disease, and genetic disorders. It is diagnosed through blood tests, imaging, and liver biopsy showing scar tissue buildup. While cirrhosis cannot be reversed, treatment focuses on managing the underlying cause to prevent further damage, and may include medications, lifestyle changes, and liver transplant for severe cases.
The document summarizes new features in the next version of Hyper-V including improved Hyper-V Manager, ability to dynamically resize VM memory and add/remove network adapters while running, support for Linux secure boot, production checkpoints, cluster upgrades without downtime, PowerShell direct on VMs, and other resiliency and container improvements. It also discusses VM versioning and manual upgrades being required when moving to new versions.
Global Medical Cures™ | USA Chartbook on HealthCare for Blacks
IMPORTANT NOTE TO USERS OF WEBSITE & DOCUMENTS POSTED ON SLIDESHARE- Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
www.globalmedicalcures.com
Global Medical Cures™ | Is my Child at risk for KIDNEY DISEASE?
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | Health Risks of Being OVERWEIGHT
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Skeletal muscles help the body move and are connected to bones by tendons. There are over 600 muscles in the human body that perform different jobs like movement, breathing, and pumping blood. Healthy muscles are important for physical activities and preventing injuries. To keep muscles healthy, one should engage in regular physical activity, eat a nutritious diet, stay hydrated, gradually increase activity levels, and rest injured muscles. Proper warmups, cooldowns, stretching and protective gear can also help prevent muscle strains or injuries.
Global Medical Cures™ | Physical Activity Guidelines for Americans
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | Handbook- Genetic Mutations & How it Impacts HealthGlobal Medical Cures™
Global Medical Cures™ | Handbook- Genetic Mutations & How it Impacts Health
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | Directory of Diabetes Organizations
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
This directory lists Government agencies and professional and voluntary associations that provide diabetes information and resources. Some of these diabetes organizations offer educational materials and support to people with diabetes and the general public, while others serve primarily health care providers.
Global Medical Cures™ | Prostate Cancer- TREATMENT CHOICES
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | BREAST CANCER- Reducing the Risk with MedicineGlobal Medical Cures™
Global Medical Cures™ | BREAST CANCER- Reducing the Risk with Medicine
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | Womens health - Sexually Transmitted InfectionsGlobal Medical Cures™
This document provides information about sexually transmitted infections (STIs), including what they are, how they are transmitted, common symptoms, and testing options. It states that STIs are common in the US, with an estimated 19 million new cases each year. While both men and women can contract STIs, the document notes that women often experience more frequent and severe complications. It emphasizes that taking protective measures like condom use can help reduce the risk of acquiring an STI. The bulk of the document then provides detailed overviews of several common STIs, including chlamydia, gonorrhea, herpes, HIV/AIDS, HPV, and others. For each STI, it outlines how transmission occurs, typical symptoms,
Global Medical Cures™ | Blood disorders
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | Medicare Payments- How Much Do Chronic Conditions Mat...Global Medical Cures™
Erdem, E., Prada, S. I., Haffer, S. C.
E6
MMRR
2013: Volume 3 (2)
1) The document analyzes differences in Medicare payments by beneficiary characteristics such as gender, age, and chronic conditions using 2008 and 2010 Chronic Conditions Public Use Files.
2) It finds that beneficiaries with multiple chronic conditions account for a disproportionate share of Medicare payments, with payments increasing significantly with the number of chronic conditions. "Stroke/Transient Ischemic Attack" and "Chronic Kidney Disease" were the most costly conditions for Part A, while "Cancer" and "Chronic Kidney Disease" were most
Global Medical Cures™ | Gastroesphageal Reflux in Infants
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global Medical Cures™ | AGING- Concerned about CONSTIPATION ?
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
Global pharmaceutical & biotechnology outlook 2013 rising stars - Reports CornerReports Corner
Innovative and improved drugs therapies developed through novel target discovery and technology platforms have originated from the backyards of small research focused companies (Rising Stars, RS, non-profitable biotech companies). The universe continues to grow despite the high risk and the wait to transform into a successful profitable Mature Biotech is long.
https://www.reportscorner.com/reports/10016/Global-Pharmaceutical-&-Biotechnology-Outlook-2013:-Rising-Stars/
The document summarizes an update to the American Geriatrics Society Beers Criteria, which lists potentially inappropriate medications for older adults. An expert panel conducted a systematic review and updated the criteria using evidence on drug-related problems and adverse drug events in older adults. The updated 2012 criteria include 53 medications or classes and are divided into three categories based on risks. The criteria are intended to improve care of older adults by reducing exposure to inappropriate medications and improving prescribing decisions.
Global Medical Cures™ | Paving way for Personalized Medicine (2013) Global Medical Cures™
Global Medical Cures™ | Paving way for Personalized Medicine (2013)
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
The Challenges Associated with Evaluating the Cost Benefit of Gene Therapies ...Covance
Despite the growing availability of approved gene therapies, decision-makers face significant challenges when evaluating pricing and reimbursement of these novel therapeutics. From determining cost-benefit ratios, setting out patient access criteria and designing reimbursement plans, this white paper explores some of the complex aspects of value assessment for gene therapies, and discusses results from a survey of key decision-makers across Germany, Sweden and the UK responsible for making pricing and reimbursement decisions.
Neglected and rare diseases traditionally have not been the focus of large pharmaceutical company research as biotech and academia have primarily been involved in drug discovery efforts for such diseases. This area certainly represents a new opportunity as the pharmaceutical industry investigates new markets. One approach to speed up drug discovery is to examine new uses for existing approved drugs; this is termed drug repositioning or drug repurposing and has become increasingly popular in recent years. Analysis of the literature reveals that using high-throughput screening there have been many examples of FDA approved drugs found to be active against additional targets that can be used to therapeutic advantage for repositioning for other diseases. To date there are far fewer such examples where in silico approaches have allowed for the derivation of new uses. It is suggested that with current technologies and databases of chemical compounds (drugs) and related data, as well as close integration with in vitro screening data, improved opportunities for drug repurposing will emerge. In this publication a review of the literature will highlight several proof of principle examples from areas such as finding new inhibitors for drug transporters with 3D pharmacophores and uncovering molecules active against Mycobacterium tuberculosis (Mtb) using Bayesian models of compound libraries. Research into neglected or rare/orphan diseases can likely benefit from in silico drug repositioning approaches and accelerate drug discovery for these diseases.
The WHO Model Lists of Essential Medicines are updated every two years by the Expert Committee on Selection and Use of Essential Medicines.
The first Essential Medicines List was published in 1977, and the first Essential Medicines List for Children was published in 2007.
The current versions, updated in September 2021, are the 22nd Essential Medicines List (EML) and the 8th Essential Medicines List for Children (EMLc).
Establishing other new medical usages for already known drugs, including approved drugs.
Drug repurposing lies in repurposing an active pharmaceutical ingredient for a new indication that is already on the market.
Drug repurposing is a promising approach and mainly applied for the treatment of both common and rare genetic diseases, and it also offers significant benefits to the pharmaceutical industries.
"At its simplest, drug repurposing is taking an existing drug and seeing whether it can be used as an effective treatment for another condition.“
“Repurposing generally refers to studying drugs that are already approved to treat one disease or condition to see if they are safe and effective for treating other diseases”.
1) The company has a diversified pipeline including hematology and immunotherapy programs with multiple clinical trials in mid to late stages.
2) Their lead hematology candidate, GALE-401, is a controlled release formulation of anagrelide for the treatment of essential thrombocythemia that has demonstrated an improved safety profile over immediate release formulations in clinical trials to date.
3) Their immunotherapy programs include NeuVax for HER2-positive breast cancer, which has shown strong CD8+ T-cell responses in clinical trials both as a single agent and in combination with trastuzumab.
The document discusses FDA approval of cancer drugs between 2002-2012. It finds that two-thirds of regular approvals were based on endpoints other than overall survival, while more than three-quarters of accelerated approvals were based on response rates. The accelerated approval program was heavily used, accounting for one-third of all oncology approvals. The FDA accepted various endpoints for approval and utilized flexibility in approval pathways, approving many drugs based on intermediate endpoints or single-arm trials.
This document provides an overview of clinical research and the clinical trial process. It discusses the various phases of clinical trials from phase 1 to phase 4. Phase 1 trials assess safety in a small group of participants, while phase 2 trials provide preliminary efficacy and safety data in patients. Phase 3 trials further evaluate efficacy and monitor safety in a larger group of patients. Phase 4 trials collect additional safety and efficacy data after marketing approval. The document outlines the objectives and requirements of each phase of clinical trials and the overall goal of generating evidence about new treatments to improve human health.
Personalized Medicines - Enhancers of Life's Quality and Their Future SindhBiotech
This lecture is presented by our volunteer Bushra Umer, she is from Karachi, Pakistan, and she is covering "Personalized Medicines - Enhancers of Life's Quality and Their Future ".
For video: https://www.youtube.com/watch?v=BSrgJaBYuxg
While the world is going through pandemic turmoil and regulatory agencies are under immense stress to approve newer pharmaceutical therapies to market. However, the classical/regular clinical trial is a hefty process hence alternative provisions like speed trials were explored for the early entry of drugs for emergency usage.
SVMPharma Real World Evidence – Real World Evidence & Adaptive PathwaysSVMPharma Limited
SVMPharma Real World Evidence (RWE) – In this paper, we are talking about Adaptive Pathways (AP), an ambitious and evolving new initiative by the European Medicines Agency (EMA) which incorporates Real World Evidence into regulatory approvals and market authorisation.
For more resources on RWE visit us at www.svmpharma.com
The document summarizes a report on medicine use and shifting healthcare costs in the United States in 2013. Key findings include:
- Healthcare and medicine utilization increased in 2013 as patient office visits rebounded after years of decline, while emergency room visits slowed. Prescriptions per capita also rose slightly.
- Out-of-pocket costs for patients continue to rise due to higher deductibles and co-payments, though most prescriptions cost $10 or less. Variations exist between insurance types.
- 36 new drugs were launched in 2013, including treatments for cancer, hepatitis C, and other diseases, transforming care for many conditions.
its not my personal work presentation but taken from lecture ppt from university of San Diego, california.
Its about the drug discovery process, its development and its commercialization.
This document discusses managed access programs (MAPs) for rare disease drugs. It provides examples of MAPs from other countries and provinces that use criteria-based access with ongoing monitoring to provide patients with drugs while collecting evidence on effectiveness and budget impact. It recommends that Canada adopt a national MAP approach, with criteria for starting and stopping drugs developed with input from experts, patients, and companies willing to risk-share costs. The goal is to give patients timely access to existing therapies while determining the long-term benefits, costs, and appropriate use.
A study on prescription pattern and rational use of statins in tertiary care ...SriramNagarajan16
Objectives
Our objectives are to evaluate prescription pattern and rational use of statins in a tertiary care corporate hospital.
Methodology
It was a prospective observational study conducted for a period of 6 months and included various departments of 300
bedded multi specialty tertiary care corporate hospital. A total of 200 patients were included and the study criteria
was inpatients and induvial more than 18 years of either gender who are prescribed with HMG-CoA reductase
inhibitors.
Results
In the present study 200 patients belonged to the age group of above 18 years, out of which about 65% were male
and 35% were female. Atorvastatin (67%) was prescribed mostly and Rosuvastatin (29.5%) was also used.
Conclusion
It is finally concluded that Rational and prophylactic use of statins can reduce further complications of Diabetes
Mellitus (DM) and cardiac events.
Statins treatment is favourable in long term treatment of diseases, it is most effectively used in treatment of serious
disease conditions which has shown its immense therapeutic role in treatment
Lipid Screening in Childhood for Detection of Multifactorial DyslipidemiaGlobal Medical Cures™
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Skin Cancer Screening
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Global Medical Cures™ | Dietary Guidelines for Americans
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Global Medical Cures™ | Aging and your Eyes
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Global Medical Cures™ | Help your Child gain control over Asthma
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Global Medical Cures™ | Drugs approved for Bone Cancer
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Global Medical Cures™ | Drugs approved for Breast Cancer
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Global Medical Cures™| Drugs approved for Head and Neck Cancer
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Global Medical Cures™ | Drugs approved for Non Hodgkin Lymphoma
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Osteoporosis - Definition , Evaluation and Management .pdfJim Jacob Roy
Osteoporosis is an increasing cause of morbidity among the elderly.
In this document , a brief outline of osteoporosis is given , including the risk factors of osteoporosis fractures , the indications for testing bone mineral density and the management of osteoporosis
share - Lions, tigers, AI and health misinformation, oh my!.pptxTina Purnat
• Pitfalls and pivots needed to use AI effectively in public health
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Travel vaccination in Manchester offers comprehensive immunization services for individuals planning international trips. Expert healthcare providers administer vaccines tailored to your destination, ensuring you stay protected against various diseases. Conveniently located clinics and flexible appointment options make it easy to get the necessary shots before your journey. Stay healthy and travel with confidence by getting vaccinated in Manchester. Visit us: www.nxhealthcare.co.uk
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These lecture slides, by Dr Sidra Arshad, offer a simplified look into the mechanisms involved in the regulation of respiration:
Learning objectives:
1. Describe the organisation of respiratory center
2. Describe the nervous control of inspiration and respiratory rhythm
3. Describe the functions of the dorsal and respiratory groups of neurons
4. Describe the influences of the Pneumotaxic and Apneustic centers
5. Explain the role of Hering-Breur inflation reflex in regulation of inspiration
6. Explain the role of central chemoreceptors in regulation of respiration
7. Explain the role of peripheral chemoreceptors in regulation of respiration
8. Explain the regulation of respiration during exercise
9. Integrate the respiratory regulatory mechanisms
10. Describe the Cheyne-Stokes breathing
Study Resources:
1. Chapter 42, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 36, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 13, Human Physiology by Lauralee Sherwood, 9th edition
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5-hydroxytryptamine or 5-HT or Serotonin is a neurotransmitter that serves a range of roles in the human body. It is sometimes referred to as the happy chemical since it promotes overall well-being and happiness.
It is mostly found in the brain, intestines, and blood platelets.
5-HT is utilised to transport messages between nerve cells, is known to be involved in smooth muscle contraction, and adds to overall well-being and pleasure, among other benefits. 5-HT regulates the body's sleep-wake cycles and internal clock by acting as a precursor to melatonin.
It is hypothesised to regulate hunger, emotions, motor, cognitive, and autonomic processes.
3. 2
Ten Year Historic Comparison
CDER’s 2013 Novel New Drugs
27 novel new drugs in CY 2013:
In Calendar Year 2013, FDA’s Center for Drug Evaluation and Research (CDER) approved 27 novel new
medicines, known as new molecular entities (NMEs). For the purposes of this report, the term NME applies to
novel new drugs approved under both New Drug Applications (NDAs) and Biologics License Applications (BLAs).
The chart below lists CDER’s 2013 NMEs.
In
2013
CDER approved
27
NMEs are often innovative new products that serve previously unmet
medical needs or otherwise significantly help to advance patient care
and public health. However, in some cases, while categorized as novel
for technical and/or administrative purposes, a particular NME may not
necessarily offer unique clinical advantages over existing therapies. This
report summarizes all NMEs of 2013, with an emphasis on those that
offer new and innovative treatments to patients in need.
The dark blue bars in the chart to the right indicate the number of
NMEs approved by CDER in each year of the past decade. CDER
approved 27 NMEs in 2013, which is similar to average totals of other
years from this time period. For instance from 2004 through 2012,
CDER has averaged about 26 NME approvals per year. In 2012, CDER
approved 39 NMEs, but this was an unusually high number compared to
any other total in more than a decade.
NMEs
Applications for new approvals remain steady
The number of applications CDER has been receiving for NMEs has not been consistently and significantly
increasing. The light blue portion of the graph to the right indicates the number of new NDA and BLA
applications for NMEs CDER has filed over the last ten years. From 2004 through 2012, CDER filed an average
of about 34 applications for NMEs per year. Although all applications submitted in 2013 had not yet been
accepted for filing as of 12/31/13, CDER projects about 36 for 2013, which is consistent with previous years in
this decade.
With a relatively steady number of applications coming in over time, it is noteworthy that FDA cannot expect a
continuing upward trend for NME approvals until a sustained increase in the number of applications for NMEs
submitted for approval is also demonstrated.
CDER’s NMEs of CY 2013: see pages 14 & 15 for what these drugs are used for.
Adempas
Anoro Ellipta
Aptiom
Breo Ellipta
Brintellix
Dotarem
Duavee
Gazyva
Gilotrif
Imbruvica
Invokana
Kadcyla
Kynamro
Luzu
Lymphoseek
Mekinist
Nesina
Olysio
Opsumit
Osphena
Pomalyst
Sovaldi
Tafinlar
Tecfidera
Tivicay
Vizamyl
Xofigo
For more details about the individual NMEs, see pages 14 & 15.
4. Ten Year Historic Comparison
From 2004 through 2012,
CDER has averaged
27 NMEs approved
in CY 2013
is similar to the
average totals approved
in the past decade
26
NME approvals per year
45
41
35
38
36
35
32
41
39
36
34
30
36 *
30
26
25
20
20
24
22
26
27
23
21
18
15
2013
2012
2011
2010
2009
2008
2007
0
2006
5
2005
10
2004
# NME Approvals and Applications Filed
40
Calendar Year
NME Approvals
NME Applications Filed
* - The filed numbers include those filed in CY 2013 plus those currently pending filing (i.e., within their 60 day filing period) in CY 2013.
- Receipts that received a “Refuse to File” (RTF) or “Withdrawn before filing” (WF) identifier are excluded.
- Multiple submissions (multiple or split originals) pertaining to a single new molecular/biologic entity are only counted once.
- There is a BLA included that does not currently have a review schedule but is known to contain a new active ingredient.
- The filed number is not indicative of workload in the PDUFA V Program.
3
5. 4
Impact on Public Health
Impact
Impact on Public Health
Many of the 27 NMEs approved by CDER in CY 2013 are notable for their potential positive impact and unique
contributions to quality care and public health.
First-in-Class
Adempas
Imbruvica
Invokana
Kadcyla
Kynamro
Mekinist
Sovaldi
Tecfidera
Xofigo
One-third (33%) of the NMEs approved in CY 2013 (9 of 27) were
identified by FDA as First-in-Class, meaning drugs which, for
example, use a new and unique mechanism of action for treating a
medical condition. First-in-Class is one indicator of the innovative
nature of a drug and a 33% First-in-Class approval rate suggests that
the group of CY 2013 NMEs is a field of innovative new products.
Noteworthy First-in-Class products include:
Invokana - for type 2 diabetes glycemic control
Kadcyla - for HER2-positive late-stage (metastatic) breast
Sovaldi - an interferon-free oral treatment option for some
cancer
patients
with chronic hepatitis C
for metastatic melanoma
Mekinist -
33
%
First-in-Class
Drugs
For more details about the individual NMEs, see pages 14 & 15.
6. Impact on Public Health
One-third (33%) of the NMEs approved in CY 2013
(9 of 27) were approved to treat rare or “orphan”
diseases that affect 200,000 or fewer Americans. This
is significant because patients with rare diseases often
have few or no drug treatment options.
Orphan Drugs
Adempas
Gazyva
Noteworthy examples of rare diseases
that now have new or additional
effective treatment options include:
Imbruvica
Mantle cell lymphoma - Imbruvica
Chronic lymphocytic leukemia - Gazyva
Homozygous familial hypercholesterolemia - Kynamro
Pulmonary arterial hypertension - Adempas and Opsumit
Opsumit
33
%
Orphan Drugs
Gilotrif
Kynamro
Mekinist
Pomalyst
Tafinlar
5
7. 6
Impact on Public Health
Notable NMEs of 2013: Another strong year for quality
In addition to the nine noteworthy examples of innovative Firstin-Class and “orphan” new products mentioned on pages 4
and 5, the 27 NMEs approved in CY 2013 also include
Gilotrif to treat late stage (metastatic) non-small
cell lung cancer, Tafinlar to treat patients with
certain forms of melanoma, Tecfidera to
Sovaldi
treat adults with relapsing forms of
multiple sclerosis, and Olysio for
patients with chronic hepatitis C.
Treatment for
patients with
chronic hepatitis C.
Olysio
Treatment for
patients with
chronic
hepatitis C.
Imbruvica
Gilotrif
Treatment for
mantle cell
lymphoma.
Treatment for
late stage (metastatic)
non-small cell
lung cancer.
For more details about the individual NMEs, see pages 14 & 15.
Invokana
Treatment for
type 2 diabetes
glycemic
control.
8. Impact on Public Health
Mekinist
Treament for
melanoma.
Adempas
Treatment for pulmonary
arterial hypertension.
Tafinlar
Treament for
melanoma.
Gazyva
Treatment for chronic
lymphocytic leukemia.
Opsumit
Treatment for
pulmonary arterial
hypertension.
Kadcyla
Treatment for HER2-positive
late-stage (metastatic)
breast cancer.
Kynamro
Tecfidera
Treatment for patients
with homozygous familial
hypercholesterolemia.
Treatment for adults
with relapsing forms
of multiple sclerosis.
7
9. 8
Innovative Methods for Expediting NMEs to Market
Innovation
Innovative methods for expediting NMEs to market
Many of the 27 NMEs of CY 2013 approved by CDER are notable for the regulatory methods CDER used to
expedite the development and approval process. From time of submission to their approval dates, some drugs
were under review for only several months prior to approval. Particularly noteworthy examples of drugs approved
rapidly are Gazyva approved in 6.3 months, Imbruvica approved in 4.5 months, and Xofigo approved in 5.0
months, all approved earlier than their goal dates for regulatory action. There are four FDA expedited review and
approval pathways: Fast Track, Breakthrough, Priority Review, and Accelerated Approval.
Fast Track
Gilotrif
Pomalyst
Imbruvica
Sovaldi
Kadcyla
Tafinlar
Mekinist
Tivicay
Olysio
Xofigo
Ten of the 27 NMEs approved in 2013 (37%) were designated by CDER as
Fast Track, meaning drugs with the potential to address unmet medical
needs. Fast Track speeds new drug development and review, for instance,
by increasing the level of communication FDA allocates to developers and
by enabling developers to use a “rolling review” process such that CDER
can review portions of an application ahead of the submission of the full
application.
Three of the 27 NMEs approved in 2013 (11%) were designated by CDER as
Breakthrough therapies, meaning drugs with preliminary clinical evidence that
demonstrates the drug may have substantial improvement on at least one clinically
significant endpoint over available therapy. A breakthrough therapy designation
conveys all of the fast track program features, as well as more intensive FDA
guidance on an efficient drug development program. Breakthrough status is
designed to help shorten the development time of a promising new therapy. This
is a new designation that went into effect after July 9, 2012. 2013 is the first
year any new drug was approved with the breakthrough designation.
Breakthrough
Gazyva
Imbruvica
Sovaldi
Priority Review
Adempas
Kadcyla
Dotarem
Olysio
Gazyva
Sovaldi
Gilotrif
Tivicay
Imbruvica
Ten of the 27 NMEs approved in 2013 (37%) were designated Priority Review,
in which CDER determines the drug to potentially provide a significant
advance in medical care and sets a target to review the drug within six
months instead of the standard 10 months.
Xofigo
Two of the 27 NMEs approved in 2013 (7%) were approved under FDA’s
Accelerated Approval program, which allows early approval of a drug for serious
or life-threatening illness that offers a benefit over current treatments. This
approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other
clinical measure that FDA considers reasonably likely to predict clinical benefit.
After this approval, the drug must undergo additional testing to confirm that
benefit; this speeds the availability of the drug.
For more details about the individual NMEs, see pages 14 & 15.
Accelerated
Approval
Imbruvica
Pomalyst
10. Innovative Methods for Expediting NMEs to Market
Combined expedited approval methods
Drugs are not limited to one expedited development and
approval method. In many cases, CDER uses one or more
of these tools to speed development and approval. Almost
half (48%) of the 27 NMEs approved in CY 2013 (13 of
27) were designated in one or more categories of Fast
Track, Breakthrough, Priority Review, and/or Accelerated
Approval. Each of these designations helps expedite the
speed of the development and/or approval process and
is designed to help bring important medications to the
market as quickly as possible.
48
%
Combined
Adempas
Imbruvica
Olysio
Tafinlar
Dotarem
Kadcyla
Pomalyst
Tivicay
Gazyva
Mekinist
Sovaldi
Xofigo
Gilotrif
Fast Track
37
%
Breakthrough
11
%
Priority Review
37
%
Accelerated
Approval
7
%
9
11. 10
Approval Predictability
Predictability
Under the Prescription Drug User Fee Act (PDUFA), sponsors
are assessed user fees that provide FDA with the additional
resources needed to meet performance goals.
Throughout the year, CDER was able to meet or exceed
PDUFA goal dates for application review, agreed to with the
pharmaceutical industry and approved by Congress. In CY 2013,
CDER met its PDUFA goal dates for 100% of the NMEs approved
in CY 2013.
PDUFA Goal Dates Met
Adempas
Anoro Ellipta
Aptiom
Breo Ellipta
Brintellix
Dotarem
Duavee
Gazyva
Gilotrif
Imbruvica
Invokana
Kadcyla
Kynamro
Lymphoseek
Mekinist
Nesina
Olysio
Opsumit
Osphena
Pomalyst
Sovaldi
Tafinlar
Tecfidera
Tivicay
Vizamyl
Xofigo
Luzu
%
100
Percentage of PDUFA goal
dates met in CY 2013
for each NME.
For more details about the individual NMEs, see pages 14 & 15.
12. Approval Access
Access
CDER approved most drugs (24 of 27) on the “first cycle” of
review (89%), meaning without requests for additional information
that would delay approval and lead to another cycle of review.
89
% First Cycle
Approval
First Cycle Approval
Adempas
Gazyva
Luzu
Sovaldi
Anoro Ellipta
Gilotrif
Mekinist
Tafinlar
Breo Ellipta
Imbruvica
Olysio
Tecfidera
Brintellix
Invokana
Opsumit
Tivicay
Dotarem
Kadcyla
Osphena
Vizamyl
Duavee
Kynamro
Pomalyst
Xofigo
Ten of the 24 First Cycle Approvals listed above are also designated
as Priority Review drugs. This is particularly important because Priority
Review drugs have the potential to serve as significant medical advances in
health care.
74
% First
Approved
in U.S.
Approval in U.S. before
Other Countries
Anoro Ellipta
Imbruvica
Mekinist
Tafinlar
Breo Ellipta
Invokana
Opsumit
Tecfidera
Brintellix
Kadcyla
Osphena
Tivicay
Gazyva
Kynamro
Pomalyst
Vizamyl
Gilotrif
Lymphoseek
Sovaldi
Xofigo
Comparing approval to other countries offers another
measure of approval efficiency. Although regulatory
processes differ widely between FDA and those of regulatory
agencies in other countries, almost three-quarters (74%) of the
NMEs approved in CY 2013 (20 of 27) were approved first in the
U.S. before any other country.
11
13. 12
Summary
Overview
This document represents a broad overview of CDER
approvals of new molecular entities (NMEs) for
calendar year 2013.
A continuing upward trend for the annual number of CDER’s NME approvals
necessarily relies upon a corresponding upward trend in the number of
applications submitted for approval. Over the past decade, submissions for
NMEs by the pharmaceutical and biotechnology industry have not been
increasing. In other words, over time, CDER can only approve a number of
NMEs proportional to the number of applications for NMEs it receives.
More important than the quantity of new drugs approved in 2013 is the quality
of the new drugs the pharmaceutical industry has developed and the important
new roles these drugs are serving to advance medical care.
Also noteworthy is the efficiency with which most of these drugs were reviewed
and approved. CDER used a variety of “expedited development and approval”
regulatory tools to speed these drugs to market.
In all cases, while striving for efficiency of review and approval of applications
for new drugs, CDER does not compromise its standards for demonstration of
effectiveness and safety in the process.
More important than the quantity of
new drugs approved by CDER in CY 2013
is the quality of the new drugs and the
important new roles they are serving to
advance medical care.
For more details about the individual NMEs, see pages 14 & 15.
14. Adempas
Anoro Ellipta
Aptiom
Breo Ellipta
Fas
tT
rac
k
Bre
akt
hro
ugh
Prio
rity
Rev
iew
Acc
eler
ate
dA
ppr
Met
ova
l
PDU
FA
Goa
l Da
Firs
tes
tC
ycle
Firs
tA
ppr
ove
d in
U. S
.
Orp
han
Firs
t-
in-C
lass
Summary
13
Drug Designation
Summary
First-in-Class
Drugs with a new and unique mechanism for
treating a medical condition.
Brintellix
Orphan Drugs
Dotarem
Drugs approved for small populations of patients
with rare diseases.
Duavee
Gazyva
Gilotrif
Imbruvica
Invokana
Kadcyla
Kynamro
Luzu
Lymphoseek
Mekinist
Nesina
Olysio
Opsumit
Osphena
Pomalyst
Sovaldi
Tafinlar
Tecfidera
Tivicay
Vizamyl
Xofigo
Fast Track
Drugs that can treat unmet medical needs.
Breakthrough
Drugs with preliminary clinical evidence that
demonstrates the drug may have substantial
improvement on at least one clinically significant
endpoint over available therapy.
Priority Review
A drug is given a priority review if there is
potential to provide a significant advance in
medical care.
Accelerated Approval
Early approval based on markers that predict a
reasonable benefit, with more testing to confirm
clinical benefit after approval.
PDUFA Goal Dates
Drugs that met the Prescription Drug User Fee
Act goal dates for the year.
First Cycle
Drugs that were approved without request for
additional information that would delay approval
and lead to another cycle of review.
First Approved in U.S.
Drugs that were approved first in the U.S.
before any other country worldwide.
15. 14
The NMEs of 2013
Drugs listed by date of approval.
Drug Name
Active Ingredient
Date
What it used for
Nesina
alogliptin
1/25/2013
To improve blood sugar control in adults with type 2
diabetes.
Kynamro
mipomersen
sodium
1/29/2013
To treat patients with a rare type of high cholesterol called
homozygous familial hypercholesterolemia (HoFH).
Pomalyst
pomalidomide
2/8/2013
To treat patients with multiple myeloma whose disease
progressed after being treated with other cancer drugs.
Kadcyla
ado-trastuzumab
emtansine
2/22/2013
For patients with HER2-positive, late-stage (metastatic)
breast cancer.
2/26/2013
To treat women experiencing moderate to severe dyspareunia
(pain during sexual intercourse), a symptom of vulvar and
vaginal atrophy due to menopause.
Osphena
ospemifene
Lymphoseek
technetium
Tc 99m
tilmanocept
3/13/2013
A radioactive diagnostic imaging agent that helps doctors
locate lymph nodes in patients with breast cancer or
melanoma who are undergoing surgery to remove tumordraining lymph nodes.
Dotarem
gadoterate
meglumine
3/20/2013
For use in magnetic resonance imaging (MRI) of the brain,
spine and associated tissues of patients ages 2 years and
older.
Tecfidera
dimethyl fumarate
3/27/2013
To treat adults with relapsing forms of multiple sclerosis
(MS).
Invokana
canagliflozin
3/29/2013
Used with diet and exercise to improve glycemic control in
adults with type 2 diabetes.
Breo Ellipta
fluticasone furoate
and vilanterol
inhalation powder
5/10/2013
For the long-term, once-daily, maintenance treatment of
airflow obstruction in patients with chronic obstructive
pulmonary disease (COPD), including chronic bronchitis and/
or emphysema.
Xofigo
radium
Ra 223
dichloride
5/15/2013
To treat men with symptomatic late-stage (metastatic)
castration-resistant prostate cancer that has spread to bones
but not to other organs.
Tafinlar
dabrafenib
5/29/2013
To treat patients with melanoma whose tumors express the
BRAF V600E gene mutation.
Mekinist
trametinib
5/29/2013
To treat patients whose tumors express the BRAF V600E or
V600K gene mutations.
7/12/2013
For patients with late stage (metastatic) non-small cell lung
cancer (NSCLC) whose tumors express specific types of
epidermal growth factor receptor (EGFR) gene mutations, as
detected by an FDA-approved test.
Gilotrif
afatinib
To view this full summary online please visit the CDER web page spotlight on Drug Innovation...
16. 15
Drug Name
Active Ingredient
Date
What it used for
Tivicay
dolutegravir
8/12/2013
To treat HIV-1 infection.
Brintellix
vortioxetine
9/30/2013
To treat adults with major depressive disorder.
Duavee
conjugated
estrogens/
bazedoxifene
10/3/2013
To treat moderate-to-severe hot flashes (vasomotor
symptoms) associated with menopause and to prevent
osteoporosis after menopause.
Adempas
riociguat
10/8/2013
To treat adults with two forms of pulmonary hypertension.
Opsumit
macitentan
10/18/2013
To treat adults with pulmonary arterial hypertension (PAH),
a chronic, progressive and debilitating disease that can lead
to death or the need for lung transplantation.
Vizamyl
flutemetamol
F 18 injection
10/25/2013
A radioactive diagnostic drug for use with positron emission
tomography (PET) imaging of the brain in adults being
evaluated for Alzheimer’s disease (AD) and dementia.
Gazyva
obinutuzumab
11/1/2013
For use in combination with chlorambucil to treat patients
with previously untreated chronic lymphocytic leukemia
(CLL).
Aptiom
eslicarbazepine
acetate
11/8/2013
As an add-on medication to treat seizures associated with
epilepsy.
Imbruvica
ibrutinib
11/13/2013
To treat patients with mantle cell lymphoma (MCL), a rare
and aggressive type of blood cancer.
Luzu
luliconozole
11/14/2013
For the topical treatment of interdigital tinea pedis,
tinea cruris, and tinea corporis caused by the organisms
Trichophyton rubrum and Epidermophyton floccosum in
patients 18 years of age and older.
Olysio
simeprevir
11/22/2013
To treat chronic hepatitis C virus (HCV) infection.
Sovaldi
sofosbuvir
12/6/2013
To treat chronic hepatitis C virus (HCV) infection.
Anoro Ellipta
umeclidinium
and vilanterol
inhalation powder
12/18/2013
For the once-daily, long-term maintenance treatment of
airflow obstruction in patients with chronic obstructive
pulmonary disease (COPD).
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/default.htm