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.
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.
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).
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.
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).
Determinants of New Molecular Entity Approval by United States Food & Drug Ad...inventionjournals
This paper analyzes the relationship between research-based pharmaceutical companies’ R&D productivity, patent, pivotal trial and drug development strategy with the number of NME approval by U.S. FDA. The model was estimated using annual data, gathered from ten large pharmaceutical companies in the world. The regression analysis used pooled regression with Estimated Generalized Least Squares (EGLS) method. The result showed that R&D productivity, patent, pivotal trial, and drug development strategy are statistically significant in increasing the number of NME approval in research-based pharmaceutical companies. The relative order of significance in influencing the number of NME approval was patent, development strategy, R&D productivity, and pivotal trial.
Fixed dose combination products – rationality, status in india, development i...Dr Sukanta sen
The development of FDCs is becoming increasingly
important from a public health perspective.
•They are being used in the treatment of a wide range of
conditions and are particularly useful in the management of
human immunodeficiency virus/acquired immunodeficiency
syndrome (HIV/AIDS), malaria and tuberculosis, which are
considered to be the foremost infectious disease threats in the world today.
Discovery of Drug and Introduction to Clinical Trial_Katalyst HLSKatalyst HLS
Introduction to Discovery of Drug and Introduction to Clinical Trials in Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
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 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).
Determinants of New Molecular Entity Approval by United States Food & Drug Ad...inventionjournals
This paper analyzes the relationship between research-based pharmaceutical companies’ R&D productivity, patent, pivotal trial and drug development strategy with the number of NME approval by U.S. FDA. The model was estimated using annual data, gathered from ten large pharmaceutical companies in the world. The regression analysis used pooled regression with Estimated Generalized Least Squares (EGLS) method. The result showed that R&D productivity, patent, pivotal trial, and drug development strategy are statistically significant in increasing the number of NME approval in research-based pharmaceutical companies. The relative order of significance in influencing the number of NME approval was patent, development strategy, R&D productivity, and pivotal trial.
Fixed dose combination products – rationality, status in india, development i...Dr Sukanta sen
The development of FDCs is becoming increasingly
important from a public health perspective.
•They are being used in the treatment of a wide range of
conditions and are particularly useful in the management of
human immunodeficiency virus/acquired immunodeficiency
syndrome (HIV/AIDS), malaria and tuberculosis, which are
considered to be the foremost infectious disease threats in the world today.
Discovery of Drug and Introduction to Clinical Trial_Katalyst HLSKatalyst HLS
Introduction to Discovery of Drug and Introduction to Clinical Trials in Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
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 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).
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.
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 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.
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.
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.
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”.
Epidemiology is the study of occurrence, distribution and determinants of health and
diseases or disorders in man and its application in controlling health problems.
Epidemiology has by tradition two major areas.
First is the study of infectious diseases that spread to large populations, i.e., epidemics.
The second is the study of chronic diseases.
Epidemiological studies help to solve such health problems and provide a basis for
improving living conditions of the people.
During its progress and development, epidemiology has made available precise and
strict methodologies for the study of diseases.
Pharmacology is the study of the effects of drugs.
Clinical Pharmacology is the study of the effects of drugs in humans, It is traditionally
divided into two basic areas namely:
1. Pharmacokinetics
2. Pharmacodynamics.
Pharmacokinetics is the study of the relationship between dose administered of a drug
and the serum or blood level achieved, it deals with absorption, distribution, metabolism
and excretion.
Epidemiology is the study of the distribution and determinants of diseases in
populations.
Epidemics is the study of chronic/ infectious diseases in large populations.
Pharmacoepidemiology is the study of the use of and the effects of drugs in large
number of people.
It involves the examination of a single individual or large groups of people followed for
many years.
It involves gathering & analysis of information in order to identify possible causation &
related factors, that can be applied in clinical practice to group of people & also to
individuals undergoing treatment.
Pulmonary Thromboembolism - etilogy, types, medical- Surgical and nursing man...VarunMahajani
Disruption of blood supply to lung alveoli due to blockage of one or more pulmonary blood vessels is called as Pulmonary thromboembolism. In this presentation we will discuss its causes, types and its management in depth.
New Drug Discovery and Development .....NEHA GUPTA
The "New Drug Discovery and Development" process involves the identification, design, testing, and manufacturing of novel pharmaceutical compounds with the aim of introducing new and improved treatments for various medical conditions. This comprehensive endeavor encompasses various stages, including target identification, preclinical studies, clinical trials, regulatory approval, and post-market surveillance. It involves multidisciplinary collaboration among scientists, researchers, clinicians, regulatory experts, and pharmaceutical companies to bring innovative therapies to market and address unmet medical needs.
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...i3 Health
i3 Health is pleased to make the speaker slides from this activity available for use as a non-accredited self-study or teaching resource.
This slide deck presented by Dr. Kami Maddocks, Professor-Clinical in the Division of Hematology and
Associate Division Director for Ambulatory Operations
The Ohio State University Comprehensive Cancer Center, will provide insight into new directions in targeted therapeutic approaches for older adults with mantle cell lymphoma.
STATEMENT OF NEED
Mantle cell lymphoma (MCL) is a rare, aggressive B-cell non-Hodgkin lymphoma (NHL) accounting for 5% to 7% of all lymphomas. Its prognosis ranges from indolent disease that does not require treatment for years to very aggressive disease, which is associated with poor survival (Silkenstedt et al, 2021). Typically, MCL is diagnosed at advanced stage and in older patients who cannot tolerate intensive therapy (NCCN, 2022). Although recent advances have slightly increased remission rates, recurrence and relapse remain very common, leading to a median overall survival between 3 and 6 years (LLS, 2021). Though there are several effective options, progress is still needed towards establishing an accepted frontline approach for MCL (Castellino et al, 2022). Treatment selection and management of MCL are complicated by the heterogeneity of prognosis, advanced age and comorbidities of patients, and lack of an established standard approach for treatment, making it vital that clinicians be familiar with the latest research and advances in this area. In this activity chaired by Michael Wang, MD, Professor in the Department of Lymphoma & Myeloma at MD Anderson Cancer Center, expert faculty will discuss prognostic factors informing treatment, the promising results of recent trials in new therapeutic approaches, and the implications of treatment resistance in therapeutic selection for MCL.
Target Audience
Hematology/oncology fellows, attending faculty, and other health care professionals involved in the treatment of patients with mantle cell lymphoma (MCL).
Learning Objectives
1.) Identify clinical and biological prognostic factors that can guide treatment decision making for older adults with MCL
2.) Evaluate emerging data on targeted therapeutic approaches for treatment-naive and relapsed/refractory MCL and their applicability to older adults
3.) Assess mechanisms of resistance to targeted therapies for MCL and their implications for treatment selection
These lecture slides, by Dr Sidra Arshad, offer a quick overview of physiological basis of a normal electrocardiogram.
Learning objectives:
1. Define an electrocardiogram (ECG) and electrocardiography
2. Describe how dipoles generated by the heart produce the waveforms of the ECG
3. Describe the components of a normal electrocardiogram of a typical bipolar leads (limb II)
4. Differentiate between intervals and segments
5. Enlist some common indications for obtaining an ECG
Study Resources:
1. Chapter 11, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 9, Human Physiology - From Cells to Systems, Lauralee Sherwood, 9th edition
3. Chapter 29, Ganong’s Review of Medical Physiology, 26th edition
4. Electrocardiogram, StatPearls - https://www.ncbi.nlm.nih.gov/books/NBK549803/
5. ECG in Medical Practice by ABM Abdullah, 4th edition
6. ECG Basics, http://www.nataliescasebook.com/tag/e-c-g-basics
Tom Selleck Health: A Comprehensive Look at the Iconic Actor’s Wellness Journeygreendigital
Tom Selleck, an enduring figure in Hollywood. has captivated audiences for decades with his rugged charm, iconic moustache. and memorable roles in television and film. From his breakout role as Thomas Magnum in Magnum P.I. to his current portrayal of Frank Reagan in Blue Bloods. Selleck's career has spanned over 50 years. But beyond his professional achievements. fans have often been curious about Tom Selleck Health. especially as he has aged in the public eye.
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Introduction
Many have been interested in Tom Selleck health. not only because of his enduring presence on screen but also because of the challenges. and lifestyle choices he has faced and made over the years. This article delves into the various aspects of Tom Selleck health. exploring his fitness regimen, diet, mental health. and the challenges he has encountered as he ages. We'll look at how he maintains his well-being. the health issues he has faced, and his approach to ageing .
Early Life and Career
Childhood and Athletic Beginnings
Tom Selleck was born on January 29, 1945, in Detroit, Michigan, and grew up in Sherman Oaks, California. From an early age, he was involved in sports, particularly basketball. which played a significant role in his physical development. His athletic pursuits continued into college. where he attended the University of Southern California (USC) on a basketball scholarship. This early involvement in sports laid a strong foundation for his physical health and disciplined lifestyle.
Transition to Acting
Selleck's transition from an athlete to an actor came with its physical demands. His first significant role in "Magnum P.I." required him to perform various stunts and maintain a fit appearance. This role, which he played from 1980 to 1988. necessitated a rigorous fitness routine to meet the show's demands. setting the stage for his long-term commitment to health and wellness.
Fitness Regimen
Workout Routine
Tom Selleck health and fitness regimen has evolved. adapting to his changing roles and age. During his "Magnum, P.I." days. Selleck's workouts were intense and focused on building and maintaining muscle mass. His routine included weightlifting, cardiovascular exercises. and specific training for the stunts he performed on the show.
Selleck adjusted his fitness routine as he aged to suit his body's needs. Today, his workouts focus on maintaining flexibility, strength, and cardiovascular health. He incorporates low-impact exercises such as swimming, walking, and light weightlifting. This balanced approach helps him stay fit without putting undue strain on his joints and muscles.
Importance of Flexibility and Mobility
In recent years, Selleck has emphasized the importance of flexibility and mobility in his fitness regimen. Understanding the natural decline in muscle mass and joint flexibility with age. he includes stretching and yoga in his routine. These practices help prevent injuries, improve posture, and maintain mobilit
Lung Cancer: Artificial Intelligence, Synergetics, Complex System Analysis, S...Oleg Kshivets
RESULTS: Overall life span (LS) was 2252.1±1742.5 days and cumulative 5-year survival (5YS) reached 73.2%, 10 years – 64.8%, 20 years – 42.5%. 513 LCP lived more than 5 years (LS=3124.6±1525.6 days), 148 LCP – more than 10 years (LS=5054.4±1504.1 days).199 LCP died because of LC (LS=562.7±374.5 days). 5YS of LCP after bi/lobectomies was significantly superior in comparison with LCP after pneumonectomies (78.1% vs.63.7%, P=0.00001 by log-rank test). AT significantly improved 5YS (66.3% vs. 34.8%) (P=0.00000 by log-rank test) only for LCP with N1-2. Cox modeling displayed that 5YS of LCP significantly depended on: phase transition (PT) early-invasive LC in terms of synergetics, PT N0—N12, cell ratio factors (ratio between cancer cells- CC and blood cells subpopulations), G1-3, histology, glucose, AT, blood cell circuit, prothrombin index, heparin tolerance, recalcification time (P=0.000-0.038). Neural networks, genetic algorithm selection and bootstrap simulation revealed relationships between 5YS and PT early-invasive LC (rank=1), PT N0—N12 (rank=2), thrombocytes/CC (3), erythrocytes/CC (4), eosinophils/CC (5), healthy cells/CC (6), lymphocytes/CC (7), segmented neutrophils/CC (8), stick neutrophils/CC (9), monocytes/CC (10); leucocytes/CC (11). Correct prediction of 5YS was 100% by neural networks computing (area under ROC curve=1.0; error=0.0).
CONCLUSIONS: 5YS of LCP after radical procedures significantly depended on: 1) PT early-invasive cancer; 2) PT N0--N12; 3) cell ratio factors; 4) blood cell circuit; 5) biochemical factors; 6) hemostasis system; 7) AT; 8) LC characteristics; 9) LC cell dynamics; 10) surgery type: lobectomy/pneumonectomy; 11) anthropometric data. Optimal diagnosis and treatment strategies for LC are: 1) screening and early detection of LC; 2) availability of experienced thoracic surgeons because of complexity of radical procedures; 3) aggressive en block surgery and adequate lymph node dissection for completeness; 4) precise prediction; 5) adjuvant chemoimmunoradiotherapy for LCP with unfavorable prognosis.
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Ve...kevinkariuki227
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
263778731218 Abortion Clinic /Pills In Harare ,sisternakatoto
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ARTIFICIAL INTELLIGENCE IN HEALTHCARE.pdfAnujkumaranit
Artificial intelligence (AI) refers to the simulation of human intelligence processes by machines, especially computer systems. It encompasses tasks such as learning, reasoning, problem-solving, perception, and language understanding. AI technologies are revolutionizing various fields, from healthcare to finance, by enabling machines to perform tasks that typically require human intelligence.
Prix Galien International 2024 Forum ProgramLevi Shapiro
June 20, 2024, Prix Galien International and Jerusalem Ethics Forum in ROME. Detailed agenda including panels:
- ADVANCES IN CARDIOLOGY: A NEW PARADIGM IS COMING
- WOMEN’S HEALTH: FERTILITY PRESERVATION
- WHAT’S NEW IN THE TREATMENT OF INFECTIOUS,
ONCOLOGICAL AND INFLAMMATORY SKIN DISEASES?
- ARTIFICIAL INTELLIGENCE AND ETHICS
- GENE THERAPY
- BEYOND BORDERS: GLOBAL INITIATIVES FOR DEMOCRATIZING LIFE SCIENCE TECHNOLOGIES AND PROMOTING ACCESS TO HEALTHCARE
- ETHICAL CHALLENGES IN LIFE SCIENCES
- Prix Galien International Awards Ceremony
- Video recording of this lecture in English language: https://youtu.be/lK81BzxMqdo
- Video recording of this lecture in Arabic language: https://youtu.be/Ve4P0COk9OI
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
3. 2
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.
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.
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.
Ten Year Historic Comparison
For more details about the individual NMEs, see pages 14 & 15.
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
In
27NMEs
2013CDER approved
4. 3
0
5
10
15
20
25
30
35
40
45
36
20
22
18
24
26
21
30
32
36
41
38
41
26
35
34
36
23
#NMEApprovalsandApplicationsFiled
Calendar Year
NME Approvals
NME Applications Filed
39
27
2004
2005
2006
2007
2008
2009
2010
2011
2012
2013
*
Ten Year Historic Comparison
From 2004 through 2012,
CDER has averaged
26NME approvals per year
27 NMEs approved
in CY 2013
is similar to the
average totals approved
in the past decade
* - 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.
5. 4 Impact on Public Health
For more details about the individual NMEs, see pages 14 & 15.
33%
First-in-Class
Drugs
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
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.
Adempas
Imbruvica
Invokana
Kadcyla
Kynamro
Mekinist
Sovaldi
Tecfidera
Xofigo
Noteworthy First-in-Class products include:
Invokana - for type 2 diabetes glycemic control
Kadcyla - for HER2-positive late-stage (metastatic) breast cancer
Sovaldi - an interferon-free oral treatment option for some patients
with chronic hepatitis C
Mekinist - for metastatic melanoma
6. 5Impact on Public Health
33%
Orphan Drugs
Orphan Drugs
Adempas
Gazyva
Gilotrif
Imbruvica
Kynamro
Mekinist
Opsumit
Pomalyst
Tafinlar
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.
Noteworthy examples of rare diseases
that now have new or additional
effective treatment options include:
Mantle cell lymphoma - Imbruvica
Chronic lymphocytic leukemia - Gazyva
Homozygous familial hypercholesterolemia - Kynamro
Pulmonary arterial hypertension - Adempas and Opsumit
7. 6
Notable NMEs of 2013: Another strong year for quality
Impact on Public Health
Invokana
Treatment for
type 2 diabetes
glycemic
control.
Gilotrif
Treatment for
late stage (metastatic)
non-small cell
lung cancer.
Imbruvica
Treatment for
mantle cell
lymphoma.
In addition to the nine noteworthy examples of innovative First-
in-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
treat adults with relapsing forms of
multiple sclerosis, and Olysio for
patients with chronic hepatitis C.
Sovaldi
Treatment for
patients with
chronic hepatitis C.
For more details about the individual NMEs, see pages 14 & 15.
Olysio
Treatment for
patients with
chronic
hepatitis C.
8. 7Impact on Public Health
Tafinlar
Treament for
melanoma.
Mekinist
Treament for
melanoma.
Adempas
Treatment for pulmonary
arterial hypertension.
Gazyva
Treatment for chronic
lymphocytic leukemia.
Kynamro
Treatment for patients
with homozygous familial
hypercholesterolemia.
Kadcyla
Treatment for HER2-positive
late-stage (metastatic)
breast cancer.
Tecfidera
Treatment for adults
with relapsing forms
of multiple sclerosis.
Opsumit
Treatment for
pulmonary arterial
hypertension.
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.
Breakthrough
Gazyva
Imbruvica
Sovaldi
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.
Priority Review
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.
Adempas
Dotarem
Gazyva
Gilotrif
Imbruvica
Accelerated
Approval
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.
Imbruvica
Pomalyst
Kadcyla
Olysio
Sovaldi
Tivicay
Xofigo
For more details about the individual NMEs, see pages 14 & 15.
Pomalyst
Sovaldi
Tafinlar
Tivicay
Xofigo
Gilotrif
Imbruvica
Kadcyla
Mekinist
Olysio
Fast Track
10. 9Innovative 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.
Fast Track Priority Review
Accelerated
ApprovalBreakthrough
11%
37%
7%
Adempas
Dotarem
Gazyva
Gilotrif
Imbruvica
Kadcyla
Mekinist
Olysio
Pomalyst
Sovaldi
Combined
48%
37%
Tafinlar
Tivicay
Xofigo
11. 10 Approval Predictability
For more details about the individual NMEs, see pages 14 & 15.
Predictability
PDUFA Goal Dates Met
Adempas
Anoro Ellipta
Aptiom
Breo Ellipta
Brintellix
Dotarem
Duavee
Gazyva
Gilotrif
Imbruvica
Invokana
Kadcyla
Kynamro
Luzu
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.
Lymphoseek
Mekinist
Nesina
Olysio
Opsumit
Osphena
Pomalyst
Sovaldi
Tafinlar
Tecfidera
Tivicay
Vizamyl
Xofigo
Percentage of PDUFA goal
dates met in CY 2013
for each NME.
100%
12. 11Approval Access
Access
First Cycle Approval
Adempas
Anoro Ellipta
Breo Ellipta
Brintellix
Dotarem
Duavee
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.
Approval in U.S. before
Other Countries
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.
Anoro Ellipta
Breo Ellipta
Brintellix
Gazyva
Gilotrif
89%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.
Gazyva
Gilotrif
Imbruvica
Invokana
Kadcyla
Kynamro
Luzu
Mekinist
Olysio
Opsumit
Osphena
Pomalyst
Sovaldi
Tafinlar
Tecfidera
Tivicay
Vizamyl
Xofigo
74%
First Cycle
Approval
First
Approved
in U.S.
Imbruvica
Invokana
Kadcyla
Kynamro
Lymphoseek
Mekinist
Opsumit
Osphena
Pomalyst
Sovaldi
Tafinlar
Tecfidera
Tivicay
Vizamyl
Xofigo
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.
For more details about the individual NMEs, see pages 14 & 15.
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.
14. 13Summary
Drug Designation
Summary
First-in-Class
Drugs with a new and unique mechanism for
treating a medical condition.
Orphan Drugs
Drugs approved for small populations of patients
with rare diseases.
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.
First-in-Class
Orphan
FastTrack
Breakthrough
PriorityReview
AcceleratedApproval
MetPDUFAGoalDates
FirstCycle
FirstApprovedinU.S.
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
15. 14
To view this full summary online please visit the CDER web page spotlight on Drug Innovation...
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.
Osphena ospemifene 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.
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 tumor-
draining 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.
Gilotrif afatinib 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.
The NMEs of 2013Drugs listed by date of approval.
16. 15
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/default.htm
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).