New Drugs of 2012: A Concise Overview of the NMEs and Trends for Innovative Brand Market in the United States

2013 ◽  
Vol 6 (2) ◽  
pp. 2009-2013
Author(s):  
D Samba Reddy
Keyword(s):  
Drug Approval ◽  
The United States ◽  
New Drugs ◽  
New Combination ◽  
Drug Status ◽  
Combination Drug ◽  
Human Cord Blood ◽  
Life Saving ◽  
Genomics And Proteomics ◽  
The Face

Thirty-nine (39) new drugs have been approved by the U.S. FDA in 2012, a record highest number of approvals since 1996. The record is a sign that pharma companies are poised to tap recent advances from genomics and proteomics. This list includes novel new drugs, known as new molecular entities (NMEs), biologics and new products. Many life-saving drugs are approved for marketing. The list includes a total of 10 drugs for cancer treatment, and nearly a quarter of those approved in 2012 had orphan drug status.  Among the breakthrough drugs approved in 2012 were ivacaftor (cystic fibrosis), vasmodegib (skin cancer), HPC-C (human cord blood product), ruxolitinib (myelofibrosis) and a new combination drug to treat HIV. In addition,  several unique products were approved for the treatment of macular degeneration, chronic weight management, overactive bladder, actinic keratosis, erectile dysfunction, glaucoma, respiratory distress syndrome, and COPD. The approval of 39 drugs in 2012 underscores a robust success rate and confirms that innovation is once again beginning to pay off. In the existing climate of reduced revenues in the face of generic competitions, the future and survival of big companies rests heavily on their unique niche products. It is apparent that big Pharma and a growing number of emerging Biotechs alike have focused their attention on developing new NMEs for rare diseases. In 2012, the length of the FDA’s review is shorter than agencies in other countries. Innovative models adopted for R&D strategies, communications, and new regulatory changes appear to shorten development timelines. Despite record drug approvals, there is bleak scope for blockbusters because most of these drugs have a limited market. The pipeline for blockbusters appears very low. However, there is unmet medical need for new drugs in autism, Alzheimer’s disease and epilepsy. Overall, the new drug approval list unveils unique and reemerging trends indicating that the pharma companies are poised for big growth from new brands approved for marketing for narrow-spectrum indications.    

Right to Experimental Treatment: FDA New Drug Approval, Constitutional Rights, and the Public's Health

2009 ◽  
Vol 37 (2) ◽  
pp. 269-279 ◽  
Author(s):  
Elizabeth Weeks Leonard
Keyword(s):  
Treatment Options ◽  
Terminally Ill ◽  
Experimental Treatment ◽  
Drug Approval ◽  
New Drugs ◽  
Constitutional Rights ◽  
Court Of Appeals ◽  
Fda Approval ◽  
Life Saving ◽  
Constitutional Right

Do terminally ill patients who have exhausted all other available, government-approved treatment options have a constitutional right to experimental treatment that may prolong their lives? On May 2, 2006, a divided panel of the U.S. Court of Appeals for the District of Columbia, in a startling opinion, Abigail Alliance for Better Access to Developmental Drugs v. Von Eschenbach, held “Yes.” The plaintiffs, Abigail Alliance for Better Access to Developmental Drugs (Abigail Alliance) and Washington Legal Foundation, sought to enjoin the Food and Drug Administration (FDA) from refusing to allow the sale of investigational new drugs that had not yet received FDA approval. The terminally ill plaintiffs contended that they quite literally could not wait that long for the drugs. With no other treatment options available, the plaintiffs urged the court to recognize a fundamental, constitutional right to take potentially life-saving or life-prolonging drugs, even though the treatment had not been fully tested through human trials for safety and effectiveness and could not be legally marketed to the public.

The Timeliness of New Drug Approvals in Canada

1995 ◽  
Vol 25 (1) ◽  
pp. 153-165 ◽  
Author(s):  
Nigel S. B. Rawson
Keyword(s):  
Recent Article ◽  
Drug Approval ◽  
The United States ◽  
Regulatory Approval ◽  
New Drugs ◽  
Marketing Approval ◽  
The Public ◽  
The United Kingdom ◽  
Anti Cancer ◽  
High Level

In a recent article, Lexchin asks “who needs faster drug approval times in Canada?” and, on the basis of extremely limited and selective data, draws the conclusion that neither the public nor the pharmaceutical industry does. Whether the Canadian system is really slower is investigated by comparing Canadian and U.S. marketing approval dates and by using information on regulatory approval times from the two countries and elsewhere. Marketing approval dates in Canada are significantly later than those in the United States, although not consistently across all therapeutic categories; anti-cancer and gastrointestinal drugs have earlier approval dates in Canada. However, Canadian and U.S. regulatory approval times are not significantly different, indicating that marketing applications are submitted later in Canada, but both are considerably longer than those in the United Kingdom. The evidence shows that Canadians need faster drug approval times if individuals requiring the medications are not to suffer unnecessarily. A significant decrease in drug approval times and the establishment of comprehensive and effective postmarketing surveillance would reduce the time it takes for new drugs to be made available to Canadians while, at the same time, providing a high level of drug safety.

Drug Approval Process in the United States

2018 ◽  
Author(s):  
Stuart O. Schweitzer ◽  
Z. John Lu
Keyword(s):  
United States ◽  
Basic Research ◽  
Drug Approval ◽  
The United States ◽  
Approval Process ◽  
Animal Testing ◽  
Life Saving ◽  
Depth Analysis ◽  
Drug Approval Process ◽  
Major Drug

The drug approval process in any country involves a balancing of conflicting social objectives: safety and access. Faster approval leads to quicker access to potentially life-saving medicine, yet could also lead to false positives or, worse, unsafe products on the market. The United States has a widely respected but stringent and rigorous review process overseen by the Food and Drug Administration. This chapter performs an in-depth analysis of the pharmaceutical regulatory approval process in the United States. Standards, guidelines, and critical milestones for basic research, animal testing, and clinical trials in the drug R&D process are explained. It highlights major drug legislation since the beginning of the twentieth century and how this legislation has helped the FDA become the gold standard in pharmaceutical regulation worldwide. The registration pathways for generics and biosimilars are also discussed.

Science: Part of the Problem, Most of the Answer

1983 ◽  
Vol 17 (7-8) ◽  
pp. 566-569
Author(s):  
Jere E. Goyan
Keyword(s):  
Small Businesses ◽  
Drug Approval ◽  
The United States ◽  
New Drugs ◽  
Great Debate ◽  
History Of ◽  
Drug Approval Process ◽  
Almost All ◽  
Small Manufacturers ◽  
Scientific Questions

The history of the drug approval process in the United States includes three phases. First, the Food and Drug Act of 1906 essentially required that the labeling of drugs be truthful. The 1938 Food, Drug, and Cosmetic Act added a requirement that drugs be proven safe, and the 1962 act added the requirement that drugs be efficacious. Each of these steps required more and more sophisticated science. Subsequent to the Kefauver-Harris Amendments of 1962, the number of innovative molecular entities each year has declined, the reasons for which have been subject to great debate. This decline has paralleled decreases in innovation across almost all American industry, leading to questions about the future of our country as an industrialized society. Both the Carter and Reagan administrations attempted to address this problem in a number of ways, including cutting back on those regulations that are perceived as unnecessary. Other innovative approaches have been used, such as the establishment of an Office of Small Manufacturers Assistance in the FDA Bureau of Medical Devices, mandated by the 1976 Medical Device Amendments. The latter came about in recognition of the fact that small businesses tend to be more creative and efficient than larger industries and more adversely affected by regulation. However, the problems raised in the regulation of technology transfer almost inevitably arise because of perceived scientific questions. Such questions, in turn, can only be answered by good science performed by the sponsor and understood by the regulator. Thus, it is essential that the FDA be staffed with knowledgeable scientists who can interact easily with their peers in academia and industry. Although science is often the cause of our troubles, it is also our only hope for minimizing the costs of new drugs and other technology. In turn, minimizing such costs will maximize the opportunities for innovation.

scholarly journals Patient-Reported Outcome Labeling for Malignant Hematology Drugs Approved in the United States: 2011-2017

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2292-2292
Author(s):  
Marjorie E Zettler ◽  
Ethan Basch ◽  
Chadi Nabhan
Keyword(s):  
Drug Approval ◽  
The United States ◽  
Patient Reported Outcome ◽  
Health Condition ◽  
New Drugs ◽  
Approval Process ◽  
New Drug ◽  
Study Results ◽  
Patient Reported ◽  
Drug Approval Process

Abstract Introduction: Patient-reported outcomes (PROs), defined as any report on the status of a patient's health condition that comes directly from the patient without interpretation by anyone else, play an increasingly important role in drug development. In 2009 the FDA issued final guidance on using PRO measures to support labeling claims, to incorporate the patient perspective into the drug approval process. The 21st Century Cures Act emphasizes PROs as a differentiating element in the FDA approval process of new drugs, beyond traditional clinical outcome measures. Further, recent data has shown that intervention based on PROs can improve survival in metastatic cancers (Basch et al; 2017). The incorporation of PROs into the labeling of new drugs for malignant hematology disorders has not been studied and is the subject of this investigation. Methods: We reviewed the FDA's Novel New Drug Summaries (2011-2017) to identify drugs approved for malignant hematology indications. Drug approval packages and product labeling were accessed via the Drugs@FDA database and analyzed for PRO endpoints, measures, and labeling claims. Clinical trial designs and published study results were retrieved via the ClinicalTrials.gov website and PubMed. Results: Of 250 novel drugs approved by the FDA between 2011 and 2017, 22 (8.8%) were approved for malignant hematology indications. Interestingly, only 1 had PRO-based claims in their labeling, even though 13 of the 22 drugs (59%) collected PRO data in pivotal trials that led to their approval. Notably, the proportion of malignant hematology trials assessing PROs has increased over time, with 4 of the 5 drugs approved in 2017 for malignant hematology indications evaluating PROs in their development programs, compared with 9 of the 17 drugs approved in the preceding 6 years (80% vs. 53%). PROs evaluated included generic instruments such as the EQ-5D, and disease-specific instruments such as the EORTC QLQ-C30 (see table). Reasons cited for rejection of PRO data inclusion in drug labeling were single arm trial design, excessive missing data, statistical issues, and use of an inappropriate PRO instrument. Conclusions: While the FDA encourages PRO data submission as part of the new drug approval process, and although more than half of all malignant hematology drugs approved in the past 7 years assessed PROs during development, only 1 was able to successfully acquire labeling claims. Whether this is due to lack of PRO expertise on clinical development teams or absence of strong regulatory guidance on how best to implement PROs remains unknown and requires further research. Designing strategies to develop, validate and report PROs effectively is needed to meet regulatory requirements and enhance patients' voices in their own care. Table. Table. Disclosures Nabhan: Cardinal Health: Employment, Equity Ownership.

scholarly journals Next-generation multiple myeloma treatment: a pharmacoeconomic perspective

Blood ◽  
2016 ◽  
Vol 128 (24) ◽  
pp. 2757-2764 ◽  
Author(s):  
S. Vincent Rajkumar ◽  
Jean Luc Harousseau
Keyword(s):  
Multiple Myeloma ◽  
Quality Care ◽  
The United States ◽  
New Drugs ◽  
Personal Finances ◽  
Life Saving ◽  
Health Insurance Premiums ◽  
Rapid Pace ◽  
The Individual ◽  
New Treatments

Abstract Advances in the diagnosis and treatment of multiple myeloma have come at a rapid pace, especially with several new drugs entering the market in the last few years. However, access to and affordability of new treatments poses a major challenge, both in the United States and around the world. High costs of life-saving drugs are detrimental to both the personal finances of the individual patient, as well as society which must bear the increasing costs in terms of increased health insurance premiums, taxes, or both. The challenges are not unique to myeloma, but are commonly encountered in several other cancers as well. But to some extent these pharmacoeconomic concerns are amplified in myeloma due to the need for multidrug regimens that combine 2 or more expensive new drugs, continuous therapy, and the prolonged disease course in most patients. We examine current myeloma therapy from a pharmacoeconomic perspective, and discuss the costs involved. We outline the underlying reasons why cancer drugs are so expensive, the measures that are required to lower cost, and propose potential ways in which costs can be reduced while still delivering high-quality care.

Managing Uncertainty in Drug Approval

2019 ◽  
pp. 108-116
Author(s):  
Charles F. Manski
Keyword(s):  
Drug Approval ◽  
The United States ◽  
New Drugs ◽  
Similar Process ◽  
European Medicines Agency ◽  
Pharmaceutical Firm ◽  
The European Union ◽  
Approval Process ◽  
New Drug ◽  
Management Of Uncertainty

This chapter considers management of uncertainty in drug approval. In the United States, the approval process of the Food and Drug Administration (FDA) determines whether a drug can legally be sold within the country. A similar process occurs in the European Union, with approval performed by the European Medicines Agency. To obtain approval for a new drug, a pharmaceutical firm must provide to the FDA information on treatment response through the performance of randomized trials that compare the new drug with an existing treatment or a placebo. The FDA makes a binary (yes/no) approval decision after reviewing the findings of these trials. Approval decisions are made with incomplete knowledge of the effectiveness and side effects of new drugs. The chapter describes how the FDA deals with uncertainty and suggests how the approval process might be improved.

Verán ustedes las chozas más humildes. El discurso de pobreza en la diplomacia pública cardenista dirigida a Estados Unidos

2019 ◽  
Vol 35 (2) ◽  
pp. 255-281
Author(s):  
Sylvia Dümmer Scheel
Keyword(s):  
United States ◽  
New Deal ◽  
Public Diplomacy ◽  
The United States ◽  
The Public ◽  
The Poor ◽  
The Face ◽  
The Government ◽  
Lazaro Cardenas ◽  
The New Deal

El artículo analiza la diplomacia pública del gobierno de Lázaro Cárdenas centrándose en su opción por publicitar la pobreza nacional en el extranjero, especialmente en Estados Unidos. Se plantea que se trató de una estrategia inédita, que accedió a poner en riesgo el “prestigio nacional” con el fin de justificar ante la opinión pública estadounidense la necesidad de implementar las reformas contenidas en el Plan Sexenal. Aprovechando la inusual empatía hacia los pobres en tiempos del New Deal, se construyó una imagen específica de pobreza que fuera higiénica y redimible. Ésta, sin embargo, no generó consenso entre los mexicanos. This article analyzes the public diplomacy of the government of Lázaro Cárdenas, focusing on the administration’s decision to publicize the nation’s poverty internationally, especially in the United States. This study suggests that this was an unprecedented strategy, putting “national prestige” at risk in order to explain the importance of implementing the reforms contained in the Six Year Plan, in the face of public opinion in the United States. Taking advantage of the increased empathy felt towards the poor during the New Deal, a specific image of hygienic and redeemable poverty was constructed. However, this strategy did not generate agreement among Mexicans.

Terrified

2014 ◽  
Author(s):  
Christopher A. Bail
Keyword(s):  
Public Discourse ◽  
The United States ◽  
September 11 ◽  
Theoretical Argument ◽  
Muslim Americans ◽  
Media Messages ◽  
American Media ◽  
The Face ◽  
September 11 Attacks ◽  
The Public Sphere

In July 2010, Terry Jones, the pastor of a small fundamentalist church in Florida, announced plans to burn two hundred Qur'ans on the anniversary of the September 11 attacks. Though he ended up canceling the stunt in the face of widespread public backlash, his threat sparked violent protests across the Muslim world that left at least twenty people dead. This book demonstrates how the beliefs of fanatics like Jones are inspired by a rapidly expanding network of anti-Muslim organizations that exert profound influence on American understanding of Islam. The book traces how the anti-Muslim narrative of the political fringe has captivated large segments of the American media, government, and general public, validating the views of extremists who argue that the United States is at war with Islam and marginalizing mainstream Muslim-Americans who are uniquely positioned to discredit such claims. Drawing on cultural sociology, social network theory, and social psychology, the book shows how anti-Muslim organizations gained visibility in the public sphere, commandeered a sense of legitimacy, and redefined the contours of contemporary debate, shifting it ever outward toward the fringe. The book illustrates the author's pioneering theoretical argument through a big-data analysis of more than one hundred organizations struggling to shape public discourse about Islam, tracing their impact on hundreds of thousands of newspaper articles, television transcripts, legislative debates, and social media messages produced since the September 11 attacks. The book also features in-depth interviews with the leaders of these organizations, providing a rare look at how anti-Muslim organizations entered the American mainstream.

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