7. Managing Uncertainty in Drug Approval

2019 ◽  
pp. 108-116
Keyword(s):  
Drug Approval

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.    

Regulatory requirements and comparison in approval of new and generic drugs in United States of America and Japan:-Implication for Future strategies

2020 ◽  
Vol 07 ◽  
Author(s):  
Paramjeet Malik ◽  
Neelam Pawar ◽  
Kavita Bahmani
Keyword(s):  
Generic Drugs ◽  
Drug Product ◽  
Drug Approval ◽  
Approval Process ◽  
Regulatory Requirements ◽  
Team Members ◽  
Regulatory Authorities ◽  
Effective Manner ◽  
Review Team

: Safety, efficacy and quality of a therapeutic product is the major concern for the pharmaceutical companies. FDA and PMDA are the main regulatory authorities in USA & JAPAN respectively that ensures the maintenance of these required parameters by forming standard guidelines and process for drug approval. These regulatory authorities’ reviews each step of a pharmaceutical drug product from its discovery phase to marketed product. Dossier plays an important role during the approval process of a drug product, as it allows both applicant and review team members to evaluate the data in an effective manner. A dossier consists of five modules containing informative data of various stages of a drug product but in a brief pattern with folders and subfolders. In the present paper, the authors focus on in-depth review of approval process for new and generic drugs in USA and Japan.

COMPARATIVE STUDY FOR GENERIC DRUG APPROVAL PROCESS AND THEIR REGISTRATION AS PER CTD IN EUROPE, USA AND BRAZIL

2016 ◽  
Vol 4 (2) ◽  
pp. 1-9
Author(s):  
Lincy Joseph ◽  
Mathew George ◽  
Kalpesh K Malaviya ◽  
Kalpesh K Malaviya ◽  
Bincy K Chacko ◽  
...  
Keyword(s):  
Generic Drug ◽  
Drug Approval ◽  
Evaluation Agency ◽  
Approval Process ◽  
Registration Process ◽  
Regulatory Guidelines ◽  
Food Drug Administration ◽  
Government Websites ◽  
Drug Approval Process ◽  
Regulatory Sites

This aims to compare the generic drug approval and registration process in the regulatory market of Europe, USA andBrazil. Based on the information collected from various sources such as regulatory sites, Government websites,discussion with regulatory agent, interviewing pharma professionals and literature survey from various journals, aclear picture on the generic drug approval and registration process of each country was drawn. The differentauthorities’ viz. European Medicines Evaluation Agency (EMEA) of Europe, Food Drug Administration (FDA) ofUSA and National Health Surveillance Agency (ANVISA) of Brazil carried out the generic drug approval andregistration process in the respective countries. After analysing the various requirements for the generic drug approvalin the above stated countries, it was concluded that the regulatory guidelines of Europe and Brazil was not welldefined. But FDA gives very much well defined requirements. 

MARKETING AUTHORIZATION OF GENERIC DRUG: GLOBAL ISSUE AND CHALLENGES

2013 ◽  
Vol 1 (3) ◽  
pp. 1-6
Author(s):  
Pankaj Kashyap ◽  
Eshant Duggal ◽  
Parveen Budhwar ◽  
Jitendra Kumar Badjatya
Keyword(s):  
Generic Drug ◽  
Patent Protection ◽  
Drug Approval ◽  
Approval Process ◽  
Generic Medicines ◽  
Healthcare Provision ◽  
Pharmaceutical Markets ◽  
Global Issue ◽  
Drug Approval Process ◽  
High Level

Generic medicines are those whose patent protection has expired, and which may be produced by manufacturers otherthan the innovator company. Use of generic medicines has been increasing in recent years, primarily as a cost savingmeasure in healthcare provision. Generic medicines are typically 20 to 90% cheaper than originator equivalents. Theobjective is to provide a high-level description of what generic medicines are and how they differ, at a regulatory andlegislative level, from originator medicines. It describes the current and historical regulation of medicines in theworld’s two main pharmaceutical markets, in addition to the similarities, as well as the differences, between genericsand their originator equivalents including the reasons for the cost differences seen between originator and genericmedicines. This article refers to the general generic drug approval process in India, USA, and Japan. They havedifferent regulation and approval process. 

Measuring Effectiveness

2018 ◽  
Author(s):  
Jacob Stegenga
Keyword(s):  
Outcome Measures ◽  
Relative Risk Reduction ◽  
Drug Approval ◽  
Target Population ◽  
Risk Difference ◽  
General Context ◽  
Measuring Instruments ◽  
Clinical Settings ◽  
Medical Interventions ◽  
Regulatory Standards

There are three methodological challenges to measuring the effectiveness of medical interventions: the choice of good measuring instruments, the use of appropriate analytic measures, and the use of a reliable method of extrapolating measures from an experimental context to a more general context. In practice each of these challenges contributes to overestimating the effectiveness of medical interventions. These challenges suggest corrective principles. The instruments employed in clinical research should measure patient-relevant and disease-specific parameters. Effectiveness always should be measured and reported using absolute outcome measures (such as ‘risk difference’), and we should employ caution when interpreting relative outcome measures (such as ‘relative risk reduction’). Extrapolating from research settings to clinical settings should more rigorously take into account possible ways in which interventions can fail to be effective in a target population. Current regulatory standards for drug approval are insufficient to manage these problems of measurement.

scholarly journals A 3D Bioprinted Material That Recapitulates the Perivascular Bone Marrow Structure for Sustained Hematopoietic and Cancer Models

Polymers ◽  
2021 ◽  
Vol 13 (4) ◽  
pp. 480
Author(s):  
Caitlyn A. Moore ◽  
Zain Siddiqui ◽  
Griffin J. Carney ◽  
Yahaira Naaldijk ◽  
Khadidiatou Guiro ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Bone Marrow ◽  
Translational Medicine ◽  
Drug Response ◽  
Drug Approval ◽  
3D Models ◽  
3D Bioprinting ◽  
Effective Evaluation ◽  
Experimental Systems ◽  
Cancer Models

Translational medicine requires facile experimental systems to replicate the dynamic biological systems of diseases. Drug approval continues to lag, partly due to incongruencies in the research pipeline that traditionally involve 2D models, which could be improved with 3D models. The bone marrow (BM) poses challenges to harvest as an intact organ, making it difficult to study disease processes such as breast cancer (BC) survival in BM, and to effective evaluation of drug response in BM. Furthermore, it is a challenge to develop 3D BM structures due to its weak physical properties, and complex hierarchical structure and cellular landscape. To address this, we leveraged 3D bioprinting to create a BM structure with varied methylcellulose (M): alginate (A) ratios. We selected hydrogels containing 4% (w/v) M and 2% (w/v) A, which recapitulates rheological and ultrastructural features of the BM while maintaining stability in culture. This hydrogel sustained the culture of two key primary BM microenvironmental cells found at the perivascular region, mesenchymal stem cells and endothelial cells. More importantly, the scaffold showed evidence of cell autonomous dedifferentiation of BC cells to cancer stem cell properties. This scaffold could be the platform to create BM models for various diseases and also for drug screening.

AIDS Drugs & the Pharmaceutical Industry: A Need For Reform

1991 ◽  
Vol 17 (4) ◽  
pp. 363-410
Author(s):  
Mary T. Griffin
Keyword(s):  
Pharmaceutical Industry ◽  
Research And Development ◽  
Drug Approval ◽  
Hiv And Aids ◽  
Price Regulation ◽  
Drug Industry ◽  
Drug Companies ◽  
Aids Drugs

AbstractThe pharmaceutical industry has long enjoyed substantial profits despite increased requirements for drug approval and various attempts to regulate the industry. Drug companies have avoided effective regulation by blaming high prices on the costs of research and development. The search for drugs effective in combatting HIV and AIDS related illnesses has provided a stark background on which to view the actions and justifications of drug companies. Despite increased cooperation between government and the drug industry and expedited approval of several useful drugs, these drugs are still prohibitively expensive. This Article explores the history and economics of the drug industry and proposes a system of national price regulation for all drugs.

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