scholarly journals FDA's drug regulatory pathways, its development strategies and regulatory considerations

2021 ◽  
Vol 9 (2) ◽  
pp. 6-15
Author(s):  
Iva Dhulia ◽  
Himani Patel ◽  
Narendra Chauhan ◽  
Nidhi Pardeshi
Keyword(s):  
Prescription Drugs ◽  
Active Ingredient ◽  
Drug Product ◽  
The United States ◽  
Development Strategies ◽  
New Drugs ◽  
Regulatory Pathways ◽  
New Drug ◽  
Generic Products ◽  
Abbreviated New Drug Applications

People who are interested in drug development may be aware that New Drug Applications (NDA) and Abbreviated New Drug Applications (ANDA) are 2 of the FDA's regulatory pathways for how prescription drugs can be approved and ultimately reach the market. In basic terms, NDAs are for new drugs that have not yet been approved and ANDAs are for generic products. NDA, also called 505 (b)(1), is the format that manufacturers use to bring a formal proposal to the FDA that a new drug should be approved and made available for use by patients in the United States. Under 505(b)(1), all investigations supporting safety and effectiveness, both clinical and nonclinical, are conducted by or on behalf of the sponsor. The other pathway is termed as abbreviated because preclinical and clinical trials are not required. The abbreviated approval pathways are described in section 505(j) and 505(b)(2) of the FD&C Act and known as ANDA and Hybrid applications respectively. Hatch-Waxman amendments in 1984 provided for a suitability petition that allows the application of ANDA for a drug product that differs from the RLD in its dosage form, route of administration, strength, or active ingredient (in a product with more than one active ingredient). The differences allowed for suitability petition and 505(b)(2) application are same but ANDA filed through suitability petition can contain only those differences that do not need clinical evidence for efficacy and safety. This article identifies considerations to help potential applicants determine the appropriate submission pathway, its development strategies to support approval under those pathways.

Fake Pharmaceuticals: How They and Relevant Legislation or Lack Thereof Contribute to Consistently High and Increasing Drug Prices

2003 ◽  
Vol 29 (4) ◽  
pp. 525-542
Author(s):  
Merri C. Moken
Keyword(s):  
United States ◽  
Prescription Drugs ◽  
The United States ◽  
Pharmaceutical Products ◽  
New Drugs ◽  
Pharmaceutical Companies ◽  
Brand Name ◽  
Potential Factors ◽  
Counterfeit Pharmaceuticals ◽  
New Applications

The use of pharmaceutical products in the United States has increased more than the use of any other health resource from 1960 to 1990. In excess of 9,600 drugs were on the market in 1984, and the Food and Drug Administration (“FDA”) approves approximately 30 new drugs and countless new applications for alterations of already existing drugs each year. In 2001, the $300 billion pharmaceutical industry sold $154 billion worth of prescription drugs in the United States alone, nearly doubling its $78.9 billion in sales in 1997. With such a rapid increase in market domination and expenditures, the U.S. government and many hospitals have focused their attention on the sales and pricing practices of pharmaceutical companies, as well as other potential factors contributing to these escalating prices. One such cause of the steadily increasing prices of brand name pharmaceuticals is the sale of fake or counterfeit pharmaceuticals (also called “look-alike” drugs).

2020 New Drug Update

2020 ◽  
Vol 35 (4) ◽  
pp. 151-161
Author(s):  
Daniel A. Hussar ◽  
Laura A. Finn
Keyword(s):  
Rheumatoid Arthritis ◽  
The United States ◽  
New Drugs ◽  
Rating System ◽  
Chronic Obstructive ◽  
Medical Problems ◽  
Idiopathic Constipation ◽  
New Agents ◽  
Obstructive Pulmonary Disease ◽  
New Drug

Five new drugs for medical problems often affecting older people and marketed in the United States in 2019 have been considered in this review. The uses and most important properties of these agents are discussed, and a rating for each new drug is determined using the New Drug Comparison Rating system developed by the author (DAH). Advantages, disadvantages, and other important information regarding each new drug are identified and used as the basis for determining the rating. The drugs considered include new agents indicated for the treatment of patients with Parkinson's disease, rheumatoid arthritis, chronic obstructive pulmonary disease, osteoporosis in postmenopausal women, and chronic idiopathic constipation.

scholarly journals Generic Drugs Not as Safe as FDA Wants You to Believe

2019 ◽  
Vol 54 (3) ◽  
pp. 283-286 ◽  
Author(s):  
C. Michael White
Keyword(s):  
United States ◽  
Prescription Drugs ◽  
Generic Drugs ◽  
The United States ◽  
Drug Supply ◽  
Active Pharmaceutical Ingredients ◽  
Pharmaceutical Ingredients ◽  
Time Horizons ◽  
Long Time ◽  
Generic Products

Food and Drug Administration (FDA) rules for the production of prescription drugs are very rigorous and, if followed, guarantees a safe drug supply. For many years, foreign manufacturers have produced substandard generic products and active pharmaceutical ingredients and shipped them into the United States. If the FDA had inspected them with the same rigor as they do domestic manufacturers, they would have found many of these egregious deviations from ethical manufacturing much earlier. Although the FDA is finally stepping up the number of inspections, their current processes still rely on preannounced inspections with long time horizons, so quality issues can be temporarily corrected and documents altered or destroyed.

2020 New Drug Update

2020 ◽  
Vol 35 (4) ◽  
pp. 151-161
Author(s):  
Daniel A. Hussar ◽  
Laura A. Finn
Keyword(s):  
Rheumatoid Arthritis ◽  
The United States ◽  
New Drugs ◽  
Rating System ◽  
Chronic Obstructive ◽  
Medical Problems ◽  
Idiopathic Constipation ◽  
New Agents ◽  
Obstructive Pulmonary Disease ◽  
New Drug

Five new drugs for medical problems often affecting older people and marketed in the United States in 2019 have been considered in this review. The uses and most important properties of these agents are discussed, and a rating for each new drug is determined using the New Drug Comparison Rating system developed by the author (DAH). Advantages, disadvantages, and other important information regarding each new drug are identified and used as the basis for determining the rating. The drugs considered include new agents indicated for the treatment of patients with Parkinson's disease, rheumatoid arthritis, chronic obstructive pulmonary disease, osteoporosis in postmenopausal women, and chronic idiopathic constipation.

Public Policy Considerations in the Pricing of Prescription Drugs in the United States

1974 ◽  
Vol 4 (1) ◽  
pp. 171-179 ◽  
Author(s):  
T. Donald Rucker
Keyword(s):  
United States ◽  
Prescription Drugs ◽  
Drug Product ◽  
Economic Value ◽  
Economic Cost ◽  
The United States ◽  
Third Party ◽  
Accounting System ◽  
Consumer Sovereignty ◽  
Cost Accounting System

The economic value of prescribed drugs consumed in the United States probably reached $10 billion in 1973. Public interest in pricing practices is traced to the involuntary nature of patient illness in general and prescribing in particular, the intrinsic attributes of drug therapy, and the growing role of third-party programs in determining reimbursement for prescription items. Drug product pricing is criticized because of the large proportion of sales revenue consumed by indirect expenses. Nine branded products are cited where the median value of revenue consumed by indirect expenses is 91 per cent. Pricing problems related to increases in active ingredients, quantities sold, and package size are also examined. Pricing at the dispensing level is assessed in terms of its relationship to the true economic cost of this function. Significant imperfections are noted and a uniform cost-accounting system for pharmacies is recommended. The issue of prescription price posting is reviewed in some detail, and it is suggested that the posting of a unique dispensing fee in each pharmacy would optimize consumer sovereignty.

The Effect of Price on Pharmaceutical R&D

2009 ◽  
Vol 9 (1) ◽  
Author(s):  
Abdulkadir Civan ◽  
Michael T. Maloney
Keyword(s):  
United States ◽  
Drug Development ◽  
Price Elasticity ◽  
The United States ◽  
Mortality Rates ◽  
New Drugs ◽  
New Drug ◽  
Development Pipeline ◽  
New Drug Development

Abstract This work extends prior research that finds drug development is driven by demand factors such as mortality rates of the diseases new drugs are aimed at. Here we find that the number of drugs in the development pipeline is strongly positively related to the price of existing drugs treating those diseases. This gives us a direct price elasticity measure from which we can draw some inference about the effect on new drug development that might occur if the pricing regime in the United States were to change.

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.

Assessment of Purity Parameters of Generic and Brand Name Losartan Potassium

2020 ◽  
Vol 17 (1) ◽  
pp. 129-139
Author(s):  
Patricia Mattiazzi ◽  
Denise Bohrer ◽  
Carine Viana ◽  
Emilene Becker
Keyword(s):  
Active Ingredient ◽  
Active Substance ◽  
Generic Drugs ◽  
The United States ◽  
Losartan Potassium ◽  
Brand Name ◽  
Elemental Impurities ◽  
Organic Impurities ◽  
Generic Products ◽  
Brand Product

Background: Generic products must be bioequivalent to the innovator brand product. Nevertheless, in addition to meeting bioequivalence standards, attention must be paid to the content of the active substance and contaminants in generic drugs. Objective: This study compared the pharmaceutical quality of four generic losartan potassium formulations with the brand-name product: Cozaar®. Methods: The United States Pharmacopeia (USP) losartan potassium standard was used as reference material. The products tested (all 50 mg formulations) included four generic tablet formulations and the innovator brand product Cozaar®. Active substance content, organic impurities, and elemental impurities were assessed following the USP monograph for losartan potassium tablets and USP Chapter <233> on Elemental Impurities. Results: The results showed that three of the four generic products had low content of the active ingredient. The values ranged from 86.4 to 93.8%, being acceptable not below 95% of the labeled amount. Organic impurities were not detected in any of the products, and of the 13 elemental impurities tested, only four elements were detected. The elemental impurities Cr, Ni, Cu, and As were, however, in amounts within the limits established by the USP monograph. The only concern on the generic drugs analyzed was the low content of the active ingredient in 75% of the products. Conclusion: Since losartan is a drug of continuous use, lower content of the active ingredient may go unnoticed by the users of the generic product and entailed clinical consequences during long-term therapy.

scholarly journals Anesthetic and Analgesic Drug Products Advisory Committee Activity and Decisions in the Opioid-crisis Era

2020 ◽  
Vol 133 (4) ◽  
pp. 740-749 ◽  
Author(s):  
Ronald S. Litman
Keyword(s):  
United States ◽  
Advisory Committee ◽  
Drug Administration ◽  
Drug Product ◽  
Food And Drug Administration ◽  
The United States ◽  
New Drugs ◽  
Analgesic Drug ◽  
Drug Products ◽  
United States Food

The United States Food and Drug Administration is tasked with ensuring the efficacy and safety of medications marketed in the United States. One of their primary responsibilities is to approve the entry of new drugs into the marketplace, based on the drug’s perceived benefit–risk relationship. The Anesthetic and Analgesic Drug Product Advisory Committee is composed of experts in anesthesiology, pain management, and biostatistics, as well as consumer and industry representatives, who meet several times annually to review new anesthetic-related drugs, those seeking new indications, and nearly every opioid-related application for approval. The following report describes noteworthy activities of this committee since 2017, as it has grappled, along with the Food and Drug Administration, to balance the benefit–risk relationships for individual patients along with the overarching public health implications of bringing additional opioids to market. All anesthesia advisory committee meetings since 2017 will be described, and six will be highlighted, each with representative considerations for potential new opioid formulations or local anesthetics.

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.

Sign in / Sign up

Export Citation Format

Share Document