Terms Beginning With 'b'

Breakthrough Therapy Designation

What is a Breakthrough Therapy designation?

Breakthrough Therapy designation is a process devised for accelerating the development and evaluation of drugs/medicines meant for the treatment of severe diseases. To obtain a Breakthrough Therapy designation, a drug must have initial clinical data indicating it may show considerable improvement over already existing treatments based on clinically significant endpoint(s).

For establishing whether the improvement over the existing treatment is significant is based on judgment. The decision considers the extent of the treatment effect (including duration) and the significance of the observed clinical outcomes. Generally, the initial clinical data should demonstrate a clear advantage of the drug over existing treatment.

The clinically substantial endpoint generally indicates an endpoint that gauges an effect on symptoms indicating severe consequences of the indication or irreversible mortality or morbidity (IMM).

A clinically substantial endpoint can also look at the findings indicating an impact on IMM or severe signs, comprising-

  • An impact on a recognized surrogate endpoint.
  • An impact on a surrogate endpoint or a clinical endpoint deemed likely to forecast a clinical advantage.
  • A substantially superior safety profile as compared to already existing treatments (like for an oncology agent less dose-limiting toxicity), with proof of similar efficacy.
  • An effect on pharmacodynamic biomarkers that does not fulfil standards for an acceptable surrogate endpoint but indicates the possibility for a clinically profound impact on the underlying indication.

The Food and Drug Administration (FDA) has established four distinctive and successful methods for speeding the availability of drugs that are for treating serious or life-threatening diseases. Breakthrough Therapy designation is part of the four fast-track approaches. The other three approaches are priority review, accelerated approval, fast track designation.

What are the benefits of Breakthrough Therapy designation to the drugs?

The pharmaceutical company or a drug manufacturer request for Breakthrough Therapy designation. If a Breakthrough Therapy designation has not been applied for, the Food and Drug Administration might recommend that the sponsor submits an application requesting for the designation if-

  • After assessing submitted data, including initial clinical data), the Agency believes the drug development program could meet the Breakthrough Therapy designation criteria.
  • The designation would aid the rest of the drug development process.

To achieve any characteristic of the designation, The FDA should preferably receive the Breakthrough Therapy designation request before the end-of-phase-2 meetings.

Since the primary objective of Breakthrough Therapy designation is developing evidence required to support approval effectively, the FDA does not expect that the designation requests would be made after submitting an original new drug application or biologics license applications (BLA) or a supplement. Once the Breakthrough Therapy designation request is received, the FDA will respond within the next 60 days.

A drug that has been granted Breakthrough Therapy designation is qualified for:

Differences between the benchmarks for fast track designation and Breakthrough Therapy designation

Fast Track designation and Breakthrough Therapy are intended for advancing the drug development as well as the review of drugs for lethal diseases.

However, there are differences related to the requirements to be determined for qualification for the programs.

A Breakthrough Therapy designation is for a drug that treats a life-threatening or serious disease, and initial clinical data shows that the treatment might show considerable improvement on a clinically significant endpoint(s) as compared to the existing therapies. 

A Fast Track designation is for medicines that is for a fatal or serious disease, and clinical/nonclinical data show the potential for tackling the unmet medical needs.

What is the deadline for a sponsor to submit a request for a Breakthrough Therapy designation?

The Food Drug and Cosmetic Act says that a Breakthrough Therapy designation request could be made simultaneously with, or later, the application submission for the investigational new drug (IND).

To increase the advantages of the program, the FDA urges drug manufacturers to submit the applications by the end-of-phase-2 meeting. The application should be submitted before clinical trial commencement that will help assess the efficacy of the drug.

Few Recent Examples of Drugs Receiving Breakthrough Therapy designation

Novartis Pharmaceutical Corp Granted Breakthrough Therapy designation for capmatinib

Novartis Pharmaceutical received a grant from the FDA for capmatinib. The drug is being developed for treating adult individuals having metastatic non-small cell lung carcinoma.

Genentech Inc Received Breakthrough Therapy designation for two drugs

Genentech received Breakthrough Therapy designation for bevacizumab and atezolizumab. These drugs, in combination, are used for treating patients suffering from unresectable or metastatic hepatocellular carcinoma who have not had any prior systemic therapy.

Boehringer Ingelheim Pharmaceuticals Inc received Breakthrough Therapy designation for avapritinib

The FDA granted Breakthrough Therapy designation to Boehringer Ingelheim Pharmaceuticals Inc for the development of avapritinib. The drug is indicated for the treatment of adults having unresectable or metastatic gastrointestinal stromal tumor harboring a platelet-derived growth factor receptor alpha exon 18 mutation, as well as PDGFRA D842V mutations.

What is Globalization? Globalization has long been supported in view of the economic, political and cultural benefits, supporting the exchange of ideas, talent, technology, culture, trade and investment. However, the trending phenomenon of Globalization is under the scanner, with increasing difficulties faced by policymakers in preventing the national economic crisis and economic depression from creating havoc across the globe. Be it 1930s Great Depression, 2008’s Global Financial Crisis or 2020’s Global Virus Crisis, Globalization has played a major role in the geographical spread of financial and economic turmoil. Globalization is defined as a phenomenon of the world becoming interconnected by the exchange of goods, services, people, trade, culture, money, ideas, technology and policies. It refers to the interdependence of human resources, corporates and governments across the world. Though it has been taking place for hundreds of years, the Globalization process has expedited over the last half-century, encompassing economic, cultural and political exchanges across the globe. How Do We Trace Back the Early Signs of Globalization? In the history books, the inter-state trade has its mention from the earliest civilizations dating back to roughly 1600s. Communities and states enlarged interdependence equations and trade, resulting in key exchange of social ideas and practices. Back in the third millennium BC, the economic trades happened between Sumer and Indus Valley Civilisation with the commercial urban centres opening between the Greek to Indians. This inter-state trade phenomenon was not only driven by Europe but also Old World centres - India, China, Japan.  Further, free-trade agreements, global political and economic discussions, and free-flow of money, people and technology promoted the evolution of Globalization in different themes. How Do We See Current Globalization Trends? Since the beginning of the 19th century, Globalization has accelerated with the breakthrough in transportation and communication technology. In the late 20th and early 21st century, Globalization has also increased for the sports and entertainment sector.  Besides, digital trade as a component of Globalization is gaining importance between the nations. Investment flows for accessing new markets in developing economies and associated investment, people and technology flows also shape the growing Globalization trends. Financial markets including commodity, debt, currency and stock markets are increasingly interconnected, with market players tapping buoyant investment opportunities across the world. On cultural exchanges, movement of students for academic purposes and learning is further supporting global interconnection trends. Although Globalization is probably serving as a bridge between countries to create more wealth, studies have shown it is not reducing the economic gap between the poorest countries and the richest. Conflicts between nations and irregular diplomacy have played a large part in the history of Globalization, and it continues to do so.   What are the Key Drivers of Globalization? Transportation: The speed, length and breadth of Globalization has increased for a number of reasons. Developments in Information Technology, transport and communication sectors have rapidly grown over the past 40 years. The internet has been transformative in closing the gap in communication with providing fast and 24/7 access world over. The containerization system where large quantity goods and commodities are shipped using intermodal containers has also enabled trade around the globe at extremely low cost.  Communications: The sharp rise in social and digital media has made the national boundaries absolutely irrelevant as any company can communicate and market its products and services in any part of the world directly connecting with its target audience. Easy access to virtual global market trade has grown substantially after the emergence of the internet. New electronic payment systems such as e-wallets, mobile banking, pre-pay have facilitated the increased global trade. For better or worse, globalised trade, supply chain, outsourcing have changed the way the world operates, and its impact on organizational operations and practices continues to grow.  Free trade: Following the collapse of communism ideology, the trade has become increasingly free with many former communist countries opening for Foreign Direct Investment (FDI) and global trade. The emergence of global players such as Google, Facebook, Apple, Microsoft, etc are by-products of Globalization. Is Globalization Changing the World for Better or Worse?   Globalization have several pros and cons attached to the international linkages between economies and markets. Current Globalization trends have largely integrated developing economies with less developed economies through Foreign Direct Investment (FDI); with the decrease in trade barriers like import quotas and tariffs as well as economic reforms like policies and regulations, and also at a large extent, immigration.  International standards have created efficiency in the trade for goods and services, and the International Organization sets these standards. Currently, the most significant free-trade area is the European Union. EU policies implement the free movement of resources (human, capital and ideas) within the European Union market. Access to broader markets means higher demand for products and services.  Though it can also lead to a lack of product diversity, like most of the world's computers, use Microsoft's Windows operating system, presenting barriers to small, local producers. Besides, global players are exerting downward pressure on wages below fair market equilibrium level.  How is Globalization Influencing Culture and Political behaviors?  At present, most of the cultures have been defused because of the internet, popular culture media and travel. As people are more exposed to other cultures, transition of ideas, values, meanings take place, which is also extended into fashion, food, languages and even religion. While Globalization’s impact on art and culture is clearly visible, but it also creates uncertainty in ideologies and disorientation in identities. Critics of Globalization argue that global connectivity poses thread to prevalent indigenous cultures.  Globalization has also interlinked state governments, intergovernmental organisations, social organisations etc. This integration between national and international levels of authority have pushed the emergent global political economy. Some countries also allow its citizens to take citizenship of other countries. Another essential aspect of Globalization is the movement of people, not only limited to immigrants but also encompassing movement for travel and business. Foreign students' revenue across different courses and learning programs add to country’s economic growth, both financially and culturally. But overdependent on these interlinkages in terms of trade, money and culture can be harmful as the system may collapse when there is a sudden downturn in the world. Novel coronavirus (COVID-19) pandemic completely halted peoples’ movement, which impacted tourism, education, hospitality industries severely. 

What are clinical trials? Clinical trials are experiments on human volunteers to evaluate the safety and efficacy of a new medical treatment. In other words, clinical trials are carefully designed studies which test the benefits along with risks of a specific medical therapy or intervention, such as a new chemical entity. The trials are conducted by healthcare institutions and pharmaceutical/biotech companies, usually in collaboration. The studies are headed by a lead investigator, known as principal investigator. Early-stage studies are conducted at a couple of sites in a single geography while late-stage trials are carried out at multiple locations across different geographies. Several healthcare and biotech companies perform clinical trials in collaboration with institutes or universities. Large-cap healthcare players, with a market capitalisation greater than US$10 billion, generally have the geographical presence and financial capability to conduct large-scale clinical trials on their own. Small-cap companies, however, have limited resources and typically enter license agreements with their bigger counterparts to conduct large-scale trials. What are the various phases of clinical trials? To understand the various phases of clinical trials, let us the example of the biggest healthcare market in the world, the US. After scientists and researchers complete the rigorous screening and preclinical testing process, the Company files for an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA). After approval of this application by the FDA, the investigational drug can be tested in human volunteers under clinical trials. If the preclinical research findings are promising, the FDA approves the IND application of the drug for clinical trials in human volunteers. Clinical trials take place in several phases that are discussed below- Phase 0- Phase 0 clinical trial is conducted with a minimal number of people, usually fewer than 15. In this phase, the clinical trial investigators use a very small dose of the drug to make sure it is not harmful to human beings before they commence next stage studies with higher doses of the drug. If there is a case that the drug or treatment acts differently than anticipated, the investigators will likely do additional preclinical research before deciding whether to continue the trial. Phase 1- During Phase 1 clinical trial, investigators spend several months looking at the effects of the medication on about 20 to 100 healthy volunteers. This phase mainly aims at figuring out the maximum dose humans can take without any serious side effects. Investigators examine volunteers carefully to observe how they react to the drug during this phase. In addition to evaluating the safety and ideal dosage, investigators also look at the best way to administer the drug, like orally, intravenously (IV), Intradermally or topically, among other routes. According to the FDA, nearly 70% of medications move on to Phase 2 clinical trial. Phase 2- This phase of the clinical trial involves several hundred volunteers who have the disease or condition that the new drug is intended to treat. In Phase 2 clinical trial, usually the same dose, that was found to be safe in Phase 1, is administered to the participants. Investigators examine participants for several months or years to find out how effective the treatment is and collect more information about any side effects the new drug might cause. While Phase 2 involves more participants than in earlier phases, it is still not large enough to demonstrate the overall safety of a medication. However, the information collected during this Phase help researchers come up with methods and procedures to conduct Phase 3 trials. As per the FDA, approximately 33% of medications move on to Phase 3 clinical trial. Phase 3- Phase 3 clinical trials usually involves 300 to 3,000 volunteers having the condition or disease that the new drug/treatment is intended to treat. Some studies can even have participants in five digits depending on the prevalence of the disease being studied. Phase 3 clinical trials can last for several years (from 1 to 4 years). The main objective of Phase 3 clinical trials is to assess how the new drug works in comparison to already existing medications for the same disease. To go ahead with the clinical trial, investigators are required to demonstrate that the medication is at least as safe & effective as existing treatment options. The FDA estimates that approximately 25-30% of drugs move to the Phase 3 clinical trial. Phase 4- Phase 4 clinical trials take place after the drug is approved by the FDA. The trial involves monitoring the safety and efficacy of a device or a drug in a large population.  In Phase 4 clinical trial, investigators get more information about the long-term safety, effectiveness, and any other benefits of the approved drug. The selection of participants, age groups, sex, ethnicity etc. for the clinical trials is based on the disease or condition being studied. As an example, for diseases where prevalence is high among the old age population, a higher number of baby boomers will be enrolled in the studies. What are the benefits and risks involved for the volunteers? All clinical trials have risks as well as benefits. The risks of participating in clinical trials depend both on the treatment being evaluated and the health of the participants. Clinical researchers will describe the well-known risks before commencing the trial when a participant receives an informed consent form. The volunteers will be informed by the researchers if they find any new risk information during the trial. The risk involved during the trials are the same as routine medical care, and the activities of daily living do. When assessing the risks of research, one can consider two factors: Chance of any harm occurring during clinical trials. The level of harm that could result from participating in the clinical trials. Every clinical trial has its benefits and risks, depending on the type of trial and what it is trying to achieve. Potential Benefits- Well-designed and properly executed clinical trials offer the best approach for participants to- Get access to a treatment that isn't available yet- This treatment may be more effective or have fewer side effects than the treatments that are currently available. Volunteers could perform an active role in their health care. The clinical trials gain access to new research for treatments before they are widely available in the market. During the clinical trials, participants receive consistent and careful medical attention from a research team of doctors and other health professionals. The treatment may be free or at a lower cost. Contributing to the clinical trial might save lives in the upcoming time by contributing to medical research. Potential Risks- Risks for volunteers during the clinical trials include: During the clinical trials, there may be unpleasant, severe, or even life-threatening side effects to investigational drug/treatment. The clinical trials may require more time and attention than standard treatment would, such as visits to the study site, several blood tests, more treatments, hospital stays, or complex dosage requirements. The new treatment may not work for everyone, even if it is beneficial for some other volunteer in the trial. It may be possible that the new medication is not as effective as the currently available treatment. Volunteers are not being able to choose which drug/treatment they are going to receive during the trial. In randomised trials, volunteers are randomly assigned to get any particular treatment. In some trials, subjects may be assigned to get a placebo or sugar pills. In a randomised, double-blinded study, neither the volunteer nor the investigator would know on which treatment the testing is being performed (although the information is available if it is needed). What are the steps after each phase of clinical trials? Once a clinical trial or study has completed, the investigators or researchers will carefully collect and analyse the data to find out what next steps are required as an outcome of the findings. Before a trial starts, the participants should be provided information about how long it is going to last, whether participants will continue receiving the study treatment after the trial ends, if applicable, and how people will be kept informed about the results of the study.

What is Drug Development? Drug development is the procedure of getting a novel treatment, therapy, or a drug, to the market after identification of a lead compound. Pharmaceutical regulatory authorities play a significant role throughout the process of drug development. These authorities are designed to make sure the safety, efficacy, accessibility & security of approved drugs. Taking an example of the United States, the United States Food and Drug Administration (FDA) is a regulatory body that is responsible for the approval of the drug candidate post the drug discovery and development process. What are the Various Stages of Drug Development? Process of drug development comprises five key stages- Drug Discovery & Development; Preclinical research; Investigational New Drug Application; Clinical Research; and Regulatory Review, Approval & Post-marketing Surveillance. Drug Discovery & Development Drug discovery is the process of finding new, innovative medication. During the process of drug development, there may be thousands of compounds as potential candidates for development as a treatment or therapy. After the initial assessment, though, only a few compounds show promising activity and call for further study. After identification of a promising drug compound for development, the researchers conduct experiments to collect information related to the absorption, distribution, metabolism, and excretion (ADME). Researchers will also find the potential benefit, risk, side effects and adverse events along with the mechanism of action of that specific compound. Preclinical Research Before assessing a new drug in human subjects, scientists must find out whether it has the potential to cause severe harm, also known as toxicity. Preclinical research is of two types: in-vitro and in-vivo. The research is designed to provide essential information related to the safety and efficacy of a novel drug candidate before its testing in human volunteers. Both in-vitro and in-vivo models are typically used to provide evidence of the biological effect of a new drug candidate. Preclinical trials are required by regulatory authorities such as the FDA and MHRA before submitting an IND (investigational new drug application), which is needed to move to the clinical research. In preclinical studies, pharmacodynamics and pharmacokinetics of a novel drug candidate are addressed. It is crucial that the most suitable animal models are used to evaluate initial safety information of drug during the preclinical study. FDA requires scientists to make use of GLP (good laboratory practices) for preclinical laboratory studies.  Generally, preclinical research is not very extensive. However, preclinical studies must give detailed information on drug dosing as well as toxicity levels. After completion of preclinical testing, scientists review their outcomes and determine whether the drug should be tested in humans. Investigational New Drug Application or IND The FDA divides investigational new drugs (INDs) into three different types- An Investigator IND is submitted by a doctor who initiates as well as investigates. Under the doctor’s direction, the investigational drug is administered.  An investigator or physician might submit a research IND to study an unapproved or a new drug. In Emergency Use IND the FDA authorises the use of a new investigational drug in an emergency that does not allow time for submission of an IND in accordance with 21CFR (Sec. 312.23 or Sec. 312.20).  It is also applied for patients who do not fulfil the standards of the current study protocol, or if there is no existing approved study protocol. Treatment IND is for experimental drugs which show promise in clinical testing for severe or immediately life-threatening diseases while the final clinical work is conducted, and the FDA review takes place. After submission of the IND, the investigator or sponsor must wait for thirty days before starting any clinical research. During this one month, the FDA review the IND for safety to make sure that research subjects will not be subjected to any unreasonable risk. The IND application should comprise information in three areas, including animal pharmacology & toxicology studies, manufacturing details, along with the information related to clinical protocols & study investigator. Drug developing companies are free to seek help from the FDA at any point in the process of drug development, including: Pre-IND application for the review of FDA guidance documents as well as find answers for augmenting their research. After Phase 2 clinical trial, to obtain guidance on the design of large Phase 3 clinical trials. During the process to get an evaluation of the IND application. IND Approval The FDA review team has thirty days for the review of the original IND submitted by the drug developer. This process protects the clinical trial participants from any substantial and unnecessary risk during clinical trials. For the IND applications, the US regulator provides its response in two aspects: Approval to commence the clinical trials. Clinical hold for halt or delay of the examination. For the clinical hold, the FDA can put the trial on hold for specific causes, including: Trial subjects are exposed to significant or any unreasonable risk. Investigators of the clinical are not qualified. Materials for the study volunteers are misleading. If the IND application does not comprise sufficient information related to the risks of the clinical trial. However, a clinical hold is unusual, and as an alternative to the hold, FDA often offers comments to improve the clinical trial quality. In most instances, if the regulatory agency is convinced that the clinical trial fulfils the Federal standards, the applicant is permitted to continue the proposed clinical study. Moreover, the drug developer is accountable for informing the FDA review team about new protocols, along with the serious side effects observed during the study. This information makes sure that the review team can monitor the clinical trials thoroughly to signal any side effects. After the completion of the clinical trial, investigators must submit the reports of the study. The process of informing the FDA about the trial’s new protocols continues until the applicant decides to end clinical trials or files a marketing application. Before filing a marketing application, the drug developer must have sufficient information from two large, controlled clinical trials. Clinical Research Several types of clinical research are done depending on what research is going on. Some types of clinical research are- Source: Copyright © 2020 Kalkine Media Pty Ltd, Data Source: FDA Other types of clinical research Other than the above-mentioned research, there are some other clinical studies that do not involve drug investigation, and a person’s regular medications may not require to be altered. Besides, healthy volunteers are also needed to compare their results to the results of patients being studied. Below are a few examples of other kinds of research: Long-term clinical research that includes brain scans or psychological tests. A genetic study, including blood tests but without any change in medication. A clinical study of family history involving family members to learn about medical needs and history of people. When clinical research is used for evaluation of any medication or device, it is conducted in four clinical trial phases- Phase 1 Clinical trials- In Phase 1, researchers test an investigational drug or treatment in a small group of volunteers for the first time. Safety and efficacy of the drug are evaluated in this phase with the identification of side effects. Phase 2 Clinical trials- In this phase, an experimental drug is given to a larger group of subjects to see if it is effective and to evaluate its safety further. Phase 3 Clinical trials- The experimental study drug or treatment is given to large groups of people. Researchers confirm the effectiveness of the new drug, monitor side effects, compare it to commonly used medications, and collect information that will permit the experimental drug or treatment to be used safely.  Phase 4 Clinical trials- Phase 4 clinical trial comprises post-marketing studies that are organized after approval of a drug or therapy for use by the FDA. Post-marketing studies provide some additional information, including the risks, adverse effect and best use associated with the treatment. To Know More About Clinical Trials Click Here. New Drug Application or NDA An NDA reveals the complete story of a drug, and its objective is to determine the safety and efficacy of the drug for its anticipated application in the population studied. In an NDA, a drug developer must incorporate every information related to a drug, from preclinical study data to Phase 3 clinical trial data. With the results of the clinical trials, drug developers should include the below-mentioned information- On receiving an NDA, the FDA review team determines whether it is complete or not. If the application is incomplete, the review team has the right to reject to file the New Drug Application. If it is complete, the team has six to ten months to decide the drug approval. In instances where the FDA finds that a drug has been demonstrated safe and effective for its intended use, then the agency will work with the applicant for the development as well as refining of the prescribing information. This process is termed as labeling. Labeling precisely and accurately describes the basis for approval and the best use of the drug. Regulatory Review, Approval & Post-marketing Surveillance Suppose a drug manufacturing company has evidence from its preliminary tests, preclinical and clinical research that the innovative drug/treatment is safe and effective for its intended use. In that case, the drug developer may file an application for the marketing of the drug. FDA approval for a medicine/drug means that the Center for Drug Evaluation and Research (CDER) has reviewed the information on the effects shown by the new drug, and during the review, the drug provided benefits outweighing its known risks for the intended population. If clinical trials provide vital information on the safety and efficacy of a drug, it is not possible to have complete information related to the safety of a drug at the time of approval. Hence, the FDA reviews reports of problems with prescription and over-the-counter (OTC) drugs. Based on these reports, the FDA decides to add cautions to the dosage or the usage information of the drug, along with some other measures related to serious issues or adverse effects. Post-marketing safety surveillance is the monitoring of a drug after receiving the approval and market launch. The surveillance is designed for the evaluation of long-term safety along with the drug efficacy. Besides, the FDA is developing a new national system to spot potential safety issues more swiftly under the Sentinel Initiative. This system will utilize extremely large existing electronic health databases such as electronic health records (EHR) systems, administrative and insurance claims databases, and registries to track approved drugs’ safety in real-time. According to the FDA. This tool will be an addition to the existing post-market safety assessment tools of the FDA. Drug development is a time, and knowledge-intensive process and several healthcare companies outsource the entire process, or parts of it, to organizations known as Contract Manufacturing Organizations (or CMOs). Click here to learn about one such Australian player, Sypharma.

What is a New Drug Application? A New Drug Application (NDA) of the US FDA (Food and Drug Administration) is an application by which drug developers or sponsors formally seek the FDA approval for a new drug for sale and commercialization in the United States. A New Drug Application (NDA) informs the complete information of a new drug molecule. The purpose of NDA is to prove that a drug is safe & effective for its expected use in the large population study. According to the FDA, 30% or less of initial drug candidates proceed through the complete multi-year drug development process, concluding with an NDA approval, after successful completion of the clinical studies and then the review of the clinical data. New Drug Applications are usually regulated by the Center for Drug Evaluation and Research (CDER) of the FDA. The filing of an NDA, after successful completion of clinical trials, represents a crucial breakthrough for a new drug. An NDA filing is a critical milestone for any organization, and investors keep a close watch on the process. The fate of any pharmaceutical company can change when the FDA passes an NDA approval of a new drug. This step is more crucial for small-cap healthcare companies, as their market value can change once their drug candidate receives approval. After the submission of the NDA, the probability of a drug getting approved is very high. Accordingly, healthcare companies that file NDA witness a rise in their share prices even before the response from the FDA. What is included in a New Drug Application? The objectives of the NDA are to provide adequate information to permit FDA reviewers to determine the comprehensive history of the new drug candidate. Some facts that are required for the new drug application are- Information related to patent, manufacturing, and safety of the drug. Specific effectiveness for the proposed use(s) of the drug when used as directed. Reports on the design, compliance, and conclusions after completion of clinical trials by the Institutional Review Board. Susceptibility of the drug to abuse. Proposed labeling and directions for drug use. An NDA comprises sufficient information for the FDA to determine the safety & efficacy of a new drug. The information should justify whether the benefits of the drug outweigh its risks, the proposed drug label (package insert) is appropriate or not, and whether manufacturing standards are adequate. The information included in a New Drug Application is based on laboratory, preclinical testing on animals as well as human clinical trials. Once an NDA is filed, the Food and Drug Administration reviews the product label (package insert) to be confident that the clinical information needed to use this drug safely is outlined. The agency also takes action to examine the manufacturing facilities where the drug will be manufactured. What are the resources required for an NDA Submission? The documentation needed for an NDA is intended to describe the complete information related to the drug. This information also includes what happened during the clinical trials, ingredients of the drug, results of the animal trials, pharmacodynamics, and manufacturing, & packaging of the drug. The subsequent resources provide summaries on NDA content, format, and classification, including the NDA review process- Guidance Documents for NDAs- Guidance documents for the new drug application represent the current thinking of the agency on a particular subject. Guidance documents are made for FDA review staff as well as sponsors to give guidelines to the content, processing, and evaluation of applications. These documents also help to design, manufacture, and test regulated products. Prescription Drug User Fee Act (PDUFA)- Some pharmaceutical companies pay for an expedited review with the FDA through a procedure known as Prescription Drug User Fee Act (PDUFA), that is enacted in 1992. This Act permits the FDA to access more resources to fast-track the process of approval. Several essential drugs have been approved through the Prescription Drug User Fee Act, including treatments for cancer, heart disease and AIDS. Advisory Committees- Advisory committees offer independent advice along with recommendations to the FDA on scientific & technical matters related to the development as well as evaluation of products regulated by the agency. Though advisory committees only provide suggestions to the agency, final decisions are made by the FDA. Laws, Regulations, Policies and Procedures. NDA Forms and Electronic Submissions. Final Drug Approval After the final approval, the drug becomes accessible for prescription by doctors. However, drugs might not come to the market with immediate effect because of patents disagreements, manufacturing issues, or controlled substance designation from the DEA. Usually, the pricing is revealed after approval. However, the FDA generally does not consider the pricing of drug or any economics as part of the approval process. In contrast, many other nations consider the economic effects of new drugs in their society.

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