- Developing New Drugs
- FDA Approval: What it means
Developing New Drugs
American consumers benefit from having access to the safest and most advanced pharmaceutical system in the world. The main consumer watchdog in this system is FDA’s Center for Drug Evaluation and Research (CDER).
The center’s best-known job is to evaluate new drugs before they can be sold. CDER’s evaluation not only prevents quackery, but also provides doctors and patients the information they need to use medicines wisely. The center ensures that drugs, both brand-name and generic, work correctly and that their health benefits outweigh their known risks.
Drug companies seeking to sell a drug in the United States must first test it. The company then sends CDER the evidence from these tests to prove the drug is safe and effective for its intended use. A team of CDER physicians, statisticians, chemists, pharmacologists, and other scientists reviews the company’s data and proposed labeling. If this independent and unbiased review establishes that a drug’s health benefits outweigh its known risks, the drug is approved for sale. The center doesn’t actually test drugs itself, although it does conduct limited research in the areas of drug quality, safety, and effectiveness standards.
Before a drug can be tested in people, the drug company or sponsor performs laboratory and animal tests to discover how the drug works and whether it’s likely to be safe and work well in humans. Next, a series of tests in people is begun to determine whether the drug is safe when used to treat a disease and whether it provides a real health benefit.
For more information about the drug development and approval process, see How Drugs Are Developed and Approved.
FDA Approval: What it means
FDA approval of a drug means that data on the drug’s effects have been reviewed by CDER, and the drug is determined to provide benefits that outweigh its known and potential risks for the intended population. The drug approval process takes place within a structured framework that includes:
- Analysis of the target condition and available treatments—FDA reviewers analyze the condition or illness for which the drug is intended and evaluate the current treatment landscape, which provide the context for weighing the drug’s risks and benefits. For example, a drug intended to treat patients with a life-threatening disease for which no other therapy exists may be considered to have benefits that outweigh the risks even if those risks would be considered unacceptable for a condition that is not life threatening.
- Assessment of benefits and risks from clinical data—FDA reviewers evaluate clinical benefit and risk information submitted by the drug maker, taking into account any uncertainties that may result from imperfect or incomplete data. Generally, the agency expects that the drug maker will submit results from two well-designed clinical trials, to be sure that the findings from the first trial are not the result of chance or bias. In certain cases, especially if the disease is rare and multiple trials may not be feasible, convincing evidence from one clinical trial may be enough. Evidence that the drug will benefit the target population should outweigh any risks and uncertainties.
- Strategies for managing risks—All drugs have risks. Risk management strategies include an FDA-approved drug label, which clearly describes the drug’s benefits and risks, and how the risks can be detected and managed. Sometimes, more effort is needed to manage risks. In these cases, a drug maker may need to implement a Risk Management and Mitigation Strategy (REMS).
Although many of the FDA’s risk-benefit assessments and decisions are straightforward, sometimes the benefits and risks are uncertain and may be difficult to interpret or predict. The agency and the drug maker may reach different conclusions after analyzing the same data, or there may be differences of opinion among members of the FDA’s review team. As a science-led organization, FDA uses the best scientific and technological information available to make decisions through a deliberative process.
In some cases, the approval of a new drug is expedited. Accelerated Approval can be applied to promising therapies that treat a serious or life-threatening condition and provide therapeutic benefit over available therapies. This approach allows for the approval of a drug that demonstrates an effect on a “surrogate endpoint” that is reasonably likely to predict clinical benefit, or on a clinical endpoint that occurs earlier but may not be as robust as the standard endpoint used for approval. This approval pathway is especially useful when the drug is meant to treat a disease whose course is long, and an extended period of time is needed to measure its effect. After the drug enters the market, the drug maker is required to conduct post-marketing clinical trials to verify and describe the drug’s benefit. If further trials fail to verify the predicted clinical benefit, FDA may withdraw approval.
Since the Accelerated Approval pathway was established in 1992, many drugs that treat life-threatening diseases have successfully been brought to market this way and have made a significant impact on disease course. For example, many antiretroviral drugs used to treat HIV/AIDS entered the market via accelerated approval, and subsequently altered the treatment paradigm. A number of targeted cancer-fighting drugs also have come onto the market through this pathway.
More information on Accelerated Approval is here.
Drug Development Designations
The agency also employs several approaches to encourage the development of certain drugs, especially drugs that may represent the first available treatment for an illness, or ones that have a significant benefit over existing drugs. These approaches, or designations, are meant to address specific needs, and a new drug application may receive more than one designation, if applicable. Each designation helps ensure that therapies for serious conditions are made available to patients as soon as reviewers can conclude that their benefits justify their risks.
- Fast Track is a process designed to facilitate the development and advance the review of drugs that treat serious conditions, and fill an unmet medical need, based on promising animal or human data. Fast tracking can get important new drugs to the patient earlier. The drug company must request the Fast Track process. More information about the Fast Track process is here.
- Breakthrough Therapy designation expedites the development and review of drugs that are intended to treat a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy. A drug with Breakthrough Therapy designation is also eligible for the Fast Track process. The drug company must request a Breakthrough Therapy designation. More information about Breakthrough Therapy designation is here.
- Priority Review means that FDA aims to take action on an application within six months, compared to 10 months under standard review. A Priority Review designation directs attention and resources to evaluate drugs that would significantly improve the treatment, diagnosis, or prevention of serious conditions. More information about Priority Review is here.
How To Invent A Catchy Drug Name
Cialis, Celebrex, Ambien, Symbicort — such fanciful and evocative names! Who comes up with these? NPR’s Rachel Martin speaks with Gary Martin, president of a pharmaceutical naming agency.
RACHEL MARTIN: Cialis, Celebrex, Ambien, Abilify, Symbicort – such fanciful and evocative names. Who makes these things up? Turns out there’s a whole cottage industry for pharmaceutical naming. Gary Martin formed his Gary Martin Group to do just that. He joins us from the studios of KALW in San Francisco. Welcome to the program, Gary.
GARY MARTIN: Well, thank you.
R. MARTIN: So you do this for a living. What makes a good drug name?
G. MARTIN: A great drug name needs to be memorable. It needs to be easy to say. It needs to be easy to write and understand. And it also needs to be clear of any existing names that may be on the registers.
R. MARTIN: Those names I just rattled off, there is something science-y about them, for lack of a better descriptor.
G. MARTIN: The starting place for pharmaceutical naming is dealing with the science. What we’re prohibited from doing in naming drugs in making any kind of claim that may be unsubstantiated or making any kind of promise. So for example, for a weight-loss drug, we could not call it Othina because that would be an overpromise. So the regulations restrict us from what we can do with names. And we have to adhere to that.
R. MARTIN: Can you tell me the story of Rogaine?
G. MARTIN: It was originally called Regain. Regain was originally proposed to the FDA for this hair-growing product. And it was thrown back at the manufacturer Upjohn because it was overpromising, suggesting that everybody could grow hair. So what they did was they just changed one vowel, and it became Rogain.
R. MARTIN: So the FDA does step in sometimes and overrules your creative process.
G. MARTIN: Yes, it does.
R. MARTIN: Can you give us an example of a drug that, in your professional opinion, was just given the perfect name.
G. MARTIN: I think a name that I was associated with years ago is Enbrel.
R. MARTIN: Remind us what it does.
G. MARTIN: Enbrel is for arthritis. Its science is very complex. And so what we wanted to do was make that complex, injectable drug very friendly. And so it became Enbrel, enabling really.
R. MARTIN: How do you come up with names? What’s your starting point in a brainstorming session?
G. MARTIN: The starting point is probably the science. What we have to do is understand what that drug is so then we can transform it into some kind of – I’ll call it a platform for hope and confidence. In other words, a name is not just a name.
R. MARTIN: Gary Martin is president of the Gary Martin Group. He joined us from the studios of KALW in San Francisco to talk about naming pharmaceuticals. Thanks so much, Gary.
G. MARTIN: Thank you.
R. MARTIN: We’d love to hear you indulge your creative process. Tell us what you think would be the perfect name for a drug. Send your suggestions to our Facebook page, or you can send us a tweet @NPRWeekend. I am @RachelNPR. This is NPR news.
NPR transcripts are created on a rush deadline by Verb8tm, Inc., an NPR contractor, and produced using a proprietary transcription process developed with NPR. This text may not be in its final form and may be updated or revised in the future. Accuracy and availability may vary. The authoritative record of NPR’s programming is the audio record.
Street names aside, who comes up with crazy non-words like Zyrtec, tenofovir and Xeljanz?
By Rebecca Boyle | Published Apr 23, 2013 6:00 PM
Say it with me: Xeljanz. OK, at least try and say it with me. Shell-jance? Zell-johns? Ghel-yahns? Who knows. It’s a new arthritis drug, and I have no idea how to pronounce it, but one thing is definitely clear: It could be worth billions for its maker, Pfizer. (That name you probably know how to say.)
Though awkward to pronounce, Xeljanz is more memorable than its generic name, tofacitinib citrate. And it’s much more noteworthy than its chemical name, some unpronounceable string of characters. When the patent expires on Xeljanz a long time from now, people will ask their doctors for tofacitinib citrate instead.
Drugs, in other words, have at least three names. And coming up with these names, both a chemical name and its commercial brand, requires a drug name decoder. It’s somewhat like learning English words based on their roots: Once you know bio comes from the Greek for life, you know biosphere, biology, biography and so on have to do with that subject.
For drugs, the key is chemistry. Drug makers propose generic names according to their compound’s chemical makeup. While the chemical formula is what truly defines the drug, the name’s stem describes its structure, function and molecular targets. Tofacitinib citrate includes an epidermal growth factor receptor (-nib), which describes how the drug works. Something with a -mab ending, like lots of cancer drugs, are monoclonal antibodies; they target specific cells, viruses or bacteria, and you can identify those by the rest of the name. Something ending in a -vir is, predictably, an antiviral.
As an example, look at tenofovir. This is a drug used to treat HIV infection, and its chemical name is (<[(2R)-1-(6-amino-9H-purin-9-yl)propan-2-yl]oxy>methyl)phosphonic acid. Want to read that over the phone to a pharmacist? Neither does any human anywhere. So instead, the people who discovered it came up with tenofovir. Given the right stem, describing structure and function–the -vir– researchers can tack on syllables of their choice.
While the names sound strange, this is very serious business, according to the Food and Drug Administration. Mis-prescribed drugs are the most common error in health care, and thousands of people die this way every year. New generic names must meet standards set by the World Health Organization’s International Nonproprietary Names (INN) and the United States Adopted Names for pharmaceuticals, and brand names must pass muster with the FDA.
As for commercial names, pharmaceutical companies are reluctant to share their strategies. These are hugely important brands, even after the patents expire–nobody knows what Sildenafil citrate is, but everyone knows its brand name of Viagra. And the odds are you know someone who insists on brand-name Tylenol instead of discount-store acetaminophen.
Though pharmaceutical companies contacted by PopSci wouldn’t share their marketing secrets, you can still derive a drug’s molecular and popular heritage just by studying its name. Like, for example, Tylenol. Its chemical name is N-acetyl-p-aminophenol–see the tyl and -ol in there? Then there’s Viagra. It doesn’t sound like sildenafil, but it certainly suggests vigor and virility–exactly what Pfizer wants you to think. Xeljanz, which was approved earlier this year after almost 20 years of research, targets a protein called Janus kinase. It works in a different cellular pathway from other arthritis drugs. That’s where you get the -jan part of that name, which, to someone with a drug decoder, connotes its uniqueness.
As for the Shell, we’ll have to leave that to the imagination.
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Drug establishments are required to provide FDA with a current list of all drugs manufactured, prepared, propagated, compounded or processed for sale in the U.S. at their facilities. Drugs are identified and reported using a unique, three-segment number called the National Drug Code (NDC) which serves as the FDA’s identifier for drugs. FDA publishes the listed NDC numbers in the NDC Directory which is updated daily.
The NDC Directory contains information on active and certified finished and unfinished drugs submitted to FDA in structured product labeling (SPL) electronic listing files by labelers. A labeler may be a manufacturer, including a repackager or relabeler, or the entity named on the product label.
The NDC Directory contains product listing data submitted for all finished drugs including prescription and over-the-counter drugs, approved and unapproved drugs and repackaged and relabeled drugs.
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Drugs often have several names. When a drug is first discovered, it is given a chemical name, which describes the atomic or molecular structure of the drug. The chemical name is thus usually too complex and cumbersome for general use. Next, a shorthand version of the chemical name or a code name (such as RU 486) is developed for easy reference among researchers.
When a drug is approved by the Food and Drug Administration (FDA—the U.S. government agency responsible for ensuring that drugs marketed in the United States are safe and effective), it is given a
Generic (official) name
Brand (proprietary or trademark or trade) name
For example, phenytoin is the generic name and Dilantin is a brand name for the same drug, which is a commonly used antiseizure drug.
The generic name is assigned, in the United States, by an official body—the United States Adopted Names (USAN) Council.
The brand name is developed by the company requesting approval for the drug and identifies it as the exclusive property of that company.
When a drug is under patent protection, the company markets it under its brand name. When the drug is off-patent (no longer protected by patent), the company may market its product under either the generic name or brand name. Other companies that file for approval to market the off-patent drug must use the same generic name but can create their own brand name. As a result, the same generic drug may be sold under either the generic name (for example, ibuprofen ) or one of many brand names (such as Advil or Motrin).
Generic and brand names must be unique to prevent one drug from being mistaken for another when drugs are prescribed and prescriptions are dispensed. To prevent this possible confusion, the FDA must agree to every proposed brand name.
Government officials, doctors, researchers, and others who write about the new compound use the drug’s generic name because it refers to the drug itself, not to a particular company’s brand of the drug or a specific product. However, doctors often use the brand name on prescriptions, because it is easier to remember and doctors usually learn about new drugs by the brand name.
Generic names are usually more complicated and harder to remember than brand names. Many generic names are a shorthand version of the drug’s chemical name, structure, or formula. In contrast, brand names are usually catchy, often related to the drug’s intended use, and relatively easy to remember, so that doctors will prescribe the drug and consumers will look for it by name. Brand names often suggest a characteristic of the drug. For example, Lopressor lowers blood pressure, Glucotrol controls high blood sugar (glucose) levels, and Skelaxin relaxes skeletal muscles. Sometimes, the brand name is simply a shortened version of the drug’s generic name—for example, Minocin for minocycline .
The term generic, when applied to such items as foods and household products, is used to describe a less expensive, sometimes less effective or lower-quality copycat version of a brand-name product. However, most generic drugs, although less expensive than the comparable brand-name drug, are as effective and of the same quality as the brand-name drug (see Bioequivalence and Interchangeability of Generic Drugs). In fact, generic drug makers manufacture many brand-name products for companies that control the brand names. Sometimes, more than one generic version of a drug is available. For example, many manufacturers sell versions of acetaminophen , a nonprescription drug commonly used to relieve pain and fever.
There’s a method to the madness of naming drugs atorvastatin and fluoxetine
Stephen Chernin/Getty Images
Asvasiran. Bremabecestat. Gedatolisib. Lulumab pegol. Nexbolizumab. Uprosertib. Orilotimod. Vepoloxamer.
What are they? Aliens? Goblins? Diseases? A spelling bee contestant’s nightmare?
Nope. They’re names for drugs.
Not brand names, though. They’re the names for the active ingredients.
You may have noticed that every brand-name drug has a second name — for instance, Prozac® (fluoxetine). That second name, fluoxetine, is a name for the active ingredient, which is the same whatever the brand or generic form.
And believe it or not, these syllable-heavy second names are actually convenient nicknames. It’s much easier to say fluoxetine than to say (RS)-N-methyl-3-phenyl-3-[4-(trifluoromethyl)phenoxy]propan-1-amine.
When a drug company researches and patents a drug, it gets to suggest what the generic name should be. It makes an application to the United States Adopted Names Council (other countries have similar councils and they generally try to cooperate). And that is what those alien-looking words are that I started with: generic names that have been applied for and are under consideration.
So can these drugs be named just any old thing? Is there anything preventing a company from calling its active ingredient supercurol? Well, yes. The U.S. Adopted Names Council. It has some rules, including the following:
- “Prefixes that imply ‘better,’ ‘newer,’ or ‘more effective;’ prefixes that evoke the name of the sponsor, dosage form, duration of action or rate of drug release should not be used.”
- “Prefixes that refer to an anatomical connotation or medical condition are not acceptable.”
- Certain letters or sets of letters also aren’t allowed at the beginning of new generic names. These include me, str, x, and z.
Every name has two main parts. The back half of the drug name is the same for all drugs in a particular class — for instance, there are a whole raft of cholesterol-lowering drugs that end in -vastatin: atorvastatin (Lipitor), fluvastatin (Lescol), rosuvastatin (Crestor), simvastatin (Zocor), and several others. Some other class suffixes include:
- -oxetine for a class of antidepressants, such as fluoxetine (Prozac)
- -sartan for a class of blood-pressure-lowering drugs, such as losartan (Cozaar)
- -afil for a class of drugs used for erectile dysfunction, such as sildenafil (Viagra)
- -lukast for a class of anti-asthma drugs, such as montelukast (Singulair)
- -azepam for a class of anti-anxiety medications, such as diazepam (Valium)
- -coxib for a class of anti-inflammatory pain relievers, such as celecoxib (Celebrex)
- -dronate for a class of drugs that prevent calcium loss, such as alendronate (Fosamax)
- -formin for one class of diabetes drugs, such as metformin (Glucophage), and -glitazone for another class, such as rosiglitazone (Avandia)
- -prazole for a class of stomach acid reducers, such as esomeprazole (Prilosec)
- -conazole for a class of anti-fungals
- -vir for antivirals, with a number of subclasses, including -amivir for a class that includes the anti-flu drug zanamivir (Relenza), -ciclovir for a class that treats herpes (such as famciclovir (Famvir)), and -navir for antiretrovirals for HIV treatment, such as indinavir (Crixivan)
- -stat for enzyme inhibitors, with a whole bunch of subclasses — for instance, –becestat means it’s a beta secretase inhibitor (doesn’t that help you? It would if you were a pharmacist)
So where do these suffixes come from? They used to often be based on the full chemical name, but now they’re sometimes based on particular descriptive terms: -mab for monoclonal antibodies, -sertib for serine/threonine kinase inhibitors, -mer for polymers, or -imod for immunomodulators. And sometimes they’re just made up. If you’re the first company to come up with a drug in that class, you may get to set the pattern that all the others who come after have to follow.
As to the prefix (what comes before the suffix to identify the individual drug), you can make it pretty much whatever you want — subject to the USANC’s rules and approval. And then it’s just up to the clinical trials to show it’s effective… and to doctors, patients, and marketing departments to make it successful. Or not. But if it is, you can bet everyone will call it by the brand name anyway.
You’ve seen the TV ads — the latest drugs touting treatment (along with what seems a never-ending, speedy narration of possible side effects).
And, of course, with each new drug comes a new name.
Who comes up with these strange drug names?
Actually, there is a method to the madness, even though the names of many drugs seem to have come right out of a science-fiction movie (see the quiz below).
For instance, Lunesta (whose generic name is even stranger — Eszopiclone) is used to treat insomnia. It makes sense that the name evokes the moon, which in turn makes you think of night and sleep.
Many drug names are derived from the name of their generic equivalent, chemical composition or what they’re designed to treat. For example, -vir at the end of a drug name such as acyclovir means that the drug is designed to treat viruses.
Every drug has three names: a brand name, a generic name, and a long chemical name. Drug companies submit proposed names to the Adopted Names Council, a group with representation from the American Medical Association, United States Pharmacopeia and the American Pharmacists Association, along with representation from the U.S. Food and Drug Administration and an at-large member.
Drug name criteria: Short and to the point
According to the USAN, a drug name should have no more than four syllables and meet three suitability criteria:
- Safety for use in routine prescribing, ordering, dispensing and administering drugs in the U.S.;
- Use in educational programs for medically oriented professions and for scientific and lay publications;
- Use internationally in drug identification, the exchange of information and translation into different languages.
Is it a drug or from ‘Star Wars,’ ‘Star Trek’
Because the names of these drugs are sometimes so strange, and because we could all use a little fun, here is a short quiz. Choose whether the drug name is an actual drug name or the name of an alien planet, race or thing. See how you did below.
1. Real drug, used to treat psoriasis. (Not to be confused with Maltz, a Klingon from “Star Trek III: The Search for Spock,” played by John Larroquette.)
2. Alien race of pale aliens with cute antennae from “Star Trek.”
3. Real drug, used to treat bipolar disorder. (It just sounds like it should be the name of an alien world).
4. Real drug, used to treat symptoms of diabetes. (You may remember the catchy jingle, to the tune of the ’70s Pilot song “Magic.”)
5. Real drug, used to treat skin tumors (not to be confused with Alderaan, Princess Leia’s home planet in “Star Wars”).
6. Alien race from “Star Trek: Voyager,” known for their short lifespans.
7. Real drug, used to treat macular degeneration and other disorders.
8. Alien race from “Star Trek,” known for their computer skills.
9. Although it sounds like an alien planet, it’s a real drug, used to treat pneumococcal pneumonia.
10. Alien moon from “Star Wars. Home of the famous Ewoks.” (OK, this was an easy one.)
Quite Involved in Discussions
My company has been acquired by another company but the new owner is still retaining the name of our existing company except there is a change in ownership to the new owner.
Therefore, with regards to establishment registration, how do I communicate this to FDA ? Could I retain the same owner / operator number ? Is there anything beyond just notifying to FDA ?
Re: How to notify FDA of change of ownership ?
Go to the DLRM/FURLS site (https://www.access.fda.gov/oaa/), log in to open your Registration page, and change your operating and ownership page as needed.
Quite Involved in Discussions
Re: How to notify FDA of change of ownership ?
I have been though that but didnt see anything on the ownership. Like the name, DUNS and everything would remain the same but under a new management (from the acquired company).
How do I notify FDA that the current management is replaced with a new management.
Sorry for not being clear earlier.
Thank you very much (could I say TYVM ?)
Re: How to notify FDA of change of ownership ?
The second choice from the top at the DRLM Main Menu page allows you to see your facility information (which I understand is not changing), your owner/operator information (which I understand you want to change), and your official correspondent information.
Facility information is changed, if you want, via the third choice in that menu.
Ownership information is changed, as I think you want in this instance, via the seventh choice in that menu.
Note that, if your DRLM account has a primary registrant and one or more secondary registrants, the facility and ownership information can only be changed by the primary registrant.