Hvis nu et medicinalfirma opfinder en ny og revolutionerende medicin - hvordan er forløbet så egentlig helt fra start, til påbegyndt komercielt salg ?
Er der mon en venlig sjæl der groft kan ridse forløbet op ?
Fx. Hvad ligger der egentlig i begreberne fase 1 2 & 3 ?
Og hvordan kunne en grov gennemsnitlig tidslinie evt. se ud ?
Jeg er klar over at det nok er et meget omfattende spørgsmål - men jeg er fint tilfreds med en ultra kort og firkantet opridsning af forløbet. :)
På forhånd tak
Er der mon en venlig sjæl der groft kan ridse forløbet op ?
Fx. Hvad ligger der egentlig i begreberne fase 1 2 & 3 ?
Og hvordan kunne en grov gennemsnitlig tidslinie evt. se ud ?
Jeg er klar over at det nok er et meget omfattende spørgsmål - men jeg er fint tilfreds med en ultra kort og firkantet opridsning af forløbet. :)
På forhånd tak
21/4 2010 11:34 1
28382 Det helt utroligt kolde hurtige svar er:
Fase 1: Dosering
Fase 2: effekt
Fase 3: Pålidelighed
Phase 1 trial
A Phase 1 trial is the first step in testing a new treatment in humans. These studies test the best way to give a new treatment (for example, by mouth, intravenous infusion, or injection) and the best dose. The dose is usually increased a little at a time in order to find the highest dose that does not cause harmful side effects. Because little is known about the possible risks and benefits of the treatments being tested, Phase I trials usually include only a small number of study volunteers.
Phase 2 trial
In cancer studies, a Phase 2 trial is a study to test whether a new treatment has an anticancer effect (for example, whether it shrinks a tumor or improves blood test results) and whether it works against a certain type of cancer.
Phase 3 trial
A Phase 3 study compares the results of people taking a new treatment with the results of people taking the standard treatment (for example, which group has better survival rates or fewer side effects). Phase 3 trials may include hundreds of people and are designed to prove the safety and efficacy of a new treatment.
Fase 1: Dosering
Fase 2: effekt
Fase 3: Pålidelighed
Phase 1 trial
A Phase 1 trial is the first step in testing a new treatment in humans. These studies test the best way to give a new treatment (for example, by mouth, intravenous infusion, or injection) and the best dose. The dose is usually increased a little at a time in order to find the highest dose that does not cause harmful side effects. Because little is known about the possible risks and benefits of the treatments being tested, Phase I trials usually include only a small number of study volunteers.
Phase 2 trial
In cancer studies, a Phase 2 trial is a study to test whether a new treatment has an anticancer effect (for example, whether it shrinks a tumor or improves blood test results) and whether it works against a certain type of cancer.
Phase 3 trial
A Phase 3 study compares the results of people taking a new treatment with the results of people taking the standard treatment (for example, which group has better survival rates or fewer side effects). Phase 3 trials may include hundreds of people and are designed to prove the safety and efficacy of a new treatment.
Fandt en længere smøre, hvor der også er en fase 4.
Phase I
Early clinical stage Phase I studies are designed to examine the safety of a new medication and understand how it will work in humans by gathering extensive data on how it is absorbed, distributed, metabolized and eliminated from the human body; other data include assessment of how quickly therapeutic concentrations is achieved, how long the drug remains in the body, and what, if any, the effect drug metabolite by-products may have; with step-by-step increases in dose, the optimal dosage is determined where minimum side effects are coupled with maximum therapeutic effect, termed the toxic-therapeutic window; a trial to determine the best way to give a new treatment and what doses can be safely given; phase 1's involve 20-80 subjects and generate data on toxicity and maximum safe dose, to later allow a properly controlled trial; FDA's review at this point ensures that subjects are not exposed to unreasonable risks; phase I studies generally enroll only healthy persons to evaluate how a new drug behaves in humans, but may enroll Pts with the disease that the new drug seeks to treat; candidates are enrolled in a study only after a review of their Hx and physical confirms eligibility and informed consent for treatment is given; the number of Pts enrolled in a phase I trial will vary depending on the step-wise progression established for achieving optimal dosing as well as prior clinical experience with similar compounds and approaches; followup time periods may range from just a few days to 6+ months; further trials may continue only if phase 1 results indicate that the new therapy is reasonably safe in humans, and the FDA approves further work; the ultimate goal of phase 1 trials is to obtain sufficient information about the drug's pharmacokinetics and pharmacological effects to permit the design of well-controlled, sufficiently valid phase 2 studies; other examples of phase 1 studies include studies of drug metabolism, structure-activity relationships, and mechanisms of actions in humans, as well as studies in which investigational drugs are used as research tools to explore biological phenomena or disease processes Glossaries, nih.gov-6/99
Phase 2
Later clinical stage Phase 2 studies are designed to evaluate the short-term therapeutic effect of a new drug in Pts who suffer from the target disease, and confirm the safety established in phase I trials; phase 2 studies are sometimes placebo-controlled, often double-blinded, enroll a larger number of Pts than in phase 1 and Pt followup may be for longer periods; phase 2 studies are tailored to specific treatment indications for which the company plans to seek broader approval; where compelling scientific evidence is presented, the FDA expedites review of a company's application for market clearance; expedited review of phase 2 clinical data, and clearance of that early application, can obviate requirements for phase 3 trials; phase 2's involve several hundred Pts and generate enough data to 1. at least suggest?if not prove that the drug actually works?efficacy and 2. demonstrate the most common side effects Glossaries, nih.gov-6/99; phase 2 trials are sometimes divided into phase 2a pilot trials and phase 2b well-controlled trials
Phase 3
Final clinical stage Phase 3 trials are designed to demonstrate the potential advantages of the new therapy over other therapies already on the market; safety and efficacy of the new therapy are studied over a longer period of time and in many more Pts enrolled into the study with less restrictive eligibility criteria; phase 3 studies are intended to help scientists identify rarer side effects of treatment and prepare for a broader application of the product; phase 3 trials enroll 1,000-3,000 Pts to verify efficacy and monitor adverse reactions during longer-term use?sometimes divided into Phase 3a trials, conducted before regulatory submission and Phase 3b trials, conducted after regulatory submission, but before approval
Phase 4
Post-FDA approval/post-marketing Phase 4 studies involve many thousands of Pts and compare its efficacy with a gold standard; some agents have been withdrawn from the market because they increase the mortality rate in treated Pts; concurrent with marketing approval, FDA may seek agreement from the sponsor to conduct certain postmarketing?phase 4 studies to delineate additional information about the drug's risks, benefits, and optimal use, and could include, but would not be limited to, studying different doses or schedules of administration than were used in phase 2 studies, use of the drug in other Pt populations or other stages of the disease, or use of the drug over a longer period of time 21 CFR §312.85
Note: Occasionally, an agent's benefit is so obvious, eg zidovudine?AZT that the need for phase 3 studies, a stage immediately preceding an official NDA, may be obviated; NDA?new drug applications were often rejected because the data from the phase 2 and 3 trials revealed study design flaws, forcing the sponsor to repeat work, which has been largely eliminated by the '1987 rewrite' of the IND status
Phase I
Early clinical stage Phase I studies are designed to examine the safety of a new medication and understand how it will work in humans by gathering extensive data on how it is absorbed, distributed, metabolized and eliminated from the human body; other data include assessment of how quickly therapeutic concentrations is achieved, how long the drug remains in the body, and what, if any, the effect drug metabolite by-products may have; with step-by-step increases in dose, the optimal dosage is determined where minimum side effects are coupled with maximum therapeutic effect, termed the toxic-therapeutic window; a trial to determine the best way to give a new treatment and what doses can be safely given; phase 1's involve 20-80 subjects and generate data on toxicity and maximum safe dose, to later allow a properly controlled trial; FDA's review at this point ensures that subjects are not exposed to unreasonable risks; phase I studies generally enroll only healthy persons to evaluate how a new drug behaves in humans, but may enroll Pts with the disease that the new drug seeks to treat; candidates are enrolled in a study only after a review of their Hx and physical confirms eligibility and informed consent for treatment is given; the number of Pts enrolled in a phase I trial will vary depending on the step-wise progression established for achieving optimal dosing as well as prior clinical experience with similar compounds and approaches; followup time periods may range from just a few days to 6+ months; further trials may continue only if phase 1 results indicate that the new therapy is reasonably safe in humans, and the FDA approves further work; the ultimate goal of phase 1 trials is to obtain sufficient information about the drug's pharmacokinetics and pharmacological effects to permit the design of well-controlled, sufficiently valid phase 2 studies; other examples of phase 1 studies include studies of drug metabolism, structure-activity relationships, and mechanisms of actions in humans, as well as studies in which investigational drugs are used as research tools to explore biological phenomena or disease processes Glossaries, nih.gov-6/99
Phase 2
Later clinical stage Phase 2 studies are designed to evaluate the short-term therapeutic effect of a new drug in Pts who suffer from the target disease, and confirm the safety established in phase I trials; phase 2 studies are sometimes placebo-controlled, often double-blinded, enroll a larger number of Pts than in phase 1 and Pt followup may be for longer periods; phase 2 studies are tailored to specific treatment indications for which the company plans to seek broader approval; where compelling scientific evidence is presented, the FDA expedites review of a company's application for market clearance; expedited review of phase 2 clinical data, and clearance of that early application, can obviate requirements for phase 3 trials; phase 2's involve several hundred Pts and generate enough data to 1. at least suggest?if not prove that the drug actually works?efficacy and 2. demonstrate the most common side effects Glossaries, nih.gov-6/99; phase 2 trials are sometimes divided into phase 2a pilot trials and phase 2b well-controlled trials
Phase 3
Final clinical stage Phase 3 trials are designed to demonstrate the potential advantages of the new therapy over other therapies already on the market; safety and efficacy of the new therapy are studied over a longer period of time and in many more Pts enrolled into the study with less restrictive eligibility criteria; phase 3 studies are intended to help scientists identify rarer side effects of treatment and prepare for a broader application of the product; phase 3 trials enroll 1,000-3,000 Pts to verify efficacy and monitor adverse reactions during longer-term use?sometimes divided into Phase 3a trials, conducted before regulatory submission and Phase 3b trials, conducted after regulatory submission, but before approval
Phase 4
Post-FDA approval/post-marketing Phase 4 studies involve many thousands of Pts and compare its efficacy with a gold standard; some agents have been withdrawn from the market because they increase the mortality rate in treated Pts; concurrent with marketing approval, FDA may seek agreement from the sponsor to conduct certain postmarketing?phase 4 studies to delineate additional information about the drug's risks, benefits, and optimal use, and could include, but would not be limited to, studying different doses or schedules of administration than were used in phase 2 studies, use of the drug in other Pt populations or other stages of the disease, or use of the drug over a longer period of time 21 CFR §312.85
Note: Occasionally, an agent's benefit is so obvious, eg zidovudine?AZT that the need for phase 3 studies, a stage immediately preceding an official NDA, may be obviated; NDA?new drug applications were often rejected because the data from the phase 2 and 3 trials revealed study design flaws, forcing the sponsor to repeat work, which has been largely eliminated by the '1987 rewrite' of the IND status
21/4 2010 14:04 our 028387 Tusind tak for svar til jer begge. :)
Men jeg formoder at vejen mod endelig godkendelse er markant længere end "blot" fase 1, 2, 3 og 4. :)
Mon ikke man tester på dyr før mennesker - og måske celler før dyr ?
Og hvad kommer efter fase 4, før medicinen rent fysisk står på apotekets hylder ?
Det jeg fisker efter er, om man lidt groft kan anslå, hvor lang tid en ny type medicin ville være undervejs, helt fra start til slut.
Jeg formoder at forløbet tager flere år ?
Men jeg formoder at vejen mod endelig godkendelse er markant længere end "blot" fase 1, 2, 3 og 4. :)
Mon ikke man tester på dyr før mennesker - og måske celler før dyr ?
Og hvad kommer efter fase 4, før medicinen rent fysisk står på apotekets hylder ?
Det jeg fisker efter er, om man lidt groft kan anslå, hvor lang tid en ny type medicin ville være undervejs, helt fra start til slut.
Jeg formoder at forløbet tager flere år ?
21/4 2010 20:57 kosmokrator 328399 fase 4 er post marketing studier. Det vil sige medikament er på markedet og der laves opfølgende undersøgelser.
Før phase I ligger der prekliniske studier, eller som det nu så smukt hedder non-kliniske. Det primære der er toksikologiske studier på dyr. Alt afh. af drug , hvor tit det skal gives og dosis, varierer disse noget i design. Her gives der en ordentlig gang drug, mange gange mere end man ngensinde vil give patienter. Toks er dog ikke overstået ved indgang til phase I, dyrene følges og andre studier laves. Specielt studier der skal søge at fastslå om et drug er kræftfremkaldende er langvarige, og kan trække sport heni phase 2, 3 og også til NDAen. Bare sprøg Novo, de dykkede hårdt i 2002 på problemer med toks data for et fase 2 drug og havde også lidt slåskampe med Victoza.
Endnu før dette vil man gerne se en effekt af drug i en dyremodel, dels for firmaets egen skyld så de ikke brænder penge af på løse ideer, men også for at retfædiggøre at drug kunne være aktivt spå det er rimeligt at udsætte patienter for den risko det er at prøve et nyt drug.
Endnu før dyrestudier for effekt (såkaldt animal proof of concept (PoC)) laves der stuider på celler eller isolerede receptorer o.lign.
Timelines er svære og stigende men 8-10 år fra ide til drug er absolut i den lave ende.
Før phase I ligger der prekliniske studier, eller som det nu så smukt hedder non-kliniske. Det primære der er toksikologiske studier på dyr. Alt afh. af drug , hvor tit det skal gives og dosis, varierer disse noget i design. Her gives der en ordentlig gang drug, mange gange mere end man ngensinde vil give patienter. Toks er dog ikke overstået ved indgang til phase I, dyrene følges og andre studier laves. Specielt studier der skal søge at fastslå om et drug er kræftfremkaldende er langvarige, og kan trække sport heni phase 2, 3 og også til NDAen. Bare sprøg Novo, de dykkede hårdt i 2002 på problemer med toks data for et fase 2 drug og havde også lidt slåskampe med Victoza.
Endnu før dette vil man gerne se en effekt af drug i en dyremodel, dels for firmaets egen skyld så de ikke brænder penge af på løse ideer, men også for at retfædiggøre at drug kunne være aktivt spå det er rimeligt at udsætte patienter for den risko det er at prøve et nyt drug.
Endnu før dyrestudier for effekt (såkaldt animal proof of concept (PoC)) laves der stuider på celler eller isolerede receptorer o.lign.
Timelines er svære og stigende men 8-10 år fra ide til drug er absolut i den lave ende.
