De er i US igang med at forslå en lidt lempeligere godkendelse procedure, måske især for de allerede godkendte stoffer. Og her tænker vi jo på i Genmabs tilfældese: udvidelse af indikationer
Senator Hagan: har fremlagt følgende:
S. 2113. A bill to empower the Food and Drug Administration to ensure a clear and effective pathway that will encourage innovative products to benefit patients and improve public health; to the Committee on Health, Education, Labor, and Pensions.
Mr. President, today I am proud to introduce the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act.
This bill empowers the Food and Drug Administration to ensure consistent processes and a clear and effective pathway that will encourage the development of innovative treatments to benefit patients, particularly subpopulations and those with rare diseases, and improve the public health.
Without question, the FDA plays a critical role in helping to ensure that new medicines are safe and effective. At the same time, by promoting investment in and development of innovative treatments for unmet medical needs, the FDA can positively influence our national strategy to identify and treat serious and life-threatening diseases and improve the quality of life for millions of Americans.
In order for FDA to accomplish this goal, however, Congress needs to give the agency the tools necessary to transcend existing barriers, reform its processes, and provide greater clarity, consistency, and transparency to industry.
The bill accomplishes this in three ways.
First, it provides the FDA with the authorities and tools that are reflective of the agency's responsibilities and that are necessary to ensure maximum operational excellence by updating FDA's mission statement and creating a management review board.
Second, it advances regulatory science and innovation within FDA to ensure that evaluations of innovative treatments, therapies, and diagnostics are conducted by those who have the best available knowledge. To do this, the bill creates a chief innovation officer and chief medical policy officers, and expands participation on advisory committees by those experts most familiar with the disease being considered.
Finally, the bill promotes the utilization of modern scientific tools and methodologies to ensure patients have timely access to innovative products by creating a clinical informatics coordinator, providing more information to drug sponsors when an application has not been approved, and enhancing and codifying the accelerated approval process.
In the nearly 2 decades since the accelerated approval mechanism was established by FDA to more expeditiously approve treatments, advances in medical sciences, including genomics, molecular biology, and bioinformatics, have provided scientists with an unprecedented understanding of the underlying biological mechanisms and pathogenesis of disease.
A new generation of modern, targeted, personalized medicines is currently under development to treat serious and life-threatening diseases. Some apply drug development strategies based on biomarkers or pharmacogenomics, predictive toxicology, clinical trial enrichment techniques, and novel clinical trial designs, such as adaptive clinical trials that can be altered based on observed patient outcomes in the interim.
In order to ensure these scientific advances are translated into treatments that benefit patients, Congress should allow FDA to implement a more effective process for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions.
FDA is already doing this, to some extent. However, application of the accelerated approval process has been somewhat limited, largely to HIV and oncology drugs, and inconsistently applied to other disease targets. For example, a 2011 report by the National Organization for Rare Disorders compared the approval process for 135 non-cancer orphan therapies approved by FDA from 1983 through June 2010. The report found that 45 went through the conventional approval process; 32 were approved with some sort of administrative flexibility; and 58 were approved on a case-by-case flexibility process. This report illustrates that while FDA does have the authority to approve these treatments with some flexibility, there does not appear to be uniformity or consistency in employing this flexibility.
The TREAT Act allows FDA to tap into modern scientific advances by using a broad range of surrogate or clinical endpoints and modern scientific tools earlier in the drug development cycle, when appropriate, to approve treaments for patients. Employing these modern scientific tools may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation without compromising or altering FDA's existing high standards for the approval of drugs.
It is the patients suffering from these serious and life-threatening diseases that benefit from expedited access to safe and effective innovative therapies. For the 30 million Americans living with rare diseases, new advances in science and medicine cannot come fast enough. That is why I am proud that this bill has the support of the National Organization for Rare Disorders (NORD) and Friends of Cancer Research. The TREAT Act provides FDA with the tools needed to modernize its processes and encourage the development of innovative products to benefit patients, particularly subpopulations and those with rare diseases.
I urge my other colleagues to join us in supporting this important bill.
-----------
Hun bliver støttet af National organisation for rare disorder (Nord):
NORD supports this proposed legislation which, among other things:
enhances and codifies the accelerated approval process
addresses concerns of the rare disease community related to conflict of interest provisions provides greater clarity, consistency, and transparency in review processes encourages innovation and adoption of modern scientific tools in regulatory science
----------------------
Her et uddrag fra Medmeme, som specifikt nævner at det kan få betydning for Arzerra:
Medmeme CEO Mahesh Naithani describes challenges: Its estimated that by 2015 the global market in hematologic cancer drugs could reach $ 14 billion. However, the extremely high cost of research and development in this category is a great challenge to pharmaceutical companies developing therapies. It is a process with a success rate of only 5-10 percent because too often late-stage clinical trials are characterized by adverse side effects, small effects on survival, and/or low response rates that lead to drugs being abandoned at a higher rate compared with drugs in other therapeutic areas. Tufts Center for the Study of Drug Development estimates a typical drug development cost at $ 1.3 billion, and needing 10-15 years to bring it to market. But the development of oncologic drugs has a tendency to amass higher costs because of these high failure rates. Hopefully, the TREAT Act currently proposed in the U.S. Congress could accelerate a review and approval process for drugs that are highly targeted therapies for serious or life-threatening diseases or conditions. That could help companies trying to tackle the hematologic cancers challenge.
Examples of firms Medmeme reports on which have undertaken the oncologic drug development challenges are: Celgene, Takeda/Millennium, Novartis, Merck, Bristol-Myers Squibb, Roche, Pfizer, GlaxoSmithKline, Sanofi, and Abbott, among others such as Pharmacyclics, Onyx Therapeutics, Keryx Biopharmaceuticals, Ambit Biosciences, Clavis Pharma, and Amgen. Celgene is a dominant company in terms of sales, and its Revlimid is the dominant drug. Celgene reports Revlimids worldwide sales for 2011 were $ 3.2 billion, a 30 percent increase over 2010. Revlimid will dominate the MM market far into the future, Medmeme predicts, and has also shown efficacy against certain subtypes of NHL and AML.
Drug re-profiling, which is the application to FDA for the use of approved drugs to treat new indications, is also discussed in the report. It can open new options for treatment and has become increasingly popular within the hematologic cancer category. For example, in addition to Celgenes Revlimid and Takeda/Millenniums Velcade, the following drugs in this process on Medmemes watch list are: Mercks Zolinza, Bristol-Myers Squibbs Erbitux, GlaxoSmithKlines Arzerra, and Teva Pharmaceuticals Treanda.
----------------------------
Her en diskussion af om det gør Biotek virksomheder mere uafhængige(which we like) af Big Pharma
http://www.newsobserver.com/2012/03/15/1931952/hagan-bill-could-speed-approval.html
Senator Hagan: har fremlagt følgende:
S. 2113. A bill to empower the Food and Drug Administration to ensure a clear and effective pathway that will encourage innovative products to benefit patients and improve public health; to the Committee on Health, Education, Labor, and Pensions.
Mr. President, today I am proud to introduce the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act.
This bill empowers the Food and Drug Administration to ensure consistent processes and a clear and effective pathway that will encourage the development of innovative treatments to benefit patients, particularly subpopulations and those with rare diseases, and improve the public health.
Without question, the FDA plays a critical role in helping to ensure that new medicines are safe and effective. At the same time, by promoting investment in and development of innovative treatments for unmet medical needs, the FDA can positively influence our national strategy to identify and treat serious and life-threatening diseases and improve the quality of life for millions of Americans.
In order for FDA to accomplish this goal, however, Congress needs to give the agency the tools necessary to transcend existing barriers, reform its processes, and provide greater clarity, consistency, and transparency to industry.
The bill accomplishes this in three ways.
First, it provides the FDA with the authorities and tools that are reflective of the agency's responsibilities and that are necessary to ensure maximum operational excellence by updating FDA's mission statement and creating a management review board.
Second, it advances regulatory science and innovation within FDA to ensure that evaluations of innovative treatments, therapies, and diagnostics are conducted by those who have the best available knowledge. To do this, the bill creates a chief innovation officer and chief medical policy officers, and expands participation on advisory committees by those experts most familiar with the disease being considered.
Finally, the bill promotes the utilization of modern scientific tools and methodologies to ensure patients have timely access to innovative products by creating a clinical informatics coordinator, providing more information to drug sponsors when an application has not been approved, and enhancing and codifying the accelerated approval process.
In the nearly 2 decades since the accelerated approval mechanism was established by FDA to more expeditiously approve treatments, advances in medical sciences, including genomics, molecular biology, and bioinformatics, have provided scientists with an unprecedented understanding of the underlying biological mechanisms and pathogenesis of disease.
A new generation of modern, targeted, personalized medicines is currently under development to treat serious and life-threatening diseases. Some apply drug development strategies based on biomarkers or pharmacogenomics, predictive toxicology, clinical trial enrichment techniques, and novel clinical trial designs, such as adaptive clinical trials that can be altered based on observed patient outcomes in the interim.
In order to ensure these scientific advances are translated into treatments that benefit patients, Congress should allow FDA to implement a more effective process for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions.
FDA is already doing this, to some extent. However, application of the accelerated approval process has been somewhat limited, largely to HIV and oncology drugs, and inconsistently applied to other disease targets. For example, a 2011 report by the National Organization for Rare Disorders compared the approval process for 135 non-cancer orphan therapies approved by FDA from 1983 through June 2010. The report found that 45 went through the conventional approval process; 32 were approved with some sort of administrative flexibility; and 58 were approved on a case-by-case flexibility process. This report illustrates that while FDA does have the authority to approve these treatments with some flexibility, there does not appear to be uniformity or consistency in employing this flexibility.
The TREAT Act allows FDA to tap into modern scientific advances by using a broad range of surrogate or clinical endpoints and modern scientific tools earlier in the drug development cycle, when appropriate, to approve treaments for patients. Employing these modern scientific tools may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation without compromising or altering FDA's existing high standards for the approval of drugs.
It is the patients suffering from these serious and life-threatening diseases that benefit from expedited access to safe and effective innovative therapies. For the 30 million Americans living with rare diseases, new advances in science and medicine cannot come fast enough. That is why I am proud that this bill has the support of the National Organization for Rare Disorders (NORD) and Friends of Cancer Research. The TREAT Act provides FDA with the tools needed to modernize its processes and encourage the development of innovative products to benefit patients, particularly subpopulations and those with rare diseases.
I urge my other colleagues to join us in supporting this important bill.
-----------
Hun bliver støttet af National organisation for rare disorder (Nord):
NORD supports this proposed legislation which, among other things:
enhances and codifies the accelerated approval process
addresses concerns of the rare disease community related to conflict of interest provisions provides greater clarity, consistency, and transparency in review processes encourages innovation and adoption of modern scientific tools in regulatory science
----------------------
Her et uddrag fra Medmeme, som specifikt nævner at det kan få betydning for Arzerra:
Medmeme CEO Mahesh Naithani describes challenges: Its estimated that by 2015 the global market in hematologic cancer drugs could reach $ 14 billion. However, the extremely high cost of research and development in this category is a great challenge to pharmaceutical companies developing therapies. It is a process with a success rate of only 5-10 percent because too often late-stage clinical trials are characterized by adverse side effects, small effects on survival, and/or low response rates that lead to drugs being abandoned at a higher rate compared with drugs in other therapeutic areas. Tufts Center for the Study of Drug Development estimates a typical drug development cost at $ 1.3 billion, and needing 10-15 years to bring it to market. But the development of oncologic drugs has a tendency to amass higher costs because of these high failure rates. Hopefully, the TREAT Act currently proposed in the U.S. Congress could accelerate a review and approval process for drugs that are highly targeted therapies for serious or life-threatening diseases or conditions. That could help companies trying to tackle the hematologic cancers challenge.
Examples of firms Medmeme reports on which have undertaken the oncologic drug development challenges are: Celgene, Takeda/Millennium, Novartis, Merck, Bristol-Myers Squibb, Roche, Pfizer, GlaxoSmithKline, Sanofi, and Abbott, among others such as Pharmacyclics, Onyx Therapeutics, Keryx Biopharmaceuticals, Ambit Biosciences, Clavis Pharma, and Amgen. Celgene is a dominant company in terms of sales, and its Revlimid is the dominant drug. Celgene reports Revlimids worldwide sales for 2011 were $ 3.2 billion, a 30 percent increase over 2010. Revlimid will dominate the MM market far into the future, Medmeme predicts, and has also shown efficacy against certain subtypes of NHL and AML.
Drug re-profiling, which is the application to FDA for the use of approved drugs to treat new indications, is also discussed in the report. It can open new options for treatment and has become increasingly popular within the hematologic cancer category. For example, in addition to Celgenes Revlimid and Takeda/Millenniums Velcade, the following drugs in this process on Medmemes watch list are: Mercks Zolinza, Bristol-Myers Squibbs Erbitux, GlaxoSmithKlines Arzerra, and Teva Pharmaceuticals Treanda.
----------------------------
Her en diskussion af om det gør Biotek virksomheder mere uafhængige(which we like) af Big Pharma
http://www.newsobserver.com/2012/03/15/1931952/hagan-bill-could-speed-approval.html
