Gottlieb Targets Drug Development Costs, Clinical Development Efficiencies

Posted 11 September 2017 By Zachary Brennan

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FDA commissioner Scott Gottlieb on Monday explained to attendees of RAPS’ Regulatory Convergence conference some steps FDA is taking to make the clinical end of drug development more efficient and effective.

Opening with a discussion of the ways in which the gap of time between the discovery of the science behind new treatments and the adoption of such treatments has been shrinking, Gottlieb outlined a few of the ways in which the agency is modernizing its approach to collecting and evaluating clinical information.

And on a day when the discussion of how much it costs to develop a new oncology drug is being hotly debated with the release of a new study, Gottlieb also discussed how the costs of drug development “are also high, and growing.

“There’s been criticism of the various estimates of how much it costs to develop a new drug,” he said, according to the transcript of his speech. “Moreover, on a relative basis, in many cases the costs of early stage drug development has grown at a proportionally faster rate than the cost of late stage drug development. In other words, inflation in early stage drug trials is rising faster than inflation in late stage development.

“By front-loading the cost of drug discovery, the broader biomedical community is making it harder to advance new ideas. It’s economically harder to capitalize the cost of an early stage drug program, relative to funding a later stage project. So frontloading the costs are a recipe for reducing the amount of new ideas that can be advanced.”

 

Read More information: http://snip.ly/6ude0#http://www.raps.org/Regulatory-Focus/News/2017/09/11/28442/Gottlieb-Targets-Drug-Development-Costs-Clinical-Development-Efficiencies/

Making sense of the FDA’s GMP and Regulatory Expectations for Early IND Products

The FDA’s recent guidance documents covering GMP requirements for Phase I products have considerably reduced some of the complexities early phase products face. These guidance documents are in addition to those that cover the CMC sections for IND submissions at Phase I.

Although these new guidelines appear to remove the need to follow GMPs for Phase I products; the same still exists in the Food, Drug, and Cosmetic Act. As a result, the need for GMP requirements for Phase I products has not been done away with; rather, it has been altered. Now, the nature and extent of GMP-related activities will depend upon the nature of the investigational drug and the extent of the study that is planned.

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Get trained to understand these aspects

GlobalCompliancePanel, a leading provider of professional trainings for the areas of regulatory compliance, will be organizing a two-day seminar to help Directors, Managers and Supervisors in Regulatory Affairs, Quality Assurance and Quality Control get a grasp of these aspects. To enroll for this highly valuable training, please log on to http://www.globalcompliancepanel.com/control/globalseminars/~product_id=900695SEMINAR?GMP-regulatory-expectations-San-Diego. Steven S. Kuwahara, Founder and Principal, GXP BioTechnology LLC, will be the Director of this webinar.

Advice on the GMP guidance document

This presentation, which has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion, will review the GMP guidance document and discuss how it may be integrated with the recommendations of the guidance documents on CMC requirements. In one source, it will present the regulations and guidelines that apply to early phase products. In some cases these may not be regulations, but needs that, if met, will increase the efficiency of activities as a product proceeds through the development process. Steven will present these items in the order of product development from the point of R & D activities to the completion of Phase 2 clinical trials.

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This course is very useful for any pharmaceutical personnel who must deal with products both in early and later phases of development, as it will make them aware of the regulatory requirements that will affect operations dealing with these products. The modifications to the GMPs for early phase products have altered the GMPs in such a way as to reduce requirements to allow more efficient work. At the same time, some of the things that may appear to have changed, have not, and personnel in the pharmaceutical sector should be aware of this. This is the learning that Steven will emphasize at this seminar.

It will cover the following areas:

  • Very Early Stages
  • GLP requirements
  • Early Pre-IND Studies
  • Meetings and Preparing for the IND
  • GMPs for Phase 1 IND products
  • Requirements for Phase 2 INDs
  • Preparing for IND Meetings

Medical products need to be validated for Radiation Sterilization

Do it right the first time” should be the mantra for medical device manufacturers that plan to make radiation products. Choosing the most functional and radiation resistant materials for their medical device will help them avoid the trouble of going through the post launch cycle of product revisions.

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To get this right, one simple line of thinking is crucial for medical device manufacturers to inculcate: To “think like a molecule”. This is the basis on which to plan and design around radiation induced changes in the qualities such as color, odor and brittleness that go into the materials.

Learning the right method

Professionals in the medical devices industry, who work on these aspects will benefit immensely from a two-day seminar on the topic, “Validating Radiation Sterilization for Medical Products”, which is being organized by GlobalCompliancePanel, a highly popular provider of professional trainings for all areas of regulatory compliance.

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The Director of this seminar, Karl J. Hemmerich, President of Ageless Processing Technologies, who brings over 35 years of experience in medical device product design, development, manufacturing, and sterilization, will offer a range of learning on this topic.

Identifying the best suited materials

Apart from helping participants identifying the materials that perform best upon radiation, keeping color and odor out of their irradiated products and enhancing product and packaging designs to take advantage of radiation; he will also offer understanding to them on how to avoid the materials that are certain to fail.

This seminar, for which medical device professionals can enroll by visiting http://www.globalcompliancepanel.com/control/globalseminars/~product_id=900685SEMINAR?validating-radiation-Washington-DC, has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion.

Understanding optimum sterilization modality

Participants will also learn the basis for choosing the optimum sterilization modality based on materials, product design, bioburden, and logistics and understand which modality (Gamma, E-beam, or X-ray) will perform best for their product.

Karl will cover the areas relating to validation of radiation sterilization for medical products, such as Materials Guidances – AAMI TIR # 17, ASTM, sterilization validation and bioburden, Shelf Life Test Methods – Accelerated Aging design, Test Design, Product Design, the influences of product assembly (molding, automation, etc.), material selection and post irradiation degradation, Regulatory Guidances – AAMI/ISO 11137, TIR #17, packaging design and materials, and biological polymers – tissue, serum at this two-day in person seminar.

The following areas will be covered at this seminar:

  • Polymers Chemistry – choosing the best polymer candidate
  • Gamma, E-beam, X-ray sterilization
  • Accelerated Aging
  • Product Qualification
  • Sterilization Validation – Establishing the Minimum Sterilization dose (VDmax)
  • Sterilization Modality Selection Criteria
  • Biocompatibility
  • Preventing Plastic Part Failure Post Irradiation

Ensuring compliance with healthcare laws is of utmost importance for healthcare providers

Compliance with the many laws, as well as implementation of the necessary compliance initiatives are the means by which healthcare providers and entities or organizations involved in any type of healthcare transaction, especially those who bill or are involved with services payable by a CMS program, play a part in the protection of the integrity of  the CMS programs.

Developing and maintaining the necessary compliance programs that place a special emphasis on auditing and monitoring, appropriate training, receiving and responding to complaints and conducting investigations is necessary for providers, entities and organizations that bill Medicare, Medicaid or other government payor programs. Those whose compliance program fails to incorporate the necessary control processes risk inviting potential audits that could lead to civil, monetary and criminal penalties.

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Understanding risks is necessary for healthcare practices

The ways by which to understand potential risks relating to their practice or organization and responding and mitigating deficiencies are all-important for healthcare organizations if they have to sustain their success and viability. This is also necessary if the organization has to avoid or neutralize the impact of negative findings by an external audit or investigative agency.

Moreover, with the many impactful changes taking place in the healthcare delivery; healthcare provider reimbursement could be affected. Quality of care and reduced costs will bring about changes into provider reimbursement for services. Healthcare providers have to understand how these changes are going to affect them.

Learning ways of implementing compliant programs

A two-day in person seminar by GlobalCompliancePanel, a highly reputable provider of professional trainings for all areas of regulatory compliance, will offer understanding on all these areas of healthcare compliance. This course, at which Gail Madison-Brown, a Registered Nurse and attorney who has spent over 25 years in the healthcare industry, and is Chief Clinical Trials Officer at UTHSCSA will be the Director,  will focus on ways by which healthcare organizations can devise means by which to stay compliant with the regulatory guidelines and laws.

To enroll for this seminar and to get a thorough understanding of how to implement practices that are compliant with regulatory requirements, just visit http://www.globalcompliancepanel.com/control/globalseminars/~product_id=900544SEMINAR?ensure-healthcare-compliance-Seattle-WA.

Ways of building a robust and pragmatic compliance program

Attending this seminar will help participants understand how to build a solid and practical compliance program. Gail will introduce the basic healthcare compliance infrastructure necessary to establish a comprehensive and proactive compliance program. She will then discuss current government auditing agencies and audits that are underway, as well as government agencies responsible for protecting the Medicare Trust Fund from fraud, waste and abuse.

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She will teach participants the ways of identifying risks in their organizations, no matter what their size, by conducting a risk assessment and developing a work plan based upon risk and will help with the mitigation efforts. Apart from OCR audits and how to conduct their own self-assessment in preparation for an audit, as well as ways of addressing any identified deficiencies; participants will also learn how to conduct auditing and monitoring activities and what to do with findings. Ways of putting in place a complaint management system and conducting an investigation will also be discussed. The Director will also offer user friendly templates and tools, as well as numerous case scenarios to the participants, which they can use to enhance current compliance programs.

 

Drug safety and pharmacovigilance are two faces of a coin

If one were to describe the relationship between drug safety and pharmacovigilance; the most concise way of describing it is by calling them as two sides of the same coin. The aspects of drug safety and pharmacovigilance are intricately and inseparably bound together. The whole purpose of pharmacovigilance is the assurance of drug safety. It is precisely to ensure drug safety, more than anything else, that pharmacovigilance has come into being as a discipline.

Pharmacovigilance (PV in drug industry parlance) is a means to ensuring drug surveillance across the entire process, right from procuring of raw materials to consumption and the effects of consumption. So, pharmacovigilance has to be implemented right across the chain of activities that go into drug manufacturing. It has to ensure compliance with regulation at all stages, namely before the product is manufactured, during the manufacture, and after it enters the market. It is an indispensable aspect of a Quality System, and plays a central role in inspections and audits.

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Drug safety and pharmacovigilance are tied together at the level of regulatory bodies. There are different PV rules for different markets. For instance, while the FDA has its own set of PV guidelines; the EU has its own. A clinician or drug company involved in drug safety and pharmacovigilance has to be aware of the regulations in the markets into which the company’s products are sold and has to abide by them.

In order to ensure drug safety, pharmacovigilance is carried out by agencies and governments across the globe in accordance with strict guidelines. Drug safety and pharmacovigilance pair together in a number of areas. These are some of them:

Clinical trial:The work of ensuring drug safety through pharmacovigilance starts at the stage of the clinical trial itself. Pharmacovigilance ensures that drug safety is built into the drug right at the clinical trial stage. PV sets out a number of processes and methods by which a pharmaceutical company involved in clinical trials has to go in order to ensure drug safety.

Marketing:PV is set out in the marketing stage of a drug, too. Drug safety brought about by pharmacovigilance is to be implemented at the marketing stage of the drug, including processes for its safe storage, handling and transit. Throughout all these stages, drug safety and pharmacovigilance guidelines are to be strictly implemented.

Governmental drug distribution:Drug safety and pharmacovigilance are also built in by governments in their interactions with each other. For instance, when the government of a country or an agency such as the UN is shipping drugs to another country to support a health program; utmost drug safety is ensured through the principles of pharmacovigilance.

Drug safety and pharmacovigilance in disease management:Several governments across the world, along with international agencies conduct disease control management or emergency handling across the globe, especially in developing countries. Pharmacovigilance is the means to ensure that the drugs administered at these programs are safe for human consumption.

Learn more on this topic by visiting:

http://bit.ly/Safety-Reporting-in-Clinical-Trials-San-Diego

http://bit.ly/Validation-and-21-CFR-11-Compliance-of-Computer-Systems

http://bit.ly/During-an-Inspection

FDA Requirements for ensuring Premarketing Clinical Trial Safety

The FDA has set out requirements for sponsors and organizations that carry out clinical trial to ensure premarketing clinical trial safety. This is a very vital requirement because this is the stage at which the database that goes into clinical trials is formed. Its integrity and safety is an important ingredient for assessing the risks and benefits that go into the clinical trial, and errors need to be identified and corrected at this stage. Wrong data could lead to disastrous consequences for the study, the subjects that are part of it, the organization and eventually, patients.

Basic nature of FDA requirements for premarketing clinical trial safety

The FDA has a set of requirements for premarketing clinical trial safety, but these are mostly informal and loose. They are more of an advisory nature than being stringent regulatory requirements that are legally enforceable. Most FDA guidance is on a case-by-case nature.

Basically, the FDA’s guidance is based on its working with large to very large clinical trials. It has thus far not seriously considered working with small groups for assessing premarketing clinical trial safety. At its barest, the FDA seeks to:

  • Advise sponsors or organizations undertaking the clinical trial about ways by which their data collection can be simplified so as to ensure that it is neither too huge nor too small, and should lead to giving insights about the drug’s safety. Essentially, the FDA guideline on premarketing clinical trial safety seeks to prevent sponsors from collecting data that is not relevant.
  • Get sponsors to consult the FDA’s review division for its premarketing clinical trial safety.

The FDA has different requirements for different kinds of studies that relate to clinical trials. For example:

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Learn more  on this topic by visiting: http://bit.ly/Clinical-Trial-Practice-for-Drugs-and-Biologics

Preclinical Toxicology & Safety and its core elements

Preclinical toxicology and safety occupies a position of eminence in a clinical research program. A study of preclinical toxicology and safety is absolutely important because this leads researchers to an understanding of what causes adverse effects. Adverse effects form the backbone of preclinical toxicology and safety, because they can come from any source of toxicology.

Toxicology, which is the study of adverse effects, can happen from any source ranging from chemicals to biological agents, as well as physical, or the environment.

So, preclinical toxicology and safety has to take safety into consideration both before the dose is administered in humans, and before clinical trials.

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Good Laboratory Practices (GLPs) relating to preclinical toxicology and safety

A Good Laboratory Practice that relates to preclinical toxicology and safety is one that lists out regulations and practices that ensure data integrity from nonclinical studies. Since these are practices and conventions that have evolved over time; there are different GLPs prescribed by different regulatory bodies such as the FDA, the Organization for Economic Co-operation and Development (OECD) and the Environmental Protection Agency (EPA).

In the US, the FDA prescribes preclinical toxicology and safety GLPs under 21 CFR Part 58. At its simplest, 21 CFR Part 58 requires preclinical studies to be compliant with GLP relating to:

  • In vitro toxicology
  • In vivo toxicology
  • Animal models

Very broadly, preclinical toxicology and safety requirements for each of these types of toxicology can be understood thus:

  • In vitro

Preclinical toxicology and safety relating to in vitro toxicology is used for screening and ranking chemicals and the study of cell and tissue. GLPs for in vitro preclinical toxicology and safety are centered on improving future study design/s.

  • In vivo

The purpose of in vivo toxicology is to establish a starting dose for clinical studies that is safe.

So, GLPs for in vivo preclinical toxicology and safety are meant to offer information on a drug that spews the least toxicity.

  • Animal studies

Finally, preclinical toxicology and safety in animal studies are assessed to serve treatment outcomes better in humans by testing drugs on animals. This is based on the assumption that these organisms serve as a proper indicator of how these drugs could act in humans.

Read More: https://www.linkedin.com/pulse/preclinical-toxicology-safety-its-core-elements-ronald-gardner