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Complex Medicines: Science, Regulation, and Accelerating Development

Available via

WEBINAR

Complex Medicines: Science, Regulation, and Accelerating Development

Monday, May 13, 2019, 8:00 AM - 6:15 PM

The New York Academy of Sciences, 7 World Trade Center, 250 Greenwich St Fl 40, New York

The rise of bio- and nano-technologies has accelerated the development of complex medicines, and at the same time it has revealed new hurdles in regulatory science and accelerating patient access to new therapies. Complex medicines include biologics (e.g. vaccines, gene therapies, recombinant proteins) and non-biological complex drug products (e.g. glatiramoids, iron-carbohydrate complexes, polymeric micelles, complex ocular emulsions, and liposomes). Their diverse nature poses challenges for the development of regulatory guidelines. While complexity is not new in medicines, our technical capacity to measure and analyze data has increased. This requires a determination of which measurements are relevant to demonstrate therapeutic efficacy and safety. Further, many obstacles remain in regulatory harmonization across global authorities, given their different approaches and legal frameworks.

To advance research and build consensus, it is necessary to engage together key stakeholders from academia, regulatory bodies, industry, and drug manufacturing. The Non Biological Complex Drugs Working Group, the Nanotechnology Characterization Laboratory, and the New York Academy of Sciences will present the conference Complex Medicines: Science, Regulation, and Accelerating Development to stimulate this discussion. This convening is a follow up to the 2016 event, Equivalence of Complex Drug Products: Scientific and Regulatory Challenges. The conference will identify the best scientific approaches for complex medicines development and regulation, will outline outstanding challenges in the assessment of equivalence, and will discuss how to improve timely patient access for new medicines and building a sustainable healthcare system. The end goal is to facilitate the translation of scientific findings into advancements in medicine for the benefit of patients.

Registration

Member
By 04/01/2019
$90
After 04/01/2019
$130
Nonmember Academia, Faculty, etc.
By 04/01/2019
$180
After 04/01/2019
$260
Nonmember Corporate, Other
By 04/01/2019
$250
After 04/01/2019
$350
Nonmember Not for Profit
By 04/01/2019
$180
After 04/01/2019
$260
Nonmember Student, Undergrad, Grad, Fellow
By 04/01/2019
$100
After 04/01/2019
$145
Member Student, Post-Doc, Fellow
By 04/01/2019
$50
After 04/01/2019
$70
Member
$30
Nonmember Academia, Faculty, etc.
$65
Nonmember Corporate, Other
$85
Nonmember Not for Profit
$65
Nonmember Student, Undergrad, Grad, Fellow
$45
Member Student, Post-Doc, Fellow
$15
Earlybird Registration:
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days
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Deadline:
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Keynote Speaker

Gregory W. Daniel, PhD, MPH, RPh

Duke-Margolis Center for Health Policy at Duke University

Speakers

Henning Blume, PhD

SocraTec R&D

Michael J.W. Johnston, PhD

Health Canada

Akm Khairuzzaman, PhD

US Food and Drug Administration (FDA)

Scott McNeil, PhD

Nanotechnology Characterization Lab

Sesha Neervannan, PhD

Allergan

Saraswathy V. Nochur, PhD

Alnylam Pharmaceuticals

Vinod Shah, PhD

Non Biological Complex Drugs Working Group

Beatriz Silva Lima, PhD

University of Lisbon

Florian Turk, PhD

Sandoz Biopharmaceuticals

Gillian Woollett, MA, DPhil

Avalere Health

Scientific Organizing Committee

Jon de Vlieger, PhD

Lygature

Beat Fluhmann, PhD

Vifor Pharma

Scott McNeil, PhD

Nanotechnology Characterization Lab

Sesha Neervannan, PhD

Allergan

Vinod Shah, PhD

Non Biological Complex Drugs Working Group

Gillian Woollett, MA, DPhil

Avalere Health

Melanie Brickman Borchard, PhD

The New York Academy of Sciences

Alison Carley, PhD

The New York Academy of Sciences

Monday

May 13, 2019

8:00 AM

Registration and Breakfast

8:45 AM

Opening Remarks

Speakers

Alison Carley, PhD
The New York Academy of Sciences
Jon de Vlieger, PhD
Lygature and the Non Biological Complex Drugs Working Group

Session I: Outline of the Future of Complex Drugs

Session Chairperson
Jon de Vlieger, PhD, Lygature and the Non Biological Complex Drugs Working Group
9:00 AM

Innovation in Complex Therapies to Treat Complex Diseases

Speaker

Sesha Neervannan, PhD
Allergan
9:25 AM

Regulatory Experience with ONPATTRO® (patisiran), a First-in-Class RNAi Therapeutic

Speaker

Saraswathy V. Nochur, PhD
Alnylam Pharmaceuticals
9:50 AM

Complex Drugs — The Future of Medicine

Speaker

Scott McNeil, PhD
Nanotechnology Characterization Laboratory

Complex drugs enable promising therapeutic breakthroughs, but also pose challenges for characterization and regulatory evaluation. The formulations being developed to treat cancer and other diseases have been specifically designed such that their physicochemical characteristics can curtail off-target toxicities and optimize pharmacokinetics. As these products increase in complexity, so too must the technologies and methods for characterization and evaluation. The Nanotechnology Characterization Laboratory has been helping developers for more than a decade by providing state of the art characterization services to help advance these products into clinical trials. This talk aims to highlight the emerging trends in complex drug characterization as well as the scientific challenges they face. Challenges in identifying critical quality attributes, defining bioequivalence for generic products, as well as strategies aimed at addressing regulatory questions and commercialization will be discussed.

10:15 AM

Discussion

10:30 AM

Coffee Break

Session II: The Importance of Regulatory Science

Session Chairperson
Vinod Shah, PhD, Non Biological Complex Drugs Working Group
11:00 AM

Introduction to the Importance of Regulatory Science

Speaker

Vinod Shah, PhD
Non Biological Complex Drugs Working Group
11:10 AM

EU Regulatory Perspective on Complex Medicines

Speaker

Beatriz Silva Lima, PhD
University of Lisbon

Since more than one decade,nanoparticle-based formulations are emerging with multiple objectives, raising challenges on their development, characterization and standardization related to quality and biological activity. These so designated “complex drugs/medicines”raise specific questions needing regulatory support, related to eg the relationships between their quality attributes and the biological activity, including target interaction, dose-response, biodistribution patterns. Some of the referred components are the basis for the development of these “complex drugs/formulations”. The complexity is often driven by the difficulties behind a thorough characterization and analytical measuring of nanoparticles and the drug-particle complexes, and the incomplete knowledge on how changes (even small) in attributes of the particles or drug-particle conjugates impacts on the bioactivity of the product, related to target access as well as target interaction. Of particular relevance for the activity of any nanoparticle-based medicine is the understanding on how some changes in the attributes can impact on the pattern of tissue distribution, and the potential for changing the pattern of activity, efficacy and safety. These aspects and the science behind have been considered by Regulators in Europe and abroad, eg the United States FDA.Several guidance documents have been issued to help Companies and Researchers on developing innovative or follow on “Complex drug formulations”. The thinking behind the produced documents,by the European Regulators and some practical examples will be discussed. Key words: nanomedicine, nanoparticle, liposome, bioequivalence, generics, CHMP and FDA regulatory guidance.

11:35 AM

Scientific and Regulatory Challenges with Complex Generic Medicine

Speaker

Akm Khairuzzaman, PhD
US Food and Drug Administration

Over the last 30 years, new drug development in pharmaceutical industries has shifted significantly, moving towards more complex formulation design, for better disease management, adherence to patients, adoption of new technology as well as in part for better patent protection. As a result, it is becoming challenging for generic companies to come up with viable option for these products to be available in the market at an affordable price. Therefore, the generic industry is becoming smarter in its approach, increasingly pursuing more paragraph III/IV type applications and, in some cases, taking regulatory paths such as 505(b)(2). In recent years, the FDA has received generic applications for drug device combinations (e.g. pulmonary and nasal delivery), nano technology-based products (e.g. generic Rapamune/sirolimus), biosimilars (e.g. filgrastim, Inflectra), complex transdermal patches, implants, ophthalmic, long acting injectables, soft gels and many other products that did not exist in the past in generic market. This presentation will focus on the key scientific and regulatory issues with respect to complex generic drug development.

12:00 PM

Complex Drugs — the Health Canada Perspective

Speaker

Michael J.W. Johnston, PhD
Health Canada

Health Canada is a department within the Canadian Federal Heath Portfolio (which comprises Health Canada, the Public Health Agency of Canada, the Canadian Institutes of Health Research, the Patented Medicine Prices Review Board and the Canadian Food Inspection Agency) and is responsible for the regulation of consumer products, environmental and workplace health, food and nutrition, cannabis as well as drugs and medical devices. This presentation will outline Health Canada’s current perspective on complex drugs (specifically non-biological complex drugs) as well as follow-on complex drug products. Additionally, an overview of the activities of the International Pharmaceutical Regulators Programme Nanomedicines Working Group, currently co-chaired by Health Canada, will be provided.

12:25 PM

Science-driven Regulations for Complex Drugs and Necessity for Harmonization

Speaker

Henning Blume, PhD
SocraTec R&D and the University of Frankfurt

Very different medicinal products are allocated to the group of non-biological complex drugs (NBCDs), such as solutions for injection containing glatiramer acetate, iron sucrose complexes, or low molecular heparins as well as liposomal parenterals with encapsulated drugs like doxorubicin.


Due to their complex structure and composition approval procedures well established for generic drugs (i.e. abridged application/ANDA) are not appropriate here as long as the essential condition of identical active ingredients cannot be confirmed. Therefore, comprehensive comparability exercise is needed between the innovator product and its follow-on versions in order to conclude on their similarity in qualitative and quantitative composition. Considering the obvious differences in structure and complexity between the various NBCDs identical regulations cannot be applied to all these products. Thus, one consistent NBCD guideline including requirements for all these products cannot be adequate, but product-specific guidances should be more appropriate.


During the last couple of years such guidances have been developed for the European Union and the United States of America. There are, however, certain differences in the requirements between both legislations. Bridging the gap between deviating viewpoints and harmonization of the regulations should be the intention of regulatory agencies and industry. Essential condition for such initiative is the availability of experimental findings in order to establish science-driven regulations.


A detailed comparison of the current requirements suggested by the existing guidances indicates more consistency between the recommendations than expected considering the international debate on the most adequate approach for this heterogeneous group of products. However, reports published on experimental comparisons between generic alternatives which received the marketing authorization based on these regulations and their innovator reference products showed differences in certain quality attributes or, in some cases, also in clinical efficacy/safety outcomes.


It will be the mission for scientists from academia, industry and regulatory agencies to reconsider the current regulations in the light of the published findings in order to find out which quality attributes are clinically relevant and, thus, may be considered "critical" for the therapeutic effects. The most appropriate way to achieve this goal needs to be defined case-by-case considering the significant deviations between products of the NBCD group. Aspects relevant in this context will be highlighted.

12:50 PM

Discussion

1:05 PM

Networking Lunch

Session III: Toward a Sustainable Healthcare System

Session Chairperson
Beat Fluhmnn, PhD, Vifor Pharma
2:20 PM

Keynote Lecture: Supporting Drug Development and Higher Value Use with Real World Data and Evidence

Speaker

Gregory W. Daniel, PhD, MPH, RPh
Duke-Margolis Center for Health Policy at Duke University
3:00 PM

The Morality Gene — a European Perspective on Collective Sustainable Action

Speaker

Florian Turk, PhD
Sandoz Biopharmaceuticals
3:25 PM

Coffee Break

Session IV: Regulatory World Future Directions

Session Chairperson
Daan J.A. Crommelin, PhD, Utrecht University
3:55 PM

Implementing the Safe-by-design Concept to Polymeric Nanobiomaterials in Drug Delivery

Speaker

Cintia Marques, MSc
School Pharmaceutical Sciences Geneva-Lausanne, University of Geneva, University of Lausanne, Switzerland
4:10 PM

Connecting the Dots: Global Development of Complex Drugs and Biosimilars using Consistent Science to Enable Efficient Regulation

Speaker

Gillian Woollett, MA, DPhil
Avalere Health
4:35 PM

Panel Discussion

Speakers

Moderator: Daan J.A. Crommelin, PhD
Utrecht University
Gregory Daniel, PhD, MPH, RPh
Duke-Margolis Center for Health Policy at Duke University
Sesha Neervannan, PhD
Allergan
Florian Turk, PhD
Sandoz Biopharmaceuticals
Gillian Woollett, MA, DPhil
Avalere Health
5:05 PM

Closing Remarks

5:15 PM

Networking Reception and Poster Session

6:15 PM

Adjourn

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