Bioeconomy and Regulatory Environment of Biopharmaceuticals

Bioeconomy and Regulatory Environment of Biopharmaceuticals

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Description: Bioeconomy I: - Biotechnology is any technological application that uses biological systems, living organisms, or derivatives thereof, to make or modify products or processes for specific use. Biobased economy/economy refers to all economic activity derived from scientific and research activity focused on biotechnology. Bioeconomy II: - Covers all industries and sectors, which develop, produce, process, reprocess or use them in any form biological resources such as plants, animals, and microorganisms (produce/manage/use biological resources).

Focuses on developing industrial viable, safe and ecologically friendly biobased (renewable biological sources) solutions to build a sustainable society, involving food, chemicals, energy, and materials.

 
Author: Ismail Mert Vural M.D.  | Visits: 379 | Page Views: 538
Domain:  Medicine Category: Biotech/Pharma 
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Contents:
Bioeconomy and
Regulatory Environment of
Biopharmaceuticals
İsmail Mert VURAL M.D.
Assoc. Prof.
Turkish Medicines and Medical Devices Agency
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Source: How t0 define bioeconomy?
Maciejczak M, Hofreiter K

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Bioeconomy I
• Biotechnology is any technological application that uses biological
systems, living organisms, or derivatives thereof, to make or modify
products or processes for specific use.
• Biobased economy/bioeconomy refers to all economic activity
derived from scientific and research activity focused
on biotechnology.

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Bioeconomy II
• Covers all industries and sectors, which develop, produce,
process, reprocess or use them in any form biological resources
such
as
plants,
animals
and
microorganisms
(produce/manage/use biological resources).
• Focuses on developing industrial viable, safe and ecologically
friendly biobased (renewable biological sources) solutions to build a
sustainable society, involving food, chemicals, energy and
materials.

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Why We Need Bioeconomy?

Major driving forces:
• growing and aging population
• increased demand for feed,
sustainable food production
• limited resources
• increased incidence of various
disorders
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

What We Hope

Major benefits:
• improved health
• a cleaner environment
• improved innovation capacity
• efficiency on resource use
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

It is estimated that in
2030 biotechnological
products will
constitute;
35% of the chemical and
industrial products,
50% of the agricultural
products,
80% of the
pharmaceutical and
diagnostic products.

Source: Scientific American Worldview (published by Scientific American Custom Media)
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Evaluation Criteria
Public company revenues, the number of
companies
Intellectual property rights

The ratio of biotechnology patents on total
patents
The amount spent on biotechnology and
R & D in the field

Education and workforce
Allocated to venture capital in biotechnology
/ support
Funds
Politics and stability
Source: Scientific American Worldview
(published by Scientific American Custom Media)
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Source: Scientific American Worldview
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

%87,7

%88,4

Source: Scientific American Worldview
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

In
the
sectorial
distribution
of
the
world's Research and
Development (R & D)
spending,
pharmaceutical
and
biotechnology
applications are located
in the first place with
19.3%.

Biopharmaceuticals I
• Biological medicines/biopharmaceuticals are comprised of
proteins such as hormones, enzymes that are naturally produced in
the human body, monoclonal antibodies, blood products,
immunological medicinal products such as sera and vaccines,
allergens, and advanced technology products such as gene and cell
therapy products.

• Biopharmaceuticals can be tailor-made to fit the desired target.

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Biopharmaceuticals II
• Biotechnology uses living systems (plant or animal cells, bacteria,
viruses and yeast) and modern technologies to produce biological
medicines to treat diseases and genetic disorders in humans.
• Many biopharmaceuticals, are made using genetically-modified
cells.
• Each manufacturer has its own unique cell lines and
develops its own proprietary (unique) manufacturing processes.

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Pharmaceutical Market (World)

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Biopharmaceutical Market (World)
Biotech medicine sales
were an estimated $289
billion in 2014 and are
projected to grow to $445
billion by 2019.
Biotech’s share of
worldwide prescription
medicine and over-thecounter pharma sales is
projected to increase
from 23 percent in 2014
to 26 percent in 2019

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Pharmaceutical Market (Turkey)
20

2,5

18

2,11
1,85

14

1,48

Billion TL

1,89

12

1,28

1,57

1,61

1,91

1,97

2,0

1,67
1,5

1,36

10

18,31
8

0,72

0,81

0,90
12,11

6

9,26

4

5,49
2

13,45

14,20

13,86

14,17

13,52

14,33

1,0

15,70

10,34
0,5

6,36

3,99

0

0,0
2002

2003

2004

2005

2006

2007

2008

2009

2010

2011

Source: IMS-Health Turkey
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

2012

2013

2014

2015

Billion (Box Sale)

16

Biopharmaceutical Market (Turkey) I

Source: IMS-Health Turkey
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Biopharmaceutical (Turkey) II
• One of the remarkable point in biopharmaceuticals is highness of
unit costs and this continues increasingly on yearly basis. Average
value of biotechnological medicines is 57,72 TL at 2003 and 121,76
TL at 2015.
• While box sales of biopharmaceuticals increased by 5,9 times
between 2003 and 2015 and value of sales increased by 12,4
between same years.

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Republic of Turkey Ministry of Health
Turkish Medicines and Medical Devices Agency
ANALYSIS OF THE EFFECTS OF PUBLIC
INTERVENTIONS ON THE FIRST 100
MEDICINES ON VALUE BASIS IN 2008-2013
PERIOD
Health Technologies Assessment Report – I
Health Technologies Assessment Unit

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Republic of Turkey Ministry of Health
Turkish Medicines and Medical Devices Agency
ASSESSMENT OF THE FIRST 100 MEDICINES
ON VALUE BASIS IN 2014 AND COMPARISON
WITH THE YEARS 2012-2013
Health Technologies Assessment Report – II
Health Technologies Assessment Unit

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Biopharmaceutical Market (Turkey) III
• The share of
biotechnological
medicines in
medicines which
have a high
amount of sales is
increasing by
years.

Source: IMS-Health Turkey
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Major challenge for biological medicines;
• the financial sustainability in healthcare
systems.

Solutions;
• biosimilar medicinal products (quality, safety,
efficacy)
• risk sharing agreements (performance based,
volume based)
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

• Based on the information published on the EMA website;
 No specific safety issue has been identified for approved and marketed
biosimilar medicines.
 There is relatively little published data available on the number of
patients that have been switched between biopharmaceuticals in clinical
practice.
 There is no evidence or scientific rationale to suggest that biosimilar
medicines are likely to cause more immune reactions than their
reference medicines.

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Regulatory Environment (World) I
• In the European Union, marketing authorisation applications for
biotechnology-derived medicinal products, including biosimilar
medicinal products, are by law reviewed centrally by the European
Medicines Agency (EMA).
• The European Commission issues the Decisions concerning the
authorisation of these medicinal products on the basis of the
scientific opinions from the EMA.

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Regulatory Environment (World) II
• The resulting marketing authorisation is valid in all EU Member
States.
• The EU have a framework and a regulatory pathway for biosimilars.

• National authorities
substitution

decides

on

interchangeability

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

and/or

Regulatory Environment (World) III
• Overview in USA; biologics regulated by FDA include human
blood and plasma and their derivatives, bacterial and viral vaccines,
toxin, antitoxin, growth factors, monoclonal antibodies, somatic
cells and gene therapy.
• Preclinical research; FDA is charged with ensuring that drugs,
biologics and medical devices are safely and thoroughly tested and
that commercial products are safe and effective for their intended
use.
• Preclinical testing can include genotoxicity screening, drug
absorption and metabolism, toxicity of drug and metabolites, and
speed at which drug and metabolites are excreted from the body.

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Regulatory Environment (World) IV
• Clinical trials; Depending on the product, clinical testing may be
required prior to product approval. Requirements include: written
investigator agreement; investigator training; approval by
institutional review board (“IRB”); informed consent; reporting
(periodic and special reports including reporting of adverse events);
recordkeeping; and study monitoring to ensure compliance with
study protocol, investigational plan, FDA and IRB requirements
• Example of FDA clinical study initiatives/issues relevant to
biotechnology products; In response to gene therapy trial death,
FDA has stepped up inspection of clinical trials, suspended trials
that raise concern, issued warning letters citing compliance issues,
begun disqualification proceedings against investigator and
requested implementation of improved quality assurance measures
in gene therapy research.
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Regulatory Environment (World) V
• NIH’s Office of Biotechnology Activities (“OBA”) is responsible for
monitoring progress in human genetics research. The OBA administers the
Recombinant DNA Advisory Committee (“RAC”). The RAC reviews gene
transfer protocols and recommends changes to NIH.
• Product approvals;
▫ “Fast track” for drugs and biologics: FDA standard for “fast track” is when
the agency determines the product has an effect on a surrogate clinical endpoint
that is reasonably likely to predict clinical benefit. Under this approach, FDA
balances the medical need for a new therapy against approving a product with
possible unknown risks.
▫ Priority review for drugs and biologics; Drug/biologic applications can get
“priority review” if they provide a significant improvement in the safety and
effectiveness of treatment, diagnosis or prevention of a serious or life threatening
disease.
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Regulatory Environment (Turkey) I
• Licensing, pricing, auditing and directing activities are executed by
Turkish Medicines and Medical Devices Agency (TITCK) and
reimbursement is performed by Social Security Institution (SGK).
• Retail sales price of medicines is determined by TITCK through
external reference pricing system (France, Spain, Italy, Portugal,
Greece).
• Reimbursement decisions in SGK are arranged by commissions
which have representatives of relevant ministries (Ministry of
Health, Ministry of Finance, Ministry of Development,
Undersecretariat of Treasury) as well in them. Public prices of
medicines are arranged considering the different discount rates.

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

Regulatory Environment (Turkey) II

18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya

THANK YOU !!!
18th International Pharmaceutical Technology Symposium –IPTS 2016; Antalya