This document summarizes information presented at a conference on access to haemophilia care post humanitarian aid. It discusses global plasma collection and FVIII production, examples of humanitarian aid programs providing factor concentrates, and comparisons of factor prices between different countries and over time. Barriers to increasing global access to treatment and potential strategies for overcoming these barriers are also examined.
ICIC 2014 Patent Landscape Analysis as a Tool for Public Policies Adjustment:...Dr. Haxel Consult
The innovative multinational pharmaceutical industry is highly dependent on the release and promotion of new drugs. However, recent economic evidence demonstrates a continuing decrease in new drugs’ market approval. Moreover, the industry is challenged by the “patent cliff”, where many blockbuster drugs are losing patent protection and facing ferocious competition.
It seems that the shortage of new drugs points to an intensification of drug development based on molecules already known, leading to incremental patents. However, there is a suspicion that many incremental patents are actually trivial, because they add little or nothing to existing therapies, but still impose high drug costs. To analyze that hypothesis, the present study has drawn the profile of all patents filed in Brazil in the antiretroviral (ARV) field up to 2012. Using VantagePoint® and Questel Orbit® softwares, a patent matrix was constructed with quali-quantitative data. Next, the patent applications' claims were analysed in order to detect incremental patents and classified according to their incrementalities. Finally we looked for evidence of triviality. As a result, it was demonstrated that the ARV market is highly concentrated and patent applications basically belong to six countries. Evidence that many incrementalities are actually trivialities and act as entry barriers, was found. Patent landscape studies such as this one can be extrapolated to other areas or countries, and can be used as a tool for public policy’s analysis to really fuel technological advance.
Reimbursement of Coagulation Medications in Latvia in 2014Baiba M. Ziemele
In 2014, spending or reimbursements for coagulation disorders reached 2.74 M EUR, which allowed to purchase
4 207 750 IU of FVIII concentrate and 617 000 IU of FIX concentrate, as well as ensure treatment for patients with hemophilia A and B inhibitors and von Willebrand disease. First time in history, Factor VIII consumption in Latvia surpassed 2 IU per capita, which is much closer to EDQM recommendations of 3 IU per capita as ever before. Medications were prescribed to 64 people with hemophilia A, 11 people with hemophilia B and 41 patient with von Willebrand disease. There are 283 people with bleeding disorders in Latvia.
ICIC 2014 Patent Landscape Analysis as a Tool for Public Policies Adjustment:...Dr. Haxel Consult
The innovative multinational pharmaceutical industry is highly dependent on the release and promotion of new drugs. However, recent economic evidence demonstrates a continuing decrease in new drugs’ market approval. Moreover, the industry is challenged by the “patent cliff”, where many blockbuster drugs are losing patent protection and facing ferocious competition.
It seems that the shortage of new drugs points to an intensification of drug development based on molecules already known, leading to incremental patents. However, there is a suspicion that many incremental patents are actually trivial, because they add little or nothing to existing therapies, but still impose high drug costs. To analyze that hypothesis, the present study has drawn the profile of all patents filed in Brazil in the antiretroviral (ARV) field up to 2012. Using VantagePoint® and Questel Orbit® softwares, a patent matrix was constructed with quali-quantitative data. Next, the patent applications' claims were analysed in order to detect incremental patents and classified according to their incrementalities. Finally we looked for evidence of triviality. As a result, it was demonstrated that the ARV market is highly concentrated and patent applications basically belong to six countries. Evidence that many incrementalities are actually trivialities and act as entry barriers, was found. Patent landscape studies such as this one can be extrapolated to other areas or countries, and can be used as a tool for public policy’s analysis to really fuel technological advance.
Reimbursement of Coagulation Medications in Latvia in 2014Baiba M. Ziemele
In 2014, spending or reimbursements for coagulation disorders reached 2.74 M EUR, which allowed to purchase
4 207 750 IU of FVIII concentrate and 617 000 IU of FIX concentrate, as well as ensure treatment for patients with hemophilia A and B inhibitors and von Willebrand disease. First time in history, Factor VIII consumption in Latvia surpassed 2 IU per capita, which is much closer to EDQM recommendations of 3 IU per capita as ever before. Medications were prescribed to 64 people with hemophilia A, 11 people with hemophilia B and 41 patient with von Willebrand disease. There are 283 people with bleeding disorders in Latvia.
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Thelansis known to provide therapy specific market insight reports which essentially covers Epidemiology and Market Forecast of 8 key markets (US, Germany, France, Spain, Italy, UK, Japan and China) in a advance excel based dynamic forecast model, enables client to understand the differnet scenario of the market over a period of 10 years.
Competitive landscape analysis covered in the Thelansis reports allows our clients to monitor competitor’s activity around targeted indications during a different phase of development (Early Phase, Phase I, II, and III) and post-approval phase.
Our services include epidemiology study, Market forecasting, clinical trial analytics, Pipeline assessment, enrollment projections, trialsite prioritization, investigator profiling, and trial completion date projection, launch date estimation, and essentially the key product positioning.
Thelansis reports now coupled with KOL insights gathered from the the Primary market research (PMR) through in-house programmed web-based survey tool which helps our clients to fill the gap of real-time data, unmet needs, and critical strategic insights right from development to commercialization.
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Primary biliary cholangitis (PBC) Epidemiology and Compitive landscape_Thelan...Thelansis
Thelansis known to provide therapy specific market insight reports which essentially covers Epidemiology and Market Forecast of 8 key markets (US, Germany, France, Spain, Italy, UK, Japan and China) in a advance excel based dynamic forecast model, enables client to understand the differnet scenario of the market over a period of 10 years.
Competitive landscape analysis covered in the Thelansis reports allows our clients to monitor competitor’s activity around targeted indications during a different phase of development (Early Phase, Phase I, II, and III) and post-approval phase.
Our services include epidemiology study, Market forecasting, clinical trial analytics, Pipeline assessment, enrollment projections, trialsite prioritization, investigator profiling, and trial completion date projection, launch date estimation, and essentially the key product positioning.
Thelansis reports now coupled with KOL insights gathered from the the Primary market research (PMR) through in-house programmed web-based survey tool which helps our clients to fill the gap of real-time data, unmet needs, and critical strategic insights right from development to commercialization.
The ‘Basic Recommendations in Brief for Treating Physicians’, based on and complementing the ‘Guidelines for the Management of Transfusion Dependent Thalassaemia (4th Edition – 2021)’, is an easy-to-understand, user-friendly resource for thalassaemia-treating clinicians, especially for those who are not fully experienced and well-knowledgeable of the management of patients with this serious chronic disorder, or those operating in Urgent and Emergency Care (UEC) settings admitting haemoglobinopathy patients.
These key recommendations offer valuable guidance on a variety of thematics pertaining to thalassaemia management, ranging from blood transfusion and iron overload and chelation to the assessment of cardiovascular and liver disease, pregnancy management, and much more.
PPPI - the involvement of patients or people in the design and development o...ipposi
Dr Jean Saunders, University of Limerick, and IPPOSI Board Member (Science) presents at the 5th World Congress on Advanced Clinical Trials and Clinical Research on public and patient involvement in clinical trials.
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Global Regulatory Issues: one BA method, one validation, one report ...Peter van Amsterdam
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Title: Sense of Taste
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the structure and function of taste buds.
Describe the relationship between the taste threshold and taste index of common substances.
Explain the chemical basis and signal transduction of taste perception for each type of primary taste sensation.
Recognize different abnormalities of taste perception and their causes.
Key Topics:
Significance of Taste Sensation:
Differentiation between pleasant and harmful food
Influence on behavior
Selection of food based on metabolic needs
Receptors of Taste:
Taste buds on the tongue
Influence of sense of smell, texture of food, and pain stimulation (e.g., by pepper)
Primary and Secondary Taste Sensations:
Primary taste sensations: Sweet, Sour, Salty, Bitter, Umami
Chemical basis and signal transduction mechanisms for each taste
Taste Threshold and Index:
Taste threshold values for Sweet (sucrose), Salty (NaCl), Sour (HCl), and Bitter (Quinine)
Taste index relationship: Inversely proportional to taste threshold
Taste Blindness:
Inability to taste certain substances, particularly thiourea compounds
Example: Phenylthiocarbamide
Structure and Function of Taste Buds:
Composition: Epithelial cells, Sustentacular/Supporting cells, Taste cells, Basal cells
Features: Taste pores, Taste hairs/microvilli, and Taste nerve fibers
Location of Taste Buds:
Found in papillae of the tongue (Fungiform, Circumvallate, Foliate)
Also present on the palate, tonsillar pillars, epiglottis, and proximal esophagus
Mechanism of Taste Stimulation:
Interaction of taste substances with receptors on microvilli
Signal transduction pathways for Umami, Sweet, Bitter, Sour, and Salty tastes
Taste Sensitivity and Adaptation:
Decrease in sensitivity with age
Rapid adaptation of taste sensation
Role of Saliva in Taste:
Dissolution of tastants to reach receptors
Washing away the stimulus
Taste Preferences and Aversions:
Mechanisms behind taste preference and aversion
Influence of receptors and neural pathways
Impact of Sensory Nerve Damage:
Degeneration of taste buds if the sensory nerve fiber is cut
Abnormalities of Taste Detection:
Conditions: Ageusia, Hypogeusia, Dysgeusia (parageusia)
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Neurotransmitters and Taste Threshold:
Effects of serotonin (5-HT) and norepinephrine (NE) on taste sensitivity
Supertasters:
25% of the population with heightened sensitivity to taste, especially bitterness
Increased number of fungiform papillae
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Introduction
Many have been interested in Tom Selleck health. not only because of his enduring presence on screen but also because of the challenges. and lifestyle choices he has faced and made over the years. This article delves into the various aspects of Tom Selleck health. exploring his fitness regimen, diet, mental health. and the challenges he has encountered as he ages. We'll look at how he maintains his well-being. the health issues he has faced, and his approach to ageing .
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Humanitarian aid and access to haemophilia care
1. Access to haemophilia care
Post humanitarian aid
Albert Farrugia
Adjunct Professor, School of Surgery, University of Western Australia
Senior Scientific and Regulatory Advisor, Kedrion S.p.A
The 10th WFH Global Forum on Research and Treatment Products for Bleeding Disorders
Montreal – November 8 - 10, 2017
2. DISCLOSURES FOR:
(SPEAKER NAME)
Conflict Disclosure - if conflict of interest exists
Research Support None
Director, Officer, Employee Senior Director of Biologics consulting company
Shareholder None
Honoraria None
Advisory Committee None
Consultant Consultant to manufacturers of haemophilia
therapies
3. G l o b a l v o l u m e o f p l a s m a f o r f r a c t i o n a t i o n 1 9 9 0 - 2 0 1 4
R e c o v e r e d a n d S o u r c e –
To t a l Vo l u m e i n 2 0 1 4 : 4 5 M i l l i o n l i t r e s
5. Estimates
• In 2014, 45 Million Litres of plasma were collected.
• Using current technology, a yield of 200 IU of FVIII per litre is expected.
• This would result in 9 billion IU (the current total amount of FVIII provided globally)
• 4 billion IU of pd VIII were actually provided
• It is clear that around half the possible amount of FVIII is not being converted into
concentrate.
• In contrast, similar estimates for other products indicate that approximately 85% of
the available IG (probably an underestimate) and albumin are being extracted
6. Conver genc e of players
The Italian exampl e – Septem ber 2017
6
http://www.centronazionalesangue.it/node/582
• 750,000 IU of Factor VIII exceeding the regional and national
requirements.
• Made available by the Lombardy Region.
• Provides for cooperation:
• Developing standards for optimum use of blood components in
Armenia
• Introduction of a blood collection system and components from
voluntary and unpaid donors based on the Italian model.
• Research projects and exchange of information.
• Technical support and logisitics provided by Kedrion.
• Based on the agreement of the Italian Regions of 7 February 2013 for the
promotion and implementation of cooperation agreements for the export
Memorandum of Cooperation
between the Italian National Blood
Center and the Ministry of Health of
Armenia signed on 6 June.
7. In recent years, over 10 million units of factors have been donated
by Italy to countries such as Afghanistan, Albania, Armenia and
India.
Why isn’t this happening in other national blood systems with
national fractionation arrangements eg:
France
The Netherlands
Spain
Australia
8. «Humanitarian Aid»
• Identify stakeholders and INTERESTS
• Political, commercial, organisational
• Create a coalition – needs an owner
• Forge international links – EU, WHO
9. What peopl e are paying ……
….in Sri Lanka
…in Brazil
10. Prices paid in Lebano n
Product Presentation Supplier Price $US
BERIATE 1,000IU CSL Behring GmbH 575
OCTANATE 1000IU
Octapharma Pharmazeutika
Produktions GesmbH 556
FACTANE 1,000IU LFB Biomedicaments 595
HAEMATE P 1000IU CSL Behring GmbH 601
KOGENATE FS antihemophilic factor (recombinant) 1,000IU Bayer HealthCare LLC 816
NOVOEIGHT 1,000IU Novo Nordisk AS 942
NUWIQ 500IU Octapharma AB 555
RECOMBINATE 500IU Baxter SA 463
WILFACTIN 1000IU LFB Biomedicaments 1,181
11. Prices for FVIII CFCs paid by the
Australian NBA 2015-16
Factor VIII
(plasma derived -
domestic)
Biostate
250 IU
CSL Behring
(Australia) Pty Ltd
$215.12
68 US cents/IU500 IU $430.25
1000 IU $860.50
Factor VIII
(recombinant -
imported)
Advate
250 IU
Baxalta Australia
Pty Ltd
$75.00
24 US cents/IU
500 IU $150.00
1000 IU $300.00
1500 IU $450.00
2000 IU $600.00
3000 IU $900.00
https://www.blood.gov.au/pubs/1516report/part-5-appendices/appendix-3-plasma-and-recombinant-products-supplied-under-contract-2015-16.html
12. 12
F A C T O R V I I I P R I C E P E R U N I T I N T H E U N I T E D S T AT E S F R O M 1 9 8 0 T O 2 0 1 5
( R e c o m b i n a n t & P l a s m a - d e r i v e d )
13. 13
• Infusing twice weekly with rFVIIIFc at 42.7 IU/kg per dose required less clotting factor than
infusing every 56 h with rFVIII 33.75 IU/kg per dose
• Annual bleeding rates were similar.
• Base case analysis suggested that total FVIII costs were equated when rFVIIIFc cost 1.18 times
more per IU than rFVIII, assuming similar adherence.
• In 2016, average Elocate price was 15.7 % more expensive than average rVIII price (MRB)
14. Cost considerations for FIX-Fc regimens
FIX
Prescribed prophylaxis regimens
Standard factor replacement
Label prophylaxis regimens EHL factor replacement
Strategy #1
40 IU kg−1 twice
weekly
Strategy #2
67 IU kg−1 twice
weekly
Strategy #1
50 IU kg−1 once
weekly
Strategy #2 100 IU kg−1 every
10 days
Price per unit $1.06 $1.06 $2.85 $2.85
Factor cost per year per adult
patient (70 kg)
$308 672 $517 026 $518 700 $728 175
Comparative premium cost 1 ×1.7 ×2.4
Number of infusions saved per
year
Baseline – 52 67
Haemophilia (2015), 21, 285–288
•Label strategy #1 and #2 differ by 15 infusions per year
•Premium cost for #2 is $13,900 per dose saved
•Annual factor usage 2600 IU/kg for #1 vs. 3650 IU/kg for #2
•Unnecessarily high peaks, lower troughs with 10 day regimen
15. Biosimil ar s
• The rationale behind the introduction of biosimilars is to increase price
competition, an effect of which is often reduced prices.
• The increased competition resulting from biosimilars entering the market
affects not just the price of the respective biosimilars reference product, but
also the price of the whole product class.
• It can have almost as large an impact on the total market price as it has on
the biosimilar/reference product price.
«The Impact of Biosimilar Competition in Europe» Quintiles May 2017
16. To t a l c h a n g e i n p r i c e p e r T D s i n c e t h e e n t r a n c e o f b i o s i m i l a r s
f o r e a c h t h e r a p y a r e a i n t h e E u r o p e a n E c o n o m i c A r e a
16
«The Impact of Biosimilar Competition in Europe» Quintiles May 2017
17. In India………
17
According to the Economic Times, the price gap for the originator
version is INR1,200 ($17.97) with the Roche price at INR29,500 for
100mg injection, compared to INR28,300 for the Reliance version
http://www.fiercepharma.com/pharma-asia/india-s-reliance-life-sciences-brings-first-bevacizumab-biosim-to-market
18. «Biosimilar» CFCs ?
• Novoeight
• Rexubis
• Ixinity
• (Greengene)
• Coagil-VII
• Octofactor
• Innonafactor
Why are there not more
Biosimilar CFs in eg
China, India?
19. Ekaterina Shiller et al. Blood 2015;126:4703
Safety and Efficacy of Octofactor – biosimilar BDD rVIII ("GENERIUM", Russia) in Prophylactic
Treatment in Adolescent Patients with Severe and Moderate Hemophilia A
20. H e a l t h s p e n d i n g b y c o u n t r i e s a n d c u r r e n t E s s e n t i a l
M e d i c i n e s L i s t ( U S a n d S o u t h E a s t A s i a n R e g i o n o f W H O )
20
21. ATC Classification of drugs in WHO EML (2011) and
India EML ( 2011)
21
*Items that are not
included in ATC classes
(e.g., coal tar,
calamine,
cryoprecipitate, etc.)
22. Clinical trials -em be ddi ng treatment
• All clinical trials should be done according to the
principles of Good Clinical Practices (GCPs)
• These include the principles of the Declaration of Helsinki
Including the continuation of treatment in trial subjects after the trial
completion
Including the principle of not exposing subjects to harm
• Many clinical triasl to «prove» prophylaxis in haemophilia
are being done in the emerging countries
• By continuing treatment in the young patient population,
a number of future leaders can be saved for the
haemophilia community
23. Reflecti ons
• Much of the plasma FVIII donated globally is not going to
patients
• To address this, strategies recognising the interests and
stakeholders in blood service delivery are needed
• Nevertheless, the cost of established and some new CFCs is not
increasing in real terms
• The retreat of governments from the provision of health care is