therisktaker247
Badges: 6
Rep:
?
#1
Report Thread starter 1 year ago
#1
Can someone help me with this essay, it has a word limit of 3000?
0
reply
macpatgh-Sheldon
Badges: 20
Rep:
?
#2
Report 1 year ago
#2
If you are asking someone to write 3000 words for you, you must be in cuckooland, man - having said that, I too live dangerously, which makes two of us, so I will provide some info below for you. Go through it and see what you think is useful.

:goodluck:


SYMPTOMS

BACKGROUND: Polymorphonuclear
neutrophil (PMN)-dominated inflammation is prominent in the airways of subjects
with cystic fibrosis (CF) and chronic bronchitis (CB). Interleukin (IL)-8,
myeloperoxidase (MPO), and DNA are markers of neutrophilic inflammation. We
hypothesized that sputum MPO, DNA, and IL-8 concentrations would negatively
correlate with pulmonary function and sputum transportability. METHODS: We
measured pulmonary function and analyzed sputum IL-8, MPO, and DNA concentrations,
as well as the transport properties of sputum samples obtained from 16 subjects
with CF and 15 subjects with CB. We also evaluated changes in these
measurements in paired sputum samples from these subjects obtained 2 to 12
months apart. RESULTS: IL-8 and MPO concentrations in the sputum of CF subjects
was inversely correlated with FEV(1) percent predicted (IL-8: r = -0.40; p =
0.003; MPO: r = -0.38; p = 0.003) and FVC percent predicted (IL-8: r = -0.4; p
= 0.02; MPO: r = -0.4; p = 0.02). IL-8 and DNA concentrations were inversely
correlated with sputum cough transportability (CTR) [IL-8: r = -0.4; p = 0.02;
DNA: r = -0.36; p = 0.048]. Changes in DNA concentration in sputum samples from
CF subjects over time were inversely correlated with changes in FEV(1) percent
predicted (r = -0.58; p = 0.02), FVC percent predicted (r = -0.74; p = 0.002),
and CTR (r = -0.59; p = 0.02). There was no correlation among pulmonary
function, sputum properties, and inflammatory markers in the sputum from
subjects with CB. CONCLUSIONS: The sputum concentrations of IL-8, MPO, and DNA
appear to be closely associated with pulmonary function in subjects with CF but
not in subjects with CB.1



Prompt detection and treatment of
lower respiratory tract infection are essential in the management of patients
with cystic fibrosis (CF), who often have signs or symptoms of respiratory
infection without any pathogens being isolated from sputum or cough swab
specimens. The aims of this study were to assess the efficacy and clinical
value of obtaining sputum and oropharyngeal cough swab samples following
induction with hypertonic saline (HS) in this group of patients. Forty-three
outpatients with CF, mean age 7.2 years (range, 1.8-12.9 years), were recruited
over a 2-year period. Nebulized salbutamol was administered, followed by 6% HS.
Sputum was preferentially obtained before and after HS induction if possible.
If the patient was not able to expectorate, oropharyngeal cough swabs were
taken instead. Four patients were able to expectorate sputum before and 19
after HS induction. The procedure was tolerated in 41 of 43 patients. Pathogens
were isolated from 13 patients' HS-induced samples, but not from their
corresponding preinduced specimens, and 4 patients' preinduced specimens
cultured organisms which were not identified from their HS-induced samples.
Significant changes were made in the management of 13 (30.2%) patients directly
resulting from the positive culture of pathogens only from HS-induced samples.
Cultures from oropharyngeal cough swab or expectorated sputum specimens
following inhalation of HS provide additional microbiological information which
is of clinical value and may lead to changes in patient management.2



Young children with cystic fibrosis
(CF) are generally very well, cough free and non-productive, and are often
incapable of spontaneously expectorating sputum even if actively coughing
during an exacerbation. Obtaining a meaningful airway sample for
microbiological analysis is therefore problematic, yet essential if lower airway
infection is to be detected and adequately treated. Recently there has been
increasing interest in the use of sputum-induction in young children with CF,
as a simple, cost effective, well tolerated and frequently repeatable approach
to sampling the lower airway, and the relative merits of this approach to
bacterial sampling are discussed. Culture-independent microbiology has
increased our understanding of the respiratory microbiota and has challenged
the current paradigm of "single pathogen causes disease".
Understanding how to diagnose infection using these new, highly sensitive
technologies will be important. How we should best intervene to optimise,
manipulate and prevent disruption of the respiratory microbiota is likely to
greatly influence how we manage infection in the future.3









TREATMENT

BACKGROUND AND PURPOSE: Drug treatment
of cystic fibrosis (CF) is associated with significant costs. To help ensure
sustainable care, this study assesses the costs associated with outpatient
treatment of adult CF patients in Germany. It identifies main cost drivers,
evaluates the potential for cost savings from "aut idem" substitution
and presents a projection of lifelong medication costs. METHODS: The analysis
is based on a complete set of prescriptions for adult CF patients from the
outpatient clinic of the university hospital of Frankfurt am Main during 2007
(n = 124 patients). Annual treatment costs were calculated on the basis of the
"Rote Liste", while the potential for cost savings from "aut
idem" drug substitution was obtained through ABDATA Pharma Data Service.
RESULTS: The annual outpatient drug costs for an adult patient with CF averages
euro 17,219 (n = 124), which increases to euro 21,782 if i.v. therapies are
included. With an average life expectancy at birth of 39.7 years, total
lifetime drug treatment costs amount to euro 824,159 (reference year 2007,
inflation rate 2.7%, 3% discount rate). "Aut idem" substitution with
cheaper drugs could reduce pharmaceutical expenditures by 4.1%. CONCLUSION: Our
results confirm the costly nature of drug treatment for CF patients, both on an
annual and in particular on a lifelong basis. At the same time, the potential
for cost savings through "aut idem" substitution with cheaper drugs
remains limited. The added transparency around a small set of costdriving drugs,
which is offered in this study, represents a solid contribution to assess
treatment choices and financing options to help secure adequate yet sustainable
care for CF patients.4



Pseudomonas aeruginosa is a
non-capsulate and non-sporing gram-negative bacillus that most commonly affects
the lower respiratory system in humans. Burkholderia (previously Pseudomonas)
cepacia has emerged as an important respiratory pathogen in patients with
cystic fibrosis (CF). The ability of P. aeruginosa to persist and multiply in
moist environments and equipment, such as humidifiers in hospital wards,
bathrooms, sinks and kitchens, maybe of importance in cross-infection. P.
aeruginosa infections of the lower respiratory tract can range in severity from
colonisation (without an immunological response) to a severe necrotising
bronchopneumonia. Infection is seen in patients with CF and other chronic lung
diseases such as non-CF bronchiectasis. In patients with CF, once P. aeruginosa
is established in the airways it is almost impossible to eradicate, but prior
to this, aggressive treatment can delay the development of chronic infection.
30 to 40% of the present paediatric population with CF will have chronic
pseudomonal infection. B. cepacia has a particular predisposition to infect
patients with CF and may be distinguished from P. aeruginosa by accelerated
lung disease in about one- third of patients. Overwhelming septicaemia and
necrotising pneumonia are well described (cepacia syndrome); events that are
rare with P. aeruginosa. With the propensity for social cross-infection,
segregation policies have been accepted as means of controlling outbreaks. A
number of antipseudomonal agents are available. The most commonly used are the
extended-spectrum penicillins, aminoglycosides, cephalosporins,
fluoroquinolones, polymixins and the monobactams. An aminoglycoside with a
beta-lactam penicillin is usually considered to be the first line treatment. No
trial has shown any significant clinical advantage of any particular
combination regimen over another. The emergence of resistance continues to be a
concern. Pipericillin, piperacillin/tazobactam and meropenem have good but
equivalent antibacterial activity against P. aeruginosa. However, B. cepacia is
characterised by in vitro resistance to colistin (colomycin), aminoglycosides
and ciprofloxacin but better susceptibility to ceftazidime. Nebulised delivery
of antipseudomonal antibiotics is thought to prevent recurrent exacerbations,
reduce antibiotic usage and maintain lung function, particularly in patients
with CF. Colistin, tobramycin and gentamicin are currently the most commonly
prescribed nebulised antibiotics. Much effort is directed at treating chronic
P. aeruginosa infection but as chronic infection is seldom if ever eradicated
when first established, prevention is preferable. Early intensive treatment for
P. aeruginosa infection is advocated in order to maintain pulmonary function
and postpone the onset of chronic P. aeruginosa infection.5



AIM: It was the aim of the study to
develop self-emulsifying drug delivery systems (SEDDS) with the ability to
change their zeta potential towards higher values at the adsorption membrane
and in this way facilitate the release of the DNA-cetrimonium complex and
enhance transfection. METHODS: Plasmid DNA was complexed via hydrophobic ion
pairing utilizing various surfactants and the complex was incorporated into
SEDDS achieving a payload of 1% (m/v). Log PSEDDS/water of the complex was
determined. SEDDS were characterized regarding droplet size, zeta potential,
stability and toxicity. Alkaline phosphatase presented in the sputum of cystic
fibrosis patients was quantified using 4-nitrophenyl phosphate disodium salt
and 5-bromo-4-chloro-1H-indol-3-yl phosphate dipotassium salt as substrates.
SEDDS containing 0.4% (m/v) 1,2-dipalmitoyl-sn-glycero-3-phosphate monosodium
salt were characterized regarding their zeta potential changing properties
utilizing isolated alkaline phosphatase and cystic fibrosis sputum. The mucus
permeating properties of SEDDS were evaluated via Transwell method using cystic
fibrosis sputum. Finally, the transfection efficiency of incorporated plasmid
DNA was investigated. RESULTS: Cetrimonium bromide showed the highest
precipitation efficiency of 99.5+/-2.72% for the complexation of pDNA. SEDDS
containing propylene glycol, Capmul PG-8, Captex 300, Captex 355, Captex 8000,
Cremophor EL, Cremophor RH-40 and Brij O10 showed stable emulsions with a
droplet size between 20 and 100nm and zeta potential <-3mV over 4h. SEDDS
demonstrated highly protective effect against enzymatic degradation and
moderate cell viability on freshly obtained pulmonary tissue. The
pDNA-cetrimonium complex incorporated into SEDDS revealed a log PSEDDS/water of
about 2. A concentration of 0.879+/-0.103U/g alkaline phosphatase was found in
the sputum of cystic fibrosis patients. SEDDS containing
1,2-dipalmitoyl-sn-glycero-3-phosphate monosodium salt showed a high potential
of changing the zeta potential by applying isolated alkaline phosphatase as
well as cystic fibrosis sputum along with high mucus permeating properties.
Formulation C demonstrated the highest transfection efficiency with a 7.2-fold
increased fluorescence intensity compared to naked pDNA. CONCLUSION: The novel
developed zeta potential changing SEDDS are opening versatile opportunities for
the treatment of cystic fibrosis caused by gene mutation.6



The European Pediatric Pharmaceutical
Legislation wants children to benefit more from pharmaceutical progress. In
rare diseases, concerns have been raised that this legislation might damage
research and stymie drug development. We discuss the role of the European
Medicines Agency (EMA) and its Pediatric Committee (PDCO) in the development of
ivacaftor, first-in-class for cystic fibrosis (CF) patients with the G551D
mutation (and eight other mutations later) and of lumacaftor and ataluren, two
more potential break-through CF medications. Ivacaftor was USA-approved early
2012 and six months later in the EU. Registration was based on the same data.
We analyzed these drugs' EU pediatric investigation plans (PIPs) and compared
the PIP-studies with the pediatric CF studies listed in www.clinicaltrials.gov.
The ivacaftor PIP studies appear to reflect what the developer planned anyway,
apart from a study in 1-23-month-olds, which has not yet started. The total
negotiation time for the current PIP version was approximately 5.5 years. For
companies that develop drugs in pediatric diseases, e.g., CF, PIPs represent
considerable additional procedural workload with minimal or no additional
benefit for the patients. New drugs for pediatric diseases should not be
hampered by additional, unnecessary and costly bureaucracy, but be registered
as rapidly as possible without compromising safety.7









1. Kim JS, Okamoto K,
Rubin BK. Pulmonary function is negatively correlated with sputum inflammatory
markers and cough clearability in subjects with cystic fibrosis but not those
with chronic bronchitis. Chest. 2006;129(5):1148-1154.

2. Ho SA, Ball R, Morrison LJ, Brownlee
KG, Conway SP. Clinical value of obtaining sputum and cough swab samples
following inhaled hypertonic saline in children with cystic fibrosis. Pediatr Pulmonol. 2004;38(1):82-87.

3. Forton JT. Detecting respiratory
infection in children with cystic fibrosis: Cough swab, sputum induction or
bronchoalveolar lavage. Paediatr Respir
Rev. 2019;31:28-31.

4. Baltin CT, Smaczny C, Wagner TO.
[Drug treatment of cystic fibrosis - cost patterns and savings potential for
outpatient treatment]. Med Klin (Munich).
2010;105(12):887-900.

5. Banerjee D, Stableforth D. The
treatment of respiratory pseudomonas infection in cystic fibrosis: what drug
and which way? Drugs. 2000;60(5):1053-1064.

6. Griesser J, Hetenyi G, Federer C, et
al. Highly mucus permeating and zeta potential changing self-emulsifying drug
delivery systems: A potent gene delivery model for causal treatment of cystic
fibrosis. Int J Pharm. 2019;557:124-134.

7. Rose K, Spigarelli MG. Cystic
Fibrosis Treatment: A Paradigm for New Pediatric Medicines, Globalization of
Drug Development and the Role of the European Medicines Agency. Children (Basel). 2015;2(1):108-130.


NB: JUST ONE TIP: I have just copied and pasted abstracts after a couple of searches on Endnote - I WOULD ADVISE YOU TO WRITE YOUR ASSINMENT ION YOUR OWN WORDS - the unis/schools have very sophisticated ways of detecting plagiarism).
0
reply
therisktaker247
Badges: 6
Rep:
?
#3
Report Thread starter 1 year ago
#3
(Original post by macpatgh-Sheldon)
If you are asking someone to write 3000 words for you, you must be in cuckooland, man - having said that, I too live dangerously, which makes two of us, so I will provide some info below for you. Go through it and see what you think is useful.

:goodluck:


SYMPTOMS

BACKGROUND: Polymorphonuclear
neutrophil (PMN)-dominated inflammation is prominent in the airways of subjects
with cystic fibrosis (CF) and chronic bronchitis (CB). Interleukin (IL)-8,
myeloperoxidase (MPO), and DNA are markers of neutrophilic inflammation. We
hypothesized that sputum MPO, DNA, and IL-8 concentrations would negatively
correlate with pulmonary function and sputum transportability. METHODS: We
measured pulmonary function and analyzed sputum IL-8, MPO, and DNA concentrations,
as well as the transport properties of sputum samples obtained from 16 subjects
with CF and 15 subjects with CB. We also evaluated changes in these
measurements in paired sputum samples from these subjects obtained 2 to 12
months apart. RESULTS: IL-8 and MPO concentrations in the sputum of CF subjects
was inversely correlated with FEV(1) percent predicted (IL-8: r = -0.40; p =
0.003; MPO: r = -0.38; p = 0.003) and FVC percent predicted (IL-8: r = -0.4; p
= 0.02; MPO: r = -0.4; p = 0.02). IL-8 and DNA concentrations were inversely
correlated with sputum cough transportability (CTR) [IL-8: r = -0.4; p = 0.02;
DNA: r = -0.36; p = 0.048]. Changes in DNA concentration in sputum samples from
CF subjects over time were inversely correlated with changes in FEV(1) percent
predicted (r = -0.58; p = 0.02), FVC percent predicted (r = -0.74; p = 0.002),
and CTR (r = -0.59; p = 0.02). There was no correlation among pulmonary
function, sputum properties, and inflammatory markers in the sputum from
subjects with CB. CONCLUSIONS: The sputum concentrations of IL-8, MPO, and DNA
appear to be closely associated with pulmonary function in subjects with CF but
not in subjects with CB.1



Prompt detection and treatment of
lower respiratory tract infection are essential in the management of patients
with cystic fibrosis (CF), who often have signs or symptoms of respiratory
infection without any pathogens being isolated from sputum or cough swab
specimens. The aims of this study were to assess the efficacy and clinical
value of obtaining sputum and oropharyngeal cough swab samples following
induction with hypertonic saline (HS) in this group of patients. Forty-three
outpatients with CF, mean age 7.2 years (range, 1.8-12.9 years), were recruited
over a 2-year period. Nebulized salbutamol was administered, followed by 6% HS.
Sputum was preferentially obtained before and after HS induction if possible.
If the patient was not able to expectorate, oropharyngeal cough swabs were
taken instead. Four patients were able to expectorate sputum before and 19
after HS induction. The procedure was tolerated in 41 of 43 patients. Pathogens
were isolated from 13 patients' HS-induced samples, but not from their
corresponding preinduced specimens, and 4 patients' preinduced specimens
cultured organisms which were not identified from their HS-induced samples.
Significant changes were made in the management of 13 (30.2%) patients directly
resulting from the positive culture of pathogens only from HS-induced samples.
Cultures from oropharyngeal cough swab or expectorated sputum specimens
following inhalation of HS provide additional microbiological information which
is of clinical value and may lead to changes in patient management.2



Young children with cystic fibrosis
(CF) are generally very well, cough free and non-productive, and are often
incapable of spontaneously expectorating sputum even if actively coughing
during an exacerbation. Obtaining a meaningful airway sample for
microbiological analysis is therefore problematic, yet essential if lower airway
infection is to be detected and adequately treated. Recently there has been
increasing interest in the use of sputum-induction in young children with CF,
as a simple, cost effective, well tolerated and frequently repeatable approach
to sampling the lower airway, and the relative merits of this approach to
bacterial sampling are discussed. Culture-independent microbiology has
increased our understanding of the respiratory microbiota and has challenged
the current paradigm of "single pathogen causes disease".
Understanding how to diagnose infection using these new, highly sensitive
technologies will be important. How we should best intervene to optimise,
manipulate and prevent disruption of the respiratory microbiota is likely to
greatly influence how we manage infection in the future.3









TREATMENT

BACKGROUND AND PURPOSE: Drug treatment
of cystic fibrosis (CF) is associated with significant costs. To help ensure
sustainable care, this study assesses the costs associated with outpatient
treatment of adult CF patients in Germany. It identifies main cost drivers,
evaluates the potential for cost savings from "aut idem" substitution
and presents a projection of lifelong medication costs. METHODS: The analysis
is based on a complete set of prescriptions for adult CF patients from the
outpatient clinic of the university hospital of Frankfurt am Main during 2007
(n = 124 patients). Annual treatment costs were calculated on the basis of the
"Rote Liste", while the potential for cost savings from "aut
idem" drug substitution was obtained through ABDATA Pharma Data Service.
RESULTS: The annual outpatient drug costs for an adult patient with CF averages
euro 17,219 (n = 124), which increases to euro 21,782 if i.v. therapies are
included. With an average life expectancy at birth of 39.7 years, total
lifetime drug treatment costs amount to euro 824,159 (reference year 2007,
inflation rate 2.7%, 3% discount rate). "Aut idem" substitution with
cheaper drugs could reduce pharmaceutical expenditures by 4.1%. CONCLUSION: Our
results confirm the costly nature of drug treatment for CF patients, both on an
annual and in particular on a lifelong basis. At the same time, the potential
for cost savings through "aut idem" substitution with cheaper drugs
remains limited. The added transparency around a small set of costdriving drugs,
which is offered in this study, represents a solid contribution to assess
treatment choices and financing options to help secure adequate yet sustainable
care for CF patients.4



Pseudomonas aeruginosa is a
non-capsulate and non-sporing gram-negative bacillus that most commonly affects
the lower respiratory system in humans. Burkholderia (previously Pseudomonas)
cepacia has emerged as an important respiratory pathogen in patients with
cystic fibrosis (CF). The ability of P. aeruginosa to persist and multiply in
moist environments and equipment, such as humidifiers in hospital wards,
bathrooms, sinks and kitchens, maybe of importance in cross-infection. P.
aeruginosa infections of the lower respiratory tract can range in severity from
colonisation (without an immunological response) to a severe necrotising
bronchopneumonia. Infection is seen in patients with CF and other chronic lung
diseases such as non-CF bronchiectasis. In patients with CF, once P. aeruginosa
is established in the airways it is almost impossible to eradicate, but prior
to this, aggressive treatment can delay the development of chronic infection.
30 to 40% of the present paediatric population with CF will have chronic
pseudomonal infection. B. cepacia has a particular predisposition to infect
patients with CF and may be distinguished from P. aeruginosa by accelerated
lung disease in about one- third of patients. Overwhelming septicaemia and
necrotising pneumonia are well described (cepacia syndrome); events that are
rare with P. aeruginosa. With the propensity for social cross-infection,
segregation policies have been accepted as means of controlling outbreaks. A
number of antipseudomonal agents are available. The most commonly used are the
extended-spectrum penicillins, aminoglycosides, cephalosporins,
fluoroquinolones, polymixins and the monobactams. An aminoglycoside with a
beta-lactam penicillin is usually considered to be the first line treatment. No
trial has shown any significant clinical advantage of any particular
combination regimen over another. The emergence of resistance continues to be a
concern. Pipericillin, piperacillin/tazobactam and meropenem have good but
equivalent antibacterial activity against P. aeruginosa. However, B. cepacia is
characterised by in vitro resistance to colistin (colomycin), aminoglycosides
and ciprofloxacin but better susceptibility to ceftazidime. Nebulised delivery
of antipseudomonal antibiotics is thought to prevent recurrent exacerbations,
reduce antibiotic usage and maintain lung function, particularly in patients
with CF. Colistin, tobramycin and gentamicin are currently the most commonly
prescribed nebulised antibiotics. Much effort is directed at treating chronic
P. aeruginosa infection but as chronic infection is seldom if ever eradicated
when first established, prevention is preferable. Early intensive treatment for
P. aeruginosa infection is advocated in order to maintain pulmonary function
and postpone the onset of chronic P. aeruginosa infection.5



AIM: It was the aim of the study to
develop self-emulsifying drug delivery systems (SEDDS) with the ability to
change their zeta potential towards higher values at the adsorption membrane
and in this way facilitate the release of the DNA-cetrimonium complex and
enhance transfection. METHODS: Plasmid DNA was complexed via hydrophobic ion
pairing utilizing various surfactants and the complex was incorporated into
SEDDS achieving a payload of 1% (m/v). Log PSEDDS/water of the complex was
determined. SEDDS were characterized regarding droplet size, zeta potential,
stability and toxicity. Alkaline phosphatase presented in the sputum of cystic
fibrosis patients was quantified using 4-nitrophenyl phosphate disodium salt
and 5-bromo-4-chloro-1H-indol-3-yl phosphate dipotassium salt as substrates.
SEDDS containing 0.4% (m/v) 1,2-dipalmitoyl-sn-glycero-3-phosphate monosodium
salt were characterized regarding their zeta potential changing properties
utilizing isolated alkaline phosphatase and cystic fibrosis sputum. The mucus
permeating properties of SEDDS were evaluated via Transwell method using cystic
fibrosis sputum. Finally, the transfection efficiency of incorporated plasmid
DNA was investigated. RESULTS: Cetrimonium bromide showed the highest
precipitation efficiency of 99.5+/-2.72% for the complexation of pDNA. SEDDS
containing propylene glycol, Capmul PG-8, Captex 300, Captex 355, Captex 8000,
Cremophor EL, Cremophor RH-40 and Brij O10 showed stable emulsions with a
droplet size between 20 and 100nm and zeta potential <-3mV over 4h. SEDDS
demonstrated highly protective effect against enzymatic degradation and
moderate cell viability on freshly obtained pulmonary tissue. The
pDNA-cetrimonium complex incorporated into SEDDS revealed a log PSEDDS/water of
about 2. A concentration of 0.879+/-0.103U/g alkaline phosphatase was found in
the sputum of cystic fibrosis patients. SEDDS containing
1,2-dipalmitoyl-sn-glycero-3-phosphate monosodium salt showed a high potential
of changing the zeta potential by applying isolated alkaline phosphatase as
well as cystic fibrosis sputum along with high mucus permeating properties.
Formulation C demonstrated the highest transfection efficiency with a 7.2-fold
increased fluorescence intensity compared to naked pDNA. CONCLUSION: The novel
developed zeta potential changing SEDDS are opening versatile opportunities for
the treatment of cystic fibrosis caused by gene mutation.6



The European Pediatric Pharmaceutical
Legislation wants children to benefit more from pharmaceutical progress. In
rare diseases, concerns have been raised that this legislation might damage
research and stymie drug development. We discuss the role of the European
Medicines Agency (EMA) and its Pediatric Committee (PDCO) in the development of
ivacaftor, first-in-class for cystic fibrosis (CF) patients with the G551D
mutation (and eight other mutations later) and of lumacaftor and ataluren, two
more potential break-through CF medications. Ivacaftor was USA-approved early
2012 and six months later in the EU. Registration was based on the same data.
We analyzed these drugs' EU pediatric investigation plans (PIPs) and compared
the PIP-studies with the pediatric CF studies listed in www.clinicaltrials.gov.
The ivacaftor PIP studies appear to reflect what the developer planned anyway,
apart from a study in 1-23-month-olds, which has not yet started. The total
negotiation time for the current PIP version was approximately 5.5 years. For
companies that develop drugs in pediatric diseases, e.g., CF, PIPs represent
considerable additional procedural workload with minimal or no additional
benefit for the patients. New drugs for pediatric diseases should not be
hampered by additional, unnecessary and costly bureaucracy, but be registered
as rapidly as possible without compromising safety.7









1. Kim JS, Okamoto K,
Rubin BK. Pulmonary function is negatively correlated with sputum inflammatory
markers and cough clearability in subjects with cystic fibrosis but not those
with chronic bronchitis. Chest. 2006;129(5):1148-1154.

2. Ho SA, Ball R, Morrison LJ, Brownlee
KG, Conway SP. Clinical value of obtaining sputum and cough swab samples
following inhaled hypertonic saline in children with cystic fibrosis. Pediatr Pulmonol. 2004;38(1):82-87.

3. Forton JT. Detecting respiratory
infection in children with cystic fibrosis: Cough swab, sputum induction or
bronchoalveolar lavage. Paediatr Respir
Rev. 2019;31:28-31.

4. Baltin CT, Smaczny C, Wagner TO.
[Drug treatment of cystic fibrosis - cost patterns and savings potential for
outpatient treatment]. Med Klin (Munich).
2010;105(12):887-900.

5. Banerjee D, Stableforth D. The
treatment of respiratory pseudomonas infection in cystic fibrosis: what drug
and which way? Drugs. 2000;60(5):1053-1064.

6. Griesser J, Hetenyi G, Federer C, et
al. Highly mucus permeating and zeta potential changing self-emulsifying drug
delivery systems: A potent gene delivery model for causal treatment of cystic
fibrosis. Int J Pharm. 2019;557:124-134.

7. Rose K, Spigarelli MG. Cystic
Fibrosis Treatment: A Paradigm for New Pediatric Medicines, Globalization of
Drug Development and the Role of the European Medicines Agency. Children (Basel). 2015;2(1):108-130.


NB: JUST ONE TIP: I have just copied and pasted abstracts after a couple of searches on Endnote - I WOULD ADVISE YOU TO WRITE YOUR ASSINMENT ION YOUR OWN WORDS - the unis/schools have very sophisticated ways of detecting plagiarism).
Thank you so much, this was very helpful. Also, I didn’t mean write the essay for me, I just Needed some advice on what to talk about in the essay
0
reply
X

Quick Reply

Attached files
Write a reply...
Reply
new posts
Back
to top
Latest
My Feed

See more of what you like on
The Student Room

You can personalise what you see on TSR. Tell us a little about yourself to get started.

Personalise

What factors affect your mental health the most right now?

Anxiousness about lockdown easing (175)
4.95%
Uncertainty around my education (517)
14.63%
Uncertainty around my future career prospects (393)
11.12%
Lack of purpose or motivation (490)
13.87%
Lack of support system (eg. teachers, counsellors, delays in care) (172)
4.87%
Impact of lockdown on physical health (214)
6.06%
Loneliness (300)
8.49%
Financial worries (129)
3.65%
Concern about myself or my loves ones getting/having been ill (140)
3.96%
Exposure to negative news/social media (160)
4.53%
Lack of real life entertainment (197)
5.58%
Lack of confidence in making big life decisions (317)
8.97%
Worry about missed opportunities during the pandemic (329)
9.31%

Watched Threads

View All