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International Clinical Trials
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Paediatric trials not only present a huge challenge – both
scientifically and ethically – they are also exceptionally demanding
from a logistic perspective. After several false starts, the market is
gaining traction with ‘the FDA rule’ finalised in 1998 – essentially
requiring all new products to undertake paediatric registration.
Global Metrics
It
has been a turbulent decade in the clinical trial environment – as it
has in other sectors – driven by the global recession, which had a
profound effect on R&D investment. Pharma R&D growth in terms of
spend dropped from historical figures of 12-14% per annum into negative
figures for the first time in living memory. Many predictions indicate
growth may not recover to beyond 5% per annum anytime soon or, indeed,
ever.
Figure 1 (see full PDF) shows that in total, the number of
clinical trials initiated between 2006 and 2014 has grown by only 7%.
Looking specifically at the paediatric market, this has in fact fared
even worse, showing a decline of -1.5% in the number of trials taking
place from 2006 to 2014. However, the make-up of the paediatric clinical
trial pipeline has changed considerably. When looking specifically at
rare diseases in paediatrics, there has been a 94% growth in the number
of trials initiated in the same period.
Across the entire
R&D pipeline, the number of indications has grown by 12%,
outstripped by a growth in paediatric indications of 31%. This is the
result of, one assumes, a sharp shift towards cost control in
pharmaceutical and biotech companies’ R&D budgets, resulting in
their investment being spread over a much larger set of indications,
many of which are rare or orphan types.
Figures 2 and 3 throw a
bit more light on these trends. Figure 2 (see full PDF) shows that in
2006, of the top 10 indications initiated, 60% were infectious diseases
and 20% were psychiatric indications – with only 10% being rare diseases
(diphtheria prophylaxis). Figure 3 (see full PDF) implies that by 2014,
the basic therapeutic areas had shifted slightly to 50% infectious
disease and 30% psychiatric indications, due to the entry of paediatric
bipolar disease into the list. More importantly, it also shows that
three of the top 10 paediatric indications had become rare diseases –
duchenne muscular dystrophy, meningococcal infections and diptheria
prophylaxis – ranking fifth, sixth and seventh respectively. It can also
be seen that the top 10 rare diseases in 2014 were 50% gene defects and
30% infectious disease.
This indicates that the pattern of
research in paediatric indications is evolving away from traditional
concepts of childhood disease into the newer and, perhaps, more
contentious areas of psychiatry, and within the rare disease category,
gene defects are now predominating. It seems clear from these metrics
that the regulatory push to increase the paediatric pharmacopoeia is
working. However, sociologically, the types of mainstream conditions
being addressed may be controversial – attention deficit (hyperactivity)
disorder (ADD/ADHD), depression, human papilloma virus (which only
enters the lists in 2015, so is not reported here) all challenge
societies’ attitudes to the normal mental and sexual behaviour of
children. Perhaps this is inevitable as this process redefines childhood
disease by making therapies available, throwing a medical spotlight on
where challenges exist – which society may struggle to accept.
The
heightened interest in paediatric clinical trials has manifested itself
in challenging and complex pieces of work. In particular, they have
required long patient visits for multiple pharmacokinetic blood sampling
in the earlier phases of clinical research, or the home administration
of complex biologicals as subcutaneous injections or infusions. The
specialist nature of these trials, in addition to the fragile patient
population, has meant that home clinical trial support is an invaluable
resource for paediatric clinical trials.
As for paediatric
studies in indications that are not rare, or do not require parenteral
investigational medicinal product (IMP) administration, the trends are
less obvious. In 2014, more than 4,660 trials were initiated, with just
over 5% being paediatric. The numbers are too small to observe whether
these studies are as yet increasing their utilisation of in-home
clinical trial visits.
Study Results
Research
attempted to find out why clinical trial sponsors use home healthcare
services. Using the definition of ‘patient-centric’ as any element of a
trial that is designed to appeal to patients in order to make it easier
for them to take part in the study, these reasons can be analysed as
follows:
- To improve recruitment and retention of patients in
trials. Providing the option to be seen at home recognises that the
patients have a choice to participate in clinical studies. Giving
patients the opportunity to undertake some significant elements of the
trial in their own home is intuitively attractive to them, and will thus
make it easier for them to consent to join and more likely to remain in
the trial
- It is expected the drug will be administered in the
home when marketed. This is a scientific objective, but derives directly
from a marketing patient-centric objective. One way a biopharma company
can make their product more desirable when on the market is to make it
suitable for use in the home, and so it is logical for them to research
it in the same environment
- To make the lives of the patients
better in return for participating in the research. Some companies seem
to simply want to reduce the impact of the trial on the patient to
enhance the relationship they have with them, which they see as
important and worth investment in its own right
- To allow access
to patients who would otherwise find it hard to participate. Some
companies want to broaden access to their research products, and find
these services are a good way of promoting access to patients who would
otherwise struggle to participate, and thus gain access to more
experimental medicines
Patient-Centric Approach
Children
need care in the home to prevent them from losing time in school, and
to allow their parents to continue working, so everyone can lead as
normal a life as possible. They also need to be treated, where possible,
in a way that allows them to feel ‘normal’ without multiple visits to
the hospital.
There are specific considerations in home
healthcare in paediatric trials. For one, an enhanced model is needed
due to stronger bonds between the treating doctors, their teams, the
child and their family. Home healthcare introduces another party into
this complex set of inter-relationships, which all the stakeholders view
as critically important. This leads to greater demands on the home
healthcare providers than in standard models, particularly in terms of
error rates and the quality of communication. The sources of error in
home healthcare can be, for example, the inability to find a nurse near
the patient with availability in their schedule; a visit interrupted by
weather or traffic; the nurse being unwell; a fault in the equipment;
disruption in the investigational medicinal product cold chain; or a
failure in venepuncture or cannulation.
Personalised Touch
In
today’s world, anything is possible: more intense models of nursing
care than those mentioned for paediatric studies can be operated in
highly complex trials in adults; full-time employed nurses can be
utilised, who work internationally to support a specific clinical trial,
and are permanently ‘on call’ for the trial in whatever trial site or
patient location is required; staff may provide patient care where their
licence permits, but in other countries or states, they simply train
and advise local site staff on the trial-specific elements of very
complex studies, or maintain highly complex equipment to reduce
variability in the trial.
The paediatric R&D clinical trial
pipeline is changing, showing significant growth in the number and
proportion of rare diseases and products that require parenteral
administration. Analysis shows there is significant demand for home
healthcare services in such studies, simultaneously creating more
complex demands on the service provider, as the objectives of the
sponsor and site focus more on the patient experience.
It is
predicted that high-intensity and basic home trial support models will
be increasingly applied to paediatric trials in the future, as patient
experience becomes an ever-more important factor.
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