Why I think rare disease companies are more patient-centric
Guest blogger Kamlesh Sheth believes companies that specialise in rare disease tend to be more patient-centric.
All pharmaceutical/biotech companies
are of course, in essence, patient-centric. However, I feel that companies
specialising in rare diseases are often more patient-centric.
It’s important to mention what I mean by patient-centric –
that is – directing the company’s resources mainly to the activities which
patients consider important. Moreover, ensuring that company’s culture as well
processes are conducive to dealing with patients’ needs and being flexible as
the needs change.
The company size plays a big role. Rare disease companies are generally
smaller, and this can allow individualised focus and dedication. The company’s
(real) mission – historically the rare disease companies have started off as
focused – often on a single disease – so their mission was to find an effective
and safe treatment for a particular disease.
The funding source of specialised companies also comes into
play – historically many rare disease companies been funded by certain people
or organisations who believed in doing some good for rare diseases community
which have been traditionally neglected. The same tends to apply to new
specialised companies being set up now. On this note, from my experiences, I
think the type of employees who chose to work for rare disease company is often
key. Rare disease companies perhaps tend to attract employees who consider
themselves too, patient-centric.
Considering processes within companies, larger company processes tend to
be much more complex – and this sometimes takes the
decision-making away from the grassroots. Perhaps in this way it is easier
for smaller, rare-disease companies to be quicker and more adaptable and thus
better able to meet patients’ changing needs.
Rare diseases and patient-centricity go hand in hand and need
adaptability at each stage – discovery, clinical trials, regulatory, marketing,
post-marketing and financing. Perchance the bigger the company, the harder it
is to be adaptable at every stage. To be truly patient-centric, the whole chain
would need to function as patient-centric whilst larger companies can often
look to being patient-centric in post-marketing phase.
In medical training we are taught very early on that paediatric medicine is
not just miniature adult medicine – the difference between the two is not just
due to size but also due to difference in anatomy, physiology, psychology
between adults and children. The same concept applies to the rare disease
community. The companies specialising in rare diseases know this, whilst
perhaps those outside tend to think of it is as difference in scale – and maybe
that is why it can be harder for them to be as patient-centric. I
hardly hear the word ‘patient-centric’ in rare disease companies – because it
is just natural to them whilst other companies do sometimes remind themselves
that they need to be patient-centric!
Author:
Kamlesh Sheth, Director of PharmacoMedics, is a physician with over 25 years of
experience in healthcare, including within clinical medicine, academic
research, pharmaceutical & the med-tech industry and healthcare
entrepreneurship. His interests span orphan and ultra-orphan diseases, medical
devices and digital healthcare. He has experience working across the world in
Asia, Europe and the USA.
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