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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!

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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1st September 2017



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