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Resolve to prioritise rare diseases treatments may weaken as prices hinder 'value for money' decision making, patient advocate warns

by Natalie Morrison
LONDON, 15 May (APM) - The will to prioritise rare diseases treatments could be weakening due to some "grossly over priced" treatments and the resulting access issues from "value for money" based decision making, rare disease patient Rebecca Tvedt Skarberg said.
Skarberg - an adviser to the patient organiation the Osteogenesis Imperfecta Federation Europe (OIFE), Norway - was speaking at the European Conference on Rare Diseases and Orphan Products (ECRD) on Thursday, where she agreed with other experts that rare diseases are now at the forefront of policymakers' decision processes more than ever before.
There is now more recognition of the problems faced by patients with rare diseases and more treatments available which can increase life choices and opportunities, she said.
"But we also see some very scary developments, such as that our treatment is being grossly overpriced and is in the hands of the few instead of the many," she said.
She went on to comment on resulting access issues, calling access a "mind game" between private stakeholders and public funding structures.
"It's a balancing act of priorities and it makes us who live with these rare conditions sick to our stomach," she said, blaming politicians and bureaucrats for putting price tags on lives and trying to calculate what will give them most value for money.
This is problematic since "my life doesn't really fit into a spreadsheet", she said.
"If there is a medicine out there, how dare someone say that our life is not worth the price of it. Is my health less worth than yours? I refrain from putting price tags on lives and treating medicine like it was some kind of lottery," she continued.
Echoing other experts at the conference, she added that new and more effective treatments will not do any good if they "sit on a shelf while companies argue".
The issues are dividing patient organisations and weakening their joint missions, she added.
"Maybe the will to recognise the need to prioritise rare disease is weakening," she warned.

Future scenarios

Skarberg went on to criticise unfettered belief in the ability of technology to treat rare diseases - a belief that "tech will save you".
"Private companies are selling the fantasy that you can pay your way out of poor health. The worse the prognosis, the easier it is to offer false hope and dreams at a top price," she said. "This kind of development is certainly not sustainable in the long run, and if tech will save you, it requires you to give up a lot of data."
She aired concerns over patient data sharing. "Of course we are all for the development of big data, but who owns this data and how do we ensure safety of this data in the future?" she asked.
"If the price is right, out data can fall into the hands of people who want to force our lives down these financial spreadsheets again. But without data collection, maybe we will all be back to square one."
The way forward, she suggested, is to make innovation a joint responsibility, with development that "leaves no one behind," in line with the United Nation's 2030 sustainability goals (APMHE 67333).
Priorities should be made together with the understanding each stakeholder depends on each other.
"Rare disease can happen to anyone and will always exist," she said. "The impact of a rare disease cannot be lessened all by yourself, or with lots of euros, it can only be managed together. […] The lives of people with a rare disease cannot be measured and forced into spreadsheets."
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