Decentralised clinical trials, a potential solution for the complexity of rare disease clinical trials, but what impact may place have?

Since the commencement of our project, SPINs have increasingly taken the main stage in a variety of conferences in Canada.

From Big Pharma to Commons, insight into social innovation in rare disease from a French perspective, a book written by Gaëlle Krikorian

In our latest blog post, we look at the role of Health Technology Assessments in the distribution and allocation of health resources.

Crowdfunding has been an increasingly common way for patients with rare diseases to access expensive treatments around the world.

The first allogenic CAR-T treatment is one step closer to market authorisation, a development that is certain to have a substantial impact.

Ultra rare diseases make it a necessity to review the entanglement of scientific innovation industrial development, and healthcare.

A transnational initiative approved in 2018 a document to ethically guide scientific collaborations involving high and low-income countries.

The public and private investment in genome sequencing and surveillance will likely have an impact on patients with rare diseases.

The wicked problem of orphan drugs, a new way of understanding the lenalidomide, a drug with several rare disease indications.

It is the need for new kinds of policies and infrastructures that require our attention, and are a research focus of our SPIN consortium.

Earlier this week, SPINRAZA was granted a negative reimbursement for patients with type II and III SMA.

Earlier in 2022, in the Netherlands, two key stakeholders started a pilot of the Orphan Drug Access Protocol, bringing SPIN to the forefront

Patient organizations and their national coalitions are already at work developing a 4th National Plan for Rare Disease in France.

In the rare disease field, Partnerships for Productive Development (​​PDP) have gained prominence in recent years

On July 6 it was announced that a group of researchers had identified the cells that cause tumours in patients Tuberous Sclerosis Complex

The aim here is to focus on the type of cooperation and clusturization involved in the development of drugs for rare diseases.

With so much emphasis on qualitative experiential data, it is worth bearing in mind that sociology tells us that setting shape experiences.

The point is, SPIN is possible. It is taking place.

As of January 1st, 2022, the Dutch collective basic benefits package contains Kaftrio. Kaftrio is a drug for cystic fibrosis, a rare...