A bright Dutch perspective for the Rare Disease Patient in 2030
For rare disease patients, these are both promising and challenging times. Over the last 15 years, society’s awareness of the impact of rare diseases has increased and new orphan drugs were introduced. Today, more orphan drugs are in development than ever before, offering new perspectives to patients.
But we are not quite there yet. With an estimated total of 5000-8000 rare diseases and only 93 orphan drug treatments currently approved on EMA-level, there is still a long way to go to tackle all unmet needs. Patients suffering from rare conditions should have the same right to benefit from healthcare systems as other patients do.
The way forward presents significant challenges. Not only do we need to develop more orphan drugs at a faster pace, we also need to ensure that patients will have access to these new and existing treatments in a way that is sustainable to healthcare systems. Over the last decades, scientific progress has paved the way for a new generation of innovative medicines that can really change the lives of patients suffering from rare diseases. In order to ensure a fast uptake on the market for these breakthrough therapies and subsequent timely access for patients, it is of utmost importance that this scientific evolution is accompanied by a similar evolution in the regulatory framework for registration and market access. To overcome today’s challenges, a joint ambition and shared commitment is therefore required.
We are committed to our ambition to ensure that the rare disease patient in 2030 is far better off than he is today. During the Rare Disease Symposium on March 30th 2017, we jointly identified a number of key elements that need to be addressed throughout the drug development path to gain momentum towards 2030:
- Active involvement of the patient in every stage of drug development and beyond.
- Investigating methods to fasten the personalized development of orphan drugs.
- Exploring alternative approaches for timely registration of and market access to orphan drugs, where possible based on a case-by-case assessment.
- Maintaining a sustainable health care system in which there is a balance between costs and value of care on one hand, and sufficient incentives to stimulate continuous innovation on the other hand.
The outcome of the Rare Disease Symposium serves as a starting point for an ongoing and constructive dialogue to identify and realise solutions for each of the four key elements listed above. In partnership and guided by a shared agenda, we will work together to shape the future for rare disease patients.