World Orphan Drug Congress, Brussels, 15th-17th November 2016
On 15th November, I travelled to Brussels to represent Vasculitis Ireland Awareness at the 7th World Orphan Drug Congress in Europe. I secured a free place along with a colleague from the Northern Ireland Rare Disease partnership (James Cauldwell, Rare Cancers support group) and we travelled together to find out more about this area and how we as patient groups could expedite access to these important medications.
Perhaps at this stage it would be good to define Orphan drugs – according to European Medicines Agency they are ‘for the diagnosis, prevention, or treatment of a life threatening or chronically debilitation condition that is rare (affecting not more than 5 in 10,000 people in European Union) or where the medicine is unlikely to generate sufficient profit to justify research and development costs.
We attended the Pre Congress workshops on the 15th, which concentrated on accelerating commercialisation of rare disease treatments. Two suggestions that stood out were having patient registries funded by Industry and the use of Expanded access (the drug is made available before reimbursement pricing is agreed). If patient registries are to be funded by Industry, we need to be vigilant that the information stored is only used for what has been consented to by the participants.
Day 1 of the Congress opened with short presentations on European Reference Networks (ERNs), assessing the value for money of orphan medicines, guidelines re ‘significant benefit’ of orphan medicines and updates on European Medicines Agency work on ‘PRIME’ (PRIME aims to bring promising innovative mediines to patients faster by optimising and supporting medicine development).
Following the morning network break, we selected two interactive roundtable sessions. I attended ‘the voice of the patient and caregiver in protocol design” and ‘Building a patient centric model for repurposed drug development” These were attended by various delegates including representatives from the pharmaceutical industry. It was interesting to discuss these areas with others from different backgrounds, hearing each other’s perspectives and making suggestions that may make a difference (i.e. rather than discarding failures in drug development, sharing these and learning from them).
I attended the Science and Industry sessions in the afternoon which were chaired by Dr Eric Tambuyzer, co-founder and chair of BIOPONTIS Alliance – an international non profit organisation set up to advance promising science into potential treatments for rare neurological diseases, involving patients in the development of their own treatments through alliances with patients organisations worldwide. These are my take home messages –
- I was shocked to learn that less than 5% of rare diseases have a designated therapy despite 25% of the population being affected by rare disease. It is hoped that the development of ERNs will get patients more involved and improve this situation.
- We need to look at alternative ways of improving patient access to innovation by increasing public awareness of diseases and their symptoms by talks, media, podcasts, blogs etc.
- There are various types of research projects, different styles of clinical trials can yield necessary information when selected/used appropriately, and thus save time and resources.
Treatments and assessments re their effectiveness should be designed according to patient priorities, what makes scientific sense may not be practical in daily life.
- Adaptive pathways in Rare Disease are essential to address unmet needs, these need to have controlled access only, be closely monitored re benefit/risk aspects, efficacy, and effectiveness.
- Patient/carer reported outcomes important to help understand disease, treatment benefits and to select outcome measurements.
- Patient networks have a vital role to play in addressing unmet medical need to establishing a therapeutic strategy.
Day 2 started with talk about personalised medicine becoming the norm and if this was financially viable (Not at present). Again we broke into interactive roundtables, I went to ‘Opportunities to improve patient access to licensed medicines through patient involvement and ‘How can a patient group more effectively approach sponsors’. I broached the subject of the difficulties in talking to Pharma, where both sides are wary of being compromised. This is overcome in USA by the Sunshine law, where all parties declare any appropriate information to avoid misrepresentation. I also spoke of problems in being aware of current research projects worldwide as a small patient organisation. At the end of the day, I received an email containing links to relevant Vasculitis research from one of the group.
After our networking lunch, I attended the ‘Rare Disease Advocacy world’ sessions, and heard some very inspirational speakers talk of their patient/carer journeys from diagnosis to talking to pharma, being involved throughout the whole clinical trial process and importance of partnering with each other as patient groups.
While the whole event was informative and good for networking, I feel there is still quite a distance to go before the patient voice is really heard and research is truly patient led.
Vasculitis Ireland Awareness,/ Northern Ireland Rare Disease Partnership