On 22 October 2012 11:45, Allan Pompe <firstname.lastname@example.org> wrote:
I’m sorry that it’s taken so long for me to reply to your proposal for an industry partner’s committee; the IPA board were unanimous that this was a logical extension to the Industry round-table that Tiffany organised in Texas last year. I can now confirm that both Genzyme and BioMarin are in support of the proposal and so we can make the first steps towards holding an inaugural face-to-face meeting.
It has been suggested that we try to meet at international symposia, next year we have two occasions that may offer suitable opportunities for face-to-face meetings:
- 9th Annual WORLD Symposium, Orlando, Florida – February 12-15, 2013, www.lysosomaldiseasenetwork.org
- International GSD Conference, 28-30 November 2013, Heidelberg, Germany. http://worldgsd.org
We should aim to meet a minimum of three times per year and so we should schedule a teleconference for July.
At our first meeting we should agree the following:
- Committee members: industry and IPA
- Chair, other officers and their responsibilities
- Committee terms of reference
- Ground rules for discussions
- Priorities for 2013/14
The meetings will provide opportunities to share knowledge and experience and to define collaborative projects that will benefit the Pompe community. We should draw up a list of topics that we could discuss collectively and then choose priorities when we’re together. Below are a few discussion points that spring to mind:
- Patient reported outcomes: Quality of life measures
- How to ease the regulatory burden for ultra-orphan diseases – Individualised drugs?
- Preparing general arguments for orphan drug reimbursement (ethical, legal, etc.)
- Understanding diagnostic pathways (targeting patient group information and educating professionals)
- Patient registries
- Erasmus Late-onset Pompe survey (e.g. value to national health providers).
- Welfare of participants enrolled into drug trials
- Patient information: leaflets, websites, other
- Raising global awareness of Pompe disease
- IPA future development
- Collaborative research initiatives – could be e.g. diet, exercise, lifestyle, mindfulness, rather than drug therapies
- Patient advocacy –support group vs. Industry – where to draw the line
Does this sound to you like a sensible way to proceed?
Chairman, International Pompe Association