2012-10-22 Amicus/IPA Rotterdam Meeting Follow-Up

Allan Muir
Category: Archive
October 22, 2012

On 22 October 2012 11:45, Allan Pompe <allan@pompe.org.uk> wrote:

Dear Jayne,

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?


Allan Muir
Chairman, International Pompe Association

Back to news articles