Congratulations and Thank You!
To all our One SMALL Step Hosts and volunteers, congratulations for making 2014 another successful year! We thank you for your support and commitment to raising funds through this, our signature fundraising event. Thank you for being there year after year in support of Prader-Willi Syndrome research. Your efforts are vital to our success. The dedication, time, and effort you put into organizing and running OSS is truly inspiring and does not go unnoticed.
Thank you to our participants. We are grateful for your support and donations to our PWS community. We know there are many kind and generous people in the world but, time and again, we are amazed by the encouragement we receive. Without you, we would not be able to work toward reaching our Mission, “To eliminate the challenges of PWS through the advancement of research.”
We look forward to seeing you again at next year’s One SMALL Step campaign in your community.
Clinical Trials and Global Registry
There are several new and ongoing opportunities for PWS families to participate in clinical studies. For more information on the opportunities below, as well as others, please visit http://www.fpwr.org/category/clinical-trials or clinicaltrials.gov. We encourage members of the PWS community to seek out information to help decide whether participation is right for them. Please contact the study coordinators directly for important details about each study and for answers to any question you may have. Some trials may have funds available to support travel to study sites.
Testing is underway on the registry and will allow it to be ready for use by the end of the year 2014. The registry will accelerate clinical trials by gathering information on the PWS community into a central location. This will facilitate the enrolment process by allowing researchers to quickly find patients matching the requirements of their study. Registry participants will have the option of being notified of opportunities to participate in PWS related research studies.
The Registry will advance fundamental and preclinical PWS research by identifying data trends, gaps in knowledge, and new questions, which will direct the research priorities in the field.
It will also provide each family with an online record of their child’s history, for more information go to www.fpwr.org.
FPWR Canada Growth Hormone Access Working Group
In June 2014, the FPWR Canada Board of Directors approved the formation and implementation of the Growth Hormone Access Working Group. Its purpose is to create a comprehensive plan to ensure access to growth hormone for every person in Canada who has Prader-Willi Syndrome.
The initial deliverables are:
- By December 31, 2014 create a comprehensive plan to achieve the purpose.
- By September 30, 2014 report on its activities to the FPWR Canada Board of Directors for review and discussion.
In May 2014, during our National Conference, parents were invited to indicate the areas in which they wished to provide support. The Growth Hormone Access Working Group was an area where five parents expressed an interest. Carole Barron, Executive Director serves as the Chair. An initial Meeting was conducted in July 2014 at which time issues such as GH Access, use of payment through employer drug plan/personal payment, our work with the Canadian Organization of Rare Diseases (CORD) and, next steps to move the GH Access forward (either nationally or by province) were discussed.
From this initial Meeting broad objectives were determined for action:
- To remove Stim testing requirements in general
- To attain label claim with Pfizer and Sandoz (pharmaceutical companies)
- To remove Stim test failures for government funding
Studies reviewed to date include:
- The Growth Hormone Consensus Guidelines – prepared November 2012 and accepted by The Endocrine Society March 2013 – 43 experts agreed that once individuals are genetically diagnosed with PWS they should consider treatment with GH.
- Dutch 2013 (Growth Hormone Therapy for Children and Adolescents with PWS is Associated with Improved Body Composition and Metabolic Status in Adulthood)
- French 2013 (Eight Years of Growth Hormone Treatment in Children with PWS Maintaining the Positive Effects)
- Australian 2009 (Response to growth hormone treatment in Prader–Willi syndrome: Auxological criteria versus genetic diagnosis)
- Public Health Agency Economic Burden of Illness in Canada: 2005-2008
In addition to reviewing these studies, we have held discussions with:
- Canadian Organization of Rare Diseases (CORD) to attain key contacts and be up-to-date on the finalization and release of Orphan Drug legislation in Canada. Orphan Drug legislation will improve the opportunity for drug companies to leverage reduced clinical trial data to support label claims for rare diseases.
- Pediatric Endocrinologist in Canada regarding updating Clinical Practice Guidelines based on the published study data.
- Ontario Drug Benefit Exceptional Access Program to determine the policy process and steps to building our business case in order to access ON Government Drug Benefit Program. The outcome would be to improve the Special Access Funding requirements.
- Pfizer and Sandoz regarding moving to GH label claim in Canada.
We will use these studies and other sources of relevant information to build a business case to support the removal of Stim testing. The plan is to present this business case to the provinces and work towards removing Stim testing as a requirement for Growth Hormone funding. At this time we are also targeting pharmaceutical companies (Pfizer and Sandoz) to support the label claim of Prader-Willi Syndrome. (These companies currently have label claim in the USA but not Canada). This is a complex process of government lobbying, government funding and commercial/financial incentives for a small population. There is a process in place to achieve our goals and we believe it will be accomplished with the support of FPWR Canada and Growth Hormone Access Working Group.