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Message from the President |
 Much has been accomplished since the last CFA general meeting held in May 2006 in Halifax, Nova Scotia where the CFA was officially established and the first slate of directors elected. For those that were there, remember the two gov't officials who clearly stated that access to ERT was imminent. We all know now that was not the case!
The executive committee and board of CFA has met and been very active since May 2006 with the priority being access and funding of ERT and diagnosis of other patients. It has been a long frustrating road since the adhoc group of patients and supporters first started working together back in August 2003. However significant progress has been made, but many challenges and tasks remain!
The good news is the Fabry disease story has made politicians realize that they must engage patients in the decision making process. The bad news is that access to drugs and therapies in Canada is at least 5 years behind the European Union and other countries. I believe that education leads to understanding and understanding leads to action and eventually to changes and progress!
Fund raising, membership and activities have all increased from a year ago. Thank you to all of our members and supporters. The successful and well attended AGM was held on December 17, 2007 via telephone conference call. Nearly 150 people attended the regional meetings in Red Deer, Vancouver, Montreal and Cambridge and provided great opportunities with many new patients in attendance. We still hope to have other regional meetings in Corner Brook, NL, Prince George, BC and Ottawa, ON. In addition plans are to host a second National CFA meeting for late 2008 or early 2009.
The CFA would like to prepare and issue a newsletter once every six months. In order to do so, your articles and personal stories are required and we are always interested to hear from you. Any relevant topic, article or story will be considered by our newsletter committee. Please consider preparing and sending the CFA your story!
We are currently involved in collecting and analyzing data from Canadian patients with a lysosomal storage disorder (Gaucher’s, Fabry’s MPS, Pompé’s and other). We are asking about their experience with healthcare services, including diagnosis, access to drug therapy, including enzyme replacement therapy, access to other healthcare services and psycho-social support for them and their family. We will summarize the findings to help make the case for the quality of care for patients with lysosomal storage disorders, and what else is needed. Our plan to submit a poster for the Lysosomal Disease Network World Symposium scheduled for February 13-15, 2008, in the USA has been accepted. So please take a few minutes to complete the survey if you are a Fabry patient. It can be found at: http://surveymonkey.com/s.aspx?sm=sNpG8RhJ3YC2P0oBDOL7IA_3d_3d
The past 18 months have also provided the CFA the opportunity to speak at various conferences and meetings to increase awareness of the situation in Canada and the issue of poor access to drugs and therapies available in many countries. A major problem to accessing ERT in Canada available in many other countries, is that Canada is the only developed country in the world without an Orphan Drug Policy (ODP).
For Fabry patients, ERT is still currently the only effective treatment but on average costs about $250,000/patient/year. It was approved by the FDA in the USA in April 03 and was already available in over 40 countries including Croatia, Argentina, Brazil, Turkey, Bulgaria. Health Canada (Federal Government) approved ERT for safety and efficacy in Jan./Feb. 04 but we only had very limited access starting Oct. 06!
You may be aware of the challenges we have had overcoming the negative decision by the CDR made back in Nov 04 & again in May 05 to not fund ERT for Fabry because they said it is not cost effective and that there is insufficient clinical evidence.
The CFA worked with many of you in a process to access ERT & focused on inequalities of patients with Fabry and other rare disorders. We had to become experts on Fabry/ERT/Health Care System. We also had to learn what was happening in other countries, while remaining independent from other stakeholders i.e. mfgs, physicians etc. The CFA also became a member of the Fabry International Network incorporated in late 2005 under Dutch law. We sent over 2000 letters to Health Ministers. As well as used media exposure we were able to secure many meetings and campaigns.
During this time of advocacy patients in the provinces of NS & ON were cut-off for over 1 year from ERT. Finally the Nova Scotia Health Minister agreed to work with us but required assistance and a guarantee for support from the Federal Gov’t Health Minister. In October 2005 at their Fed/Prov/Territorial Health Ministers meeting they agreed that the CDR is not correct body to evaluate orphan drugs and agreed to expedite approval.
To summarize we still have Health Canada at the Federal Gov’t level, the 10 Provincial Governments which actually fund the public health care and prescription drug programs and the Common Drug Review (CDR)! A positive CDR decision is still a maybe, and a no is still a no. There are actually ten different programs (provinces) depending on where you live in Canada and in the end it is still up to each and every Provincial Heath Minister on which drugs and therapies to fund.
Access to ERT in Canada today is very restrictive and limited. We do now have a limited 3 Year Funding Agreement between Prov/Fed/Mfg which was approved in Aug 2006. In addition we now have a separate Canadian Fabry Registry called the Canadian Fabry Disease Initiative (CFDI) which started only in October 2006. The announcement for the CFDI only became public in September 2007. We are very concerned as this is only a 3 year funding agreement. It required various ethics review board’s approval across Canada. The CFDI provides limited access to ERT with a Canadian made criteria which is the most stringent in the world
The CFA’s position remains that Canada should administer ERT for Fabry as a preventative therapy prior to organ failure with the international community (Fabry Reg/FOS).
The current government plans are now to develop an Expensive Drugs for Rare Disorders (EDRD) Policy. Health Ministers agree not to repeat Fabry model & to resolve ‘EDRD’ committed as 1/5 priorities of National Pharmaceutical Strategy (NPS). The good news is that the F/P/T HM & bureaucrats now recognize role of patients and the CFA as well as CORD were invited to an EDRD policy meeting in Toronto, ON on Sept. 19/07 and Edmonton on Sept 27/07. The bad news is that the current proposal is not acceptable and it seems that the governments priority is cost containment!
Your help is required to advocate and educate the Provincial and Federal Health Ministers and decision makers that Canada requires an Orphan Drug Policy which must address drug approval process, access & funding using int’l accepted standards, criteria and protocols including cost effectiveness with societal values in an ethic framework. CFA is working with the Canadian Organization for Rare Disorders (CORD) which has initiated meetings and discussions even at the senior Fed Gov’t level for what we call the Chance for Life Fund.
The Fabry file and advocacy has had other impacts. Not only has there been changes to the National Pharmaceutical Strategy (NPS) but there are now 2 public people on the CDR. The CDR committee still evaluates drugs only on cost-effectiveness only and they rely on the Canadian Expert Drug Advisory Committee, a small select few physicians. Drugs still are only approved based on cost effectiveness model with no ethical or societal values considered. In addition all discussions and meetings are still held with no access by the public allowed.
The aim and goal of CFA continues to be to find and diagnose Fabry patients in Canada and lead them to proper treatment. With the help and support of many, the CFA will continue to grow, develop and network with other organizations in an effort to improve the lives of Fabry patients, their families and care givers in Canada and around the world. Fabry patients and their families require a comprehensive care program that fits their particular needs to address not only the physical aspects of the disease but the mental, psychological, social, and financial aspects as well.
Thank you to all and appreciate your input, suggestions and support!
Ed Koning
President, CFA
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