Optimalisatie van de werkingsprocessen van het Bijzonder ... - KCE

26 Special Solidarity Fund KCE Reports 133
Patient organizations
To select Belgian patient organizations to be contacted for the qualitative research, we
started with Rare Disease Organization Belgium (Radiorg.be), established in January
2008 and recognized by Eurordis. Radiorg.be, the Belgian alliance for rare diseases is an
umbrella organization, representing more than 80 Belgian rare disease organizations
(http://www.radiog.be).
To further extent the number of patient organizations we used the following criteria:
• The patient organization has to be known at international level. We limit
ourselves to the members of the European organization for rare diseases.
• The patient organizations represent a variety of SSF submissions.
o The decentralized SSF procedure
Since 2001 the decentralized SSF procedure is applicable to patients
with EB (Epidermolysis Bullosa). The treatments reimbursed via the
SSF procedure are mostly ointments, bandages,… Therefore the
decision was taken to include their patient organization, Debra
Belgium.
o The centralized procedure
One patient organization needed to represent patients for whom the
treatment was mainly focused on expensive drugs. Therefore we
included PH Belgium, the patient organization for pulmonary
hypertension. SSF files are mostly related to the reimbursement of the
very expensive drugs Remodulin® and/or Flolan® .
One patient organization needed to focus on children. BOKS, the
patient organization for adults and children with metabolic diseases
has a clear focus on children as more then 80% of their
families/members are linked to illness of children.
As a last patient organization, we included NEMA, the patient
organization for neuromuscular diseases, as the organization covers a
huge group of rare diseases and a neurological focus was not yet
realized via the other patient organizations.
The 5 selected patient organizations were contacted by phone to ask for their
collaboration. All organizations confirmed their participation and meetings were
scheduled.
The initial idea to include also patient organizations representing rare diseases, often
confronted with a rejection of SSF submissions was not possible. The necessary data to
identify and select these patient organizations could not be extracted from the SSF
database.
Pharmaceutical industry representatives
During the project execution the question was raised to include representatives of the
pharmaceutical industry in the qualitative data collection. The project team contacted
pharma.be, the Belgian pharmaceutical industry organization, who distributed an email
to the members of their “orphan drug working group” as these member companies are
often confronted with the SSF. Thirteen companies reacted positively (prepared to
cooperate by means of an interview) of which 4 indicated they had never or seldom
experience with the SSF. Due to time restrictions, it was decided to meet a small
number of representatives of the pharmaceutical industry. From the remaining 9
companies, the project team selected 4 companies, who were contacted to schedule a
telephone interview. This selection was made following the advice of the general adjunct
director of pharma.be, who indicated the companies that are most familiar with the SSF
thanks to their professional experience or because they produce orphans or cancer
drugs for which requests to the SSF are frequently introduced.