IPA Positon Papers

Patient Support Group Collaboration with Industry

In the support of patients and their families living with rare diseases, the work of patient groups is vital for the collection of information and establishing network links between patients, medical experts, and industry.

Orphan Drug Safety and Supply

Predicting the demand for an orphan drug is fraught with difficulty; the prevalence figures of patients are very unreliable and ethnically heterogeneous, and the willingness of health providers to reimburse such expensive therapies is often inconsistent.

Patient Access to Approved Therapies

An Enzyme Replacement Therapy (ERT) for Pompe disease developed by the Genzyme Corporation, and several others are currently under development. For the commercial ERT, approval has been given by many nations and is reimbursed by health providers within those nations. However, we are aware that some nations have not approved this therapy and obstacles to reimbursement exist even within nations that have approved the therapy.