Times of austerity always affect those who are already most in need and vulnerable. For this reason 2010 may become a year with serious setbacks for Rare Disease patients. Already a number of disappointing news confirm this apprehension: Recently we learned that the compromise on the directive on cross boarder health care will not bring the progress for Rare Disease patients that we had hoped for. On the contrary there is absolutely no mention of rare disease patients in the final version of the proposal.
In addition we start seeing examples that access to reimbursement of new drugs will be delayed or denied at national level. In Sweden a committee (TLV) evaluates the benefit of drugs reimbursed by the healthcare system, A recently authorised orphan drug, Kuvan, developed for PKU (Phenylketonuria) patients, will not be automatically reimbursed. This drug will only be reimbursed if the local authorities of the regions so decide. This is a major threat to a subset of PKU patients who have difficulty complying with the normal very restrictive diet.
In addition, the process leading to this deplorable decision violates the principles of patient involvement that we have fought hard to gain respect for. The way the Swedish and many other countries, have designed their procedure for reimbursement decision, implies that it is only for the company putting the drug on the market to produce documentation on the need for the drug. Therefore, patients have to rely solely on drug company representatives’ competence and commitment to provide this information whereas those most affected, the patients and their organisations, are not consulted and have no right to contribute separate information or challenge the decision of the committee. The decision on Kuvan has now been brought to the Swedish court. But what will be the situation while waiting for a decision?
At its meeting on July 2, the board of directors of Eurordis discussed to what extent governments will cut back on expenditure for orphan drugs. The board found it essential to monitor the development closely and decided to invite rare disease national alliances to collaborate in a survey in order to identify changes in real access to reimbursement of orphan drugs.
In the current political context, it is necessary that the new EU Committee of Experts on Rare Diseases soon becomes operational. Therefore it was seen with some concern by the board of Eurordis that the first meeting of this important new committee, initially foreseen for May of this year, has been postponed to December 1.
How can the added value of a better quality of life for rare disease patients be measured? how can their active contribution to the society (their integration at school or at work), thanks to an appropiate treatment be assessed? How can the savings of hospital stay, because a good access to an existing orphan drug be evaluated?
Governments spend a good deal of money to rescue a climber,, a fishing boat from the XXI century pirates, or to find bodies for the confort of the families, even when it was supposed to undergo a difficult financial situation Nobody questions these admirable actions, no matter how expensive they are, but everybody does when it comes tto the treatment of a person with a rare disease. Are they second row citizens?
The economical crisis may prevent new patients from partipating in clinical trials or benefit from new treatments, that is why a survey on this matter is necessary to have concrete data. The inequalities of access to treatments can even be seen among regions or hospitals in the same country, when it depends on their budget or decision, as you mention.