ISSUES
Legal Issues
One of the many issues that arise when considering the legal issues of orphan drugs is the protection that orphan drugs receive after applying for an orphan designation. Many officials are beginning to discover ways that companies have been manipulating this rule, and also discovering ways that some companies have found loopholes in this system.
One example of this is Humira. Humira is a rheumatoid arthritis treatment, but developer AbbVie asked for orphan designations to treat juvenile RA, which AbbVie reported affects only 30,000 to 50,000 Americans. The end date for market exclusivity for Humira was then pushed to 2023, but Humira declined to comment when asked why the end date was extended by such a large period of time.
On the other hand, orphan drug protection can most definitely be seen as a good thing. In fact, without considering the potential loopholes of the orphan drug protection policy, the orphan drug policy is seen as an absolutely necessary part of the Orphan Drug Act. The law allows protection that enables patients to have access to other these drugs. Without this protection, all orphan medicine may have increased prices due to the rising drug competition on the market. Orphan drug designation allows companies funding for medicines if they qualify as an orphan drug. In this manner, these groups are given incentives to produce orphan drugs and are more encouraged to find cures/treatments for rare diseases.
Social Issues
One of the most obvious issues when it comes to orphan drugs is cost. This is quite possibly the most pressing issue of orphan medicines. The cost for orphan medicines can be ridiculously high. Take Soliris, a drug with an orphan designation to treat atypical, hemolytic-uretic syndrome, or aHUS. This drug averages at about $500,000 annually, or $18,000 per dose. The average consumer would easliy consider this to be an insane price.
But it just may be that the orphan drug pricing isn't at completely unreasonable. While the price of Soliris is expensive, it can certainly be argued that Soliris is just one extremely rare case of ridiculous orphan drug pricing. But even without taking this into account, the makers of Soliris, Alexion, claim that separate factors were taken into account before coming to this final price. Biotech company Alexion states that Soliris saves consumers money that would otherwise be spent on trips to the hospital, or blood transfusions to treat aHUS.
Ethical Issues
A serious concern of orphan drugs has to do with the research and discovery portion of development. One view is mainly based on a utilitarian approach. This view is what was in mind when the Orphan Drug Act of 1983 was initially passed. Thinking in this way is generally how costs for medicines are set, but utilitarians disagree over which values are most important. The utilitarian approach involves asking, "What level of resources should be devoted to orphan disease research overall?"
Another way of seeing orphan drugs is through a rights-based lens. One of these rights that people need to consider is the right to a "decent minimum of healthcare," which assures citizens healthcare. It should be noted that citizens are given healthcare without it being based on their ability to pay. However, there are limitations and problems to this approach as well. One of these is that even if everyone is allowed access to healthcare, the definition of "healthcare" holds many interpretations.