The Role of Compounding in Orphan Drug Use
The connection between so-called orphan drugs and compounding is a contentious one. This is because compounding pharmacists can, in essence, create just about any specialized drug they choose to — even one that is suspiciously close to another that may be out there on the market. This doesn’t matter as much when a drug is already widely available and the long road towards developing it has already been traveled. But when a company is still wavering about whether or not to push forward with a drug because it of the question of whether it’s going to be profitable, this conflict can become fierce.
Orphan Drugs In Focus
Many orphan drugs are so named because they treat diseases that are very rare. This intended use makes their development potentially less profitable for drug companies, and so under normal business and marketing conditions they often get abandoned. According to the Rare Genomics Institute, a disease is “rare” if it affects fewer than 1 in 2,000 people in Europe or 1 in 200,000 people in the United States. It may not seem like many people could be impacted by orphan drugs or rare diseases, but there are approximately 7,000 rare diseases; this means that approximately 1 in 10 people in the U.S. have a rare disease — about 25 million people.
Another reason a drug might be orphaned would be that the drug’s market would not be profitable, such as a drug that would primarily find use in Third World Countries. Orphan drugs also may be drugs developed and approved for one purpose and have another use, such as the treatment of a rare disease. Because that unapproved use isn’t a profitable area, the approval is never pursued.
Orphan drugs are occasional derived from research processes that cannot receive patents. When this is the case, manufacturers will not pursue the drug. Finally, orphan drugs may have been withdrawn from the market for therapeutic reasons, but later have been found to have new treatment purposes. For example, the drug thalidomide, formerly used as a hypnotic, was banned years ago. However, we now know that it may be among the only analgesics that is effective in treating leprosy and lupus erythematosus.
In short, orphan drugs embody a conflict between public health and need on the one hand, and business and profit on the other. The 1983 Orphan Drug Act is aimed to encourage research and support of orphan drugs in the US. The Act grants seven years of exclusivity to drug companies that obtain FDA approval for orphan drugs that impact fewer than 200,000 people; they must obtain “orphan drug status” for their product first.
Compounding to Meet the Needs of Patients
When drugs are no longer available or patients have a specific, unmet pharmaceutical need, this is the most obvious situation for using compounded medications. However, this is not always a welcome response; compounding to meet the gap left by drug companies can draw their ire, as the Makena preterm pregnancy injection case shows.
With this particular case, the public learned the pitfalls of the Orphan Drug Act. Compounding pharmacists, who had been charging patients between $10 and $20 for the injections in question, were prevented from selling the medications once orphan status was granted. At that point K-V, the owner of the drug, raised the price to $1,500 per injection. Expectedly, this priced almost all users out of using the drug at all. The K-V stockholders, who experienced a drop in stock values from $9.64 to $5.39 per share over the course of two months, sued the company, but lost.
The silver lining of the Makena case has been the opportunity for more exposure of these problems in the legal arena and the public forum. Compounding pharmacists were meeting the needs of patients across the country for between $10 and $20 per dose; the medical problem as presented was being treated, and there was no real need for orphan status.
In fact, research has shown that although the actual combined treatment costs of rare diseases such as Wilson’s disease, Addmedica’s hydroxyurea, and Barrett’s oesophagus (all studied in this research) is high enough to merit support from state sources, the best approach for dealing with these kinds of diseases is not necessarily through granting orphan drug status. Instead, the best results can often be achieved via “the pragmatic use of pharmacy-compounded products and evidence-based off-label use of already available commercial products.”
For all of these reasons and more, it becomes clear that compounding plays a crucial role in the treatment of rare diseases. Orphan drug status is not always, or even usually, the right answer for patients; often the best treatment options are more easily accessible via compounding, making it a worthwhile practice to seriously consider for your business.
Compounding to treat rare diseases and to fill gaps left in the market as drugs become unavailable makes sense for patients and your business. In any work with drugs with limited availability, you have the option to purchase your products and receive information from a reliable source. For more about pharmaceutical compounding and purchasing bulk APIs, please contact Pharmaceutica North America, your trusted compounding resource.