Orphan drugs are pharmaceutical drugs designed to treat rare conditions. They are difficult to develop and finance but are important regardless of these challenges. The difficulties involved in developing orphan drugs means that pharmaceutical companies will often charge much more for these drugs than their more widely used medications. Controversy around the price and development of orphan drugs has made them a special topic in medical ethics. Those who need to use orphan drugs are often unable to afford them and finding a solution to this issue is one of the most challenging components of development.
Developing Orphan Drugs
Drug development in general is a very expensive and long process. It involves trial and error attempts as pharmaceutical companies look for important differences in the structures of drugs, the outcomes of drugs, and many other factors. Understanding all of the tasks involved in orphan drug development is confusing for many people. Xpertdox contains examples of the challenges you’ll find affecting the production of orphan drugs. They are often obscure and the medical literature surrounding them tends to be sparse. Compounding these challenges is the serious risk of market failure for orphan drugs. They don’t have lots of users to provide a reliable stream of income. This means many developers must use money they make from other mainstream drugs in order to make sure the orphan drug is fully funded. Even if they aren’t able to make a profit, this can prevent the development costs from causing any long term damage.
The Responsibility Of The Public
The exorbitant costs of producing orphan drugs makes public funding a major priority for pharmaceutical companies. Using public funds makes it much easier to produce orphan drugs but it can also add politics into the mixture. The public will decide which diseases are going to be treated and which ones will be ignored. That brings in special interest groups and increased scrutiny private funding doesn’t typically have. Some pharmaceutical companies have exploited public funding in the past leading to concerns over the potential to abuse financing.
Defining Rare Disease
Orphan drugs are drugs designed to treat small but specific demographics. The exact size of that population determines how much funding and effort will go towards treating the diseases afflicting it. If a population is relatively large, there is generally greater enthusiasm towards the idea of possible curing or treating the ailment. For instance, a disease affecting 0.5% of the population will receive more funding than something that might only affect 10,000 people in total. This brings about the challenge of defining rarity and deciding when to fund them and when to avoid funding orphan drugs.
The Benefits Of Treating Rare Diseases
Even if few people are affected by a particular disease the insights and discoveries made during the development process can provide benefits for the general public. If the people affected with a certain disease seem to be immune to other conditions, we might find a way to stomp out a more common affliction. In fact, we have managed to find treatments for diabetes, osteoarthritis, and other common ailments this way. Rare diseases often come with a few upsides to them which makes studying them more important than commonly believed. The greater insight provides a strong foundation for the advancement of medicine as a whole.