Ben’s Friends recently attended the World Orphan Drug Conference. Not only did we meet many wonderful people, but we learned a great deal as well. This article offers more information about orphan drugs in general.
Most rare diseases are treated with rare drugs. The term rare is often interchanged with orphan or specialty, fundamentally the meaning is the same. It can be very frustrating to be diagnosed with something only to then be told there is no treatment or the treatment is extremely expensive. Like it or not, there are inescapable economic reasons for the treatment issues faced by those with rare diseases.
What is Rare?
Officially a rare disease is expected to impact be one percentage or less of the world population, about 75 million people overall. Breaking it down more, it works out to be fewer than 200,000 in the United States and/or less than one person per 2,000 in Europe. Calling a disease rare and its’ medication a speciality is based on population statistics, which determine the potential future market share, as well as the cost of research and development (R&D) of treatments.
How Expense is Medication R&D?
Tuft study from 2014 found that it costs an average of $1.4 billion and 10 years to develop any new medication. There’s another 2 years in human trials, plus the associated costs, which can range upwards of $50 million. Another $312 million is spent after the drug is on the market, developing new strengths, new formulations, and making alterations.
Only 5 in 5,000 drugs entering preclinical testing progress to human testing. Final market approvals are estimated as low as 1 in 10. Just the attempt of creating a new drug treatment may exceed $2.5 billion with no return on investment. No matter how you look at it, it’s a huge amount of money.
Developing medications is high-risk, low return process, rife with failure.
What about Specialty Medications?
What makes specialty medications special are the odds of return on investment, or really, the lack of return. Specialty drugs are used to treat complex, chronic conditions like cancer, rheumatoid arthritis, and multiple sclerosis, basically any any disease which impacts an average of one percent or less of the world population (approximately 75 million). In contrast, on average, 80% of Americans (approximately 300 million people) were prescribed an antibiotic last year.
With a potential market share of 300 million in just one country, antibiotics make back the R&D costs much more quickly than specialty medications that have a worldwide potential market share of less than one third that amount. The return on investment for speciality medications simply is not there. The less return on investment, the higher the cost to consumers.
Specialty medications sometimes require special handling or administration (typically injection or infusion), and patients using a them may need careful oversight, which adds to the overall cost. Not to mention the ongoing costs of producing, packaging, shipping, and dispensing the medication. There are enormous costs all the way along, assuming the medication makes it to market, remember, most medications never make it out of trials.
But No One Should Profit from the Health and Well Being of Others
You may feel that your personal health should not be a money maker for someone else. Keep in mind the only true mother of invention is competition and the promise of the possibility of profits. No one is going to invest time, money, and effort into something without the possibility of a good return on said investment, particularly when the investment may reach into billions of dollars as new drug development does on a regular basis.
Capitalism really does makes the world go round. It’s not all about the profits, though. Many drugs manufacturers offer patient assistance programs (PAP). These programs range from coupons to saving cards, and even free samples.
What do You do?
As a person with a rare disease you need to be your own best advocate. You need to educate yourself about what insurance plans and programs are available. An excellent listing of PAPs can be found through the information gathered here:
Insurance programs are as widely varied as the people who use them. The one thing they have in common is how details are available. Check the back of your insurance card for a web site and a member services phone number. Often the prescription insurance is seperate from the medical insurance, be sure to research both.
If you live in America your employer may offer a flexible or health savings plan. Typically this allows you to set aside pre-tax dollars for health care, reducing your taxable income while giving you a way to save for medical expenses.
Most importantly, remember that your insurance is not personal to you. The initial information you find is what is called “umbrella coverage.” This is the coverage that applies to everyone on the plan. You then have the opportunity to do authorizations and appeals through the plan to customize the coverage to your particular needs.
With education and persistence you can create a specialized treatment plan and coverage for your speciality medication!
This article has been written by azurelle, whose home community is Living With Chronic Facial Pain. She lives in the United States with a flock of three rescued parrots. When she’s not working on Ben’s Friends, she can be found writing short fiction or avoiding regular house work by doing miniature house work in her dollhouse