Why are Rare Disease Drug Prices Still Rising? Doctors and Advocates Speak Out

According to a story from Charcot-Marie-Tooth News, the continuously escalating cost of drugs in the United States is still plaguing rare disease patients and chronically ill people across the country. The US healthcare system has a lot going for it, with many of the latest treatment techniques, devices, and drugs being developed by American companies. Nevertheless, you won’t find many people who rely in the health system to stay alive that will tell you that they are comfortable with the extreme costs.

Preying on the Sick

Unfortunately, it is not like this is a new problem. They power of the pharmaceutical industry has only grown in recent years, and they spend millions in the halls of Congress each year to ensure that there is nothing stopping them from raking in profits by arbitrarily charging steep prices for drugs. They have also thoroughly infiltrated regulatory bodies like the FDA. Perhaps no one embodies this greed in recent years more than Martin Shkreli, who gained notoriety in 2015 for raising the prices of a drug essential for treating HIV and rare infections just because he could. Thankfully he is rotting in prison, at least for now.

Many rare disease advocates, such as Ron Bartek of the Friedreich’s Ataxia Research Alliance, are concerned about the immense cost of rare disease therapies, which routinely run into the hundreds of thousands of dollars just for a year of treatment or, in some cases, a single dose.

Why so Expensive?

Industry reps typically say that drug prices are based on precise calculations that take into the account the drug’s relative value as well as other factors, such as manufacturing and development costs. These costs, are, unfortunately, quite high, especially for many rare disease drugs. Unfortunately, there are no regulations in place that require companies to accurately explain the costs of their drugs.  Dr. Markus Peters, a consultant for the industry, hopes that gene therapies can help bring down costs for rare disease patients in the long run, as these therapies may only require a single administration.

A Platform For Lowering Costs

According to a report from Physicians For a National Health Program, there are several steps that should be taken to bring costs down to a reasonable level.

  1. Universal coverage of drugs without point-of-use fees.
  2. Compulsory manufacture of generic drugs and forcing drug makers to negotiate prices.
  3. Patent reform to enhance innovation as well as the establishment of public departments to spur development.
  4. Increase standards for safety and approval. 
  5. Build popular support in order to curtail the political dominance of the pharmaceutical industry.