Letters to the Editor: What makes drug research so expensive, even before the FDA approval process?
-
Click here to listen to this article - Share via
- A rare-disease patient questions what drives research costs behind $41,000-per-month medication, even before FDA approval process begins.
- Readers debate balancing faster drug access for rare diseases against ensuring thorough safety and efficacy data through multiple clinical trials.
- FDA approval standards emerged after 1930s sulfanilamide tragedy killed over 100 Americans, establishing requirements for documented safety before marketing.
To the editor: I was diagnosed with a rare disease, eosinophilic granulomatosis with polyangiitis, around five years ago (“By loosening standards, the FDA isn’t doing rare-disease patients any favors,” Oct. 19). In my case, it manifested with a horrid skin rash, red and itchy, all over my body.
My rheumatologist figured it out, and I am now taking Nucala by clinic-administered shots every four weeks, which seems to be doing the trick. Nucala is a biologic medication also used for eosinophilic asthma. It seems to be effective and useful for different types of allergic chronic conditions.
The only downside to Nucala is the cost: My treatment costs about $41,000 per month. There’s a “Nucala Cares” program that helps, depending on family income. I am on Medicare with a Medigap private insurer, so I pay nothing.
My question to the authors of this op-ed is: What are the costs of the drug research and trials to get to the point where my particular treatment is? That is a big part of the “why and how” of these rare-disease drug developments. Not just Food and Drug Administration approvals, but what comes before.
I would hope all rare diseases would be researched carefully and treatments prescribed as carefully. It’s not fun to have something wrong that no one can help you with.
Leslie Tillmann, Palm Desert
..
To the editor: The op-ed by Holly Fernandez Lynch and Reshma Ramachandran describes the dilemma of utilizing drugs under development for rare diseases that are not FDA-approved for which evidence of their effectiveness is incomplete.
The authors highlight the problems of inadequate efficacy data; equally important is safety data on adverse events and toxicity. Undetermined safety data further complicates decision-making on safe and effective drug use.
The FDA drug-approval process is based upon legislation enacted in the wake of the progressive era in the U.S. to address the poisoning deaths of more than 100 Americans who took sulfanilamide elixir in the 1930s. This tragedy highlighted the need for the government to require more rigorous new drug testing to document safety before approval for marketing. As a result, President Franklin D. Roosevelt signed into law the Federal Food, Drug and Cosmetic Act of 1938.
All drugs can have adverse side effects (even placebo ones); the therapeutic use of any drug therefore is a balance between documented beneficial effects and its adverse effects/toxicity. As such, multiple robust clinical trials are typically needed to establish the benefit vs. risk ratios for prescribed drugs. In absence of these studies, the risk-benefit balance is unknown, making decision-making complicated.
Robert Deamer, Thousand Oaks
