Government regulation is stifling America’s vibrant pharmaceutical industry. A recent report by the President’s Council of Advisors on Science and Technology estimates that it costs an average of $1.2 billion to win FDA approval and bring a new drug to market. Given that biopharmaceuticals account for roughly two percent of the economy, this is no small matter.
The chief problem is the complex process of clinical trials, in particular “Phase 3,” in which a drug is tested and retested to prove its its effectiveness in treating conditions across a broad population. These trials have a strong track record, but they are poorly suited to new biopharmaceuticals, which are often very effective in smaller, targeted groups despite a lower success rate in the public at large. Under the current system, many of these drugs may fail their trials despite their effectiveness when prescribed correctly.
This is a set of problems against which folks have been railing for decades. The ginormous cost and length of the approval process is a huge barrier to:
The introduction of new products;
Innovation by small companies (though “Big Pharma” sure is a handy whipping boy for demagogic pols and cable/satellite-channel conspiracy-theorist snake oil salesmen!);
Development of treatments for rare diseases (when the potential market for a drug is a few thousands or 10s of thousands worldwide, a $1.2B development cost will never be recouped, let alone a profit made).
Public safety and medication effectiveness is a valid concern, but these have also been used/misused, resulting in delays and lack of treatments, also costly to US citizens (in lives, disabilities and $$). This is a bit of a sensitive issue to me. I’m not exactly young, and should diseases develop, I wouldn’t mind effective treatments with minimal side effects being available. Also, one of my children had (has?) a disease for which there are but a few thousand diagnoses a year worldwide. With many such patients dying every year - usually in childhood - there may not be 50,000 people with that disease (or set of similar diseases) in the world. Drug companies are not going to be developing drugs specifically for that disease(s), for obvious reasons. Fortunately several common chemotherapy agents are effective against the disease my child had, so after 6 months of chemo (at age 2), my child has been clear for over 20 years. My opinion is that the time and $$ cost for developing small-market drugs could be reduced, without detriment to the public, allowing more rare diseases to be the subject of research and possible treatments (some of which might be existing drugs whose risks are already known - the FDA specifies conditions for which a drug can be used, and if a rare disease is not on the list for a drug …!).