15 of 44 Health Economists Oppose Current FDA Approval System

The article asked a wonderful set of questions.  I decided I'd stick my neck out and give my initial thoughts (italicized) on these questions and even throw in a few comments designed to "stimulate" the conversation.  So don't believe everything I say.  (Like I needed to tell you that.)

1. The policy that requires pre-market approval of new pharmaceuticals and medical devices may prevent harm, and it also may suppress would-have-been benefits (by suppressing drug development, causing delays, or increasing costs).  This may be particularly true for less common conditions.
2. Consider now only the second policy effect, suppression of would-have-been benefits.  Which of the following statements best describes your view of the public discourse about this effect?  The Orphan Drug Act addressed rare, but not common, conditions.  Many people may turn to alternative, relatively untested, therapies because they don't have good FDA-approved options.
3. Which best describes your attitude toward the U.S. government policy that requires pre-market approval of new pharmaceuticals and devices?  Greater access to information and shared expertise will continue to shape my opinion.
4. Do you think there is a sound market-failure rationale for the policy that requires premarket approval?  
5. Given that you believe there is no sound market failure rationale for the policy that requires pre-market approval, please explain why you do not oppose the policy.  Much better access to information
6. Do you believe that consumers or patients systematically err when coping with uncertainties related to health and treatment?  Most definitely.
7. In what ways does consumer or patient erring manifest itself?  Sham treatments remain a big business.
8. Do you believe that doctors systematically err when selecting and prescribing therapies?  If you include value (i.e., cost per successful therapy), then this occurs frequently.  
9. In what ways does doctor erring manifest itself?  In addition to costs we still see a lot of prescriptions for drugs that simply don't work or have much better alternatives. 
10. Say the policy that requires pre-market approval was eliminated and, in its place, a policy was implemented allowing new pharmaceuticals/devices and initially classifying them as requiring a doctor’s prescription (pending a review process to consider dropping the prescription requirement). Do you think such a liberalized system would be superior to the current system?  I don't think that individual physicians have the time to track down and evaluate whether a new drug offers a better alternative to existing therapies.
11. Do you believe that uncertainty per se constitutes a market failure?  No, even the current system has uncertainty.  The common occurrence of off-label prescribing relies on the possibility the drug offers better patient value.
12. How in your view does uncertainty per se constitute a market failure?
13. Knowledge is often said to have public-goods aspects. Imagine a new pharmaceutical being developed within the current regulatory system and brought all the way through to FDA approval. Do you think the policy that requires pre-market approval induces the generation of more knowledge about the new pharmaceutical than there would have been in the absence of the policy?  Yes.  Barriers to entry require the company to work harder to prove the benefits of their product.
14. As compared to the current system, would you favor making approval of a new drug automatic upon completion of the safety and efficacy testing processes specified by the FDA, regardless of what those testing results turned out to be?  That depends on how the results of those processes get summarized, evaluated and communicated.  Groups like the Cochrane Collaboration help us get closer to that possibility.
15. Relative to doctors and consumers, do you believe the government has superior ability to judge the safety and efficacy of pharmaceuticals (assuming, that is, that the government does not have exclusive access to certain research information)?  Much better than individual doctors and consumers.  But other groups (e.g., Cochrane, Drug Effectiveness Review Project, etc.) add additional expertise to the FDA review.  I'll admit that I've been very impressed with the quality of the FDA expert reviews.
16. How would you describe or explain the government's superior ability to judge safety and efficacy?  Compared to individual doctors and consumers that is their job.
17. As compared to the current system, would you favor a reform so that pharmaceuticals approved by the FDA counterparts in Europe, Japan, Canada, or Australia were automatically approved for the United States?  Why not?  They do a good job.
18. Which of the following describes the nature or source of the market failure that justifies the policy requiring pre-market approval?  Presently, the market cannot force the drug companies to extensively test and communicate all results to the public.
19. Please describe the 'other' source of market failure that justifies the policy requiring pre-market approval.
20. Do you believe that consumers or patients systematically err when coping with uncertainties related to health and treatment?  I know I have.  New evidence leads to changed opinions.  I still don't know if resveratrol (e.g., red wine) has a significant benefit.
21. In what ways does consumer or patient erring manifest itself?  Homeopathy
22. Do you believe that doctors systematically err when selecting and prescribing therapies?
23. In what ways does doctor erring manifest itself?
24. Say the policy that requires pre-market approval was eliminated and, in its place, a policy was implemented allowing new pharmaceuticals/devices and initially classifying them as requiring a doctor’s prescription (pending a review process to consider dropping the prescription requirement). Do you think such a liberalized system would be superior to the current system?  The choice of individual prescribers does not help us evaluate the benefits of a new drug.
25. Do you believe that uncertainty per se constitutes a market failure?  No.  Physicians that have good information make good decisions.   
26. How in your view does uncertainty per se constitute a market failure?
27. If neither consumers nor doctors err systematically in these matters and uncertainty per se does not constitute a market failure, then in what sense do you believe that imperfect information is a source of the market failure that justifies the policy requiring pre-market approval?  That has been the primary problem.  But how do we create the incentives for physicians and consumers to access and evaluate good information?  
28. In addition to systematic error on the part of consumers/doctors and/or uncertainty per se, is there any other sense in which you believe imperfect information to be a source of the market failure that justifies the policy requiring pre-market approval?
29. You indicated that public-goods aspects of knowledge do not justify the policy requiring premarket approval. Is that because you think such aspects are better addressed by subsidizing the generation of knowledge, e.g., via the National Institutes for Health?  I have trouble believing that a single organization can guess right when prioritizing their research.  
30. You indicated that public-goods aspects of knowledge are a source of the market failure that justifies the policy requiring pre-market approval. Do you think that this source of market failure would be better addressed with a policy that subsidizes the generation of knowledge, e.g., via the National Institutes for Health?
31. Imagine a new pharmaceutical being developed within the current regulatory system and brought all the way through to FDA approval. Do you think the policy that requires pre-market approval induces the generation of more knowledge about the new pharmaceutical than there would have been in the absence of the policy?  Yes.  Otherwise a lot of the knowledge would look like abstracts from early research.
32. How is it, then, that the public-goods aspect of knowledge provides a rationale for the current system?
33. As compared to the current system, would you favor making approval of a new drug automatic upon completion of the safety and efficacy testing processes specified by the FDA, regardless of what those testing results turned out to be?  This might work if the results were readily available.  But would advertising drown out the analysis performed by the third-party expert groups?
34. You indicated that a superior ability of government to assure the safety and efficacy of pharmaceuticals justifies the policy requiring pre-market approval. Does that superiority stem from the FDA having special expertise in evaluating safety and efficacy?
35. Why is it that doctors and consumers have inferior judgment in evaluating safety and efficacy?  Lack of time, lack of information, lack of training.
36. Would you say that impartiality or commitment to the public good are sources of the government’s superior ability to assure safety and efficacy?  I wouldn't call it impartial, just less partial.
37. In what ways does the FDA’s impartiality or commitment to the public good render it superior to doctors and patients in judging safety and efficacy?
38. What is the basis of government’s superior ability to assure safety and efficacy, if not the FDA’s special expertise, impartiality, or commitment to public good?
39. Prior to 1962, the FDA did not consider efficacy when making drug approval decisions. Doctors today are at liberty to prescribe drugs for “off-label” use—that is, for use where there has been no FDA evaluation of the drug’s efficacy. However, as part of the current pre-market approval process, the FDA requires proof of efficacy in the drug’s “on-label” use(s). Would you favor dropping efficacy requirements from the pre-market approval process?  I want to know that.  How else will we get that information?
40. Efficacy requirements were introduced in 1962. Do you believe that the pre-1962 record shows systematic failure in assuring efficacy?  I'm still concerned about the post-1962 record.
41. What evidence can you cite for systematic failure in assuring efficacy prior to 1962?
42. Do you have any general comments about this questionnaire or further thoughts about the matters treated?