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FDA approval debate

FDA approval debate

Jack Scannell, friend to the Empower: Access to Medicine campaign, enters a debate in the British Medical Journal on patients wanting faster access to treatment and the perceived motivations of the drug industry. Below is his response to an article in the BMJ entitled The FDA's New Clothes.

Light and Lexchin write that patients “are said to want” faster regulatory reviews for important new drugs (1). This word choice suggests the authors heard about patients’ views second hand, from sources that they did not entirely trust. Why not ask a few patients and settle the matter? I know some who are, to quote their website, campaigning to “change the drug development process in order to speed up the delivery of life-saving and life-enhancing medicines to people with life-threatening and rare illnesses” (2). I think there are others with similar views. I would be happy to try to arrange introductions.

The $85,000 launch price of sofosbuvir shows why it is a bad thing to have only one breakthrough drug with no “me too” competitors. At long last, some health systems are learning to use competition, via therapeutic substitution (choosing drug a ahead of therapeutically similar drug b), to force lower prices and to improve access (e.g., bevacizumab versus ranibizumab in AMD in France, AbbVie versus Gilead in hepatitis C, competitive tendering for insulin supply to Brazil, UNICEF’s vaccine procurement, Norway’s procurement of biosimilar infliximab, NHS procurement of erythropoietin, etc., etc.). I am therefore puzzled by Light and Lexchin’s (1) negative view of the approval of drugs that are not “any more effective than existing products”. I think health systems need more opportunities to force manufacturers to compete on the basis of price, and not fewer.

The story of insulin in type 1 diabetes has been one of incrementalism for nearly 100 years. No patients today would prefer the breakthrough that Banting and Best offered in 1922. Boeing has produced a series of me-too aircraft since the first jetliner, the 707. Few of us would now prefer to cross the Atlantic in the 1958 original. I am therefore puzzled by Light and Lexchin’s (1) negative view of incremental innovation. I would like to see more frequent incremental gains in the pharmacopeia, not less frequent ones.

I agree with Light and Lexchin (1) that bias is a major problem. As I have written elsewhere, “Phase III trials have become a messy mixture of science, regulation, public relations and marketing.” (3) However, there is a problem with the cost and speed of clinical development, independent of commercial bias (see, for example: (4) (5)). There is also the problem that clinical trials and regulatory decisions lack “ecological validity.” The usefulness, or uselessness, of drugs often becomes clear only after years of real world use (6). A great deal of therapeutic innovation occurs as doctors and patients make the most of the drugs at their disposal (7). The pharmacopeia will improve more rapidly if more acceptably safe “chemical diversity” reaches patients and doctors, at lower R&D cost, and if it is then evaluated in its natural environment. I do therefore see a useful role for some of the expedited programmes in the US, and for adaptive regulatory pathways in Europe. To quote Mao Tse Tung, who I confess is an unusual source of advice on drug regulation: “Letting a Hundred Flowers Blossom and a Hundred Schools of Thought Contend is the Policy for Promoting Progress.”

1. Light D, Lexchin J. The FDA's new clothes. BMJ. 2015; 351: p. h4897.

2. Empower. Access to Medicines. [Online].; 2015 [cited 2015 October. Available from:

3. Scannell J, Blanckley A, Boldon H, Warrington B. Diagnosing the decline in pharmaceutical R&D efficiency. Nat Rev Drug Discov. 2012; 11: p. 191-200.

4. Coates A, Halls G, Hu Y. Novel classes of antibiotic or more of the same? British Journal of Pharmacology. 2011; 163: p. 184-194.

5. IDSA. IDSA comments on FDA draft guidance for industry on hospital-acquired and ventilator-associated bacterial pneumonia. Federal Register 73106, November 29, 2010. [Online].; 2011. Available from:

6. Scannell J. What bad forecasts say about pharmaceutical and biotech innovation. [Online].; 2014 [cited 2015 September. Available from:

7. DeMonaco HJ, Ali A, Von Hippel E. The major role of clinicians in the discovery of off-label drug therapies. Pharmacotherapy. 2006; 26: p. 323-332.

Competing interests: Jack Scannell is a director of J W Scannell Analytics Ltd., which provides consulting services related to drug R&D to the public and private sectors.


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