Sir Michael Rawlins: Regulation and Reputation

The last 50 years have witnessed extraordinary growth in the world's pharmacotherapeutic armamentarium. Nowhere is this more evident than in the state of medical science depicted in the first edition of the Laurence and Moulton text, "Clinical Pharmacology" (1960). For managing cardiovascular disease in 1960 there were no effective ways to reduce blood lipids; antihypertensive therapy relied on bretylium, reserpine, hydrallazine, and pempidine; and thiazides were the only oral diuretics. There were no oral contraceptives, beta blockers, H₂ receptor antagonists, proton pump inhibitors, benzodiazepines, angiotensin converting enzyme inhibitors, and little in the way of drug treatments for malignant diseases.

Sir Michael Rawlins

The effective treatments we now possess owe almost everything to the pharmaceutical industry. Although my colleagues in academia have been responsible for identifying many drugable targets, it is the pharmaceutical and biopharmaceutical industries that have converted these ideas into effective therapeutic agents.

Nevertheless, despite the substantial increase in expenditure in R&D over the past two decades, the industry has disappointed. The numbers of new active substances reaching the market has not increased proportionately and drug hunting has unquestionably become more difficult. It has also become more expensive, and the costs of clinical development are unsustainable. Many senior executives in the industry accept that this "business model" needs to change—but so, too, does the current regulatory model.

We need new innovative products that not only serve an "unmet need," but that make a real difference to quality and length of life. It is not enough to show that a novel product acts on some novel target. Unless it makes a real difference to patients, and comes at a price the healthcare system can afford, it does not represent an innovation.

Added to these difficulties is the fact that the industry has forfeited the respect of many physicians, patients, and the public. This is in part due to the ways that companies promote—directly and indirectly—their products, as well as the persistent failure to place results of clinical studies in the public domain. I detect some improvement in the industry's performance, in both of these areas, but the drive for change has largely come from external critics rather than any obvious internal recognition of the problems.

I don't think I have really done anything much to transform the way that the pharmaceutical and biopharmaceutical industries conduct their business. I did, many years ago, publish an article in the Lancet criticizing my medical colleagues for colluding with the industry in the industry's marketing practices. It won me few friends at the time, even though the excesses that I described have now largely been eliminated, at least in the UK.

Likewise, as chairman of the Committee on Safety of Medicines (1992 to 1998) I did too little to reduce regulatory demands. Indeed, during those years, the numbers of patients in premarketing trials doubled, even though I hope it was not due to anything I said or did.

Chairing NICE since its inception in 1999 has been the most exciting, rewarding, and invigorating part of my professional career. If during those years I have done anything to influence the industry, it is—I hope—making industry understand that it is competing for healthcare resources with other healthcare providers. And that if a healthcare system spends substantial sums on a few patients, others will be deprived of cost-effective care. Moreover, those people that forgo effective healthcare will often be the marginalized members of our societies who lack powerful advocacy groups or much public sympathy.

In this latter context I hope that the industry now realizes that giving covert support for patient advocacy groups, who are unhappy when NICE advises against the use of a particular intervention, will be exposed. I fully accept the right of advocacy groups to lobby on behalf of their members. But if I ever again learn that their public relations campaigns are being supported by particular companies I will be very noisy in my condemnation of both the importunate company and the guilty advocacy group.

Looking ahead, the global economic meltdown is likely to have a serious impact on healthcare generally and on medicine in particular. The notion that we cannot only just continue—but increase—our current levels of expenditure on healthcare will be impossible. The pharmaceutical and biopharmaceutical industries will only be able to continue providing patients with safe and effective medicines if the price is affordable. This will involve changes in attitudes by the industry, regulators, politicians, and the public. I am more than willing to help.

Sir Michael Rawlins Chairman of the UK National Institute for Health and Clinical Excellence