Drug Innovation 5: The US PCAST Report on Propelling Drug Discovery

* This is my article yesterday in the online magazine,
http://thelobbyist.biz/index.php/perspectives/less-government/item/110-medicines-innovation-and-the-fda
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There was a new report, Report to the President on Propelling Innovation in Drug Discovery, Development and Evaluation made by the President’s Council of Advisors on Science and Technology (PCAST), Office of the President of the United States, dated September 2012. The members of this Council seem to be high profile scientists and academics in some known US universities. The 110 pages report can be downloaded at www.whitehouse.gov/ostp/pcast. 

Among the findings of the report is this chart below: While the number of new molecular entity (NME) and new biological entity (NBE) approvals was declining from their peak in 1996-97, the cost of research and development (R&D) per NME is rising. This chart is found on page 12, then page 35 of the Report.

As a result of this, the Report noted that

heart disease and stroke remain leading causes of mortality, many common cancers are still incurable unless they are caught in the earliest stages, and the vast majority of rare diseases lack effective therapies altogether. Infectious diseases, including those caused by antibiotic-resistant bacteria and viruses with pandemic potential, pose a constant threat of large-scale mortality. And treatments for psychiatric diseases, which impose a tremendous burden on society, are frustratingly limited in their efficacy, as are treatments for neurodegenerative diseases such as Alzheimer’s.

To address this problem, the Report identified two important measures of actions.

1) Scientists need better methodologies and tools for translating basic biological insights into validated therapeutic targets and leads—a gap in the drug discovery and development pipeline that academic scientists often view as “too applied” and pharmaceutical companies often eschew as “too basic” to justify private investment. And

(2) Pharmaceutical developers and regulators need to incorporate new efficiencies into clinical trials of candidate medicines—complex and costly human studies that today constitute fully 40 percent of the biopharmaceutical industry’s R&D budget.

Medicines innovation and invention is not done in the Philippines and many other poor countries due to the high costs, high risks, and high technical regulatory approvals required. For instance, Figure 1 (b) on page 35 showed that by 2010, one academic paper estimated that the cost to innovator pharma companies for each NME is between $884 million to $1.80 billion. Just for one drug alone, and there is no assurance that it will become a blockbuster, high profit drug.

We only have policy making bodies that involve the Department of Health, its attached agencies like the Food and Drugs Administration (FDA), industry players like the multinationals and local generic pharma companies, drugstores and hospitals, health professional and civil society organizations. Like the DOH Advisory Council for Healthcare, of which this writer is one of the members.

One problem with government regulatory agencies, the FDAs in particular – in the US, Europe, Philippines, etc. – is that there is too much emphasis or focus on making the newly-invented drugs to be “very safe, very effective” so that the regulatory approval processes have become so tedious, so strict, that diseases evolve faster than the government approval process. So by the time the very few new drugs that were invented to address certain new diseases have been approved by FDAs, the diseases have evolved into something more sinister and more lethal. Not for big number of people initially but for a few patients only, but has the potential to explode into a highly contagious and infectious disease someday.

If this hypothesis is plausible, then one remedy is for government FDAs to step back and relax a bit their strict drug approval procedures. Bring down the approval process of various clinical trials from 10 or 12 years on average, to only six or eight years. In the process, bring down the average cost of innovation from $0.9 to $1.8 billion per new medicine to possibly only half of it.  Encourage more big generic manufacturers to go into drug innovation business themselves.

In short, turn the focus of accountability away from FDAs and more into the drug innovators and manufacturers . The rule of law and property rights protection says that accountability should stop on whoever initiated something, and the main function of governments is to enforce existing laws, including penalties to violators of regulations governing public health and safety.

So if innovator company A released a drug that turned out to have several adverse reactions, it is incumbent on that company to quickly withraw its drugs worldwide and pay limited penalties only. On the other hand, if innovator company B has released a drug that turned out to be very effective and safe at the same time, then more patients will benefit. A one year delay in marketing a new and effective or revolutionary drug could mean a lot in terms of saving lives.

This should also be a lesson to groups and lobbyists that oppose intellectual property rights (IPR) like patents for new medicines, or at least advocate to keep such IPRs to be as short as possible. It is not easy, it is not a walk in the park, to become a drug innovator. Otherwise, it would be safe to assume or expect that huge global generic manufacturers like Teva in the US, Dr. Reddy in India, and Unilab in the Philippines would be in this business too while retaining their resources in generic drugs production.

The new Philippines FDA Director, Dr. Kenneth Hartigan-Go, is a friend. When he was newly appointed to the post, I suggested to him that the FDA should develop a strong legal and prosecution team, in alliance with the Department of Justice (DOJ) perhaps. It is simply impossible for the FDA to inspect all medicines, all food supplements, all shampoo and skin whiteners, all fat/weight reducers and boobs enhancers, all sauces and juices, all beer and wines, and tell all of us consumers in this country, that they are all safe and have no adverse side effects. Let the manufacturers, wholesalers and distributors stand by their products, to accept full accountability if any adverse results should happen to consumers and/or patients.

And this is where the FDA should come strongly and forcefully. FDA’s message would be: “Do your thing as you swore and submitted to us. If you lied and sell unhealthy products, woe unto you, you will regret that you planned or thought of such thing in the first place.”
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See also: 

Drug Innovation 1: On Cancer, Bioequivalence and Clinical Trials, March 28, 2012 
Drug Innovation 2: Sustaining Innovation via Competition, April 14, 2012 
Drug Innovation 3: Biopharmaceutical R&D and Innovation, June 01, 2012

Drug Innovation 4: New Anti-Cancer Drugs, June 08, 2012
IPR and Medicines 25: Patents, Diagnostics and Technology Transfer, October 08, 2012