Patents, Medicines, Public Funding–1

Let’s look at four areas of health “technology”: preventions, cures, facilitators, and alleviators. A prevention does just that–prevents an adverse health condition. A vaccine, for instance, prevents a disease (for many, and sometimes with adverse reactions, even deaths). Or, regular exercise may prevent cardiac disease. A cure heals or restores an adverse health condition. An infection clears up with antibiotics or a cancer goes into remission. A facilitator permits other medical interventions to take place–anesthesia, for instance, facilitates surgery and anti-nausea medication facilitates chemotherapy. Alleviators reduce symptoms or slow the progress of disease. In a sense, alleviators turn acute conditions into chronic ones.

In this list of “health technologies,” we might also consider priorities. Clearly, prevention is pretty neat. If one doesn’t get an adverse health condition in the first place, then all the rest of the medical interventions are not needed. Imagine, if you will, that we already have protection from many diseases and injuries–our immune systems, or gut biota or even the Earth’s magnetic field may prevent us from health adversities that would be caused by cosmic rays not getting deflected–just we don’t even think about these things much because we never suffer from them. Imagine–there could be a whole cloud of health adversities circling just beyond our ken that never appear because something we also don’t bother to know about keeps them at bay. Our little cocoon of not knowing. Similarly, the vitamin has cured and reduced the frequency of many deficiency diseases, just as the vaccine has eliminated some really nasty diseases from our immediate imagination–polio, say, or tetanus.

After prevention we might place cure–stopping a disease or healing an injury and restoring function and health. If one can’t prevent some bad thing, at least there’s the prospect of recovery. We do have cures–broken bones can be set and immobilized until they have knitted together, antibiotics can knock down infections, and chemicals can cure a variety of conditions–sofosbuvir, say, can cure hepatitis C (sometimes, and often with side effects, such as reactivating a latent hepatitis B infection). Cure is good–uncurable disorders are terminal or chronic, and neither makes for a happy outlook.

Facilitators are interventions that permit other medical treatments to be carried out more effectively or with less suffering for the patient. These are good things, generally, in the context of bad things happening. The challenge with facilitators is that without those bad things continuing to happen, there would not be much need for facilitators. Given, however, that we cannot be protected from every possible health adversity, cures will always be in demand, and facilitators–everything from blood tests to anesthesias to sensors to surgical tools–improve the delivery of cures and healing. If we need a vaccine stored for extended periods of time, then it’s great to have an improved preservative to facilitate that storage and not cause its own world of woe.

Finally, alleviators. Alleviators reduce symptoms–pain is less although the arthritis is still there–and extend survival times–valganciclovir, for instance, apparently can extend survival of glioma patients by one to four years. If we can’t prevent or cure/heal a condition, at least we can reduce its symptoms and make it last longer, along with the human that has it.

From a public health point of view, it appears that prevention and cure have the greatest importance, and facilitators have nearly as much importance when used in the service of preventions and cures. Alleviators, too, have their place, but as a matter of what we really want, alleviators are stop-gaps. If we can’t cure your disease, we can at least help you suffer less for longer.

When discussions throw everything together under the heading of “drugs” or “medicines,” the distinctions of prevention, cure, facilitation, and alleviation go by the wayside. The result is muddied debate about discovery, treatment, research, and development. It’s like people intensely debating how to care for a cat, thinking “cat” refers to an animal, not a bulldozer. Whatever they come up with won’t have much to do with treads and hydraulics.

The problem with this value proposition–that prevention is tops and cures are tops, too, and facilitators of prevention and cures are really keen, and alleviators that enable prevention and cures are dandy but not the primary thing–is that it runs counter to the value proposition we find if we treat adverse health conditions as a “market” for “commercial products.” From a market perspective, alleviators and facilitators are cash cows and prevention and cures are adverse outcomes. If prevention and cure had their way, the value of alleviators would be greatly reduced–in many cases, the market for alleviators would vanish–the value of potential products would fail to be realized, investors would lose their risk capital, pension funds invested in companies focused on alleviators might wobble, all the little people depending on those pensions might be hurt–the public would be hurt.

From a market perspective, the best value is in finding alleviators for widespread conditions–making them bearable or extending life. Make acute conditions chronic. Make chronic conditions bearable. The new drug Symdeko improves lung function in cystic fibrosis patients by four percentage points, for nearly $300,000 per year. The improvement is real, as is the income potential for the drug. A win-win, from a market perspective. From a public health perspective, however–and perhaps from the perspective of the contemplative patient with CF–the quarter million dollars per patient per year for a stopgap alleviator is not the desired outcome, which is a cure with restoration of normal lung function.

Here’s a chart of CF treatments vs average life expectancy with CF:

Lots of medical technology has been used to extend life–especially antibiotics. But these treatments are alleviators–they combat symptoms that arise from the disease, including bacterial infections that in a normal person would not be so life-threatening. In the US, there about 30,000 people with CF, with 1,000 more added each year. From a market perspective, extending survival means growing the market. If we pursue alleviation, then the goal would be to extend life expectancy to that of the non-CF adult–but with dependency on the alleviations for that entire period. Otherwise, if alleviators were no longer necessary, we would have something more like a cure, no?

The market perspective on the “value” of medical interventions is readily skewed from the public health or patient perspective. Yes, it’s great that CF patients live longer with 4 points better lung function. No, it’s not really a matter of price–but for each patient each year paying over a quarter million, that same money could fund a year’s worth of research into prevention and cure. With public screening, CF carrier couples might think twice about having children; with genetic editing, say, CF patients might receive genes without the mutations that could once for all restore their lung function.

Now, we might ask–where should public funding in support of the CF situation go? Should it go to prevention? cures? Or should it go to continuing to find alleviators? Of course, in a waffling mood a policy maker might argue for all of these, and facilitators too! The “commercial” perspective might well be–sure, fund prevention efforts and the search for cures, but don’t do it so effectively that there’s no longer a market for alleviators–don’t suddenly undermine the private investment that’s been made in alleviators and undo the progress to-date. Government, so this argument goes, should not destroy the value of an area of technological investment–and even global leadership. Let market interests decide the course of technology development, including in health-related areas, including for, say, CF.

Given that facilitators and alleviators both have their role in supporting prevention and cures, the policy argument is not simply that the federal government (and state governments) should fund only prevention and cure and not facilitators and especially not alleviators–but rather that the facilitators and alleviators that ought to be valued are those that work in the context of delivering prevention and cure, and not stopgap. If there’s value in stopgap medicines, the market certainly may pursue that value, but the government should not spend its primary energy facilitating that pursuit of “value” either by commissioning research or by purchasing at prices many times the cost of manufacture (and discovery, and development) the stopgap alleviators on behalf of patients.

Even if governments do decide that alleviation is the way to go, that there will never be a cure for, say, CF, and prevention is simply a bother (let lovers make love, and don’t offer them screening as CF carriers, even of the most common CF mutations)–even if all that, there’s still an important public value in people working to understand how genes control function, and how we might intervene to deal with mutations that cause problems. And those people–the ones doing the research–necessarily are betting against the market value of all those interventions that assume there won’t be a cure, at least not any time soon. Is the search for a cure merely idealistic virtue-signaling? That would appear to be the argument from a “market” perspective. From a “market” perspective, there’s more reason to expect improvements in alleviators than there is to bother with prevention or cure.

If we drill down, then, in the alleviator “market,” we may ask a further question: should public funding for alleviation be a subsidy for for-profit efforts to develop commercial alleviator products? Or should there be other expected pathways for development? Here, too, we need to sort things out carefully or we will start talking calico cats when we should be talking D9T cats. We can distinguish three pathways: (1) discovery and public development followed by commercial manufacture and distribution as needed; (2) discovery and public development followed by monopoly commercial manufacture and distribution via an exclusive license; and (3) public discovery followed by monopoly commercial development, manufacture, and distribution.

In this set of distinctions, we are also concerned with how alleviator research, development, and manufacture is controlled–should there be a “maximum” payout for investors in alleviator development? Should that payout include “maximum” pricing (which in turn we, the public, often pay for)? Will things grind to a halt if investors sense that this proposed maximum payout has been limited by policy decisions?

Next, let’s sort out these three pathways.

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