Recently, a federal right to try bill was introduced in the senate, which is a companion to a bill introduced in the house last summer, HR 3012. I want so bad to feel hopeful and even excited. I’m trying, I really am, but I was deeply involved in similar past efforts, including the bill championed by the heavy weight senators, “the Toms” (Senate Majority leader Tom Daschle and Tom Harkin) along with Congressman Peter DeFazio. I was also around when Dan Burton tried on the Republican side to pass similar legislation. The prior bills generated a lot of excitement from the health activist community, but little from the rank and file congress folk, and relatedly, little excitement or interest from pharma. My recollection is that none of those bill ever made it out of committee, which is where HR 3012 currently resides. Maybe this time it will be different because of the impressive efforts of the Goldwater Institute’s legislative achievement of passing right to try in 28 states (so far). I hope so.
The federal bill is simple. https://www.congress.gov/bill/114th-congress/house-bill/3012/text The FDA can’t interfere with a physician or drug company providing a post phase 1 investigational drug to terminal patients. Sure, there is vagueness in the bill about whether any aspects of current IND law might apply. However, for sure, if passed, the bill would do at least one important thing which is necessary to open up access to pipeline drugs: Me and my fellow health care attorneys will stop advising drug companies that shipping an investigational drug outside of a clinical trial or expanded access is a violation of federal law.
The other really good news is that presumably under this law, the physician no longer has to fill out a lengthy IND application or even the new streamlined two-page expanded access SPP form. The physician can just administer the drug, right? Does the physician have to report adverse events? What obligations does the manufacturer have once it releases the drug to the physician? The same as in an IND or expanded access? Can the sponsor charge the patient for the drug? If so how much? Cost recovery like under current FDA law? Or a greater amount? How long does the drug manufacturer have to keep on supplying the drug to the patient under right to try?
Apart from these technical questions, there is still the one overarching question/problem in the right to try universe: why would drug manufacturer agree to provide their investigational drugs outside of clinical trials or FDA expanded access? Manufacturers haven’t been pushing their drugs into the expanded access program, and there is no reason to think that right to try would change that. The most off-cited reasons for pharma’s past disinterest are: 1. Fear that open access would slow enrollment in clinical trials which would delay drug approval. 2. Fear that negative data from patients who are sicker with more co-morbidities would lessen the chance of, or slow drug approval, and 3. Limited supplies of investigational agents, which could slow approval. It is already ridiculously slow and costly to have a drug approved in this country, and pharma is worried (and rightly so, I might add) that open access will make the process even slower and more expensive.
Pharma’s past and anticipated future reluctance to embrace expanded access of any kind is for me the big head scratcher, and the reason why I’m just not feeling that federal right to try will have a major impact on the large number of cancer patients who might benefit from pipeline drugs. Under current law, and even under federal right to try, there is no incentive for drug manufacturers to provide investigational drugs outside of clinical trials. Sometimes drug companies can be embarrassed to provide access to their investigational drugs, as has happened in the past with some Facebook shaming campaigns. But that’s not the way to get pipeline drugs to large numbers of desperate patients.
So the obvious answer is to give pharma incentives to provide open access to post phase 1 drugs. Most of the time when drug manufacturers provide drugs in expanded access, they do not charge for the drugs, even though theoretically they could seek permission to do so. The main reason is probably that manufacturers can only recover the actual costs, and drug companies don’t want the FDA or the public to know how cheap the drugs are to manufacture.
Some drugs are early-stage developed by relatively small drug companies, some of whom are cash-strapped, and such companies answer to investors. Taking these two facts together suggest one possible incentive: let manufacturers charge a reasonable price above cost recovery. This might provide an incentive to some drug companies to offer their investigational products post phase 1.
The talking-head ethicists and professional policy folk might decry the fact that drug companies are charging for unapproved drugs, but again the population is terminal patients. Some of these patients might believe that the lack of efficacy data and the financial risk of using an unproven or ineffective treatment are worth taking. And let’s remember that essentially all of the almost 600,000 people who die each year of cancer had treatment which didn’t cure their disease. So by the actual measure of treatment success, which is cure, all of these people took treatment or many treatments which were unsuccessful. Someone has to decide when a dying patient should have access to an investigational drug outside of a clinical trial, either a government bureaucrat for the dying patient. Why shouldn’t it be the patient?
Is charging for the investigational drug enough of an incentive to dramatically increase pipeline drug access? Proably not. To get big pharma on board might require big-time additional incentives. Here are a couple possibilities: Give some kind of priority review to companies which provide open access to their drugs. Give consideration to any positive results achieved under expanded access/right to try, but continue the FDA stated policy of not considering negative data because the population is sicker with more co-morbidities than the patients in clinical trials.
You want to really get pharma’s attention? Throw in an extra year or two of patent protection if a drug is made widely available by the manufacturer under federal right to try. Orphan drugs already have this kind of incentive, and that program has greatly helped the development of drugs for rare conditions. The point is that we need to think creatively about incentivizing the drug companies to participate in right to try, both to assist in getting it passed in Congress, and to make it effective in providing pipeline drugs to large numbers of dying patients. The law itself as it is written simply won’t do the job.
Richard Jaffe, Esq.