On June 29 of this year, Lauren Erb, our Executive Director at the Caring for Carcinoid Foundation, will be speaking at a public hearing regarding rare diseases held by the FDA. The title of this hearing is “Considerations Regarding FDA Review and Regulation of Articles for the Treatment of Rare Diseases”. In honor of this occasion, I would like to continue the series of posts on Rare Disease Advocacy. The first part can be found here.
One of the most obvious questions a person may be asked is why they chose to advocate for a particular issue/group. When this happens, it is very satisfying to have a clear set of reasons and facts to support your position. In an earlier post I discussed the difficulties many patients with a rare disease, including carcinoid cancer, face compared to other individuals with more common conditions. In this post, I would like to go over an important issue, one that is related to the public hearing being conducted by the FDA: treatments for rare diseases.
As almost everyone knows, the Patient Protection and Affordable Care Act was signed this year, resulting in major changes for the future of health care in the United States. Among the changes are provisions that seek to prevent insurance companies from placing lifetime caps (read about this issue here) and denying patients due to pre-existing conditions. There are very strong opinions surrounding this Act and rather than adding to that discussion, I will continue to focus on the issues rare disease patients face. Of course, both of the aforementioned changes will be beneficial to patients with rare diseases, but there are still steps that must be taken on their behalf.
Health insurance is very important for receiving health care in this country, but the type of care received will ultimately be determined by what is available. I believe it is fundamentally unfair that the availability of treatments is strongly determined by the prevalence of one’s condition, a fact that is out of any individual’s control, other things being equal. Put differently, if two people are similar in all respects, including income, behaviors, age, etc, except for the fact that Person A has a rare condition and Person B a common one of equal severity, I believe A has a legitimate complaint if no treatments are available for him while many exist for B. It may be argued that any given treatment for Person B’s condition will help more people, but I hope to show that this fact alone does not sufficiently explain the disparity between the availability of care options for common and rare diseases.
In this post, I will use the term ‘standard of care’, which should not necessarily be confused with its common use in law. As I use the term, it will simply mean the standard course of treatments used by physicians to treat a disease. Specifically, I will argue that patients with rare diseases like carcinoid cancer often lack a standard of care for reasons to be shown.
First and foremost, diseases have traits which researchers must learn about before treatments can be developed and a standard of care is established. Research is an essential part of this process. Without research it is difficult to know very basic things about a condition such as its natural progression, mechanisms, causes, etc. Yet research of this kind is seriously lacking for rare diseases. Very many resources are needed to do this kind of medical research and public interest is an important component in determining the types of conditions that scientists choose to study. For this reason, I strongly encourage you to support patients of these conditions and organizations like the Caring for Carcinoid Foundation that work diligently to provide funding and support in these areas.
The importance of research doesn’t end there. Without a standard of care established by research, it is difficult to know whether something a physician does is in fact helping the patient or if it is not making any difference at all. Even worse is the possibility that a physician unknowingly does something that harms the patient. Different people with the same condition will have a different progression of that condition. In research, the effectiveness of treatments is often measured by the difference in times to some endpoint or marker, for example simple survival or progression-free survival. A physician who sees two or three carcinoid patients cannot make any generalizable conclusions because even though there may be a difference in these times, it is almost impossible to know for certain whether that difference came from something he did without a sufficiently large sample size.
Combining these two aspects, research may also be able to show why a certain treatment is more effective in one person than another. Take the following real life example. In the paper found here Dr. Matthew Kulke describes a study done on the effects of Temozolimide. This study found that only a very small number of patients with carcinoid tumors responded to the drug, compared to a much higher proportion of pancreatic NET patients. Furthermore, response to the drug was strongly associated with deficiency of a specific enzyme (MGMT). 4 out of 5 patients with this deficiency responded to treatment with Temozolimide compared to 0 of 16 patients without this deficiency. Consider the implications of a study like this one. Without knowing the influence of this enzyme, a physician might prescribe Temozolimide to a large patient group with a small response rate, as opposed to a smaller patient group with a much greater response rate.
For obvious reasons, other things being equal, selling a product that many people want or need to buy will yield higher profits than selling one that only a few want or need. As a result, pharmaceutical companies will often most heavily invest in drug treatments marketable to the largest patient population. This is not the same as the issue of whether a drug treatment for a common condition will help more people than one for a rare condition. That fact may offer reasons to do more research for a more common condition, but I do not think it is sufficient to account for the extremely large gap that currently exists between the two. For example, consider that there are four major categories of drugs for the treatment of asthma: anti-Inflammatory drugs, Leukotriene blockers and Anti-Ige medications, (source) and a much greater number of approved drugs that fall into these categories. By comparison, Sandostatin is the only FDA approved drug for carcinoid tumors, and it is only approved to manage symptoms associated with the disease, and not intended as a cure or treatment for the disease itself.
The lack of parity between rare and common diseases is prevalent in all stages of disease. Although the government has taken steps to improve this situation, for example passing the Orphan Drug Act in 1983 (offering incentives for pharmaceuticals to find treatments for rare conditions) the gap is still far too wide for comfort. Consider that the prevalence for all invasive cancer sites was roughly 11 million in the United States in 2006 (source) compared to about 25 million people with a rare disorder (source) . There is overlap between these two groups, but that figure should still be enough to convince you that more research and attention should be devoted to this issue. At the very least, I believe patients are entitled to receiving the benefit of a standard of care if not a cure. I hope you will join CFCF and other organizations in ensuring that everyone can receive treatment that has been shown to be effective and moving forward in the fight to end patient suffering.
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