Patient Perspectives

Patient Perspectives

Clinical Trials • In a placebo controlled trial, one group gets the experimental substance and the other gets a placebo. • This means that one group is receiving no treatment, even if they are sick. The US Food and Drug Administration (FDA) requires that any drug or device which will enter the human body must first go through a series of clinical trials to ensure that it is safe and effective. The gold standard of clinical trials is the randomized, double blind placebo controlled trial. 1

Patient Activism The clinical trials of AZT in the 1980s marked the beginning of serious patient activism in the US. AIDS patients demanded access to AZT treatments for AIDS even though it was still very experimental. This compromised the integrity of theclinical trials for AZT because it was too difficult to conduct placebo – controlled trials. Patients argued that it was unjust to withholda treatment if there was any chance that itcould benefit them, since the alternative wasdeath. 2

Patient Activism Credited with expanding patient access to AZT; led to other programs for other diseases From Burroughs Welcome shelf to FDA approval: 3 years Pressured the drug company to lower the price and give out drugs free Helped pressure Congress to allocate $billions to AIDS research ACT UP criticized for its aggressive tactics “by any means necessary” 3

Activist Funding • This may mean that the research that is done is the most popular, not always the most scientifically feasible or promising. • Popularity is frequently based on misleading information. • Since the AZT trials, patient activism has become a major force in the health research field. • Some examples are the breast cancer and autism movements. • Although these movements may generate large amounts of research funding, they also influence the direction of research. 4

Effect on the Field • Some scientists and doctors are disturbed bythe amount of influence patient advocacy groups have. • Because these groups are usually focused onshort-term gains for patients who are activelyill they do not always consider long-term scientific progress. • For instance, inaccurate links between autism and vaccines have had a detrimental effect on both autism research and vaccination rates. • Many patients and patient groups also advocate taking risks – for instance access to very experimental procedures – that scientists and doctors believe are too high. 5

Embedded Assessment 1 Active political and physical lobbying forced scientists to terminate placebo-controlled trials of AZT and offer the drug to a wider population, even though it was not proven safe and effective. AIDS patients equated the placebo-controlled trial with murder, since they were enduring terminal illness. Researchers argued that the long-term value of good scientific data on the safety and effectiveness of AZT would benefit many more people over a long period of time. Do you think it was right to terminate the trial and distribute an unproven treatment? Were the patients right to argue that withholding AZT in the name of research was wrong? 6

Stem Cell ‘Tourism’ These same issues exist today with unproven stem cell ‘treatments.’ Because these procedures do not have FDA approval, they cannot take place in the US. Because some people travel to undergo such procedures, the practice is called ‘Stem Cell Tourism.’ 7

Unproven ‘Treatments’ • There are some autologous stem cell treatments which are accepted and conducted in the US. • There is only one embryonic stem cell trial. • But there are a wide variety of unproven procedures offered by undocumented clinics around the world. • Examples include lumbar punctures or injections of nasal stem cells, placental cells or neural cells. • Because there are no clinical trial data on these procedures their safety and effectiveness is unknown. • However, there is little medical and scientific justification to support their success. 8

International Regulations • Some examples are Mexico, Cyprus, China, India and Thailand. These procedures are not supported by many countries that have strict medical regulations. However, there is wide international variation in the strictness of medical regulations. Clinics offering stem cell procedures are generally offered in countries with less stringentmedical treatment laws. 9

Ethical Issues • The advertisements for these clinics are frequently misleading. • Clinics claim to be able to ‘treat’ everything from Alzheimer’s to autism to spina bifida. • The targets of this advertising are a vulnerable population because they are frequently desperate on behalf of themselves or their children.

Advertising

Advertising Tactics 1) Prominent use of certifications (ISO, Joint Commission, use of locally board certified physicians) for non-treatment-related aspects of practice 2) Recruitment of scientists and physicians at prestigious institutions to serve on scientific or medical advisory boards 3) Publication of reviews and research articles in poorly reviewed journals 4) Establishment of opaquely organized professional associations and societies 5) Registration of irrelevant or redundant Phase I clinical trials or pilot studies 6) Claims of association or collaboration with major research and/or medical organizations 7) Claims of regulatory approval or de facto legal status of treatments in countries in which relevant laws have not been developed, or are not enforced 8) Reference to incomplete, slanted, or weakly researched media accounts 9) Viral and guerrilla marketing tactics (blogging, commenting) 10) Anecdotes, patient testimonials, celebrity spokespeople. Courtesy of Doug Sipp, Riken Center for Developmental Biology 14

Financials These procedures are very costly. Clinics frequently charge upwards of $20,000 for a single ‘treatment.’ This does not include travel costs, time off work and the medical expenses associated with failed procedures. Many clinics encourage potential patientsto publicize their condition and ask fordonations from their community. Often, patients mortgage their house oruse their retirement funds to pay for theseprocedures. 15

Fundraising

Hope • Patients and their families frequently say that maintaining hope is an important part of their quality of life. • Even if something is not proven medically effective it may still be beneficial if it provides hope. • Hope may also provide a placebo effect if people genuinely believe that they see benefits from stem cell procedures.

Meet Richard • Richard is a 48 year-old engineer who was born with Machado-Joseph Disease (MJD), also known as spinocerebellar ataxia type 3. • MJD is characterized by progressive clumsiness, difficulty with speech and swallowing, impaired eye movements sometimes accompanied by double vision or bulging eyes, and lower limb spasticity. • Richard is married and has a daughter who is a college student. • Richard’s life expectancy is severely shortened by his condition. • Bieke Stem Cells in China has recruited him to undergo five stem cell injections which they say will help with his symptoms. • Other ataxia patients say they have benefitted from this procedure but there is no clinical data to verify.

Embedded Assessment 2 Richard’s doctor is very skeptical of the proposed stem cell procedure. She says that Richard’s underlying condition is genetic and will not be ‘cured’ by any stem cell injection. Richard is concerned for his quality of life, which is deteriorating rapidly. Richard’s wife, Lily, is also worried about Richard’s condition and is afraid she will not be able to take care of him if it worsens. What would you do if you were Richard? If you were Lily? What about if you were Richard’s doctor? 19

Hope as Exploitation • Doctors and ethicists say that stem cell clinics are exploiting hope and using it to make a profit. • They are aware that many of these families will literally try anything. • Many have called for stem cell clinics that offer untested procedures with no medical oversight to be shut down. • Many organizations have called on local governments to regulate these clinics.

Efforts to Educate

Paternalism • However, we usually agree that paternalism should be reserved for clear and present harms. • Since we have trouble defining the harms that these stem cell clinics are causing, should we be paternalistic? • Taking individual choice away from someone ‘for their own good’ is paternalism. • Governments are frequently paternalistic. • We do not allow people to do things which are bad for them, like refuse to wear a seatbelt.

What are the alternatives? • Many patients are angry because the American medical system does not have any effective treatment to offer them. • They argue that the medical regulatory system is ‘killing them’ by restricting experimental procedures that might help them. • By disparaging or shutting down stem cell clinics the medical profession and the regulatory system are destroying hope, and thus making their condition even worse. • The lack of clinical data can also be seen as refuting the claim that these procedures are harmful. • Anecdotal evidence is seen as more powerful than a lack of data.

The Gold Standard? • These views raise some important questions: • Is our gold standard too gold? • Should the bar be lower for terminal or highly degenerative conditions? • What would ‘clear and convincing’ evidence of stem cell treatments’ effectiveness look like? • Is the FDA too slow? • Should there be expedited procedures for some treatments?

Obstacles There are other obstacles to regulating unlicensed stem cell clinics. • Internationalism. • We have no control over the governments of other countries. • Many developing countries have much more serious problems than unlicensed stem cell procedures. • Scarce resources. • Legitimate treatments also cost a lot of money. So does regulation. • Where is the most important place to put our money? • Some countries, like China, have written laws that restrict unlicensed clinical procedures but they do not have the resources to enforce them.

Embedded Assessment 3 The medical regulatory establishment tries to strike a balance between protecting the health and safety of patients and expediting the discovery and translation process. Do you think that the medical regulatory establishment is doing the right thing about stem cell treatments? Should be shut down unlicensed clinics? Should we expedite clinical trials? How much pressure, if any, should we put on patients not to travel to unlicensed clinics? 26

Summary Why do you care? In the end, all stem cell research has the goal of helping individual patients. Unlicensed stem cell clinics can be seen as exploiting a vulnerable population and potentially causing medical harm. However, some patients argue that these clinics offer hope and that to take the hope away is to harm them. There are very real questions about how our medical system handles novel treatments, especially for terminal conditions. 27

Patient Perspectives