In 1987, a group of angry HIV positive patients came together to set-up ACT UP, an outfit with the mandate for fighting for the basic human rights of AIDS patients. This marked the start of a long and lonely battle
Clinical research allows doctors and scientists to test and develop new medical diagnostic tools and treatments, so they can discover better ways of treating deadly diseases. Advocacy is required as much in clinical research as it’s required in primary care, because the subjects of research are the patients themselves. While biomedical research used to be the monopoly of scientists and clinicians, patient advocates started overseeing clinical trials in order to guard the rights of patients. The need arose when it was discovered that patients who participate in clinical trials are often in danger of being exploited by unscrupulous researchers who may be so focussed on their research objective and advancing their own careers, that they may forget that their true goal is to reduce the suffering of the patients. There are scores of real life horror stories of clinical trials gone awry, where medical ethics were violated and trial participants harmed knowingly with impunity.
In these cases, patient advocates have played a key role in protecting patient rights during clinical trials by:
There are three main reasons why hospital patients feel lost and helpless:
- Educating patients about a clinical trial’s potential benefits and harm
- Making sure that the information provided to the patient is reliable
- Confirming subjects have provided informed consent
- Promoting patient safety and confidentiality
- Identifying and addressing potential ethical issues in the trial
Once their interest and involvement in this discipline rose, patient advocates began to realise that they needed to take a much more active role in biomedical research. Their focus is now on making the research agenda more patient-friendly. This they do by sharing the patient’s perspectives with researchers and increasing their clout by:
- Lobbying with the legislature to increase funding for research on specific diseases
- Acting as a connecting bridge between scientists and society, thus helping researchers keep patients’ needs in mind, rather than furthering their narrow professional agendas
- Helping to design clinical trials which were meaningful and practical
- Expanding opportunities for patients to participate in clinical trials
- Working with media channels to raise awareness about a particular disease
This 1993 American film, draws heavily from the real life workplace discrimination faced by attorneys Geoffrey Bowers and Clarence B. Cain, who got dismissed by their law firms once their HIV-status came to be known at office. Philadelphia was one of the first mainstream Hollywood films to acknowledge HIV/AIDS, homosexuality, and homophobia. This is an inspiring film, and demonstrates what impact the media can have in influencing public perception of a disease, by giving patients a public platform to articulate their views.
An excellent example of how empowered patient advocates can influence the biomedical research community and policy makers to catalyse change is the HIV/ AIDS advocacy group - AIDS Coalition to Unleash Power (ACT UP). This is an international direct action advocacy group, whose motto is Silence = Death. This is what advocacy is about - give voice to the disempowered. When AIDS was discovered nearly four decades ago, a diagnosis of AIDS was like pronouncing a death sentence on the patient. AIDS became the leading cause of death in US men aged 25 to 44, and there was a lot of stigma attached to the disease. That’s when a group of HIV- positive patients joined forces and established ACT UP in 1987. This was a group of angry patients who were determined to stand up and fight for their rights.
They realised that anger alone cannot bring about a mindset change. They dedicated themselves to their mission of advocating for AIDS patients, and put in a lot of hard work to acquire knowledge by attending scientific meetings. They learned about drug design; biomedical research; clinical trials, the commercial interests of pharmaceutical companies, and how the Food and Drug Administration (FDA) of America and the National Institute of Health (NIH) function. Armed with this information, these patient advocates then sat down with policy makers, scientists and regulators and pushed for wide-scale changes by demanding transparency and accountability in the functioning of all these government and non-government agencies. Their hard work gradually began to pay off and they were able to:
- Accelerate the FDA approval of anti-viral drugs which were stuck in the trial phase, so that new drugs could be made available to patients when they needed these quickly
- Bring down the price of anti-viral drugs used for AIDS therapy
- The “parallel track” programme they created enabled seriously ill patients to gain access to drugs which were still in the clinical trial pipeline, but had not yet gone through clinical trials
- Increase funding for AIDS research
AIDS patient advocacy paved the way for more effective medical research that was responsive to the patient’s needs. This group showed that patients that patient advocates can actually influence the design, manner and execution of clinical trials.
AIDS garners a lot of media attention, but what happens when people suffer from rare diseases? They are treated as orphans by the medical community simply because pharmaceutical companies that fund research on developing new treatments tend to neglect these patients. They understandably give priority to studies on common diseases because these are much more likely to lead to the discovery of the next billion dollar blockbuster drug.
Thus patient advocacy groups for rare diseases are often formed and run by patients themselves or by their family members. One such group is the Lymphangioleiomyomatosis (LAM) Foundation which was started by the mother of a young woman who died from LAM. When the young woman asked her mother endless questions about her disease to which the hapless mother had no answers, she promised her daughter that she would help to find them. Based on her determination to change the system, she started the LAM foundation (www.thelamfoundation.org) with the help of the physician who treated her daughter. Such rare disease patient advocacy groups have played a crucial role in:
- Raising awareness about the disease
- Gathering information about the disease and starting a registry for the same
- Educating researchers about their disease and symptoms
- Promoting research on rare diseases by raising funds for research activities
Often patient advocates (www.rareadvocates.org) work together as a group to amplify their voice so that their demands are heard and met by researchers and the government.
Following the success of ACT UP, LAM foundation and a clutch of other such organisations, many patient advocacy groups became active participants in research. New special interest advocacy groups mushroomed and their growth was so fast and haphazard, they began to interfere with research with disastrous consequences because they forgot about Murphy’s Law of Unintended Consequences.
These loosely-formed groups naively began to expect that their participation would help in expediting the development of new drugs and that this would benefit patients by allowing them quicker access to new treatments. In one particular study, this intervention ended up playing havoc with the life of patients with metastatic breast cancer.
A therapy called high dose chemotherapy with autologous bone marrow transplantation (HDC- ABMT) was being tested for treating women with advanced breast cancer. Phase II clinical trials had been completed and phase III trials were on. Before this stage could be completed, the patient advocacy groups overseeing the trials started to pressurise insurance companies to pay for this experimental therapy in regular clinical practise. The emotional pressure from patients who were clamouring for what was presented as a medical breakthrough ; and the negative media coverage because the insurance companies refused to cover for a treatment which was unproven and very expensive, forced insurance companies to cave in. Many women with metastatic breast cancer were treated with HDC-ABMT therapy. Several years later, the investigators reached the painful conclusion that HDC-ABMT therapy caused more harm than good, and it had to be abruptly abandoned. Since insurance companies had agreed to pay for this very expensive treatment, 41, 000 women underwent this futile, painful therapy, which even cost some patients their lives. This incident serves as an example of how overzealous and well-meaning patient advocates can end up inflicting harm on the patients they are meant to protect. To guard against such disasters, it is important that patient advocates gain scientific expertise about the disease, the drugs and clinical trials, before trying to leverage their clout to influence the decisions of researchers, policy makers or insurance companies. Critically-ill patients are desperate and willing to “try anything” to get better. At such times it’s important that patient advocates advice rationality. They should keep in mind that not all scientific experiments succeed and that a clinical trial means that doctors do not have all the answers - not yet.
Medical research often progresses in an unpredictable fashion, and trying to fast forward it is not sound science. Clinical trials are conducted to gain knowledge and benefit future patients - they are not designed to benefit trial participants. In India, the Supreme Court and the NHRC Guidelines on Medical Research forbid monetary inducements for participants to safeguard their interests.
In hospitals, clinical trials must have the approval of ethical committees that also invite lay people on the decision-making panel. In essence, these committee members function as patient advocates but they need to do their homework in order to understand the nature of scientific research and its limitations, in order to be able to contribute to the research process in a meaningful fashion.