The following are his remarks:

Good Afternoon.  Thank you for inviting me to speak as a patient advocate at this meeting. Because I have a currently incurable, progressive degenerative brain disease, I am going to finally heed the advice of my neurologist and read from text for this talk. I should start by telling you a little bit about myself.  While in many ways I am increasingly the typical 21^st century patient sitting in the doctor’s waiting room or standing in line at the pharmacy, in other significant ways I am an atypical patient.  The differences between the two may present the biggest challenges to creating effective decision-making tools for patients if we are serious about giving patients as a class a meaningful seat at the health care policy table, and real power as individual patients to make, or meaningfully participate in, decisions in the management of their own health care.  

I am a typical 21^st Century patient in the following ways: I am middle-aged. I have chronic co-morbidities, including Parkinson’s disease for (which I’ve had for 12 years), type II diabetes, and hypertension. I passed by my ideal weight 20 pounds and 20 years ago.  I take nine different prescription medications multiple times daily to help manage my diseases and their symptoms.  As I am speaking, there is a small device behind and inside my left ear, which feeds an altered and minutely delayed reproduction of what I have just said into the auditory and speech channels in my brain (I also hear it consciously – it sounds a little like Mickey Mouse on helium), which makes it possible for me to speak at sufficient volume and with enough clarity to be understood despite the speech degeneration caused by my Parkinson’s. Increasingly, such complex combinations of illnesses and their symptoms and possible treatments are becoming typical of the kind of matrix that patients, doctors, and the health care system generally will have to deal with as we move from a system built on acute care to one focusing on the care management of an older population with multiple chronic health problems. 

I am also an atypical patient. I was a lawyer and a book editor, which places me above the mean in education. Both my profession and education have given me skills that help with problem solving, locating and synthesizing information, and working my way through texts that frequently include words and terms that are new to me. I have been a Parkinson’s disease advocate for a decade, which has familiarized me with the current systems and networks for the provision and regulation of health care beyond even the average well-educated lay person. I use the Internet and know how to search for information relevant to my health care. English is my native language. Lastly, I am a white male, which gives me less quantifiable but important cultural and social affinities with most health care professionals.

This conference has included patients as stakeholders and decision-makers in the management of their own health care and its regulation. The Parkinson’s Action Network (PAN) applauds this as an example of the FDA’s response to the general criticism that the missing voice at the healthcare policy table is the voice of the patient. I think it is vital that the patient voice be included not only in individual health care decisions, but at every level of decision making, including IRB’s, Advisory Committees to PhARMA and Biotech companies, and relevant government agencies.  Patient participation in health care decisions still has a long way to go before it is an accepted fact and not just a platitude, but thankfully we are making a start. 

For the patient to participate as a full-fledged stakeholder in today’s health care environment, we must overcome a health care culture stretching back generations.  This culture has regarded the patient as a passive and uninformed recipient of the specialized knowledge and training possessed in some measure by everyone else here today. A patient’s unsolicited input or questions about diagnoses or treatment protocols have traditionally been met with an attitude of “you worry about your insurance (or lack of it), and I’ll worry about how to treat you.”  More often than not, patients accepted and even encouraged the assumption that doctors, researchers, drug companies, and government regulators were responsible for and capable of fixing what ailed them.  It was comforting to think that the people in the labs coats—one part scientist, one part magician--had solutions to all of our medical problems.  Gradually, however, more and more patients have been disabused of that comforting but erroneous notion.

The idealization of medicine hasn’t completely disappeared, but two factors are hastening its demise. First, physicians increasingly have less time to spend with each patient, reducing both the time that the physician has to get a clear picture of the particular patient and the problems he or she presents, and to give the patient a clear understanding of a chosen or proposed treatment.  Secondly, we now have an active patient population that increasingly wants and needs to have a voice in the decisions made about its health care. That population--particularly the growing number with chronic illnesses like Parkinson’s that require a lifetime of treatment decisions as the disease progresses--needs tools to assess risk and benefit in managing their care. 

That said, all of us here today, including patients and their representatives, must recognize that the average patient, while integral to the processes of both individualized health decisions and the broader area of healthcare policymaking, is unlikely to have specialized training in medicine, pharmacology, biologics, the systems and organizations that provide healthcare, healthcare policy language, or the Code of Federal Regulations.  We run a great risk in simply handing over to individual patients an array of treatment options to choose from as if they were a smorgasbord without also providing the tools that they need to make informed decisions.  Likewise, we must create tools by which the unique perspective, which only patients can give as to the experience of their illness and its treatment can be captured and used to better inform the policymaking process. Creating these tools will not be simple or easy, but create them we must if we are to make the once passive recipients of health care decisions made by others into effective and meaningful collaborators in developing healthcare policies and practices which will meet the new requirements of a new century.

With regard to decision-making at the individual level, I think the most basic need of the typical patient is access to information that is relevant to his or her individual health profile. This information must be easy to find and as easy as possible to understand. Patients need a tool to find their way through the incredible mass of information they don’t need, or can’t hope to understand, and locate the information they do need and can understand without specialized training or experience.  I think that ultimately, the most usable tool will be a web-based, user-friendly data site.  It may be part of Medwatch.  It may be a clearinghouse for data already online. It may be an entirely new Web site devoted to this particular purpose. It does need to be available and usable by ordinary patients trying to make informed decisions about their care. 

The patient must also know that the tool exists.  Information about it must be broadly and consistently distributed through a variety of information portals.  These might include television and radio PSA’s, much like Direct to Consumer advertising, informational brochures distributed through doctors offices, hospitals, clinics, and pharmacies, advertisements on patient forums or patient advocacy sites, and relevant government Web sites.  Whatever the method or methods, the patient population must know about the tool in order to use it.  And if the tool does take the form of a web-based data access point for patients, there must be an agency or panel willing to step up and take responsibility for ensuring that the tool is as accurate, up-to-date, and easy to use as possible.

At the policymaking level, all of us here today will gain by creating tools that provide more data about what patients are thinking about their diseases and their treatments.  A recent study in Europe showed that information derived from online patient forums turned up serious adverse events that had not yet been reported or detected.  With due consideration for patient privacy in such forums, data mining of this type could be an extremely valuable tool in assessing post-approval responses to medications.  Polling patient populations has also uncovered otherwise unrecognized symptoms as basic as pain in Parkinson’s patients.  Finding out what patients are talking about and thinking in something like real-time is vital to move quickly in covering and responding to issues that arise after medications are in the marketplace. 

To sum up, we need both the typical and the atypical patient or patient representative as participants in healthcare decision-making.  Typical patients will increasingly be called upon to actively participate in the management of their own health.  In turn, the typical patient needs the atypical patient or patient representative, more familiar with the intricacies of the health care system and its policymaking apparatus, to provide the patient perspective on committees, councils, boards -- industrial, governmental, and academic -- wherever discussions are being held and decisions made about health care policy and practice. Having the patient involved in the process of drug trial, approval, labeling, and post-approval monitoring, provides not only invaluable information and perspective for health care providers, the drug and biotech industries, and regulators, but empowers patients, increasing their ability to make informed choices, and provide valuable data, about their own health care. 

Adding the patient perspective to policymaking will improve those policies.  Having the patient perspective at the front end of the pipeline will result in fewer problems or surprises downstream. By recognizing and addressing the need for comprehensible, easily accessible information available to all patients, we can make the average patient a genuine and effective decision-maker in the increasingly complex world of health care.