At 5:28 PM 3/28/97, Camilla Flintermann wrote: >Our Rep. John Boehner opposed "earmarking" of funds for SPECIFIC diseases >last year--he didn't thing Congress "should tell the NIH what to work on " >(!!!) He also argued that the FDA needed to be reformed so drug companies can >get new drugs approved (and make money) sooner, than we'd get all the new >meds we need, and not need money --eg=Udall. We're trying to get an appt. >with him now, but I'm not too hopeful of outcome. > >Camilla Flintermann, CG for Peter, 78/7, Oxford,Ohio >[log in to unmask] >* * * * * * * * * * * * * * >"Ask me about the CARE list for PD caregivers!" >* * * * * * * * * * * * * To Camilla, and to all: This is a response to the "earmarking" issue. What follows is an examination of the NIH review process for PD funding. It begins with a letter to Sen. Kyl, arguing, among other points, the issue of "earmarking." It includes as well our subsequent letter to Dr. Hall questioning the NINDS review process, and a short excerpt from his response. The letter to Dr. Zach Hall, attempts to demonstrate why "earmarking" is needed for PD research. Our data, and Dr. Hall's reply, points to a type of discrimination - benign, perhaps, but terribly deleterious discrimination nonethless - which faces PD researchers in the viciously competitive world of medical research funding competition. May 17, 1996 Senator Jon Kyl 702 Senate Hart Office Building Washington, D.C. 20510 Attention: Tom Alexander, Legislative Aide Dear Tom: It was good meeting with you last week to seek Senator Kyl's support for the Morris K. Udall Parkinson's Research and Education Act. Your questions were pertinent, and your request for additional cost savings data to present to the senator was reasonable and valid. I believe my responses are reasonable and valid as well. I hope you share that belief when you have finished reading them. Population We don't know the Parkinson's population in America. No one has taken a census. Nonetheless, for a number of years NIH consistently estimated the Parkinsonian population of this country at 500,000. Recently it has cited "up to 1.5 million" as a possibility. Other analysis indicates even this number may be below the true total. Whatever the current number, as the "Baby Boomers" grow older the Parkinsonian population will skyrocket. Here is the Census Bureau's numbers, with emphasis on the 55 and older population: Pop. 55Yrs. % of the Pop. %of Pop.55 Year U.S. Pop.(mil.) &Older(mil.) Increase55&Up &Older 1995 263.4 54.9 - 20.84% 2000 276.2 59.0 38.3% 21.36 2005 288.3 65.9 57.0 22.86 2010 300.4 74.6 71.9 24.83 2025 338.3 102.6 73.9 30.33 Notice the increasing rate of growth projected in the "55 and older" population. Four years from now almost one-half of our population growth will be "55&Older." By 2010 almost three-quarters will be in that category. What is significant about age 55? Fifty-seven is the average age of diagnosis for Parkinson's. As the growth of population in this age group accelerates, the incidence of Parkinson's disease in America will also accelerate. A recent study in the New England Journal of Medicine presented pertinent data on a sample of East Boston residents over 65. Applying data from that study to the American population, an estimated 2.2 million Americans over 65 have PD. Also, the incidence of "Parkinsonism" would double every ten years in that same age group. Separately, Dr. Erwin Montgomery, a reknowned University of Arizona neurologist specializing in PD, estimates some 2 million plus Americans currently have PD. By 2025, he projects a Parkinson's population at 3.7 million. For this analysis, let's assume that some 3% of Americans over 55 are victims of PD, recognizing that while this number may be somewhat high it serves to compensate for the estimated 40% of PD patients who are under 60. Costs Total annual cost of the disease is said by NIH to be about $6 billion. At 500,000 Parkinsonians, that's only $12,000 per patient, and at 1.5 million, $4,000. Either cost is fiction. Dr. Ole Isacson, a noted neuroregeneration researcher at Harvard, estimates that PD costs the nation between $31 billion and $56 billion annually. On a micro scale, in the early stages of a study designed and undertaken byThe Parkinson's Action Network to identify specific disease-related costs, the average for fifty-two Parkinsonians approximates $46,400 per person (see the enclosed "Cost of Parkinson's' Summary"). For the sake of simplicity and to be conservative, let's call it $40,000. Applying the 3% incidence rate and a constant $40,000 cost per capita to the growing American population over 55 through the year 2025, and the numbers validate Dr. Isacson's estimates: Pop. in Millions Cost in Year Over 55 w/PD $Billions 1995 54.9 1.65 $65.9 2000 59.0 1.77 70.8 2005 65.9 1.98 79.2 2010 74.6 2.24 89.5 2025 102.6 4.10 164.0 Whether these costs are met privately or through the government is only a matter of bookeeping - they are ultimately borne by all of us. Savings through Therapies Delaying the severe symptoms of the disease through improved therapies will save society considerable money. Within one year of diagnosis the typical PD patient loses his job. And, odds soon double that he will fall, causing increased disability, even his eventual death. As the illness progresses, in spite of treatments currently available, he loses more and more of the human capacities we typically take for granted. When all therapies fail, he may go on, in a vegetative state, for many years, imposing pain for family and friends and a financial burden on society. In the latter stages of the disease, if he can think, he can't communicate those thoughts. In fact, he can't do a thing for himself, not a thing. He is a prisoner encapsulated in a totally useless body and, most cruelly, with a mind that may still work. If Dr. Isacson and other specialists are correct, and new and improved therapies are developed that alleviate symptoms or delay the disease, immense savings will be achieved. To illustrate, assume that, under Udall funding, savings would not begin to accrue until the third year, and then would be rather modest booth in the number of patients and per patient cost savings. Even with this guarded optimism, the outlook for a significant and early "return on investment" from Udall funding is terrific. If during the third year - because promising therapies now being developed could be accelerated with additional funds - a mere one hundred thousand people could stay on the job one additional year (average $18,200 each), keep assisted living or a nursing home out of their lives for one year (average $8,800), and not require any "'Dis/SSI/SS" (average $11,200) for a year, savings of $1.82 billion, $.88 billion, and $1.12 billion, respectively, would accrue to the national economy. The projected $70.8 billion cost to the nation in the year 2000 would be reduced by almost $4 billion in the third year of Udall funding. That's a return of $4 billion on $300 million - 13,333% - over three years. Unrealistic? Not at all! The experimental drug I am taking has delayed my departure for places unproductive for at least a few years. It is clear that, if the research dollars proposed under Udall are authorized, appropriated and invested wisely, even an annual return of 13,333% may be conservative! Earmarking Your explanation that the senator is not fond of "earmarking," and has voted against measures on that principle, got my attention. The Udall bill may be "earmarking," but if it is I'd like to point out that it is so only because research in PD has been, and is to this minute, so terribly underfunded. As evidence, please look at the three enclosed graphs. Graph 1 shows the amount of NIH direct research funding in FY91, by disease, for six selected diseases. Graph 2 shows the incremental dollar increase in direct funding in the FY95 budget over that in the FY91 budget. Graph 3 shows the percentage increase for direct research funds between those years, by disease. Parkinson's, with the tiniest base to start with, continued to see the gap widen. PD funding had the lowest percentage increase among the six diseases. Last in FY91, a more distant last four years later! And the per patient numbers are just as pitiful: $26 for Parkinson's, $1,069 for AIDS, $295 for cancer - but, I left that data with you. If this doesn't show that we've been underfunded, too much and too long, please tell me how to prove it. Perhaps this condition is partially our fault - the Parkinson's community has not done the effective job of lobbying, both in the Congress and with the American people, that needs to be done. Maybe it is because "people don't die from Parkinson's," a common allegation even in medical circles, but seldom believed among friends and family of a late Parkinsonian. Or, maybe it is because Parkinson's has been indelibly ingrained in many minds as a disease of stumbling old people whose time has come to fall by the wayside. I don't know. But I do know that this sixty year old is not prepared to cash in his chips. Nor are the many superior people I met while in Washington, most younger than I, who are courageously fighting this insidious disease. They, and I, have faith in the research people who say - not for self-aggrandizement but because they truly believe it - that a relatively insignificant infusion of funds would result in absolutely monumental results, even a cure. "Earmarking" may be the technical term that applies to the process, but wouldn't "catching-up," or "equalizing," be a more accurate description of the result? (NOTE: Since this letter was written to Senator Kyl, we sent the letter found at the end of this letter to Dr. Zach Hall, NINDS director, concerning the preponderance of PhDs in the NINDS review process for Parkinson's funding, as well as the lack of published documents on the subject of Parkinson's disease by the committee members. The jist of our argument to Dr. Hall is "If the reviewers are not up to date in their familiarity with the disease (and they are not, according to our research), and don't understand the disease, how can they appreciate the potential the many PD applications may hold? Dr. Hall did not refute, or even seriously respond to, any of the data we submitted. If I had a scanner I'd include his entire response, but it was a "puff ball," wordy and non-responsive, technical, obtuse and conclusive without furnishing data to support his conclusiveness. A couple quotes deserve mention, however: 1) "I do not agree that Parkinson's disease is underfunded relative to other diseases. The NINDS has a rich and varied program in Parkinson's research, one which we are very proud of." 2) "Finally, I am very pleased to inform you that NINDS funding for Parkinson's disease research increased significantly in Fiscal Year 1996. The total for the past year is $54,283,000. This represents an increase of approximately 10 percent from the 1995 level of $45,396,000. Most of the increase is in the "direct" category." Are you surprised? Do you think a senator's aide, or a congressman's aide, can argue with this type of presentation? Question is, can we?) Other Data Other pertinent information concerning the disease was left with you. But if there is anything lacking, anything at all that will help convince Senator Kyl of the fiscal and human validity of our cause, we will do all in our power to provide it. I appreciate your time,Tom, and the genuine interest you have shown in our request. For the tens of thousands of Arizonans with the disease, and their many caregivers and friends, I ask that Senator Kyl join Senator McCain, and Representatives Kolbe and Pastor, to honor the man and his legacy, and become a co-sponsor of the Morris K. Udall Parkinson's Research and Education Act. Sincerely, The letter to Dr. Hall: __________________________________________________________________ ARIZONA PARKINSON'S ADVOCATES A COMMITTEE OF THE AMERICAN PARKINSON'S DISEASE ASSOCIATION 5319 E CALLE BOSQUE, TUCSON AZ 85718 TEL. 520-299-1030 FAX 520-299-8795 E-MAIL [log in to unmask] _________________________________________________________________ December 18, 1996 Dr. Zach Hall, PhD Director, NINDS Bldg 31, 31 Center Drive MSC2540 Bethesda, MD 20892-2540 Dear Dr. Hall: Thank you for speaking to us at AGENDA 97. We are honored to be the first local Parkinson's group to hear from you. Our only regret was that your visit with us passed too quickly. With more time I am sure we would have covered many issues, particularly related to Parkinson's research funding, that now perplex us. As you already knew, and heard again, many in the Parkinson's community believe research of the disease is terribly underfunded within the NIH/NINDS structure. Hence the push for the Udall bill. Increasingly they are also concluding that what funding is available is being directed more toward "basic science," which offers little promise for those with the disease, rather than "clinical" research which could result in more immediate improved treatments. Some attribute this conflict to the composition of the committees within NINDS that pass on the merits of funding proposals. They contend that the committees are far less interested in clinical research, often favored by investigators who are also "hands on" medical doctors and have a greater involvement with Parkinson's patients, and more interested in a search for "the underlying biology of the disease," more frequently identified with PhDs. And, recently some have even questioned whether those reviewing applications and determining the allocation of funds to Parkinson's research are sufficiently aware of recent developments in the field to assess their merit vis a vis applications in areas with which they are more familiar. The issue, then, is the validity of these beliefs. From information provided by your office concerning the Neurological Sciences Program Project Review Committees, NSPA and NSPB, as well as from "Medline," we have found that 1) there is compelling evidence that PhDs vastly outnumber medical doctors on these committees, resulting in a "basic science" philosophy, and 2) remarkably few members of the committees have written remarkably little for publication on Parkinson's issues, reflecting a lack of involvement in current Parkinson's research. > Education and experience of the members As illustrated in the tables immediately below, a substantial majority of the members of the NSPA and NSPB have not been medical doctors experienced in dealing with hands-on problems of Parkinson's disease. Non-medical doctors predominate the membership on the review committees. Based on education and experience alone - and no judgment is made questioning their ability or intentions - the majority of the members are simply underequipped to understand the significant therapeutic benefits a Parkinson's research proposal may hold for the ultimate consumer, the PD patient. Here is the representation by degree status - MD, PhD, both, and other - in the "A" and "B" Committees (lists from NINDS) over the last five years: "A" Committee (person years) PhDs. MDs Both Other (M.S.P.H.) 1995-96 9 5 1 1 1994-95 8 4 2 1 1993-94 9 5 1 1 1992-93 7 5 3 - 1991-92 8 5 3 - Totals 41 24 10 3 "B" Committee (person years) PhDs MDs Both Other 1996-97 10 5 1 - 1995-96 10 5 1 - 1994-95 10 4 2 - 1993-94 10 4 2 - 1992-93 11 5 - - Totals 51 23 6 - Combined Totals 92 47 16 3 Nearly twice the number of committee members were PhDs as compared with MDs. Even if we assume that all of those with "Both" degrees were more inclined toward a "hands on" approach, and added them to the MD total, PhDs would still constitute the vast majority of proposal reviewers. > Who has written about Parkinson's? Very few. Using information supplied by your office, and from "Medline," we identified publications containing one or more of four terms - Parkinson's disease, dopamine, basal ganglia, and motor control - any one of which would trigger an examination of the abstract to determine the publication's relationship, if any, with the disease. The following table presents the numbers of publications published by current members and past members (term expired during the period) of both committees from 1992. The first number is the total of all publications written by committee members. The second is the number apparently related to Parkinson's disease. The percentage that PD articles comprise of the total published is in parentheses. "A" "B" Total Current 371 - 1 348 - 8 719 - 9 Members (.27%) (2.3%) (1.25%) Past 407 - 2 327 - 4 734 - 6 Members (.49%) (1.22%) (.82%) _____ ______ ______ Totals 778 - 3 675 1,453 - 15 (.39%) (1.77%) (1.03%) Aside from Dr. Caroline Tanner's eight publication contribution on the "Current B" committee, Parkinson's clearly is not a priority among the sixty-two men and women who played such a critical role in distributing scarce research dollars. Caveat , conclusions and request While it was undertaken with all due diligence, we do not claim this analysis is without inadvertant error. Also, we were unable to obtain from your office the names of the "ad hoc" committee members, some of whom may have provided some technical assistance in reviewing PD proposals. But it is difficult to conceive of any error or errors, or any "ad hoc" committee representation, which could alter the obvious conclusion that Parkinson's research specialists are underrepresented in this review process. Further, it strains credibility to believe that research scientists who are predominantly not medical doctors, and who specialize overwhelmingly in areas other than Parkinson's research, could be capable of reviewing funding proposals for Parkinson's with the same understanding, appreciation and empathy with which they review applications in their own areas of specialization. In light of the above, we would appreciate responses to the following: 1) Would you please send us the complete description of the review process(es) for any research proposal from the moment it is received at the NIH. 2) If not contained in the description, how are reviewers named? 3) If not contained in the description, what are the criteria used to determine a proposal's "scientific excellence?" 4) How are the relative continuing costs of diseases - health costs, lost productivity, long-term care costs, etc. - factored into the review process? 5) Finally, for the most recent five year period, a list of the members of any other committees that review and determine the fate of Parkinson's funding proposals. Your review of and response to this data - and we sincerely appreciate your offer to do so - will help the Parkinson's community understand how the funding process works, and provide some insight into how to better design our appeal for additional funds for Parkinson's research. Again, thank you for taking the time and making the effort to be here for AGENDA 97. We are confident that the goal of our symposium - "Researchers and advocates carry the torch" - will be well-served by the thoughts and suggestions you articulated during your brief stay. Sincerely, Robert L. Dolezal Chairman, Arizona Parkinson's Advocates cc Morton Kondracke Joan Samuelson Paul Smedberg