> INTERNATIONAL
As you will read toward the end of this update, we are delighted to announce that Carol Grayson, of Newcastle-on-Tyne, England, came to COTT’s Anniversary Celebration. She was active from the earliest days of the AIDS epidemic, caring for community members as well as fighting government refusal to release information. Her work expanded to include hepatitis C as that became more widespread. And, for the last ten years and more, Carol has been a tireless advocate on behalf of the hemophilia community in battling Creutzfeldt-Jakob Disease (Mad Cow), to which many in the community, including her husband, may have been exposed. Her recent dissertation topic was on how the global politics of blood impacted on the UK hemophilia community.
> CONGRESS
Health Care Reform
The brief but intense month of Congress’ July session featured release or near-release of the three health care reform bills in Congress. We will detail the bills below. Both released bills had the all-important public option component, and lay down strict limits on insurance companies to eradicate past grossly unfair practices, and both cost a trillion dollars or more over time.
As you will read toward the end of this update, we are delighted to announce that Carol Grayson, of Newcastle-on-Tyne, England, came to COTT’s Anniversary Celebration. She was active from the earliest days of the AIDS epidemic, caring for community members as well as fighting government refusal to release information. Her work expanded to include hepatitis C as that became more widespread. And, for the last ten years and more, Carol has been a tireless advocate on behalf of the hemophilia community in battling Creutzfeldt-Jakob Disease (Mad Cow), to which many in the community, including her husband, may have been exposed. Her recent dissertation topic was on how the global politics of blood impacted on the UK hemophilia community.Health Care ReformThe brief but intense month of Congress’ July session featured release or near-release of the three health care reform bills in Congress. We will detail the bills below. Both released bills had the all-important public option component, and lay down strict limits on insurance companies to eradicate past grossly unfair practices, and both cost a trillion dollars or more over time.
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Neither retains earlier elements to raise this amount, we are pleased to report, through taxing health care benefits, which it was feared could be hellacious for those in the high-cost hemophilia community. Actually, it would be a tax on the value the benefit as if it were added to the employee’s income. That is, taxing the policy benefit, not any claims or payouts made under it. To help pay for the reform packages, instead, a proposal to exact millions of dollars in fees from the insurance industry itself has been substituted.
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We would like to give you here some idea of the three major bills, their characteristics on five main areas of structure, and what we know about where they stand on some elements most critical to our community. As things keep changing and evolving, however, note that not only will we be mentioning changes that have already occurred, but there will no doubt be more that happen after we finalize this Update – so you should see what we’ve laid out, but then be attentive to the news for changes as well.
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Of course, nothing technically could change while Congress was not in session, during August. There was a ten-day rush in both Chambers just before adjournment to try to pass something, even if only to successfully vote one out of committee (one already did so, earlier), although floor passage was the ideal, but that did not happen.
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THE BILLS
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In the Senate, the Health (Health, Education, Labor & Pensions: HELP) Committee met for a month, chaired by Acting Chair Sen. Chris Dodd (D-CT) in Chairman Ted Kennedy’s absence, and on July 15th passed The Affordable Health Choices Act. We will refer to this bill as the HELP bill, rather than by the chairman’s name, given the uncertainties of the time.
Also in the Senate, the Finance Committee, Chaired by Sen. Max Baucus (D-MT), gave indications of a bill taking form as early as January of this year, and has worked intensely with its members and staff, but at this writing has only begun markup. It would appear that committee Republicans and Democrats are still too far apart in their positions to expect the Committee to not produce a bill anytime soon. We will refer to this as the Baucus bill.
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In the House, the Energy & Commerce Committee, chaired by Henry Waxman (D-CA), has entered into an agreement with the Ways & Means Committee and the Education & Labor Committee to offer up one bill: all three began markup at the same time (the week ending July 17).
The bill, HR 3200, But while the other two committees finished their work in a day or two, the Energy Committee suffered an interruption of markup by some members who wanted major reductions in costs in the bill. After changes were made, the Committee barely completed its work, on a substantially modified bill, hours before Congress left town for a month at the end of July. These three bills will be collectively known as the Waxman bill.
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THE CHANGES
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The explosion of modifications to the bills just before recess was the result of an upwelling of opposition to the bills as introduced, which had several ‘waves’ to it. This began when, in the Senate, a decision was made by leadership back in the Spring, before the work began, to classify these reform bills under a category of legislation called ‘reconciliation.’ Usually reserved for technical changes, or budget bills without program specifics, this route was chosen because passage requires only 51% of votes, not 60% as in most funding bills. Accordingly, the Democratic chairs of the relevant committees were given free rein to push their bills through the mark-up (modify & vote) process, over any objections from the minority, since the percentage of majority members on each committee was well in excess of the 51% majority votes needed.
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This did not sit well with the minority members, despite the occasional case in which a minority amendment was accepted, which did not mollify them and in fact was seen as lip service. The groundwork for the blowup and stopping of progress in the last few weeks before recess was thus laid.
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This Republican discontent began in the Senate when the Health committee wrapped up 14 days of marking up its bill on July 15th over the intense opposition of its Republican members (and some 200 defeated Republican amendments). The negative groundswell so created then moved in two directions: down the hall to Senate Finance, to strengthen resolve of minority members there, as Finance got started putting together a bill; the venom also spread to the House, where the Energy & Commerce Committee was just beginning a five-day markup.
In that House markup, on Day Two Republicans raised such opposition to the terms of the bill, along with that of ‘Blue Dog’ Democrats (fiscal conservatives) of the Chairman’s own party, that markup was suspended for what became a week of negotiations. It resumed, with a heavily compromised bill. Through a concerted last-minute effort on July 31, the day before the House’s month-long recess, the bill passed.
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While all three bills have been thoroughly developed and have merit, the public eye has seen the HELP bill, the most costly as it is the most thorough, and the Waxman bill, complex and thoroughly attacked, fade from view. The Baucus bill emerges, but as September turns into October its political utility as a standard bearer of change is diminished with every passing week.
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All three bills have been weakened, under serious insurance and pharmaceutical industry lobbying.
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Removal of controls on insurance companies, and preservation of the current market-based (shareholder profits as Priority One, client health status far less important) system, and Elimination of a Public Option, or tying its hands so it doesn’t out-compete private carriers, are examples of this. So is the requirement in all three bills that years go by under current operations before any changes are begun.
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In terms of future modifications of the three bills, currently the strongest indications are the outcomes of the Town Hall Meetings last month … and the declining popularity of the President.
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AUGUST MUD-SLINGING
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We have just endured one of the ugliest months in American politics in decades. Why was it done this way? The rational way would have been, for example, routine visits to Congress in June and July by large numbers of conservatives (other than those representing insurance and providers) to object to the Public Option proposal, to register disbelief that their current care arrangements would be retained, that the cost of the proposed plans was prohibitive. No one thought the absence of such efforts meant acquiescence, but, at one after another Town Hall Meeting during August, we heard distinguished members of Congress, including Committee Chairs, being shouted down or even suffering loud and aggressive ‘in-your-face’ confrontations, with slogans like Death Panels, Obama=Hitler, and Public Option=Complete Government takeover. As one long-term Committee Chairman in the House finally said to his hecklers, “What planet do you live on?”
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The Republican, or business-first perspective on health care change is very different than what broad sections of the American people reportedly support and hopefully told their members of Congress during this recess. One recent column citing industry figures reported that profits for the top ten insurance companies are up over 400% over ten years. At the 40% per year that this works out to, they’re right up there with the pharmaceutical industry! Yet the message is, spread fear: the Obama plan will hurt your family by taking away the good health care you have now, imposing new taxes, putting a government bureaucrat between you and your Doctor, and costing the nation trillions of dollars we don’t have.
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Countering this is the real world: like most large corporations today, insurers make unconscionable profits and are always seeking ways to provide less service per dollar received.* In fact it is insurers who force themselves between doctor and patient.
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The plan is to not impose new taxes on most families, but find revenues elsewhere, not the least from reducing excess charges for unneeded procedures, and changing to pay for outcomes, not procedures.
On government intrusion: Do Medicare patients wish the program would end? One part of it, Medicare Advantage, is run by private companies, who charge $17 Billion a year yet provide no advantages for patient nor savings to the client (government). Ending this Bush-era program alone can permit reallocating the funds to providing over half the cost of beginning to cover the 45 million uninsured people in this country.
The idea of a government worker between the Medicare patient and the Doctor is a negligible issue compared to the value of the care. At least, unlike the true horror stories we have been hearing about private insurance, Medicare won’t cancel your policy for just filing a claim. The
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*For a firsthand report on the internal workings of insurance to limit care, limit costs, fight opposing plans and discredit critics, see Bill Moyers’ interview with former Cigna Vice President for Publicity on the pbs website http://www.pbs.org/moyers/journal/index-flash.html. The program originally aired earlier in July. It has already been re-aired.
specter of government intrusion has been magnified into terms like ‘government takeover’ and ‘socialized medicine.’ Business leaders know that advertising works, even if far from the truth. Caveat emptor – let the buyer beware.
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Lastly, citing the cost of the program is flawed in two ways. Usually pundits cite the figures released in July by the Congressional Budget Office. Analysts in such offices cannot include savings expected in a program from things like nutritional education or smoking reduction, for the simple reason that they get no guidance as to WHEN such savings would accrue. The Obama/Kennedy plan has many such savings built into it, including those from a substantial implementation of health information technology. CBO-reported costs lack the offsets expected from such savings. If they were included, the cost of each bill’s plan would be far less.
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The second way in which high-cost alarm logic is flawed, is that it fails to include the savings when the coverage everyone will have, no longer has to pay for so many high-cost defensive
medicine procedures, and no longer has to pay for repeat procedures when the original was in error. The savings overall from reduction in defensive medicine, and the unduplication of test after test thanks to computerization’s ability to see what the test result was even if done elsewhere, will be enormous. Sure we’re adding 50 million people to the rolls, but that’s only one-sixth of our population, and much of the funds to meet those costs is already available. The savings will accrue from all of the population’s programs, when fully operational, in addition to specific savings already mentioned.
STRUCTURE
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The sweeping plans currently under study all deal in what can be reduced to five main areas. Not every one addresses all five; but these are benchmarks from which to compare them.
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Kennedy Baucus (mod) Waxman (mod)
Public Program yes no yes but hobbled
Individual Mandate yes yes yes
Employer Mandate yes no yes (only 15%)
Financing Insurance tax Insurance tax tax on richest
Quality/Cost Controls ___ ___ ___
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With regard to the last row of this chart, Quality & Cost Controls, much of the debate in Congress in July was specifically about the juxtaposition of the high apparent cost of the program, as cited by the CBO (with none of the savings just mentioned), and the lack of cost-limiting controls and quality-increasing controls. The question being asked is how does a given plan “bend the curve” of future cost increases expected under the plan vs. under the status quo. To date, most of the plans have been light on both cost control and quality-improvement issues, although some point to the language on “Comparative Effectiveness Research” in the Stimulus package, which is a structure to improve determination of relative effectiveness of medications, as laying the groundwork for – unfortunately not estimable – savings from full outcome-oriented (vs. procedure oriented) medicine.
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A last-minute note: it seems certain, as of the end of September, that the two Senate bills will be somehow merged, and go to a Conference Committee to reconcile the results of that merger with the House legislation from Mr. Waxman. Although the Public Option was defeated in the final votes of Senate Finance, it is a strong part of the House bill, which may be enough to assure that it stays in the bill coming out of Conference. Also, the White House is expected to make its wishes known (pro Public Option) very clearly to the Conferees.
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Before we leave this discussion, let us look at two salient aspects of this reform effort.
Mandates Many eyes have been on the state of Massachusetts as it has worked to implement an employer-mandate, individual-mandate reform begun several years ago. Many adjustments were needed in the first two years of the program, to assist the lowest-income populations to enroll, and to contest with business owners and their associations at the state capitol over their reluctance to provide for their employees. True, it still costs more than expected, and costs continue to rise, but this experiment is nowhere near even ten years old – these transitions take time!
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Many eyes have been on the state of Massachusetts as it has worked to implement an employer-mandate, individual-mandate reform begun several years ago. Many adjustments were needed in the first two years of the program, to assist the lowest-income populations to enroll, and to contest with business owners and their associations at the state capitol over their reluctance to provide for their employees. True, it still costs more than expected, and costs continue to rise, but this experiment is nowhere near even ten years old – these transitions take time!
Regulation Government oversight function is not being discussed in the current debates; any such thing would be pounced upon by critics as ‘leading us down the road to a government takeover!’ Nevertheless, as Medicare from time to time takes ‘Medicare Mills’ to courts – health facilities which see patients, don’t treat them, bill Medicare for the treatment not given and pocket the payment – there must be oversight. And there is a huge opportunity here: historically oversight of the insurance industry has been a state prerogative, but now with Wellpoint serving 36 million health care customers across the 13 states in which it’s for-profit Blue Cross plans operate, maybe it’s time for a change to national regulation of the insurance industry.
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In sum, we watch and wait, and COTT keeps up its schedule of visits to key members of Congress. We monitor Sen. Baucus’ work – his staffer told COTT ‘there used to be programs in our bill to protect your and similar groups, but we took them out to reduce the cost of the bill.’ Loud actions are called for. The hemophilia community became highly respected on Capitol Hill during Ricky Ray. Here, as among those most in need, we must try to gain some protection despite the drafters’ focus on the general population. If there was ever a time to be heard, this is it!
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> AGENCIES
FDA’s Transmissible Spongiform Encephalopathy Advisory Committee (TSEAC, a term that covers all forms of Mad Cow Disease) met in June, for the most part (since it did not meet at all in 2008) due to the release in the UK earlier in the year of results showing that prions, the active agent in all TSEs, could be blood-borne. At the meeting FDA presented once again their risk model, showing the reduction of the prion count through fractionation (the process by which factor is made); unfortunately given the few people who have had the disease and the long period of incubation, science has not yet identified what level would be safe for the recipient: If there are a hundred viruses per cup of a liquid the processing plant obtains, and the processes there eliminates 83 of them, leaving 17, it is important to know if 17 is small enough to be sure the product is safe. What if the safe level is 5? Or 8? Or 16? The consumer would be at risk.
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Our regular feature of excerpts from recent FDA Enforcement Reports can be found at the end of this Update as Attachment #1.
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> INDUSTRY
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The manufacturer’s lobby association PPTA held its second Stakeholder Meeting – industry and community representatives together – in early September. A main topic was health reform and the current deadlocks that seem to threaten the legislation altogether. The various groups presented their legislative priorities, which in our case included Follow-On Biologics, renewal of the Ryan White AIDS Program, and plans to fight next year’s scheduled renewal of the Prescription Drug User Fee Act (under which industry pays FDA to evaluate industry’s proposed new drugs. New this meeting was participation by the National Organization of Rare Diseases, certainly a sister group to all blood-products-using communities.
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> COALITIONS
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The Plasma Users Coalition, which drafted the letter to Senator Baucus and others, that was included in the last COTT Washington Update, has since prepared a statement of General Principles which has been sent to all members of key health committees, and begun followup visits. A copy is included as Attachment #2 to this issue.
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> COTT Operations
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The COTT Anniversary Celebration has just taken place, here in Washington. The two-day affair consisted of a Reception on Friday October 2nd, which featured remarks by Congresswoman Lois Capps (D-CA), followed by a banquet Dinner, with speakers including our Keynote Speaker Dr. Don Francis, formerly of CDC, and an overview of current blood safety measures by Dr. Jay Epstein of the Office of Blood at FDA. Also featured was the presentation of over twenty awards; the introduction of each awardee and their comments at the podium added to the richness of the evening.
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The following morning Saturday October 3rd, the Celebration continued at the Senate Park, only a little over a block from the hotel, and adjacent to the Senate offices and the Capitol Building. After a review of COTT’s historical timeline, many reminiscences from members followed. A number of guests remarked that whole they recognized voices, they had never met one another face to face – their only contact was the Ricky Ray planning conference calls over ten years ago!
COTT acknowledges the assistance of Hemophilia Health Services and Factor Support Network
in publication of this issue of the COTT Washington Update.
Attachment #1: Recent FDA Enforcement Report excerpts
July 1
PRODUCT
Source Plasma. Recall # B-1123-09
CODE
Units: AB1119419, AB119718
RECALLING FIRM/MANUFACTURER
Bio-Save Resources of Albuquerque LLC, Albuquerque, NM, by electronic mail on September 22, 2008. Firm initiated recall is complete.
REASON
Blood products, which had not been tested for PCR Nucleic Acid Test (NAT), were distributed.
VOLUME OF PRODUCT IN COMMERCE
2 units
DISTRIBUTION
United Kingdom
___________________________________
PRODUCT
Red Blood Cells Leukocytes Reduced. Recall # B-1148-09
CODE
Unit: 3291680
RECALLING FIRM/MANUFACTURER
Florida’s Blood Centers, Inc, Orlando, FL, by telephone on January 12, 2009 and by letter dated January 13, 2009. Firm initiated recall is complete.
REASON
Blood product, collected from a donor who was at risk for variant Creutzfeldt-Jakob Disease (vCJD), was distributed.
VOLUME OF PRODUCT IN COMMERCE
1 unit
DISTRIBUTION
FL
___________________________________
PRODUCT
Source Plasma. Recall # B-1149-09
CODE
Unit: UQ032240
RECALLING FIRM/MANUFACTURER
Plasma Biological Services, Inc, Memphis, TN, by fax on June 4, 2008. Firm initiated recall is complete.
REASON
Blood product, collected from a donor whose annual physical examination was not performed, was distributed.
VOLUME OF PRODUCT IN COMMERCE
1 unit
DISTRIBUTION
NC
___________________________________
PRODUCT
1) Recovered Plasma. Recall # B-1151-09;
2) Red Blood Cells Leukocytes Reduced. Recall # B-1152-09
CODE
1) and 2) Unit: 6585642
RECALLING FIRM/MANUFACTURER
Carter BloodCare/ WE & Lela I Stewart Blood Center, Inc, Tyler, TX, by fax on April 23, 2007 and March 11, 2009. Firm initiated recall is complete.
REASON
Blood products, collected from a donor considered to be at increased risk for variant Creutzfeldt-Jakob Disease (vCJD), were distributed.
VOLUME OF PRODUCT IN COMMERCE
2 units
DISTRIBUTION
TX and Switzerland
___________________________________
PRODUCT
Source Plasma. Recall # B-1168-09
CODE
Units: 08MOHG8960, 08MOHG8189
RECALLING FIRM/MANUFACTURER
BioLife Plasma Services L.P., Lima, OH, by facsimile on February 19, 2009. Firm initiated recall is complete.
REASON
Blood product, collected from a donor with an unacceptable arm inspection, was distributed.
VOLUME OF PRODUCT IN COMMERCE
2 units
DISTRIBUTION
Austria
August 19
___________________________________
PRODUCT
Red Blood Cells Leukocytes Reduced, Recall # B-1277-09
CODE
Unit: 012FJ48930
RECALLING FIRM/MANUFACTURER
Recalling Firm: American Red Cross Carolinas Blood Services Region, Charlotte, NC, by telephone on May 8, 2007.
Manufacturer: American Red Cross Blood Services, Durham, NC. Firm initiated recall is complete.
REASON
Blood product, which was labeled as Kell negative due to incorrect testing, was distributed.
VOLUME OF PRODUCT IN COMMERCE
1 unit
DISTRIBUTION
NC
___________________________________
PRODUCT
1) Red Blood Cells Leukocytes Reduced, Recall # B-1342-09;
2) Fresh Frozen Plasma, Recall # B-1343-09
CODE
1) and 2) Units: W045008033997, W045008112160
RECALLING FIRM/MANUFACTURER
Community Blood Center of Greater Kansas City, Kansas City, MO, by letter dated May 1, 2009. Firm initiated recall is complete.
REASON
Blood products, collected from a donor with a history of high risk behavior, were distributed.
VOLUME OF PRODUCT IN COMMERCE
4 units
DISTRIBUTION
MO, KS
___________________________________
PRODUCT
Red Blood Cells Leukocytes Reduced, Recall # B-1353-09
CODE
Unit: W118809551319
RECALLING FIRM/MANUFACTURER
The Blood Alliance, Inc., Jacksonville, FL, by telephone on February 27, 2009. Firm initiated recall is complete.
REASON
Blood product, which was labeled leukoreduced, but exceeded the acceptable limit for white blood cell count, was distributed.
VOLUME OF PRODUCT IN COMMERCE
1 unit
DISTRIBUTION
GA
___________________________________
PRODUCT
1) Red Blood Cells Leukocytes Reduced, Recall # B-1375-09;
2) Platelets, Recall # B-1376-09
CODE
1) Unit: 4053319;
2) Unit: 4056171
RECALLING FIRM/MANUFACTURER
Wellmont Health System dba Marsh Regional Blood Center, Kingsport, TN, by facsimile on October 4, 2007. Firm initiated recall is complete.
REASON
Blood products, collected from a donor who was taking the medication finasteride, were distributed.
VOLUME OF PRODUCT IN COMMERCE
2 units
DISTRIBUTION
TN, MD
Sept. 23
___________________________________
PRODUCT
Red Blood Cells Leukocytes Reduced. Recall # B-1354-09
CODE
Unit: W067309002831
RECALLING FIRM/MANUFACTURER
Coffee Memorial Blood Center, Inc., Amarillo, TX, by telephone on March 13, 2009. Firm initiated recall is complete.
REASON
Blood product, manufactured from a Whole Blood unit that was overweight, was distributed.
VOLUME OF PRODUCT IN COMMERCE
1 unit
DISTRIBUTION
TX
___________________________________
PRODUCT
Red Blood Cells Leukocytes Reduced. Recall # B-1417-09
CODE
Unit: W165709006989
RECALLING FIRM/MANUFACTURER
Our Lady Of The Lake Hospital Inc., Baton Rouge, LA, by telephone and facsimile on March 19, 2009. Firm initiated recall is complete.
REASON
Blood product, which was not labeled as an autologous unit, was distributed.
VOLUME OF PRODUCT IN COMMERCE
1 unit
DISTRIBUTION
LA
___________________________________
PRODUCT
Platelets Pheresis Leukocytes Reduced. Recall # B-1573-09
CODE
Unit: W115909013585
RECALLING FIRM/MANUFACTURER
Central California Blood Center, Fresno, CA, by telephone on July 13, 2009. Firm initiated recall is complete.
REASON
Blood product, which exceeded the maximum criteria for platelet count, was distributed.
VOLUME OF PRODUCT IN COMMERCE
1 unit
DISTRIBUTION
CA
___________________________________
PRODUCT
Source Plasma. Recall # B-1581-09
CODE
Units: 08FINE8902, 08FINE0461
RECALLING FIRM/MANUFACTURER
BioLife Plasma Services, L.P., Fort Wayne, IN, by facsimile on April 13, 2009. Firm initiated recall is complete.
REASON
Blood products, collected from donors who did not receive a complete physical examination, were distributed.
VOLUME OF PRODUCT IN COMMERCE
2 units
DISTRIBUTION
CA
___________________________________
PRODUCT
1) Fresh Frozen Plasma. Recall # B-1584-09;
2) Red Blood Cells. Recall # B-1585-09
CODE
1) and 2) Unit: W120609200816
RECALLING FIRM/MANUFACTURER
Michigan Community Blood Centers, Saginaw, MI, by facsimile on April 17, 2009. Firm initiated recall is complete.
REASON
Blood products, which were quarantined subsequent to receiving information regarding a post donation illness, were distributed.
VOLUME OF PRODUCT IN COMMERCE
2 units
DISTRIBUTION
MI
___________________________________
PRODUCT
1) Red Blood Cells Leukocytes Reduced. Recall # B-1625-09;
2) Plasma Frozen within 24 hours (FP24). Recall # B-1626-09
CODE
1) and 2) Units: 7000204, 7000306
RECALLING FIRM/MANUFACTURER
Community Blood Bank of Erie County, Buffalo, NY, by facsimile on March 31, 2009 and by telephone on April 1, 2009. Firm initiated recall is complete.
REASON
Blood products, collected from a donor who was at risk for variant Creutzfeldt-Jakob Disease (vCJD), were distributed
VOLUME OF PRODUCT IN COMMERCE
4 units
DISTRIBUTION
NY, NJ
Attachment #2: General Principles from the Plasma Users Coalition
Principles for Health Care Reform and the Rare Disease Community
The Plasma Users Coalition (PUC) is a group of national patient organizations that include the Alpha-1 antitrypsin, primary immunodeficiency diseases, hemophilia, Guillain-Barré, CIDP, platelet disorders and other rare disease communities, all of which represent patients whose lives depend on plasma derived therapies. The PUC believes our health care system must provide affordable, quality coverage for all Americans, and as the health care reform debate intensifies, we have concerns regarding continued access to the specialized, expensive care and therapies for individuals with rare diseases.
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The Plasma Users Coalition (PUC) is a group of national patient organizations that include the Alpha-1 antitrypsin, primary immunodeficiency diseases, hemophilia, Guillain-Barré, CIDP, platelet disorders and other communities, all of which represent patients whose lives depend on plasma derived therapies. The PUC believes our health care system must provide affordable, quality coverage for all Americans, and as the health care reform debate intensifies, we have concerns regarding continued access to the specialized, expensive care and therapies for individuals with . .
The patients we represent have rare disorders and need life-long access to expensive, life-saving treatments. For example, the average annual cost of treating an adult male with hemophilia is $300,000 per year; the cost of treatment for an individual with alpha-1 antitrypsin deficiency is approximately $150,000 per year; and persons with Primary Immune Deficiency diseases (PIDD) who infuse or inject their therapy every four weeks costs on average $35,000 to $50,000 per year to treat. Individuals managing acute neurological disorders such as Guillan- Barré Syndrome or Chronic Inflammatory Demyelinating Polyneuropathy, who also infuse a plasma therapy on a regular basis, cost approximately $30,000 to $60,000 per year.
We applaud the recent efforts of Congress to develop a health care reform proposal. Our health care system must meet the diverse needs of all Americans, including the day-to-day needs of relatively healthy Americans, who essentially require preventative care and acute treatment for illness and injury. Likewise, the health care system must address the highly individualized needs of people with rare diseases. These Individuals present unique challenges within the health care system, as many rare diseases require expensive, complex and specialized treatments.
Rare disease patients face chronic, life threatening diseases and some of this country's highest treatment costs forcing many into personal bankruptcy and poverty! With thoughtful and reasonable healthcare reform, Congress can ensure this will never happen, ever again. The following principles highlight our unique needs and concerns:
PRINCIPLES
Private Insurance Market Reforms
The Coalition is pleased that the proposals outlined to date include guaranteed insurance coverage with guaranteed renewability, choice of health plans and no pre-existing condition clauses. We are also pleased that lifetime and annual limits of coverage are eliminated. While we applaud the annual caps on out-of-pocket costs, we are concerned that they are not adequate for the
rare disease community. Annual out-of-pocket costs and co-pays for individuals with
rare diseases are still too high and continue to thwart access to medical care. It is crucial that out-of-pocket limits are set at dollar amounts and not as percentages. High out-of-pocket costs and co-pays
disproportionately impact individuals with
rare diseases whose therapies can be extraordinarily expensive and life-long.
Affordability
Affordability can be defined as the percentage of income a household can devote to health care while still having sufficient income to address other necessities of life. Securing reasonably priced insurance and covering the uninsured is an important component of health care reform. In addition, health care reform legislation must also limit deductibles and co-payments, which can deter patients from getting the care they need and limit patient access. Co-payment percentages benchmarked at 10% or 20% that may seem appropriate for traditional pharmaceuticals are not at all reasonable for rare disease patients. With regard to individual insurance subsidy levels, users of plasma protein therapies need subsidy levels at or above 400% of the federal poverty level (FPL). Patients with rare diseases treated with plasma protein therapies are able to lead productive lives and attend school or work but they must be able to afford the therapy.
Access to Specialists
Streamlined access to specialists is critical for people with rare diseases. These individuals require services and care from members of the medical profession who have specialized knowledge of the diagnosis, treatment and management of their disorders. The current proposals do not explicitly address how individuals with rare diseases will have unobstructed access to specialists.
Access to Therapies
Individuals with rare diseases must have access to the full range of medically necessary treatments appropriate for their condition. Decisions regarding which treatments are most suitable must be reserved for the physician in consultation with the individual patient. Without appropriate treatment, individuals face detrimental health outcomes. Furthermore, payers risk facing unnecessary costs from potential complications that arise from any limitations placed on the full range of therapies.
Comparative Effectiveness ResearchThe Coalition supports comparative effectiveness research that identifies treatments and services that are effective in
clinically treating
rare diseases. We are concerned, however, that under certain circumstances patients with
rare diseases could be disadvantaged should comparative “cost” effectiveness studies occur rather than comparative “clinical” studies. To address this concern, we propose creation of a single point of contact, a “Rare Disease Ombudsman”, to serve as a liaison for patients with
rare diseases. We also support implementation of an expert
rare disease advisory panel to evaluate comparative effectiveness research studies on “orphan” therapies or services for
rare diseases. When treatment for a disease is chosen for evaluation, the research team should include expert clinicians, consumer representatives impacted by the disorder, and physicians or researchers from the relevant agency.
General Recognition of Rare DiseasesWe call upon all policymakers engaged in the current health care reform process to ensure that the system fully addresses the needs of all Americans including the specialized needs of individuals with
rare diseases. Payers and providers of health services must recognize the unique and on-going needs of the
rare disease community to have access to specialists and highly specialized therapies. For individuals with these conditions, “one size does not fit all.”
Alpha-1 Association
Alpha-1 Foundation
GBS/CIDP Foundation International
Committee of Ten Thousand
Hemophilia Federation of America
Immune Deficiency Foundation
National Hemophilia Foundation
Platelet Disorder Support Association
Patient Services Incorporated
