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Week Ending December 16, 2005
HR 2520 to provide for the collection and maintenance of human cord blood stem cells for the treatment of patients and research, and to amend the Public Health Service Act to authorize the C.W. Bill Young Cell Transplantation Program.
BRIEF
The bill would authorize the Secretary of Health and Human Services to enter into one-time contracts with qualified blood stem cell banks to assist in collection and maintenance of 150,000 units of high quality human umbilical cord blood cells to be made available for transplantation through the existing C. W. Bill Young cell Transplantation Program.
This effort, unlike a similar bill HR 810 passed in the House the same day, would not look to allow the research use of embryonic stem cells but rather similar cells from the neonatal blood remaining in the placenta and umbilical cord after separation from the baby.
The bill requires that cord blood stem cell banks must agree to participate for ten years but limits contracts to three years or those that will end on or before Sept. 30, 2010. The contract can be extended beyond three years if the Secretary finds that the 150,000 unit goal has not been accomplished or if insufficient applicants were accepted into the program.
The donating mother must agree to donate the neonatal blood.
This bill couples the effort to collect, classify and distribute cord blood with the National Bone Marrow Transplant Registry. In short the program would, for cord cells and bone marrow, organize the inventories taking into account information to determine to with whom the cells or marrow would be compatible and would create a database showing the outcome of the efforts.
A three year demonstration project is authorized through which a cord blood bank could use a portion of funding received under a contract for collection and storage of cord blood for a family where a relative has been diagnosed with a condition that will benefit from transplantation at no cost to the family. Those cells would not be counted in the 150,000 units authorized in this bill to be collected.
The Secretary of Health and Human Services would create an advisory council on the programs activities and to recognize entities that can accredit the cord blood banks and examine consent issues and issues related to educating the donor on all of her options for use and storage of the cells.
A system is required to 'identify, match and facilitate distribution from a qualified cord blood bank to candidate patients', reserve cord blood units for upcoming transplants, provide for patient advocacy, coordinate educational activities and keep track of patients at each stage of the process. The Secretary is required to create an office of patient advocacy.
The program must recruit bone marrow donors and cord blood donors while identifying underrepresented populations of potential donors, have a plan to increase donations from those populations and consider racial and ethnic minority groups to be such populations. The required educational programs would be used to recruit donors and make information available to the public and to physicians and other healthcare professionals.
The bill provides for physicians and other health professionals to search for a available bone marrow donors electronically and requires that a medical contingency response to prepare for and respond to biological, chemical and radiological attacks that could damage bone marrow be maintained and expanded.
Sponsor: Representative Christopher H. Smith (R-NJ-4th)
Vote: Passed House 431 to 0 (RC 205) (May 24, 2005) Passed Senate amended by Unanimous Consent December 16, 2005. Passed House amended 413 to 0 (RC 664) December 17, 2005. Signed into law by President Bush December 19, 2005.
Cost to the taxpayers: HR 2520 is calculated to cost $60 million for 2007 through 2010. As for S 1317, "CBO estimates that implementing S. 1317 would cost $3 million in 2006 and $212 million over the 2006-2010 period."
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FROM SENATE COMMITTEE REPORT BACKGROUND AND NEED FOR THE BILL
EXPLANATION OF BILL AND COMMITTEE VIEWS (Senate amended version)
BACKGROUND AND NEED FOR THE BILL
Human bone marrow and peripheral blood stem cell transplantation are well-established application of stem cell therapy. Human cord blood transplantation represents a variant of this therapy, in which blood, taken from the placenta and umbilical cord after birth of the baby and following separation of the cord, is used as the transplant material rather than bone marrow. All forms of stem cell transplantation can be life saving. Stem cell transplantation utilizing cord blood as the source material adds a third treatment option to the therapeutic armamentarium; providers can now balance the unique advantages and disadvantages of bone marrow/peripheral blood stem cells and cord blood to choose the optimal source material for transplantation in their adult and child patients. Note that, with each variant of therapy, the stem cells used for transplantation are derived from biological material which completely lacks all capacity for human life. In addition, all types of source material--bone marrow, peripheral blood stem cells and cord blood--are of value to the ongoing research that broadens the clinical applications of stem cell therapy.
>5.5 M adults), including routine assessment of the search function; recruitment of marrow donors, especially in underserved populations; development and maintenance of medical emergency contingency response capacity to a radiation disaster; and patient advocacy and case management services. Over 20,000 bone marrow/peripheral blood stem cell and cord bood transplants have taken place to date. The NMDP has demonstrably advanced the application of bone marrow transplantation. This legislation would modify and expand established programs, providing the Federal support that is necessary in order to more fully realize the potential of cord blood as a source material for stem cell transplantation.
Particular issues to be addressed by the legislation include the following:
The inventory of cord blood now available falls far short of the estimated need (150,000 new units);
Lack of consistent standards for donor identification, collection and storage of cord blood have made some of the current inventory unsuitable for treatment or, possibly, for research;
A common, comprehensive, readily accessible information system, designed to facilitate timely identification of compatible transplant material, would be of singular benefit to patients and providers alike;
A database which records pertinent clinical outcome measures would be of particular value to this developing therapy; and
Patients require informational/educational, advocacy and cases management services, which may be duplicated or unmet.
Technologies pertinent to bone marrow/peripheral blood stem cell and cord blood collection often differ remarkably; however, given the complimentary nature of these stem cell therapies, it makes sense to combine federal support for them into a single Program, rather than reauthorizing the NBMDR and passing new legislation pertaining to cord blood alone. Both economies of scale and decreased duplication of services (e.g. patient education and advocacy, case management, and clinical outcomes database) should help reduce the costs of broadening support to include cord blood-related considerations. Given the complexities and rapid developments in these areas of stem cell therapy, the Advisory Council will provide the Secretary with a consolidated yet comprehensive source of expert, unbiased analysis and recommendations.
A small proportion (¾5%) of the Program's funding is devoted to a demonstration project that allows for directed donation of cord blood for the benefit of first-degree relatives with conditions amenable to cord blood transplantation therapy. Cord blood units banked for this purpose must adhere to public standards and, if not used, will revert to the public inventory. Application for the demonstration project is at the discretion of the individual cord blood banks and is not a prerequisite for receiving the award of a contract for broader cord blood services.
HIPAA regulations are sufficient to guarantee proper use of both the single portal of access to transplant material and to the outcomes database.
Federal funding is necessary to create an inventory of cord blood units sufficient to meet estimated current patient needs. Funds authorized and appropriated for this purpose should be available only until the inventory goal specified is met by U.S. public cord blood banks. These funds are not intended to pay for individual clinical treatment that involves the use of cord blood; in particular, they are not intended to substitute for treatment-related reimbursement available through other federal programs (e.g. Medicaid/SCHIP, or through private insurance). The legislation also authorizes additional appropriations in support of the C.W. Bill Young Cell Transplantation Program; these latter funds are completely separate and distinct from the former and are not time-limited.
FROM HOUSE BILL HR 2520
This Act may be cited as the `Stem Cell Therapeutic and Research Act of 2005'.
(a) In General- The Secretary of Health and Human Services shall enter into one-time contracts with qualified cord blood stem cell banks to assist in the collection and maintenance of 150,000 units of high-quality human cord blood to be made available for transplantation through the C.W. Bill Young Cell Transplantation Program and to carry out the requirements of subsection (b).
(b) Requirements- The Secretary shall require each recipient of a contract under this section--
(1) to acquire, tissue-type, test, cryopreserve, and store donated units of human cord blood acquired with the informed consent of the donor in a manner that complies with applicable Federal and State regulations;
(2) to make cord blood units that are collected pursuant to this section or otherwise and meet all applicable Federal standards available to transplant centers for stem cell transplantation;
(3) to make cord blood units that are collected, but not appropriate for clinical use, available for peer-reviewed research;
(4) to submit data in a standardized format, as required by the Secretary, for the C.W. Bill Young Cell Transplantation Program; and
(5) to submit data for inclusion in the stem cell therapeutic outcomes database maintained under section 379A of the Public Health Service Act, as amended by this Act.
(c) Application- To seek to enter into a contract under this section, a qualified cord blood stem cell bank shall submit an application to the Secretary at such time, in such manner, and containing such information as the Secretary may reasonably require. At a minimum, an application for a contract under this section shall include an assurance that the applicant--
(1) will participate in the C.W. Bill Young Cell Transplantation Program for a period of at least 10 years; and
(2) in the event of abandonment of this activity prior to the expiration of such period, will transfer the units collected pursuant to this section to another qualified cord blood stem cell bank approved by the Secretary to ensure continued availability of cord blood units.
(d) Duration of Contracts-
(1) IN GENERAL- The Secretary may not enter into any contract under this section for a period that--
(A) exceeds 3 years; or
(B) ends after September 30, 2010.
(2) EXTENSIONS- Subject to paragraph (1)(B), the Secretary may extend the period of a contract under this section to exceed a period of 3 years if--
(A) the Secretary finds that 150,000 units of high-quality human cord blood have not yet been collected pursuant to this section; and
(B) the Secretary does not receive an application for a contract under this section from any qualified cord blood stem cell bank that has not previously entered into a contract under this section or the Secretary determines that the outstanding inventory need cannot be met by the one or more qualified cord blood stem cell banks that have submitted an application for a contract under this section.
(e) Regulations- The Secretary may promulgate regulations to carry out this section.
(f) Definitions- In this section:
(1) The term `C.W. Bill Young Cell Transplantation Program' means the C.W. Bill Young Cell Transplantation Program under section 379 of the Public Health Service Act, as amended by this Act.
(2) The term `cord blood donor' means a mother who has delivered a baby and consents to donate the neonatal blood remaining in the placenta and umbilical cord after separation from the newborn baby.
(3) The term `human cord blood unit' means the neonatal blood collected from the placenta and umbilical cord.
(4) The term `qualified cord blood stem cell bank' has the meaning given to that term in section 379(b) of the Public Health Service Act, as amended by this Act.
(5) The term `Secretary' means the Secretary of Health and Human Services.
(g) Authorization of Appropriations-
(1) FISCAL YEAR 2006- Any amounts appropriated to the Secretary for fiscal year 2004 or 2005 for the purpose of assisting in the collection or maintenance of human cord blood shall remain available to the Secretary until the end of fiscal year 2006 for the purpose of carrying out this section.
(2) SUBSEQUENT FISCAL YEARS- There are authorized to be appropriated to the Secretary $15,000,000 for each of fiscal years 2007, 2008, 2009, and 2010 to carry out this section. Amounts appropriated pursuant to this paragraph shall remain available for obligation through the end of fiscal year 2010.
(a) National Program- Section 379 of the Public Health Service Act (42 U.S.C. 274k) is amended--
(1) in the section heading, by striking `national registry' and inserting `national program';
(2) in subsection (a)--
(A) in the matter preceding paragraph (1), by striking `The Secretary shall by contract' and all that follows through the end of such matter and inserting `The Secretary, acting through the Administrator of the Health Resources and Services Administration, shall by one or more contracts establish and maintain a C.W. Bill Young Cell Transplantation Program that has the purpose of increasing the number of transplants for recipients suitably matched to biologically unrelated donors of bone marrow and cord blood, and that meets the requirements of this section. The Secretary may award a separate contract to perform each of the major functions of the Program described in paragraphs (1) and (2) of subsection (b) if deemed necessary by the Secretary to operate an effective and efficient system. The Secretary shall conduct a separate competition for the initial establishment of the cord blood functions of the Program. The Program shall be under the general supervision of the Secretary. The Secretary shall establish an Advisory Council to advise, assist, consult with, and make recommendations to the Secretary on matters related to the activities carried out by the Program. The members of the Advisory Council shall be appointed in accordance with the following:';
(B) in paragraph (1), by striking `except that' and all that follows and inserting `except that--
`(A) such limitations shall not apply to the Chair of the Advisory Council (or the Chair-elect) or to the member of the Advisory Council who most recently served as the Chair; and
`(B) 1 additional consecutive 2-year term may be served by any member of the Advisory Council who has no employment, governance, or financial affiliation with any donor center, recruitment group, transplant center, or cord blood stem cell bank.';
(C) by amending paragraph (4) to read as follows:
`(4) The membership of the Advisory Council--
`(A) shall include as voting members a balanced number of representatives including representatives of marrow donor centers and marrow transplant centers, representatives of cord blood stem cell banks and participating birthing hospitals, recipients of a bone marrow transplant and cord blood transplants, persons who require such transplants, family members of such a recipient or family members of a patient who has requested the assistance of the Program in searching for an unrelated donor of bone marrow or cord blood, persons with expertise in blood stem cell transplantation including cord blood, persons with expertise in typing, matching, and transplant outcome data analysis, persons with expertise in the social sciences, and members of the general public; and
`(B) shall include as nonvoting members representatives from the Department of Defense Marrow Donor Recruitment and Research Program operated by the Department of the Navy, the Division of Transplantation of the Health Resources and Services Administration, the Food and Drug Administration, and the National Institutes of Health.'; and
(D) by adding at the end the following:
`(5) Members of the Advisory Council shall be chosen so as to ensure objectivity and balance and reduce the potential for conflicts of interest. The Secretary shall establish bylaws and procedures--
`(A) to prohibit any member of the Advisory Council who has an employment, governance, or financial affiliation with a donor center, recruitment group, transplant center, or cord blood stem cell bank from participating in any decision that materially affects the center, recruitment group, transplant center, or cord blood stem cell bank; and
`(B) to limit the number of members of the Advisory Council with any such affiliation.
`(6) The Secretary, acting through the Advisory Council, shall submit to the Congress--
`(A) an annual report on the activities carried out under this section; and
`(B) not later than 6 months after the date of the enactment of the Stem Cell Therapeutic and Research Act of 2005, a report of recommendations on the scientific factors necessary to define a cord blood unit as a high-quality unit.';
(3) by amending subsection (b) to read as follows:
`(b) Functions-
`(1) BONE MARROW FUNCTIONS- With respect to bone marrow, the Program shall--
`(A) operate a system for listing, searching, and facilitating the distribution of bone marrow that is suitably matched to candidate patients;
`(B) carry out a program for the recruitment of bone marrow donors in accordance with subsection (c), including with respect to increasing the representation of racial and ethnic minority groups (including persons of mixed ancestry) in the enrollment of the Program;
`(C) maintain and expand medical emergency contingency response capabilities in concert with Federal programs for response to threats of use of terrorist or military weapons that can damage marrow, such as ionizing radiation or chemical agents containing mustard, so that the capability of supporting patients with marrow damage from disease can be used to support casualties with marrow damage;
`(D) carry out informational and educational activities in accordance with subsection (c);
`(E) at least annually update information to account for changes in the status of individuals as potential donors of bone marrow;
`(F) provide for a system of patient advocacy through the office established under subsection (d);
`(G) provide case management services for any potential donor of bone marrow to whom the Program has provided a notice that the potential donor may be suitably matched to a particular patient (which services shall be provided through a mechanism other than the system of patient advocacy under subsection (d)), and conduct surveys of donors and potential donors to determine the extent of satisfaction with such services and to identify ways in which the services can be improved;
`(H) with respect to searches for unrelated donors of bone marrow that are conducted through the system under subparagraph (A), collect, analyze, and publish data on the number and percentage of patients at each of the various stages of the search process, including data regarding the furthest stage reached, the number and percentage of patients who are unable to complete the search process, and the reasons underlying such circumstances;
`(I) support studies and demonstration and outreach projects for the purpose of increasing the number of individuals who are willing to be marrow donors to ensure a genetically diverse donor pool;
`(J) conduct and support research to improve the availability, efficiency, safety, and cost of transplants from unrelated donors and the effectiveness of Program operations; and
`(K) assist qualified cord blood stem cell banks in the Program in accordance with paragraph (3).
Subsections (c) through (e) apply with respect to each entity awarded a contract under this section with respect to bone marrow.
`(2) CORD BLOOD FUNCTIONS- With respect to cord blood, the Program shall--
`(A) operate a system for identifying, matching, and facilitating the distribution of donated cord blood units that are suitably matched to candidate patients and meet all applicable Federal and State regulations (including informed consent and Food and Drug Administration regulations) from a qualified cord blood stem cell bank;
`(B) allow transplant physicians, other appropriate health care professionals, and patients to search by means of electronic access all available cord blood units listed in the Program;
`(C) allow transplant physicians and other appropriate health care professionals to tentatively reserve a cord blood unit for transplantation;
`(D) support studies and demonstration and outreach projects for the purpose of increasing cord blood donation to ensure a genetically diverse collection of cord blood units; and
`(E) coordinate with the Secretary to carry out information and educational activities for the purpose of increasing cord blood donation and promoting the availability of cord blood units as a transplant option.
`(3) SINGLE POINT OF ACCESS- If the Secretary enters into a contract with more than one entity to perform the functions outlined in this subsection, the Secretary shall establish procedures to ensure that health care professionals and patients are able to obtain, consistent with the functions described in paragraphs (1)(A) and (2)(A), cells from adult donors and cord blood units through a single point of access.
`(4) DEFINITION- The term `qualified cord blood stem cell bank' means a cord blood stem cell bank that--
`(A) has obtained all applicable Federal and State licenses, certifications, registrations (including pursuant to the regulations of the Food and Drug Administration), and other authorizations required to operate and maintain a cord blood stem cell bank;
`(B) has implemented donor screening, cord blood collection practices, and processing methods intended to protect the health and safety of donors and transplant recipients to improve transplant outcomes, including with respect to the transmission of potentially harmful infections and other diseases;
`(C) is accredited by an accreditation body recognized pursuant to a public process by the Secretary;
`(D) has established a system of strict confidentiality to protect the identity and privacy of patients and donors in accordance with existing Federal and State law; and
`(E) has established a system for encouraging donation by a genetically diverse group of donors.';
(4) in subsection (c)--
(A) in paragraph (1), by striking `The Registry shall carry out a program for the recruitment' and inserting `With respect to bone marrow, the Program shall carry out a program for the recruitment';
(B) in paragraph (2)(A)--
(i) in the matter preceding clause (i), by striking the first sentence and inserting `In carrying out the program under paragraph (1), the Program shall carry out informational and educational activities, in coordination with organ donation public awareness campaigns operated through the Department of Health and Human Services, for purposes of recruiting individuals to serve as donors of bone marrow and shall test and enroll with the Program potential donors.'; and
(ii) in clause (ii), by striking `, including providing updates'; and
(C) in paragraph (3), by striking `the availability, as a potential treatment option, of receiving a transplant of bone marrow from an unrelated donor' and inserting `transplants from unrelated donors as a treatment option and resources for identifying and evaluating other therapeutic alternatives';
(5) in subsection (d)--
(A) in paragraph (1), by striking `The Registry shall' and inserting `With respect to bone marrow, the Program shall';
(B) in paragraph (2)(C), by inserting `and assist with information regarding third party payor matters' after `ongoing search for a donor';
(C) in subparagraphs (C), (D), and (E) of paragraph (2), by striking the term `subsection (b)(1)' each place such term appears and inserting `subsection (b)(1)(A)';
(D) in paragraph (2)(F)--
(i) by redesignating clause (v) as clause (vi); and
(ii) by inserting after clause (iv) the following:
`(v) Information concerning issues that patients may face after a transplant regarding continuity of care and quality of life.'; and
(E) in paragraph (3)(B), by striking `Office may' and inserting `Office shall';
(6) in the matter preceding paragraph (1) in subsection (e), by striking `the Secretary shall' and inserting `with respect to bone marrow, the Secretary shall';
(7) by amending subsection (f) to read as follows:
`(f) Comment Procedures- The Secretary shall establish and provide information to the public on procedures under which the Secretary shall receive and consider comments from interested persons relating to the manner in which the Program is carrying out the duties of the Program.';
(8) by amending subsection (g) to read as follows:
`(g) Consultation- In developing policies affecting the Program, the Secretary shall consult with the Advisory Council, the Department of Defense Marrow Donor Recruitment and Research Program operated by the Department of the Navy, and the board of directors of each entity awarded a contract under this section.';
(9) in subsection (h)--
(A) by striking `Application- ' and inserting `Contracts- ';
(B) by striking `To be eligible' and inserting the following:
`(1) APPLICATION- To be eligible'; and
(C) by adding at the end the following:
`(2) CONSIDERATIONS- In awarding contracts under this section, the Secretary shall give substantial weight to the continued safety of donors and patients and other factors deemed appropriate by the Secretary.'; and
(10) by striking subsection (l).
(b) Stem Cell Therapeutic Outcomes Database- Section 379A of the Public Health Service Act (42 U.S.C. 274l) is amended to read as follows:
`(a) Establishment- The Secretary shall by contract establish and maintain a scientific database of information relating to patients who have been recipients of stem cell therapeutics product (including bone marrow, cord blood, or other such product) from a biologically unrelated donor.
`(b) Information- The outcomes database shall include information with respect to patients described in subsection (a), transplant procedures, and such other information as the Secretary determines to be appropriate, to conduct an ongoing evaluation of the scientific and clinical status of transplantation involving recipients of bone marrow from biologically unrelated donors and recipients of a stem cell therapeutics product.
`(c) Annual Report on Patient Outcomes- The Secretary shall require the entity awarded a contract under this section to submit to the Secretary an annual report concerning patient outcomes with respect to each transplant center, based on data collected and maintained by the entity pursuant to this section.
`(d) Publicly Available Data- The outcomes database shall make relevant scientific information not containing individually identifiable information available to the public in the form of summaries and data sets to encourage medical research and to provide information to transplant programs, physicians, patients, entities awarded a contract under section 379 donor registries, and cord blood stem cell banks.'.
(c) Definitions- Part I of title III of the Public Health Service Act (42 U.S.C. 274k et seq.) is amended by inserting after section 379A the following:
`In this part:
`(1) The term `Advisory Council' means the advisory council established by the Secretary under section 379(a)(1).
`(2) The term `bone marrow' means the cells found in adult bone marrow and peripheral blood.
`(3) The term `outcomes database' means the database established by the Secretary under section 379A.
`(4) The term `Program' means the C.W. Bill Young Cell Transplantation Program established under section 379.'.
(d) Authorization of Appropriations- Section 379B of the Public Health Service Act (42 U.S.C. 274m) is amended to read as follows:
`(a) In General- For the purpose of carrying out this part, there are authorized to be appropriated $28,000,000 for fiscal year 2006 and $32,000,000 for each of fiscal years 2007 through 2010.
`(b) Emergency Contingency Response Capabilities- In addition to the amounts authorized to be appropriated under subsection (a), there is authorized to be appropriated $2,000,000 for the maintenance and expansion of emergency contingency response capabilities under section 379(b)(1)(C).'.
(e) Conforming Amendments- Part I of title III of the Public Health Service Act (42 U.S.C. 274k et seq.) is amended--
(1) in the title heading, by striking `NATIONAL BONE MARROW DONOR REGISTRY' and inserting `C.W. BILL YOUNG CELL TRANSPLANTATION PROGRAM'; and
(2) in section 379, as amended by this section--
(A) in subsection (a), by striking the term `board' each place such term appears and inserting `Advisory Council';
(B) in subection (c)--
(i) in the matter preceding subparagraph (A) in paragraph (1), by striking `Such program' and inserting `Such recruitment program';
(ii) in paragraph (2), by striking `program under paragraph (1)' and inserting `recruitment program under paragraph (1)'; and
(iii) in paragraph (3), by striking `program under paragraph (1)' and inserting `recruitment program under paragraph (1)';
(C) in subsection (d)(2)(E), by striking `Registry program' and inserting `Program';
(D) in subsection (e)--
(i) in the matter preceding paragraph (1), by striking `participating in the program, including the Registry,' and inserting `participating in the Program, including'; and
(ii) in paragraph (6), by striking `the program' and inserting `the Program'; and
(E) by striking the term `Registry' each place such term appears and inserting `Program'.
END
This legislation builds upon the firm foundation established by the NMDP, which has advanced the public health by facilitating the application of one form of stem cell therapy--bone marrow transplantation--to the care of patients with a variety of malignant, metabolic and other diseases. It also draws upon the strength and experience of the long-established blood banking profession in defining standards for collection and storage of blood products and accreditation of facilities. Each will do much to promote the use of this analogous therapy--cord blood transplantation. Yet the legislation acknowledges that more needs to be done in the area of cord blood transplantation.
The committee, in its deliberations on S. 1317, placed particular emphasis on several aspects of the legislation and wants to state clearly the following points of consensus.
Cord blood units collected through the program, but which subsequently prove unsuitable for use as transplant material, are still of great value for research purposes. For example, units that are collected and cryopreserved, but which later test positive on initial screening for infectious agents, as well as units with insufficient cell counts, can nevertheless be used for quality assurance testing and for ex vivo expansion, adoptive immunotherapy or graft engineering trials. The committee supports the use of some cord blood units collected under the auspices of the Program for research.
It is not the intent of the committee that all clinical expenses pertaining to cord blood transplantation should be borne by the Program. It is expected that all pertinent public and private insurance reimbursement for clinical expenses related to cord blood transplantation will be fully utilized; in particular, no funds are available through the legislation for this purpose. This pertains to both public and private cord blood banks and includes both directed and random donation of cord blood. Cord blood units are owned by the cord blood banks to which they were donated. Eventual financial self-sufficiency regarding the inventory provisions of the legislation is desired and anticipated.
The committee believes strongly in the value of Congressional oversight of the programs it authorizes and considers thoughtful, candid, timely reports to Congress to be an important part of this process. Thus, it anticipates that the reports mandated by this legislation will contain at least the following features:
Related Cord Blood Donors Demonstration Project--
Number of cord blood banks participating.
Number of cord blood units banked.
Number of units used for transplantation and for research transplant or research results.
Amount of money spent by the bank in support of this project.
Advisory Council--
Recommendations made to the Secretary on all matters related to the Program.
Actions taken by the Program on those recommendations.
The committee recognizes that cord blood transplantation is a promising, but emerging, therapy and that they have a unique opportunity to advance the science and practice through establishment of the Stem Cells Therapeutic Outcomes Database. Currently, the Bone Marrow Scientific Registry (BMSR) compiles outcomes data on transplant patients. Given the creation of the cord blood program, the BMSR is expanded and renamed the Stem Cells Therapeutic Outcomes Database. While the Advisory Council must play a leading role in its expansion, the database should contain at least the following elements: clinical diagnosis; donor selection criteria/matching considerations; preparatory regimen; transplant procedure; dosages; results; post-transplant procedures; adverse events; and long-term follow-up.
The committee thinks it is of pre-eminent importance that the Secretary promptly develops a scientifically sound definition of a `high-quality cord blood unit' and anticipates that the Advisory Council will play a prominent role in this process. Such a definition is likely to include quantitative measures of cell counts and viability, HLA typing resolution, match algorithms, and agreement on outcomes measurements; it is also likely to address quality testing, selection criteria, collection and transportation standards, confidentiality and integration of files, searches and general procedures. We feel that additional features of this legislation will facilitate the establishment of optimal standards:
FDA participation on the Advisory Council;
Establishment by the FDA of licensure requirements for cord blood units;
Continued authority of current FDA, other Federal Government and state regulations; and
Institutional accreditation.
The committee understands that the actual practice of clinical medicine is and should be a highly individualized and personalized interaction between provider and patient. Nothing in this legislation should be construed, then, to limit the provider's right and responsibility to explore any and all sources of transplant material for their patient. This legislation is intended to help physicians to determine which source of stem cell material is best for their patient. Incumbent on the program, then, is the duty to present all pertinent information clearly and without bias or conflict of interest.
To ensure equitable and efficient operation of the program, however, it is likely that the Secretary, in consultation with the Advisory Council and subject to public comment, will develop guidelines for participation, which should include at least the following:
Unit reservation policies for cord blood units;
Informed consent policies for cord blood donation;
Accreditation requirements for public and private cord blood banks; and
Standardized data requirements for the outcomes database and the single portal of access.
The committee recognizes that health information technology has revolutionized the management of medical data. Thus, as per the IOM report recommendation, the committee intends that the statute be neutral regarding whether centralized (`real') or decentralized (`virtual') models of management are employed. It anticipates that the competitive process of contracting will encourage innovative solutions to the needs of the Program, especially those unique to the transplantation of cord blood. In determining that standard dataset, the Secretary should consider HLA typing, size of the cord blood unit, sex, and blood type of the donor.
The requirements of the bone marrow and cord blood programs may differ; therefore, the Secretary is required to conduct a competition for the initial establishment of this cord blood function of the Program separate from that for the bone marrow/peripheral blood stem cell function. The contracting competition should be transparent and without preconceptions; in particular, it should not exclude, de novo, any applicant or any particular model from the process.
We emphasize, however, that any successful solution must provide for a single portal of entry for provider and patient alike, one that is accurate, comprehensive, understandable, easy to use and secure; in particular, the provider must have sufficient information readily available to choose intelligently and objectively amongst the various stem cell transplant materials available. A standardized, electronic format for presentation of the data to provider and patient will considerably facilitate this use.
The committee expects that, with passage of this legislation, consideration of cord blood banking will be given, as a matter of routine, in all of the organ donation education and awareness campaigns currently sponsored by the Secretary. Furthermore, the committee anticipates that, as with other donor organ procurement programs, the Secretary will consider carefully how best to promote donation of cord blood.
The committee strongly supports the IOM report recommendation that women be provided with a balanced perspective and clear information in order to participate, actively and knowledgably, in the choice of whether or how to donate cord blood. Informed consent is likely to include, at least, consideration of the following options: public donation or private storage; and disposal. Pertinent cost information must be conveyed to the potential donor. The discussion must be informed, objective, non-coercive and culturally sensitive; timing may be a crucial factor. Experience with other types of organ transplant may be instructive and should be considered in designing the approach to be used for cord blood donation.
Medical contingency response to a terrorist emergency is particularly pertinent to cord blood as the source of stem cell transplant material most likely to be immediately available. It is the committee's expectation that the Secretary will, through the contracting process, address developing the capacity for urgent, large scale searches and attendant logistic considerations, including transportation and security. In addition, cord blood banks must be prepared to increase collection in order to maintain their public inventory in the case of an emergency.
It is the committee's expectation that those Program functions which remain under the direct oversight of the Secretary, i.e. Patient Advocacy and Case Management, single point of access, and the Stem Cell Therapeutic Outcomes Database, will operate in a objective and unbiased manner, free from conflicts of interest, such that they can support use of the stem cell source material that is most appropriate for the individual patient.
The legislation does not presume any fixed, pre-determined distribution of funds appropriated amongst the various functions of the program, save for the limit established for the demonstrations programs; rather, that is left to the Secretary's determination, providing the Secretary with the flexibility necessary to meet the pre-eminent goal of better patient care through stem cell transplantation therapy. However, in providing this flexibility, the committee does not intend to imply that the funds should be divided equally between the operators of the bone marrow and cord blood functions. When determining how to allocate funds under this program, the Secretary should ensure that his actions do nothing that would put the bone marrow functions of the Program at risk. The allocation of funds between section 3 and the Stem Cell Therapeutic Outcomes Database should be proportional to the relative functions of each. By combining the funding for emergency contingency planning with other program operations, the committee does not intend to diminish the importance of providing sufficient funds for the Program's activities related to national security and encourages the Secretary to ensure that the emergency contingency planning activities are adequately funded.
The committee anticipated that the funding authorized for establishing and strengthening the cord blood unit inventory will be devoted primarily to defraying the start-up expenses, including developing the expanded inventory in an optimal fashion. While we feel that such activities clearly have the potential to be self-supporting in time, we also recognize that sufficient funding over an adequate period of time will be necessary for these activities to realize their full potential. It is the committee's expectation that the Secretary will closely scrutinize all costs related to this legislation, so that tax dollars are spent judiciously to achieve the maximum effect.
The legislation neither requires nor precludes the Secretary from developing new regulations, either temporary or permanent, governing the program; however, the committee expects that, if passed, the legislation will be implemented promptly.
The committee recognizes that some existing blood banks may have particular expertise in managing private or directed donation of cord blood, i.e. donations made expressly for the benefit of a first degree family member with a disease amenable to treatment with cord blood transplantation. In order to take advantage of this expertise, the committee interprets the legislation to permit applications from such blood banks for participation in the demonstration project only. These blood banks would not be required to provide analogous donor services, nor would they be required to submit an application for a more comprehensive contract. Blood banks submitting an application for the demonstration project only will, however, still need to meet all quality, accreditation and research requirements specified in the legislation. Funding for contracts awarded to these blood banks for a demonstration project would be apportioned from the same ¾5 percent of the authorization of appropriations that funds demonstration projects awarded to blood banks under a more comprehensive contract. All qualified cord blood units donated through any form of the demonstration project which are not utilized by the intended recipient will revert to the public inventory established by the C.W. Bill Young Cell Transplantation Program.
The Secretary has consistently required that the operator of the National Bone Marrow Donor Registry (NMBDR) conduct research to analyze data pertaining to blood stem cell transplantation. As part of previous contracts, the Secretary requires the contractor to establish and maintain a program of analysis and research regarding the operation of the Registry and persons' decisions regarding enrolling in the Registry, using data in the National Registry of Unrelated Marrow Transplants (renamed, in S. 1317, the Stem Cell Therapeutics Outcomes Database). Research priorities are established collaboratively with Registry committees possessing expertise in research and histocompatibility. The Secretary also requires the contractor to ensure that research conducted or sponsored by the Registry results in peer-reviewed published articles whenever possible.
The committee recognizes the important research the NMDP has accomplished while operating the Registry. We support its goal to systematically remove all barriers to transplant and to increase the likelihood of finding a match for all who could potentially benefit from transplantation. There, S. 1317 includes language authorizing the Secretary to continue having the contractor engage in the type of research projects it has historically required. The committee seeks to ensure that the contractor maintains the relationships the NMDP has established with other research organizations, donor centers, leading investigators, and the government. The committee seeks to formalize the existing research aspects of the program.
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