Policies and Procedures for Operation and the Development of Recommendations for Screening Newborns and Children for Heritable Disorders and for the Heritable Disorders Program

Printer-friendly Policies and Procedures

A. Purpose

According to its charter, the Advisory Committee on Heritable Disorders and Genetic Diseases in Newborns and Children (ACHDGDNC, Committee) purpose is to provide to the Secretary:

• Advice and recommendations concerning the grants and projects authorized under section 1109 of the PHS Act.
• Technical information to develop policies and priorities for this program to enhance the ability of State and local health agencies to provide for newborn and child screening, counseling and health care services for newborns and children having or at risk for heritable disorders.
• Advice and guidance regarding the most appropriate application of universal newborn screening tests, technologies, policies, guidelines and programs for effectively reducing morbidity and mortality in newborns and children having or at risk for heritable disorders.

The ACHDGDNC is the only entity in the Federal government which makes such recommendations. The target audience for the Committee recommendations are State-based newborn screening programs and public and private health care providers who provide health care services to newborns and children with heritable disorders, public and private officials who make newborn and child screening policy, and the public.

B. Membership

The Committee consists of 11 regular members, including the following ex-officio members: the Administrator of Health Resources and Services Administration (HRSA), the Directors of the Centers for Disease Control and Prevention (CDC), the National Institutes of Health (NIH), and the Agency for Healthcare Research and Quality (AHRQ), or their designees and two non-voting liaison members from the Secretary’s Advisory Committee on Genetics, Health and Society and Advisory Committee on Infant Mortality. The ACHDGDNC members are selected based on their expertise and qualifications necessary to contribute to the accomplishments of the Committee=s objectives. Through its recommendations regarding newborn and child screening programs, the ACHDGDNC plays a leading role in the promotion of public health in the United States. Therefore, members are selected based upon the following criteria from the general public, health care, technical, public health or scientific professionals:

• with special expertise in the field of heritable disorders;
• in providing screening, counseling, testing for newborns and children at risk for heritable disorders;
• in providing specialty services for newborns and children at risk for heritable disorders.

Members also include members of the public having special expertise about or concern with heritable disorders.

Departmental policy provides that Committee membership be fairly balanced in terms of points of view represented and the Committee's function. The Department will give close attention to the membership of the Committee to ensure that it reflects a distribution of the experience and expertise needed to understand and serve the diversity of the population served. Members are generally appointed to the Committee for a term of up to 4-years.

1. Consideration for Nomination

Committee members serve as individuals, not as representatives of organizations or interest groups. Each person is selected based on his or her expertise as noted above.

HRSA’s goal in appointing members to the ACHDGDNC is to achieve the greatest level of expertise while minimizing the potential for actual or perceived conflicts of interest and assuring public confidence in the integrity of the Committee’s advice. This can be achieved in large part by focusing on the types of expertise needed on the Committee, the ways in which individuals attain that expertise, and the value of maintaining such contacts during their tenure on the committee. Applying such an analysis, HRSA has concluded that particular personal financial interests may create conflicts, or perceptions of conflicts. When the expertise of the individual contributes little unique or additional knowledge to the Committee and the financial interests create either an actual or apparent interest in the success or failure of the products of a genetic or newborn screening test manufacturer, such interests should disqualify an individual from membership on the Committee. Disqualification is especially important if the individual personally will benefit financially from a decision that the Committee may make. Therefore, individuals with certain financial genetic or newborn screening-related interests generally will not be considered for appointment to the Committee. These conflicts would include employment by a genetic or newborn screening test or therapy manufacturer. Members are prohibited from serving as a consultant or advisor to a genetic or newborn screening test or therapy manufacturer and from accepting honoraria or travel reimbursement from a genetic or newborn screening test or therapy manufacturer during Committee tenure.

2. Solicitation for Nominees

Each year, suggestions for members are sought, generally through a federal register notice, from a variety of sources including, family organizations, professional societies, current and former ACHDGDNC members, manufacturers of technologies, therapies and tests used in screening newborns and children for heritable disorders as well as commercial providers of treatments for such screened disorders, and the general public. These individuals are encouraged to contact members of their institutions, professional organizations, and peers to develop a broad slate of candidates. During the year, suggestions for membership to the Committee are received from various sources. These submissions are compiled for consideration along with those received from the solicitation.

3. Selection of Nominees

A listing of individuals suggested for nomination for appointment to the Committee is prepared by the Executive Secretary, ACHDGDNC and forwarded to the Associate Administrator, Maternal and Child Health Bureau, HRSA and the Committee representatives from AHRQ, CDC, FDA, and NIH.

The Administrator, HRSA approves the final nomination package, which is submitted to the Secretary, Department of Health and Human Services (HHS), who appoints the member(s) to the Committee. If the appointment is confirmed by the Secretary, the new member serves for a term of up to 4 years. A member may be reappointed to serve up to an additional 4 years at the request of the Secretary, HHS. A member who is unable to fulfill the full term on the Committee may resign by submitting a letter of resignation to the Executive Secretary, ACHDGDNC. If a member resigns, a new member is appointed to fill the remainder of the unexpired term. The Chair is chosen by the Secretary, HHS.

C. Financial Interests – Financial Conflicts of Interest

Federal law (18 U.S.C. §208) prohibits Federal executive branch employees, including Special Government Employees (e.g., members of Federal advisory committees such as the ACHDGDNC), from participating, personally and substantially, in particular matters which have a direct and predictable effect on financial interests, to their knowledge, held by themselves, their spouse, minor child, general partner, organization in which they are serving as officer, director, trustee, general partner or employee. Members may have potential financial conflicts of interest because members are chosen for service based on their expertise and experience in the areas in which advice is sought by the government. Congress has recognized the need for service by these experts on Federal advisory committees, despite the inherent potential for conflicts of interest, by providing for waivers of the conflict of interest prohibition for particular matters of general applicability, under 18 U.S.C. §208(b)(3) when Athe need for the individual’s services outweighs the potential for a conflict of interest created by the financial interest involved.”

HRSA is sensitive to concerns about potential conflicts of interest by members serving on the ACHDGDNC. To assure the integrity of the Committee, HRSA has taken steps to assure that there is compliance with the ethics statutes and regulations regarding financial conflicts and the appearance of financial conflicts of interest. As described in Section C of this policy, limited 208(b)(3) waivers for particular matters of general applicability are considered for members utilizing that statute’s standard of the need for the individual’s services outweighing the potential for a conflict of interest created by the financial interest involved [18 U.S.C. 208(b)(3) may be viewed at this site:  Title 18--Crimes and Criminal Procedure

ACHDGDNC members must file OGE Confidential Financial Disclosure Reports, Form 450, as required by OGE regulations and the HRSA policy “Financial Disclosure for Federal Advisory Committee Members Appointed as Special Government Employees.” HRSA will individually evaluate and consider for waiver the related financial interests of each ACHDGDNC member in accordance with the OGE regulations at 5 CFR Parts 2635 and 2640.

D. Organizational Representatives

Appointments of organizational representatives are based upon written requests from organizations. Requests from organizations should document the commitment of the organization to providing expert input into the ACHDGDNC decision-making process, travel and per diem support to their representative, and active dissemination to their membership about ACHDGDNC activities and recommendations. A request for organizational representation suggested to the Committee should be prepared and forwarded to the Executive Secretary, ACHDGDNC, Maternal and Child Health Bureau, HRSA, for review and approval by the Committee. Because of space and time limitations at meetings, representatives must represent organizations that show interest in the Committee’s work through active involvement and participation at Committee meetings, have broad interests in relevant fields (e.g., primary care, newborn screening, genetics and other relevant specialty expertise) and represent large constituencies relevant to the Committee’s needs. Groups that represent more narrow interests or small constituencies (e.g., interest in a single disease) are invited to participate in ACHDGDNC activities on an ad hoc basis whenever issues of interest and concern are being discussed rather than requesting liaison representation. The Committee will evaluate requests for particular organizational liaisons in an ongoing manner and will consider the commitment of specific organizations to the Committee’s charter and involvement of those organizations in Committee activities.

E. Voting

For Committee meetings, the Committee shall not take a vote unless a quorum of at least eight voting members is present. Voting members are specified by the charter. Only Committee members may vote (this includes ex-officio members). Liaison members and organizational representatives may not vote.

All members, including ex-officio members, are expected to announce any conflicts of interest, as described within Federal law (18 U.S.C. §208) prior to any voting to determine if they can vote. For subcommittee meetings, only subcommittee members who are members of the Committee may vote.

F. Meetings

Regularly scheduled meetings are held up to three times a year, at the call of the Executive Secretary. Meeting dates are announced 6-12 months in advance. Meeting dates and the location of the meetings are posted on the ACHDGDNC web site as soon as the dates and location are selected. At least 15 days prior to the meeting, the meeting date, items to be discussed, and location are published in the Federal Register. Meetings traditionally are held in Washington, D.C. metro area. Except as noted otherwise in these policies and procedures, the Chair will use Roberts Rules of Order (Ninth Edition) as a guide when conducting Committee meetings.

If there is a need to consult ACHDGDNC members on an urgent or emergency basis, the Executive Secretary may request that the Chair establish an Aemergency consultation workgroup” consisting of ACHDGDNC members, to discuss the nature of the emergency and possible responses to it. The workgroup will report its findings and recommendations to the full Committee for their deliberation.

ACHDGDNC meetings are generally open to the public for their entire duration. However, there may be occasions when the nature of the information is such that a closed meeting is required. All provisions of the Federal Advisory Committee Act and Government in the Sunshine Act regarding closed sessions will be followed. All ACHDGDNC proceedings shall be published on the Committee web site.

1. Public Comment

ACHDGDNC holds open discussions and reserves meeting time for public comment. In some limited circumstances, a formal comment period is scheduled during the deliberation of a specific agenda item. Comments from the public may be received during open discussions depending on the amount of time available. These comments may be restricted in order to keep within the time allotted for the Committee to complete the agenda. Opportunity to make public comment orally on specific agenda items should be requested in advance. Members of the public who wish to address the Committee should contact the ACHDGDNC Executive Secretary to request public comment time. Brief comments will be allowed and in the event of a large volume of requests, the time allowed for each speaker may be specified in advance. It is recommended that oral comments also be submitted in writing. Public comments may be submitted to the Committee in writing, even when an individual cannot attend the meeting. Written comments are provided as handouts to the Committee and to the attendees, but are not read aloud during the meeting. The Committee may request that written comments be concise in order to facilitate the Committee’s ability to properly review and consider all comments received.

G. Working Groups and Subcommittees

ACHDGDNC is authorized to establish subcommittees. ACHDGDNC subcommittees: 1) must include two or more ACHDGDNC members, 2) must include HRSA staff members, and 3) may include as consultants, organizational representatives. On occasion, technology or disease experts who are not government employees, ACHDGDNC members, ex officio members or organizational representatives may be asked to serve as consultants to a subcommittee. Only appointed voting members may chair a subcommittee. Members with a potential financial conflict of interest cannot serve on a subcommittee. All subcommittee findings are presented to the ACHDGDNC in open meeting, and this information is openly deliberated.

ACHDGDNC utilizes subgroups of the Committee, or working groups, to review research data, published literature and expert opinion and develop options for presentation to the full Committee. The ACHDGDNC working groups are used as a resource for gathering, analyzing, and preparing information for the Committee. The Committee Chair appoints working group members and these members need not be Committee members. All working group findings are presented to the ACHDGDNC in open meeting, and this information is openly deliberated.

H. Member Responsibilities

1. Attendance at Meetings

The ACHDGDNC meeting dates are published approximately 6 months to 1 year in advance. Except in the event of an emergency, members of the ACHDGDNC assume the responsibility of attending all meetings. At the discretion of the Executive Secretary, a member may be linked to a Committee meeting by telephone or video conference, in which case their presence shall count toward the quorum. Failure by a member to participate actively in the work of the Committee, including through regular attendance at ACHDGDNC meetings, may result in a request by the ACHDGDNC Executive Secretary to the Secretary of Health and Human Services to replace the affected member.

2. ACHDGDNC Related Contacts

ACHDGDNC members may be solicited to participate in consultations or surveys on screening issues that are addressed by the ACHDGDNC. ACHDGDNC members should not participate in such consultations or surveys if they are requested to participate because of their ACHDGDNC membership status.

The Standards of Conduct for Employees of the Executive Branch (Title 5, Code of Federal Regulations, Section 2635.807) prohibit receiving compensation for speaking, teaching, or writing on matters related to an ACHDGDNC member’s official duties outside Committee or subcommittee meetings. ACHDGDNC members are prohibited from receiving compensation for any speech or publication in which the purpose is to report on the member’s work on the ACHDGDNC.

3. Media Interaction

Committee members and organizational representatives may be approached by the media for an interview. Members and representatives are free to give interviews and express their opinions, or the views of their employer, professional organization, etc., but should have HRSA approval to speak as an ACHDGDNC member or organizational representative on ACHDGDNC matters. Therefore, the Committee member should inform the Executive Secretary of such an interview to determine the appropriateness of the interview and the appropriate Committee member to participate in the interview. The ACHDGDNC Chair is the usual spokesperson for the Committee. An organizational representative is free to represent their respective organization but may not represent the ACHDGDNC.

4. Committee Correspondence

Any correspondence (letter, fax, e-mail, solicitation of articles or commentary on ACHDGDNC matters, etc.) should be routed to the Executive Secretary for the ACHDGDNC who then determines who the most appropriate respondent is. No member or organizational representative should reply to Committee correspondence without consulting the Executive Secretary. The only exception to this rule is that all members are free to respond to questions about established points of fact (e.g., meeting dates, citations for ACHDGDNC recommendations, etc.).

I. Selection of Topics

Potential topics for ACHDGDNC consideration can be suggested by anyone, but are most often proposed by HRSA program staff; AHRQ, CDC, NIH and FDA program staff; ACHDGDNC members; scientific and medical professional organizations; lay advocacy groups; or manufacturers of technologies, tests or processes for screening newborns and children for heritable disorders.

Approximately 10 weeks prior to an upcoming meeting, a memorandum requesting potential agenda items is generally sent via postal mail or e-mail to the ACHDGDNC chair and HRSA, AHRQ, CDC, FDA and NIH and HRSA program staff. A list of topics based on action or follow-up items from the last meeting or previously suggested is included in the memorandum. The person suggesting an agenda item is asked to specify the topic to be on the agenda, issues of concern, and specific questions to be addressed by ACHDGDNC.

Agenda items are accepted for discussion by the Executive Secretary in consultation with the Chair, and the Associate Administrator, MCHB, HRSA; and representatives from AHRQ, CDC, FDA and NIH

J. Process for Developing Recommendations

The ACHDGDNC process for developing recommendations is designed to be streamlined, consistent through-out the review process, transparent and evidenced based.

1. Technical Analysis

The ACHDGDNC purview explicitly includes children as well as newborns and therefore is relevant to both the screening in the neonatal and pediatric clinical settings. For the Committee’s technical analysis for recommending disorders to screen infants and children, three broad areas are considered: the condition (incidence, significance, etc); the screening test (analytical and clinical validity, etc); and the treatment (efficacy, effectiveness).

• Step 1 in the process for developing the recommendations is the submission of a completed nomination form (Appendix A) to GSB/MCHB/HRSA for evaluation by the ACHDGDNC.
• Step 2 consists of an administrative review by GSB/MCHB/HRSA to determine the completeness of the form. If the form is complete, the nomination form is sent to the ACHDGDNC.
• Step 3 consists of the ACHDGDNC review.

The Committee review will entail an internal Committee review, and if found to have sufficient evidence for each of the three components identified above (condition, test, and treatment), the nominated condition will be assigned to an external workgroup, for an evidence-based review. After the evidence-based review is completed, the Committee will review the report and reached a formal recommendation based on the quality and strength of the data as summarized in the evidence review and other factors, such as the expert opinions of voting members and other experts, and ethical and legal issues. When relevant, the Committee will also consult with other federal Advisory Committees when developing their recommendations.

• Step 4, the Committee presents its recommendations to the Secretary, HHS.

The ACHDGDNC recommendations should be accompanied by:

• Summary of evidence and strength of recommendation(s)
• Recommendation(s) of other Groups
• Discussion of rationale for ACHDGDNC recommendation(s), that will explicitly state the basis upon which the recommendations were made, i.e., a sufficient body of evidence based on results of controlled trials, observational studies, case series, expert opinion, focus groups, cost-effectiveness analysis, policy analyses, ethical analysis, and other inputs.
• Recommended subsequent surveillance, research, education, and program evaluation activities (if applicable)

When relevant, some policy recommendations will be developed in formal consultation with other national advisory committees.

2. Policy Analysis

Many of the issues addressed by the Committee are not technical but policy issues. In such cases, a simple but formal policy analysis should be considered and may be requested and/or performed by the ACHDGDNC.

3. ACHDGDNC Recommendations for the Heritable Disorders Program (HDP)

ACHDGDNC recommendations for the grants and other activities under the HDP also may be developed and voted upon. Section 1111(b)(2) of the Public Health Service Act (42 U.S.C. 300b-10) gives the ACHDGDNC the responsibility and authority to provide technical information to the Secretary for the development of policies and priorities for the administration of grants under section 1109 of the PHS Act (42 U.S.C. 300b-8).

K. Publication of Recommendations

ACHDGDNC recommendations are published on the Committee web site. Occasionally, ACHDGDNC recommendations are also reprinted in other publications.

L. Implementation and Evaluation of the Recommendations

Implementation and evaluation of the impact of the recommendations is the responsibility of the relevant HHS program, and not the ACHDGDNC. However, HHS programs will develop an implementation and evaluation plan for each set of recommendations and periodically report information relevant to the implementation and evaluation activities to the ACHDGDNC, and others who may be involved in implementing the recommendation (e.g., State public health agencies, organizations and institutions, health care payers, private practitioners, etc.).

• Attachment A: Nomination Form (acrobat/pdf)