The use of surrogate endpoints in regulating medicines for cardio-renal disease: opinions of stakeholders.

AIM: There is discussion whether medicines can be authorized on the market based on evidence from surrogate endpoints. We assessed opinions of different stakeholders on this topic. METHODS: We conducted an online questionnaire that targeted various stakeholder groups (regulatory agencies, pharmaceutical industry, academia, relevant public sector organisations) and medical specialties (cardiology or nephrology vs. other). Participants were enrolled through purposeful sampling. We inquired for conditions under which surrogate endpoints can be used, the validity of various cardio-renal biomarkers and new approaches for biomarker use. RESULTS: Participants agreed that surrogate endpoints can be used when the surrogate is scientifically valid (5-point Likert response format, mean score: 4.3, SD: 0.9) or when there is an unmet clinical need (mean score: 3.8, SD: 1.2). Industry participants agreed to a greater extent than regulators and academics. However, out of four proposed surrogates (blood pressure (BP), HbA1c, albuminuria, CRP) for cardiovascular outcomes or end-stage renal disease, only use of BP for cardiovascular outcomes was deemed moderately accurate (mean: 3.6, SD: 1.1). Specialists in cardiology or nephrology tended to be more positive about the use of surrogate endpoints. CONCLUSION: Stakeholders in drug development do not oppose to the use of surrogate endpoints in drug marketing authorization, but most surrogates are not considered valid. To solve this impasse, increased efforts are required to validate surrogate endpoints and to explore alternative ways to use them.

European Medicines Agency, CAT Secretariat & US Food and Drug Administration.

The European Medicines Agency (EMA) and the Committee for Advanced Therapies (CAT) are responsible for reviewing applications for marketing authorization for Advanced Therapy Medicinal Products (ATMP), which include (stem) cell-based medicines, for the ATMP classification and certification procedure, and to provide scientific advice to developers of ATMPs. The CAT, an expert committee dedicated to ATMPs, was established by the Regulation (EC) No 1394/2007 on Advanced Therapies. The CAT came into operation in January 2009. ATMPs are defined in this Regulation as gene therapy and cell therapy medicinal products, and tissue-engineered products. The US FDA's Center for Biologics Evaluation and Research is responsible for ensuring the safety, purity, potency and effectiveness of many biologically derived products, including blood intended for transfusion, blood components and derivatives, vaccines and allergenic extracts, and cell, tissue and gene therapy products for the prevention, diagnosis and treatment of human diseases, conditions or injury. Human cells or tissue intended for implantation, transplantation, infusion or transfer into a human recipient are regulated as human cells, tissues, and cellular and tissue-based products (HCT/Ps).

Family planning clinic services in the United States: patterns and trends in the late 1990s.

CONTEXT: Publicly funded family planning clinics are a vital source of contraceptive and reproductive health care for millions of U.S. women. It is important periodically to assess the number and type of clinics and the number of contraceptive clients they serve. METHODS: Service data were requested for agencies and clinics providing publicly funded family planning services in the United States in 1997. The numbers of agencies, clinics and female contraceptive clients were tabulated according to various characteristics and were compared with similar data for 1994. Finally, county data were tabulated according to the presence of family planning clinics and private physicians likely to provide family planning care and according to the number of female contraceptive clients served compared with the number of women needing publicly funded care. RESULTS: In 1997, 3,117 agencies offered publicly funded contraceptive services at 7,206 clinic sites. Forty percent of clinics were run by health departments, 21% by community health centers, 13% by Planned Parenthood affiliates and 26% by hospitals or other agencies. Overall, 59% of clinics received Title X funding. Agencies operated an average of 2.3 clinics, and clinics served an average of 910 contraceptive clients per year. Altogether, clinics provided contraceptive services to 6.6 million women-approximately two of every five women estimated to need publicly funded contraceptive care. The total number of providers and the total number of women served remained stable between 1994 and 1997; at the local level, however, clinic turnover was high. Some 85% of all US counties had one or more publicly funded family planning clinics; 36% had one or more clinics, but no private obstetrician-gynecologist. CONCLUSIONS: Publicly funded family planning clinics are distributed widely throughout the United States and continue to provide contraceptive care to millions of US women. Clinics are sometimes the only source of specialized family planning care available to women in rural counties. However, the high rate of clinic tumover and the lack of significant growth in clinic numbers suggest that limited funding and rising costs have hindered the further expansion and outreach of the clinic network to new geographic areas and hard-to-reach populations.

Proposed redesignation of three health service areas in Illinois--HRSA.

This notice announces the following decisions of the Administrator, Health Resources and Services Administration (HRSA), on the requests of Governor Thompson of Illinois to designate or redesignate three health service areas in Illinois: 1. The request to revise the boundary of health service area 8 (Kane, Lake, and McHenry Counties) by adding DuPage County is denied; 2. The request to establish Surburban-Cook County as a single health service area is denied; and 3. The request to designate a health service area for southwestern Illinois, to include Clinton, Madison, Monroe and St. Clair Counties, is approved. Through this Notice, entities are being invited to apply for designation as the health systems agency for the new southwestern Illinois health service area.

Designation of health services areas and health systems agency application information--HRSA. Notice designating health services areas in Ohio.

This notice is issued to announce the establishment of ten health service areas for the State of Ohio and to provide information on the application procedures to become a federally designated and funded health systems agency.

Public attitudes toward health planning under the Health Systems Agencies.

The demise of the National Health Planning and Resources Development Act of 1974 (PL 93-641) raises questions about the degree of public support it had for planning goals. The results of a 1978 nationwide public opinion poll reveal (1) low confidence in and recognition of the Act's institutional arms, the Health Systems Agencies; (2) little support for hospital cost containment strategies and their consequences; and (3) less than average support for these goals and consequences among those groups traditionally under-represented in health planning activities. The results suggest that the Act did not reconcile centralized federal goal formation with democratic local health planning.

Health systems agency and state health planning and development agency reviews; certificate of need programs--PHS. Notice of proposed rulemaking.

The Assistant Secretary for Health, with the approval of the Secretary of Health and Human Services, proposes to amend the regulations governing certificates of need reviews by State health planning and development agencies (State Agencies) and health systems agencies (HSAs). The proposed amendments would accomplish two tasks: (1) Implement amendments to the Public Health Service Act made by the Health Programs Extension Act of 1980 (Pub. L. 96-538) and the Omnibus Budget Reconciliation Act of 1981 (Pub. L. 97-35) and (2) reduce Federal regulatory burdens. Under the provisions of Title XV of the Public Health Service Act, the planning agencies are required to administer certificate of need programs consistent with the Secretary's regulations, under which they review and determine the need for proposed capital expenditures, institutional health services and major medical equipment. These regulations set forth proposed changes to the requirements for satisfactory certificate of need programs. Interested persons are invited to submit written comments and recommendations concerning these proposed rules as well as suggestions for alternative methods of implementing any of the provisions of the amendments that affect the requirements for certificate of need programs.