- Email: [email protected]
Prospects for cystic fibrosis carrier screening
478
In a radical move indicating that it means business the ABPI has provided a "hotline" to its director of medical affairs, Dr Frank Wells, to enable him to compile an informal list of doctors against whom he is satisfied there are reasonable grounds for suspicion and concern. Dr Wells tells’me that a complainant can telephone or write to him in confidence explaining his or her concerns and the grounds for suspicion and any data on which these are based. If Wells is satisfied that there is a cause for concern and that the complaints are made in good faith, he will note the name of the doctor and the complainant and his company. If contacted by a second complainant in respect of the same doctor Wells will follow the same procedure and the two complainants will be put in touch with one another to pool their data. The list will not be held on computer, to avoid the possibility of enforced disclosure under the Data Protection Act: All other background information will be held in Wells’ head. Where there are several complaints in respect of the same doctor, a rountl-table discussion may be convened at the ABPI and, if appropriate, a case for referral to the General Medical Council may be drawn up. Does this black-list procedure breach natural justicenamely, the right of an accused person to answer critics and be confronted with complaints and allegations of misconductor dishonesty and given an opportunity to answer them before an unbiased tribunal? Wells feels that the procedure is fair. He was satisfied that in each case the doctor whose actions gave rise to suspicion would have been asked to verify his results-and it was only when verification was not forthcoming that the hotline would be used. This liotline depends for its credibility to a large extent on the’ trust placed in Dr Wells=who is as popular as he is respected in pharmaceutical medicine-and on the integrity of complainants. Wells hopes thatcin time confidence in the system would grow so that the list might be circulated and brought out into the open as in the’ USA where the FDA disclosed its black list under the Freedom of Information Act. But what about libel laws in UK, which are far stricter than those in the USA? The ABPI system has been running since the beginning of the year (some months before publication of the report) and so far Wells has put complainants in touch with each other on three occasions, in respect of different doctors. In the past four years, with the mediation of the ABPI, 26 instances of research fraud involving eight doctors have been proved to the satisfaction of the GMC. But what if a name should get on the black list from a single complainant without justification? The doctor may be aware of a subtle change in attitude towards him but not able to tackle or pinpoint the cause or challenge the. suspicion. From the USA over the past decade or so have come spectacular examples of biomedical research misconduct. However, much of the fraud uncovered by the ABPI’s initiative is likely to be on a more modest scale. A few weeks ago the GMC struck off a general practitioner for fabricating data in a study for a pharmaceutical company. He claimed to have recruited five patients and was found to have improperly completed data on four of them. All eight cases to have come before the Professional Conduct Committee of the GMC in the past four years have ended in a verdict of serious professional misconduct, and a further 18 cases are being investigated. Some frauds are blatant and would become obvious on careful audit--eg, the doctor who dated the assessment of patients before the date when the drugs ’
.
were
supplied. Diana Brahams
Noticeboard Prospects for cystic fibrosis carrier screening Testing for cystic fibrosis (CF) carrier status is likely to become routine in North America within the decade, says the congressional Office of Technology Assessment (OTA).1 The OTA estimates that in the past year the number of such tests done in the USA increased nearly sevenfold, despite calls by professional societies and government agencies for caution in the introduction of the new test, especially for individuals without a family history of CF. The conclusion that the trend toward widespread testing is irreversible may lead doctors to conclude that failure to offer CF testing will soon be a mark of substandard practice, even though the personnel, technical, and public education advances needed are still lacking. Critics of the acceleration in CF testing point out that the existing test has a sensitivity of only 85-95%. Another argument is that the country is unprepared for clinical applications of molecular genetics research. There have been almost no federally funded public education programmes targeted to genetic diseases for a decade, and one estimate is that CF testing for all couples conceiving in one year could require each trained counsellor in the USA to spend 25% of his time on this task alone. The OTA noted that all but one of the steps involved in genetic diagnosis are automated or involve instrumentation under development, and efforts are being made to develop tests that identify several disorders simultaneously. Prepackaged test kits are expected in the next year or two, though there may be a delay before they are approved for marketing. But even if tests are offered to patients, the OTA found that few will accept them without reimbursement by health insurers. A CF carrier test ranges from$55 to$270 (mean$170). An OTA survey of health insurers indicated little likelihood of reimbursement for individuals without family history of the disease. The OTA also found that although few insurers expect to require genetic tests in the next 5 or 10 years, most think that it is fair for insurers to use these tests to identify individuals with increased risk of genetic disease and to use the information to determine risks. Most health insurers who responded to OTA’s survey would accept a CF carrier at standard rates, but a few would exclude benefits related to CF, impose a waiting period, or decline coverage--even though a CF carrier will never have the disease and would be expected to have CF-related costs only if his or her partner was also a carrier and they had children with CF. The results of the calculations of cost-effectiveness of CF carrier screening were impressive and will be further evaluated. Since pre-symptom treatment for CF still shows no substantial benefit, the key variable is whether testing leads to avoidance of the birth of a CF-affected child. 1 Office of Technology Assessment. carrier
Cystic Fibrosis and DNA Tests. implications of screening. Washington: Government Printing Office. 1992. Pp 301. $16
GPO stock 052-003-01291-0
US
growth
The
petition
hormone trials to be reviewed to
halt the US National Institutes of Health
(NIH)-sponsored testing of human growth hormone (hGH) on children, filed by the Foundation on Economic Trends (FOET) and the Physicians Committee for Responsible Medicine (Lancet, July 11, p 110), seems to have met with some success. In her July 24 reply to Jeremy Rifkin, president of FOET, NIH director Bernadine Healy said that "NIH has made the determination to review the hGH research through a newly constituted and independent data safety and monitoring body, which will report to within three months. We have considered the rate of patient accruel in the hGH studies, and are assured that the review will be completed before any further patients are enrolled". But the petitioners are not satisfied, since their aim is that the two hGH trials-one in non-growth-hormone-deficient children with short stature and the other in children with Turner’s syndromeshould cease. Andrew Kimbrell and Katharine Matthews of FOET me
In a radical move indicating that it means business the ABPI has provided a "hotline" to its director of medical affairs, Dr Frank Wells, to enable him to compile an informal list of doctors against whom he is satisfied there are reasonable grounds for suspicion and concern. Dr Wells tells’me that a complainant can telephone or write to him in confidence explaining his or her concerns and the grounds for suspicion and any data on which these are based. If Wells is satisfied that there is a cause for concern and that the complaints are made in good faith, he will note the name of the doctor and the complainant and his company. If contacted by a second complainant in respect of the same doctor Wells will follow the same procedure and the two complainants will be put in touch with one another to pool their data. The list will not be held on computer, to avoid the possibility of enforced disclosure under the Data Protection Act: All other background information will be held in Wells’ head. Where there are several complaints in respect of the same doctor, a rountl-table discussion may be convened at the ABPI and, if appropriate, a case for referral to the General Medical Council may be drawn up. Does this black-list procedure breach natural justicenamely, the right of an accused person to answer critics and be confronted with complaints and allegations of misconductor dishonesty and given an opportunity to answer them before an unbiased tribunal? Wells feels that the procedure is fair. He was satisfied that in each case the doctor whose actions gave rise to suspicion would have been asked to verify his results-and it was only when verification was not forthcoming that the hotline would be used. This liotline depends for its credibility to a large extent on the’ trust placed in Dr Wells=who is as popular as he is respected in pharmaceutical medicine-and on the integrity of complainants. Wells hopes thatcin time confidence in the system would grow so that the list might be circulated and brought out into the open as in the’ USA where the FDA disclosed its black list under the Freedom of Information Act. But what about libel laws in UK, which are far stricter than those in the USA? The ABPI system has been running since the beginning of the year (some months before publication of the report) and so far Wells has put complainants in touch with each other on three occasions, in respect of different doctors. In the past four years, with the mediation of the ABPI, 26 instances of research fraud involving eight doctors have been proved to the satisfaction of the GMC. But what if a name should get on the black list from a single complainant without justification? The doctor may be aware of a subtle change in attitude towards him but not able to tackle or pinpoint the cause or challenge the. suspicion. From the USA over the past decade or so have come spectacular examples of biomedical research misconduct. However, much of the fraud uncovered by the ABPI’s initiative is likely to be on a more modest scale. A few weeks ago the GMC struck off a general practitioner for fabricating data in a study for a pharmaceutical company. He claimed to have recruited five patients and was found to have improperly completed data on four of them. All eight cases to have come before the Professional Conduct Committee of the GMC in the past four years have ended in a verdict of serious professional misconduct, and a further 18 cases are being investigated. Some frauds are blatant and would become obvious on careful audit--eg, the doctor who dated the assessment of patients before the date when the drugs ’
.
were
supplied. Diana Brahams
Noticeboard Prospects for cystic fibrosis carrier screening Testing for cystic fibrosis (CF) carrier status is likely to become routine in North America within the decade, says the congressional Office of Technology Assessment (OTA).1 The OTA estimates that in the past year the number of such tests done in the USA increased nearly sevenfold, despite calls by professional societies and government agencies for caution in the introduction of the new test, especially for individuals without a family history of CF. The conclusion that the trend toward widespread testing is irreversible may lead doctors to conclude that failure to offer CF testing will soon be a mark of substandard practice, even though the personnel, technical, and public education advances needed are still lacking. Critics of the acceleration in CF testing point out that the existing test has a sensitivity of only 85-95%. Another argument is that the country is unprepared for clinical applications of molecular genetics research. There have been almost no federally funded public education programmes targeted to genetic diseases for a decade, and one estimate is that CF testing for all couples conceiving in one year could require each trained counsellor in the USA to spend 25% of his time on this task alone. The OTA noted that all but one of the steps involved in genetic diagnosis are automated or involve instrumentation under development, and efforts are being made to develop tests that identify several disorders simultaneously. Prepackaged test kits are expected in the next year or two, though there may be a delay before they are approved for marketing. But even if tests are offered to patients, the OTA found that few will accept them without reimbursement by health insurers. A CF carrier test ranges from$55 to$270 (mean$170). An OTA survey of health insurers indicated little likelihood of reimbursement for individuals without family history of the disease. The OTA also found that although few insurers expect to require genetic tests in the next 5 or 10 years, most think that it is fair for insurers to use these tests to identify individuals with increased risk of genetic disease and to use the information to determine risks. Most health insurers who responded to OTA’s survey would accept a CF carrier at standard rates, but a few would exclude benefits related to CF, impose a waiting period, or decline coverage--even though a CF carrier will never have the disease and would be expected to have CF-related costs only if his or her partner was also a carrier and they had children with CF. The results of the calculations of cost-effectiveness of CF carrier screening were impressive and will be further evaluated. Since pre-symptom treatment for CF still shows no substantial benefit, the key variable is whether testing leads to avoidance of the birth of a CF-affected child. 1 Office of Technology Assessment. carrier
Cystic Fibrosis and DNA Tests. implications of screening. Washington: Government Printing Office. 1992. Pp 301. $16
GPO stock 052-003-01291-0
US
growth
The
petition
hormone trials to be reviewed to
halt the US National Institutes of Health
(NIH)-sponsored testing of human growth hormone (hGH) on children, filed by the Foundation on Economic Trends (FOET) and the Physicians Committee for Responsible Medicine (Lancet, July 11, p 110), seems to have met with some success. In her July 24 reply to Jeremy Rifkin, president of FOET, NIH director Bernadine Healy said that "NIH has made the determination to review the hGH research through a newly constituted and independent data safety and monitoring body, which will report to within three months. We have considered the rate of patient accruel in the hGH studies, and are assured that the review will be completed before any further patients are enrolled". But the petitioners are not satisfied, since their aim is that the two hGH trials-one in non-growth-hormone-deficient children with short stature and the other in children with Turner’s syndromeshould cease. Andrew Kimbrell and Katharine Matthews of FOET me