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Theme: Regulatory issues

Drugs regulatory bodies ought to be the public’s principal defence against medicines that are useless, unproven or downright dangerous. Yet regulators increasingly depend upon financing from the pharmaceutical industry itself.  

What companies want

There is cause for concern about patients and healthcare expenditures, if the so-called regulatory authorities allow the most influential pharmaceutical companies to get their way, as the French authorities’ behaviour in the case of pegaptinib (Macugen°) has demonstrated.

One month after Pfizer was granted European marketing authorisation, via the centralised procedure, for its pegaptinib-based product Macugen°, the French marketing authorisation committee held a meeting to issue an opinion on the conditions for its sale, and its use outside the hospital setting (1).

Six outside experts, "retina specialists", attended this meeting in order to "inform the committee". Three of them declared "links with the matter under review (...) as consultants to the pharmaceutical company editor’s note: Pfizer]". But the committee felt that it was desirable to hear their opinions, given the "major scientific interest" (1). A favourable decision was handed down for outpatient use of pegaptinib. The experts predicted that the number of patients would increase and that hospitals would not be able to deal with "all the practical and financial constraints" that would result from treating them (1).

In order to prevent the risk of serious adverse effects linked to the increased use of pegaptinib, the French Health Products Safety Agency (Afssaps) created a working group on "the proper use of pegaptinib” and issued a "statement on the proper use" of the drug (2,3). And the European Medicines Agency (EMEA) accepted the company’s risk management plan, which includes a "patient education programme" on the risk of adverse effects, a programme run by the company itself (4)! In late November 2006, the French Transparency Committee (which assesses the medical benefits of new drugs and provides advice concerning drug reimbursement) issued a favourable opinion on the reimbursement of pegaptinib (at a rate of 65%), drawing conclusions that are appalling (5). According to this opinion, "the current data, although they suggest a similar degree of efficacy, do not permit to clearly distinguish the role of Visudyne° (verteporfin) and Macugen° in treatment strategy editor’s note: the two drugs were not directly compared] (...) treatment with Visudyne° is still considered the reference treatment" (5).

The Committee nevertheless concluded that "pegaptinib provides a moderate increase in therapeutic benefit ("ASMR III") to patients suffering from exsudative subfoveolar aged-related macular degeneration (...) notably in forms involving barely visible choroidal neovascularisation"! (5). And the decree announcing the high price granted for reimbursement of pegaptinib only stipulates that treatment be restricted to neovascular subfoveolar (wet) forms (6).

The Transparency Committee’s opinion also mentions the authorisation of ranibizumab in the same indication and off-label use of bevacizumab (5). As there is a trend towards intensive screening for age-related macular degeneration (7), there is reason to be concerned about patients and healthcare expenditures, if the so-called regulatory authorities, as in this case, continue to allow the most influential drug companies to get their way.

©Prescrire 2007

Source: "Ce que firmes veulent" Rev Prescrire 2007; 27 (284): 421.

1- Afssaps “Commission d’autorisation de mise sur le marché des médicaments - Réunion n°397 du 2 mars 2006”: 10 pages + “Réunion n°404 du 28 juin 2006”: 6 pages.
2- Afssaps “Mise au point sur le bon usage de la spécialité Macugen°, solution injectable intravitréenne” 26 July 2006: 4 pages.
3- “Décision DG n°2006-154 du 11 juillet 2006 portant nomination auprès de la Commission d’AMM du groupe de travail en charge de l‘élaboration de la mise au point “bon usage de Macugen” de l’Agence française de sécurité sanitaire des produits de santé” Bulletin Officiel Santé Solidarités 2006/8 du 15 septembre 2006: 25.
4- EMEA-CHMP “EPAR-Macugen (first published) Summary for the public + Scientific discussion” 41 pages posted on EMEA website 16 February 2006.
5- HAS - Commission de la transparence “Avis- Macugen” 29 November 2006: 12 pages.
6- - “Arrêté du 28 février 2007 modifiant la liste des spécialités pharmaceutiques remboursables aux assurés sociaux” Journal Officiel 20 March 2007: 5090-5092 + “Avis relatif au prix”: 5133.
7- Retina France - Communiqué de presse “SOS DMLA - Lundi 5 février, début de la campagne nationale de dépistage Retina France” 6 February 2007: 1 page.