New products or new indications eligible
for the Prescrire Drug Awards are those
evaluated during the previous year
in the New Products section of
our French edition.

About the 2017 Drug Awards
Each month, the Prescrire Editorial Staff publish systematic analyses of the data available on: new drugs, new indications authorised for existing drugs, and existing drugs marketed in a new form or with different packaging. The goal is to help readers distinguish, among the plethora of new products, those worth adding to their list of useful therapies, those worth using instead of older products, and those to be avoided.

Our analyses are based on rigorous procedures. See > How we work

The Prescrire Editorial Staff conduct these analyses free from any industry or institutional influence. Our independence is made possible by the fact that we are financed exclusively by our subscribers, carry no paid advertising in either the French or the English edition, and receive no grants or subsidies of any kind.

The Prescrire Drug Awards are compiled at the end of each year, based on the reviews published that year in our French edition, and taking into account any new data made available since the initial articles were published. These awards honour drugs that constitute a therapeutic advance, in that they offer better efficacy, less frequent or less severe adverse effects (for similar efficacy), or safer or easier administration.

Three drugs received a Prescrire Award for 2017
Three drugs received a Prescrire Drug Award this year: one that earned a place on the Honours List, and two that were deemed "Noteworthy" (see below).

Asfotase alfa (Strensiq°) in perinatal and infantile hypophosphatasia
Hypophosphatasia is a rare inherited disease caused by an enzyme deficiency. Perinatal forms have a mortality approaching 100%, while over 50% of babies who develop signs and symptoms before the age of 6 months (infantile forms) die in the first year of life.

Asfotase alfa is a recombinant protein that reproduces the activity of the deficient enzyme. The results of two non-comparative clinical trials in a total of 70 infants and children younger than 5 years old, all treated with asfotase alfa, suggest that this drug greatly reduces mortality and the bone disorders associated with this disease. None of the documents identified in our literature search give a breakdown of the results by age group, in particular for infants younger than 6 months old, who have the most serious forms of the disease. This is an important weakness in the drug's evaluation. In addition, the effects of asfotase alfa on the complications of the disease other than bone disorders are unknown. The main known adverse effects are injection site reactions, hypersensitivity reactions, and probably ectopic calcifications.

Pertuzumab (Perjeta°) in certain patients with metastatic breast cancer
In women with HER2- overexpressing metastatic breast cancer, longer-term results from a single clinical trial in 808 patients showed that adding the anti-HER2 antibody pertuzumab to the trastuzumab + docetaxel combination increases the proportion of women alive after 4 years by about 12 percentage points (54% versus 42%) and prolongs median survival by about 16 months. This benefit was observed in patients who for the most part had not previously received trastuzumab and exhibited no cardiac dysfunction before inclusion in the trial. It is for these women particularly that pertuzumab constitutes a therapeutic advance. Pertuzumab's main adverse effects are potentially severe diarrhoea, febrile neutropenia, mucocutaneous disorders, infusion reactions, and heart failure.

Emtricitabine + tenofovir disoproxil (Truvada° or other brands) to prevent HIV transmission in patients at high risk
The main methods for preventing HIV transmission between serodiscordant partners who have penetrative sex are systematic condom use and antiretroviral treatment of the infected person. However, some people engage in sexual behaviour that carries a high risk of HIV transmission but do not use condoms.

Two clinical trials conducted in men who have anal sex with men have shown that pre-exposure prophylaxis with the antiretroviral combination emtricitabine + tenofovir disoproxil, taken every day or only during periods of sexual activity, considerably reduces (without eliminating) the risk of acquiring HIV infection, and has acceptable adverse effects. This type of prophylaxis can increase high-risk behaviour in those who mistakenly believe it affords complete protection and could increase the frequency of other sexually transmitted infections. It is also associated with a poorly documented risk of developing HIV infection that is resistant to the antiretrovirals used for prophylaxis.

In summary
The three drugs that received a Prescrire Drug Award for 2017 are indicated for very different diseases. Three awards for a total of 92 drug analyses published in 2017 is a very low success rate. And for the third year running, none of the drugs examined offered a therapeutic advance worthy of a Golden Pill/Pilule d'Or.

> Download the rules governing the Prescrire Awards (pdf in French)

©Prescrire 25 January 2018

"The Prescrire Awards for 2017" Prescrire Int 2018; 27 (191): 79-83. (Pdf, free)

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