By Marcio Barra

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More than 30 new drugs, some with novel mechanisms of action which are already used in other European countries, are currently waiting for authorization by the Portuguese National Competent Authority, some for more than a year. The president of the board of APIFARMA, João Almeida Lopes, in a newspiece on the SIC tv channel commented that he believes these delays are due to economic reasons.

Some of the delays extend over a year, whereas current law sets a 75 day time limit for approval.

Below is a list of the drugs currently waiting for commercialization approval in Portugal:

 

Brand name Molecule Indication Marketing Authorization holder Reported reason for delay
Zydelig Idelalisib Zydelig is a cancer medicine that is used to treat chronic lymphocytic leukaemia (a cancer of a type of white blood cells called B lymphocytes) used in combination with another medicine (rituximab) Gilead The first contract proposal was presented by INFARMED in September 2015
Affinitor Everolimus Affinitor is a cancer treatment for advanced breast cancer in women who have been through their menopause. Novartis Contract was received. Process not yet concluded
Votubia Everolimus Used to treat benign tumours caused by the genetic disease tuberous sclerosis Novartis Economic study not yet started
Cosentyx Secukinumab Moderate to severe plaque psoriasis Novartis Parity with the comparator
Cosentyx Secukinumab Ankylosing spondylitis when conventional treatments do not work well enough. Novartis Economic evaluation not yet started
Xolair Omalizumab Chronic urticaria Novartis  –
OFEV Nintedanib Idiopathic pulmonary fibrosis (IPF) Boehringer-ingelheim Orphan indication
Vargatef Nintedanib Cancer medicine used to treat adults with non-small cell lung cancer. Boehringer-ingelheim  –
Praxbind Idarucizumab Reversal agent for Pradaxa Boehringer-ingelheim  –
Giotrif Afatinib Non-small cell lung cancer boehringer-ingelheim  –
Xofigo Radium-223 dichloride Treatment of adults with castration-resistant prostate cancer, symptomatic bone metastases and no known visceral metastases Algeta and Bayer Waiting for a cost minimization analysis from INFARMED. Waiting opinion from the Comissão Nacional de Farmácia e Terapêutica
Stivarga Bayer Gastrointestinal stromal tumor  Bayer Currently under economic evaluation and Waiting the opinion of the Comissão Nacional de Farmácia e Terapêutica
Stivarga  Bayer Colorectal cancer  Bayer Reportedly looking for solutions to assure accessibility to patients. Waiting opinion from the Comissão Nacional de Farmácia e Terapêutica
Zytiga Abiraterone acetate Treatment of prostate cancer  Janssen Accelerated approval by the EMA.

Recognition of added therapeutic value.

Imbruvica Ibrutinib Chronic lymphocytic leukaemia  Janssen Breakthrough therapy designation by the FDA. Recognition of added therapeutic value.
Imbruvica Ibrutinib Mantle cell lymphoma  Janssen Breakthrough therapy designation by the FDA. Recognition of added therapeutic value.
Olysio  Simeprevir  Chronic hepatitis C   Janssen Recognition of added therapeutic value. An estimated 300 patients missing Harvoni (sofosbuvir)
Velcade Bortezomib Patients with non-previously treated multiple myeloma  Janssen
Velcade Bortezomib Previously untreated patients who are going to receive high-dose chemotherapy followed by a blood stem-cell transplant. In this group of patients, Velcade is used in combination with dexamethasone, or with dexamethasone plus thalidomide;  Janssen Therapeutic standard in all guidelines. Recognition of added therapeutic value.
Sylvant Siltuximab Treatment of multicentric Castleman’s disease in adults who tested negative for the human immunodeficiency virus (HIV) and the human herpesvirus-8 (HHV-8)  Janssen Orphan disease –roughly 9 patients each year. Recognition of added therapeutic value
Sirturo Bedaquiline Sirturo is used in combination with other tuberculosis medicines in adults with tuberculosis that is affecting the lung and that is multi-drug resistant (resistant to at least isoniazid and rifampicin, two standard tuberculosis medicines).  Janssen New molecule for tuberculosis. Orphan disease –roughly 3 patients each year. Recognition of added therapeutic value
lynparza Olaparib First oral treatment for women with BRCA-mutated advanced ovarian cancer who have received 3 or more prior chemotherapy medicines  AstraZeneca Oprhan drug. Currently undergoing economic evaluation. Waiting opinion from the Comissão Nacional de Farmácia e Terapêutica
Toujeo Insulin glargine injection Used to treat diabetes in adult patients  Sanofi-Aventis New formulation. Waiting decision on charges limit.
Keytruda Pembrolizumab used to treat adults with melanoma that has spread to other parts of the body or cannot be surgically removed.  Merck Formal negotiations with INFARMED have begun, and a final proposal has been reached.
Zontivity Vorapaxar Used to reduce the occurrence of atherothrombotic events such as further heart attacks or strokes in adult patients who have already had a heart attack. It is given in combination with aspirin and clopidogrel.  Merck Under pharmacoeconomic evaluation
Isentress Raltegravir Isentress is indicated in combination with other anti-retroviral medicinal products for the treatment of human immunodeficiency virus (HIV-1) infection in adults, adolescents, children,toddlers and infants from the age of 4 weeks  Merck Under pharmacoeconom ic evaluation
Genoya Elvitegravir/Cobicista/Emtricitabine/Tenofovir alafenamide HIV-1 Gilead
Zerbaxa Ceftolozane and tazobactam. Antibiotic used to treat adults with complicated (difficult to treat) bacterial infections affecting:

·        tissues and organs within the belly (intra-abdominal infections);

·         the kidneys (pyelonephritis);

·         the urinary tract (structures that carry urine, such as the bladder).

 Merck Waiting opinion from the Comissão Nacional de Farmácia e Terapêutica
Adempas Riociguat It is used to increase the ability to carry out physical activity in adults with Chronic thromboembolic pulmonary hypertension and Pulmonary arterial hypertension  Bayer
Jardiance  Empagliflozin  Type 2 diabetes Boehringer Ingelheim
Pradaxa´s third indication Dabigatran etexilate Treatment of deep vein thrombosis (DVT) and pulmonary embolism (PE), and prevention of recurrent
DVT and PE in adults
Boehringer Ingelheim
Eylea  Aflibercept Diabetic macular edema  Bayer Currently in contract negotiation. Waiting opinion from the Comissão Nacional de Farmácia e Terapêutica
Eylea  Aflibercept Occlusion of the central retinal vein  Bayer Currently in contract negotiation.
Stelara  Ustekinumab  Janssen New indication. Equivalent added therapeutic value
Rezolsta Darunavir / cobicistat Human immunodeficiency virus type 1 (HIV-1)  Janssen New fixed association. Equivalent added therapeutic value
Triumeq Abacavir, dolutegravir and lamivudine Human immunodeficiency virus type 1 (HIV-1)  ViiV Healthcare New fixed association. Equivalent added therapeutic value

 

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By Marcio Barra

A new report detailing patient access to cancer drugs across Europe has shed some light on the numbers for Portugal, and how does it compare to other countries.

The report, “Comparator report on patient access to cancer drugs in Europe revisited”, conducted by the Swedish Institute for Health Economics, gives an overview on the consumption and spending on cancer drugs across European countries, divided by cancer type. The report´s data encompasses 1995 to 2014.

Highlights of the report include:

  • The number of people diagnosed with cancer continue to increase in Europe, with cancer incidence totaling 7 million in 2012, up by 30% from 1995.
  • Mortality rates are going down in relative terms, owing to advances in cancer treatments available today. These include multimodal therapy approaches, improved care by multidisciplinary teams, and improved diagnostic and screening methods that have facilitated early detection of tumors.

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By Márcio Barra

Over the years, I have created a multitude of documents to help me in my projects. I have thus decided to create a useful documents tab to go along with my articles on medicines tab, which is by far the most popular tab of Pharmupdates.

To quick start the tab, I have published both a Word template and an Endnote Style for anyone looking to submit articles to the British Medical Journal (BMJ). I will be updating the tab with new documents that I feel would be useful to someone.

British Medical Journal (BMJ) journal template and Endnote style

I created both a Word template which I used for the submission that has all the required fields for a Research Article (this includes systematic reviews as per the journal rules). I also created an Endnote style, since the link that the journal provides is broken, and the one provided in the Thomsom Reuters website is incomplete.

If you require assistance with the submission procedure (formatting, image creation, referencing) contact me.

For the BMJ Endnote style and template, send me an e-mail.

By Márcio Barra

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Six people were hospitalized in the Hospital of Rennes, France in serious condition, and one is brain dead, following a Phase I first-in-man clinical trial with a new drug  from the Portuguese pharmaceutical company BIAL.  The clinical trial was being conducted by French company Biotrial.

The French Health Minister, Marisol Touraine said on Friday that it is a “serious accident”. The minister added that the accident happened on Thursday and that the trial was suspended, with all previous volunteers called to undergo tests.

There are several reports online describing the drug as an analgesic product based on cannabis, but little to new additional details are being reported. Looking at BIAL´s R&D pipeline, the agent appears to be BIAL´s BIA 10-2474. BIA 10-2474 is described as a new small molecule drug that acts on the Central Nervous System as a painkiller, and currently undergoing Phase I trials.

pipeline_EN_09112015

Reports state that a total of 8 participants were participating in the trial, with two taking placebo. A message on Biotrial´s website reads:

During a FIM study which was being conducted for a sponsor, serious adverse events related to the test drug  have occurred in some subjects at our CPU. The trial has been conducted in full compliance with the international regulations and Biotrial’s procedures were followed at every stage throughout the trial, in particular the emergency procedures for the transfer of subjects to the hospital. We are in close and regular contact with the Health Authorities and Ministry in France. The priority at Biotrial remains the safety of our subjects. We are very grateful for the support we have been receiving from our clients and partners today.

BIAL is still verifying the occurrence, since the drug has been used successfully in other subjects with no issues.

By Márcio Barra

savaysa

Savaysa, a novel anti-clotting agent made by Tokyo-based Daiichi Sankyo, and the first new molecular entity to be approved by the FDA in 2015

Novel drug approvals in the US reached a new high in 2015, with 45 new medicines containing new molecular entities approved by the Food and Drug Administration, above the 2014 figure of 41, the record held since 1996. Moreover, of the 45 new approved drugs, more than 40% of new approvals were for rare disease therapies.

IMS Health expects this rising trend of approvals to continue in the coming years, with with 225 new drugs expected to receive clearance between 2016 and 2020, compared to 184 for the 2011-2015 period.  Cancer treatments are expected to be the largest category of new drug approvals.

The full list (still under construction) is provided below:

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By Márcio Barra

FILE - This undated file photo provided by Gilead Sciences shows the hepatitis C medication Sovaldi. Gilead Sciences says it has reached a deal with several generic drugmakers to produce cheaper versions of its popular, expensive hepatitis C drug Sovaldi for use in developing countries. (AP Photo/Courtesy of Gilead Sciences, File)

Sofosbuvir, Gilead´s Hepatitis C drug which is making waves due to its high cure rate (approximately 90%) and high treatment cost, has already cured 651 Hepatitis C patients in Portugal since it was made available, according to the National Competent Authority INFARMED to Diário de Notícias.

The drug (marketed as Sovaldi for the Sofosbuvir-only formulation, and as Harvoni for the combination of Ledipasvir and Sofosbuvir) was made available following an agreement between the Ministry of Health, INFARMED and Gilead Sciences to treat 13,000 hepatitis C patients. The agreement was concluded on February 2015 following weeks of negotiations. So far, 5322 patients are currently undergoing treatment, with 683 reaching the end of treatment. 32 patients were not cured of Hepatitis C, and hospitals have not reported the causes for the non-response to treatment. As for the total cost of payment per patient, the amount remains undisclosed.

In Portugal, it is estimated about 100,000 people are living with hepatitis C and a large proportion of them have developed cirrhosis. The prices of Sofosbuvir across European member states- in excess of €50,000- make Sobusfuvir a heated topic of discussion among healthcare regulators.