August 28, 2013 | By Márcio Barra

Last Friday, INFARMED, the Portuguese National Competent Authority on Medicines, triggered an inspection in order to identify the causes of the reported difficulties some patients are having in accessing the Parkinson’s disease drug Azilect (Rasagiline).

According to a statement issued by INFARMED, in the course of this operation a total of 133 inspections were conducted – 129 pharmacies and four distributors – alongside telephone surveys with 111 pharmacies to collect additional information.

July 17 2013 | By Márcio Barra

NICE, the UK price regulatory agency, has released this Tuesday preliminary draft guidance rejecting Pfizer’s Bosulif (bosutinib) for previously treated patients with chronic myeloid leukaemia (CML). According to the draft release, Bosulif did not represent good enough value for use in the treatment of patients, even though the drug would be provided to the UK’s NHS patients through a patient access scheme. This follows two earlier rebuffs for new cancer drugs developed by Pfizer this year: lung cancer drug Xalkori and kidney cancer drug Inlyta.

February 09, 2013 | By Márcio Barra

The final part of my article on the orphan drug market.

The price question

Now, looking at the prices I showed in part 1 and part 2, one wonders how a patient pays for an orphan drug.

The answer to the question is that the patient doesn’t pay for the drug. First, it’s worth noting that these drugs have a different commercial circuit than normal drugs. They are usually only prescribed at hospitals, under the management of physicians that participated in the trials for the orphan drug.

In the case of the European Union, member states evaluate the drug to decide if their national health systems reimburse the drug. The pricing of the drug itself follows the same logic as drug pricing in general: the price of the orphan drug is set up by the manufacturer in an attempt to recoup R&D…

December 20, 2012 | By Márcio Barra

After Pfizer Inc. and Johnson & JohnsonBapineuzumab failure and Eli Lilly Solaneuzumab not so great results in meeting the clinical endpoints in a phase III trial back in August, it would be expected that pharma companies would steer clear from the amyloid hypothesis which has dominated alzheimer research these last two decades. However, while other theories have re-emerged – the Tau theory, a hypothesis which states if the Tau proteins are hyperphosphorylated they can lead to the creation of tangles in brain cells –,  the amyloid theory is still backed up by a number of stakeholders. Eli Lilly’s Solaneuzumab, while ineffective in lower stages of the disease, has “showed a 34% reduction in cognitive decline”, in patients with mild disease,  and so many companies are focusing to create therapies that intervene before the damage is done and identify affected people much…

http://www.bbc.co.uk/news/health-23847632

Eating lots of broccoli may slow down and even prevent osteoarthritis, UK researchers believe.

The University of East Anglia team is starting human trials following on from successful lab studies.

Tests on cells and mice showed that a broccoli compound – which humans can also get from Brussels sprouts and cabbage – blocked a key destructive enzyme that damages cartilage.

They are asking 20 patients to eat a daily dose of “super-charged” broccoli.

Super-dosed

This special cruciferous vegetable has been bred to be extra rich in nutrients – it is a cross between standard broccoli and a wild relative from Sicily.

Our body takes this glucoraphanin compound and turns it into another, called sulforaphane, which appears to protect the joints.

The volunteers will have two weeks…

April 26, 2013 | By Márcio Barra

When planning a clinical trial, the question that the sponsor wishes to address is the starting point, and where the rest of the trial planning hangs. While the objective of the trial is often obvious to the investigator designing the trial, the question itself can be phrased poorly. Thus, writing a scientific, clear research question is a most valuable action when starting and establishing the study (1).

After coming up with a sound research question, the study design has to be elected. This choice is deeply connected with the desired information that the sponsor wishes to obtain regarding the drug product.

August 27, 2013 | By Márcio Barra

Following the news yesterday on Amgen’s $10.4 billion Onyx buyout, Bayer AG announced today that the FDA has put its drug Nexavar, sold for liver and kidney cancer in the US in partnership with Onyx, on priority review for thyroid-cancer approval.

The priority review designation granted to Nexavar is for the treatment of locally advanced or metastatic radioactive iodine (RAI)-refractory differentiated thyroid cancer, with the submission being based on the results of the DECISION clinical trial. In this trial, a total of 417 patients with locally advanced or metastatic, RAI-refractory, differentiated thyroid cancer – a type of cancer that doesn’t respond to treatment with radioactive iodine – that hadn’t receive prior therapy for thyroid cancer were randomized to receive 400 mg of oral Nexavar twice daily (207 patients) or matching placebo (210 patients). Nexavar doubled progression-free survival, to a median of 10.8 months…

http://phys.org/news/2013-08-alzheimer-cancers.html

A new chemical discovery will lead to better monitoring and treatment for cancers and degenerative diseases, according to latest research by scientists.

In a paper published today in ChemComm an international team of researchers from the Universities of Birmingham, Bath and the East China University of Science and Technology in Shanghai outline a new approach to detecting ‘reactive oxygen’ using fluorescence.

Reactive oxygen, which includes peroxides or oxygen ions, causes cell damage and degeneration in the body and its presence in high doses can be a sign of diseases such as Alzheimer’s or cancers. The importance of reactive oxygen has led researchers to find new approaches for its quick and simple detection.

This latest research paves the way for new systems to detect changes in reactive oxygen levels which could help target interventions. Mapped using fluorescent imaging, the concentration of reactive oxygen in cells and tissue would…

August 26, 2013 | By Márcio Barra

Amgen has announced today that it has agreed to buy Onyx Pharmaceuticals for $10.4 billion, giving Amgen, the largest biotechnology company by sales, access to a new blood cancer drug, Onyx’s Kyprolis. In an all-cash takeover, Amgen is to pay $125 a share for Onyx’s outstanding stock, with the acquisition expected to close at the beginning of the fourth quarter.

Krypolis (Carfilzomib), from Kypolis is an injectable tetrapeptide epoxyketone and a selective proteasome inhibitor, approved by the FDA on 20 July 2012 to treat patients with multiple myeloma, the second most commonly diagnosed blood cancer, who have received at least two prior therapies, including Velcade (bortezomib) and an immunomodulatory therapy.