¿Cuál será en sí el efecto de este precedente jurídico en el mercado farmacéutico mexicano? México es un mercado emergente y el área con mayor crecimiento hasta la fecha es la de biocomparables. A pesar de que actualmente, sólo existen alrededor de cinco empresas que se dedican de lleno al desarrollo de biocomparables en México, el acceso a este mercado ha incrementado y seguramente crecerá rápidamente en los próximos años. El desarrollo de medicamentos biocomparables requiere el uso de células vivas, así que la imposibilidad de patentar moléculas de origen humano cambiará el ambiente regulatorio en México, afectando desde investigadores que descubren la molécula hasta autoridades regulatorias y por supuesto a pacientes. Hasta la fecha, la Comisión Federal para la Protección contra Riesgos Sanitaros, COFEPRIS, ha actualizado y agilizado los trámites para otorgar bioequivalencia a medicamentos biotecnológicos y biocomparables, mostrando así su disposición para el crecimiento de estas industrias en el país. Aunque aún existen muchas posibilidades de mejora en este aspecto, hemos visto que las autoridades mexicanas han mostrado mucha más aceptación en el desarrollo de terapias con células madre autólogas, tema que es muy debatido en países como Estados Unidos.

Frente a un mercado global de constantes cambios donde las regulaciones parecen multiplicarse y volverse más estrictas, ¿Cuál será la reacción de las autoridades regulatorias respecto al desarrollo de moléculas provenientes de células vivas? ¿O será necesario que las empresas interesadas en desarrollar este tipo de medicamentos inviertan más capital y tiempo en educar a las autoridades y al público sobre los procesos de desarrollo y beneficios de éstas terapias?

Lea el artículo sobre la decisión de la Corte Suprema Estadounidense en patentado de ADN aquí: http://www.nytimes.com/2013/06/14/us/supreme-court-rules-human-genes-may-not-be-patented.html

It’s not been a good couple of years for Alzheimer’s research. Recently, Baxter’s Gammagard joined J&J and Pfizer’s iv bapineuzumab and Eli Lilly’s solanezumab in a list of late-stage failures. Now, Lilly has voluntarily stopped a phase II trial of another of its Alzheimer’s disease drug candidates. This time, it was the company’s beta secretase 1 (BACE-1) inhibitor, LY2886721, that was given the axe.

Through inhibition of beta secretase, BACE inhibitors can theoretically prevent the production of beta amyloid. However, rather than the drug not being effective, Lilly say the decision to terminate the study was on the basis of abnormal liver biochemical tests. Perhaps the good news is that Lilly don’t believe that the abnormal liver tests were related to the BACE mechanism, and they retain interest in developing BACE inhibitors in the future. The company say they will further evaluate the data before deciding on their next steps with the drug.

“While stopping this Phase II study for our BACE inhibitor is disappointing, patient safety is of utmost importance to Lilly,” said Jan M. Lundberg, Ph.D., executive vice president, science and technology, and president, Lilly Research Laboratories. “Discovering and developing medicines for devastating diseases like Alzheimer’s is fraught with many challenges, but Lilly’s 25-year commitment to bringing medicines to the millions of Alzheimer’s disease patients who are waiting will not wane.”

Eyes will now be on the results of clinical trials with other BACE inhibitors, including Merck’s MK-8931, AstraZeneca’s AZD3293, Roche’s RG7129, and Eisai’s E2609.

Read our infographic on Alzheimer’s Drug Development in 2013.

The trial was halted at Phase II rather than at Phase III, which would have been much more costly for the company. If you want to know more about the detection, analysis and prevention of adverse drug reactions with case studies, industry experiences and global regulatory coverage of developments, you might be interested in attending the World Drug Safety Congress Europe 2013, 10-12 September 2013, London.

What do you think? When do you think the string of AD clinical trial failures will end? Why not join our discussion on LinkedIn, or leave a comment below. Want more from Total BioPharma? Sign up to our newsletter – it doesn’t cost anything and only takes a few seconds.

Download a copy here.

The eBook contains answers to the following questions:

• What do you see as the major opportunities for pharma operating in Asian markets?
• What are the biggest challenges that global organizations face in operating in Asia?
• What do you see as the top 3 must-know considerations for drug safety and pharmacovigilance in your local market?
• What is the most significant regulatory development in your local market?
• How can pharma approach drug safety trials in Asia in a better way?

Download the eBook here.

If you are interested in learning more about benefit risk communication, including how to improve your benefit risk communication strategy, you might be interested in attending the World Drug Safety Congress Europe this September. For more information visit the website.

The risk benefit strategies for all drug is incredibly important and is a critical element used to reduce potential risks occurring and enlighten patients and doctors prescribing the medications so that the decision making process is transparent and informed. Benefit risk strategies are always a central theme at the World Drug Safety Congress series of events and often look at novel and proven approaches in the real world.  

Michael Kim, PharmD Process Development Leader, Risk Management spoke at this year’s World Drug Safety Congress Americas in Boston and gave an interesting review of assessing the effectiveness of Risk Management Plans. Download the presentation here.

Dr Robin Gellar, Director of Risk Management of Global Pharmacovigilance, Baxter International, spoke at this year’s World Drug Safety Congress Americas and provided an insightful look at “The benefit risk mindset & evolving management strategies’ download it here for free.

If you are interested in learning more about risk benefit strategies, you might be interested in attending the upcoming World Drug Safety Congress Europe. View the programme here.

The timing could have almost been scripted. No sooner had an FDA panel downplayed historic safety fears over GSK’s Avandia than a new diabetes drug safety scare has reared its head. A paper, published this morning in the British Medical Journal, details the results of a joint investigation between the BMJ and Channel 4 Dispatches into the potential proliferative or inflammatory effects of GLP-1 based diabetes drugs on the pancreas, and asks whether evidence for the link has been underplayed and the regulators slow to react.

Their investigation reportedly uncovered unpublished data that pointed towards unwarranted effects of the drugs on the pancreas, and that despite published reports indicating safety concerns, companies had not performed certain critical safety studies, nor had regulators requested them to do so.

On their own, states the BMJ piece, the individual pieces of unpublished evidence might seem inconclusive, but when considered alongside other emerging and long standing evidence, a “more coherent and worrying picture emerges”.

The BMJ article highlights that three publications this year have raised safety concerns about GLP-1 based drugs, and as such both the FDA and EMA have launched a review into whether the drugs may be linked to pancreatic cancer.

The evidence, says the BMJ, is “fiercely contested” amongst scientists and manufacturers, with some arguing that the evidence against the drugs is weak. Still, the BMJ asks whether companies and regulators have done enough to address these safety concerns, and whether the doctors and patients have been adequately warned.

YOU MIGHT BE INTERESTED IN: 5 Up-and-Coming Treatments for Type 2 Diabetes

For the time being, it seems that while we still can’t be certain whether such a link does exist, the article raises a key issue relating to transparency and the sharing of data. Dr Fiona Godlee, Editor in Chief said: “All drug licensing is about balancing benefits and risks. But instead of engaging in open debate about legitimate and important scientific questions, the manufacturers have been unwilling to share their data. Meanwhile patients and doctors have not been kept properly informed about the uncertainties surrounding these drugs.” She added: “The debate would be much easier to resolve if all the information was placed in the public domain so scientists, doctors and ultimately patients could make up their own minds.”

Given the fate suffered by GSK’s Avandia after some apparently unfounded safety concerns, it seems more important than ever to really get this one right. Already the stories are published across the UK newspapers (Express: “Is diabetes super drug a cancer risk for two million people?”; The Times: “Safety fears over diabetes drug ‘linked with cancer’”), something which will no doubt be of extreme concern to all patients taking an otherwise effective drug.

Read the BMJ article here, and the press release here >

What do you think? Why not join our discussion on LinkedIn, or leave a comment below. Want more from Total BioPharma? Sign up to our newsletter – it doesn’t cost anything and only takes a minute.

If you want to know more about drug safety and addressing the key challenges for safety professionals, you might be interested in attending the World Drug Safety Congress Europe 2013, 10-12 September 2013, London.

YOU MIGHT BE INTERESTED IN: GSK’s Avandia: FDA panel recommend rosiglitazone restriction relaxation

An advisory committee to the US Food and Drug Administration (FDA) has voted to recommend market restrictions on former blockbuster drug rosiglitazone in the US. The GlaxoSmithKline drug, also known as Avandia, was once of the best-selling diabetes pill in the world, but became the centre of controversy as data emerged from the RECORD study linking the drug to cardiovascular problems. From 2010 onwards, the drug which once drew in $3.2 billion of sales annually was subject to heavy restrictions in the US and withdrawn from the market in Europe.

Following a recent re-analysis of data from the study, however, it seems that that Avandia might not actually increase the risk of heart attack more than other drugs used for type 2 diabetes. “In general, this drug doesn’t look any different than any other diabetes drug,” said Dr. William Hiatt, a cardiologist from the University of Colorado (Reuters).

The FDA panel vote was divided, with 13 members voting to modify the restrictions and 7 members voting to remove the risk evaluation and mitigation strategy (REMS) programme. 5 members voted to continue with REMS, and 1 member voted for complete withdrawal of the drug from the US market (PharmaTimes).

YOU MIGHT BE INTERESTED IN: Top 5 Most Surprising Pharma Regulatory Agency Rejections

“We continue to believe that Avandia is a safe and effective treatment option for type 2 diabetes when used for the appropriate patient and in accordance with labeling,” Dr. James Shannon, Glaxo’s chief medical officer, said in a statement (Reuters).

How does this change the future for Avandia? With the amount of new diabetes medications entering (or soon to enter) the market (5 Up-and-Coming Treatments for Type 2 Diabetes), Avandia might not find its place in the market again. What do you think? Do you think the recommendations will lead to a bounce back to former glory for rosiglitazone, or do you think they come too late to salvage the troubled drug?

Why not join our discussion on LinkedIn, or leave a comment below.

If you want to know more about drug safety and addressing the key challenges for safety professionals, you might be interested in attending the World Drug Safety Congress Europe 2013, 10-12 September 2013, London.

Want more from Total BioPharma? Sign up to our newsletter – it doesn’t cost anything and only takes a minute.

Reliance on patient self-report (patient reported outcome (PRO) measures) to gain insight into the impact of living with a disease or the outcomes of an intervention is based upon the assumption that the respondent understands the question in the same manner as the researcher.

RESPONDENTS’ TASK IN RESPONDING

Although there are a number of models proposed to explain the cognitive process on how a respondent answers a survey question they all generally share the same steps which are:

1. Understanding the question
2. Recalling the relevant behaviour or event
3. Inference and estimation
4. Mapping the answer onto the available response options
5. Editing the answer for social desirability

To understand and respond to the question, respondents will draw on a wide range of contextual information to report on their behaviour or frequency of some event or symptoms.

AUTOBIOGRAPHICAL MEMORY

When reporting a particular health event or behaviour respondents are unlikely to scan the prescribed recall period and count i.e. recall and count the instances, unless the event or behaviour is memorable such as a severe hypoglycaemic event. There are a number of factors that make the recall and count strategy unsuitable for much of the behaviours and episodes that are evaluated using PROs because:

• Memory decreases over time irrespective of its importance
• Respondent are unlikely to have detailed representations of the various episodes or behaviour when they are of frequent nature.
• Our autobiographical memory is not structured by categories of behaviour or events such as having low blood sugar levels but, is thought of as a hierarchical network in which lower-level events (was hospitalised with flu)  are nested within higher order events (such as diagnosed with diabetes) and within these are the specific or summarised events at a lower level (frequently had low blood sugar levels).

RESPONSE OPTIONS

Cognitive research has shown that the response options presented can influence respondents’ reporting of the frequency of vaguely defined events and that the set of response alternatives is treated as information in the interpretation of the question.

Our early research showed respondents presented with response alternatives discriminating at medium frequency, reported significantly fewer health-related events than those presented with high frequency response alternatives, suggesting respondents use response categories as a guide as to whether the event or behaviour in is of minor or major importance from the perspective of the investigator, with high frequency reporting response categories e.g. “Once a week” to “everyday” being associated with minor events and behaviour. Questions with lower frequency options e.g. “Less than a year” to “more than once a month” the magnitude of the event or episode is interpreted as more major.

Because response options carry meaning which can be conveyed to the respondent that influences their  interpretation of the question developers need to consider the implications of the used response scale on the respondents behaviour.

THE TEMPORAL DIRECTION OF RECALL

The ordering of questioning is also important and cognitive research suggests that asking respondents to recall the most recent event aids recall of previous episodes. This is probably due to the most recent recall serving as a cue for recalling previous events.

SUMMARY

This brief post has outlined some of the many cognitive issues regarding asking respondents question about their heath behaviour etc. Not least is the respondents comprehension of just what the question is asking and the various strategies the respondent will use to answer the question.

For more information on the cognitive processes in the PRO measurement contact:

Dr Keith Meadows

Email: info@dhpresearch.com

www.dhpresearch.com

Blog: the patientoutcomesblog.com

TransCelerate BioPharma Inc, an independent non-profit organization focused on accelerating the research and development of new medicines, has released a paper that outlines a standard approach for risk-based monitoring that can be adopted to any clinical trial, regardless of type, phase and stage (Press Release). The approach is designed to enhance patient safety and ensure quality clinical data, and could significantly modernize and streamline the way studies are conducted and monitored in the future.

“Biopharmaceutical companies often spend an extraordinary amount of effort monitoring clinical trials – data from each patient, for every study, at every global site, is reviewed – yet, there isn’t much evidence to indicate that this level of review is effective at identifying systemic errors or substantially improving the quality of data gathered,” said Dalvir Gill , PhD, Chief Executive Officer of TransCelerate, in a press release.  “Despite this, monitoring approaches have remained unchanged.  In this position paper, we have outlined a methodology – procedures, algorithms, a toolkit – for risk-based monitoring that we believe will be effective and efficient for our member companies and others.”

Current monitoring processes rely on source data verification (SDV), but the TransCelerate toolkit shifts the monitoring processes away from SDV and towards comprehensive risk-driven monitoring. TransCelerate’s recommendations are driven by centralized and off-site monitoring techniques, as well as adaptive on-site monitoring, allowing researchers to mitigate risks and detect any issues early.

“TransCelerate’s RBM methodology provides the framework and tools to manage clinical trial risks through identification, categorization and appropriate mitigation.  We are providing tools like the RACT (Risk Assessment and Categorization Tool) and the IQRMP (Integrated Quality and Risk Management Plan), which will help companies identify and plan their risk mitigation strategies.  We also are sharing an approach that enables a balance between off-site/centralized study monitoring and on-site activities at the investigative site.  This allows for a more targeted approach,” said Rehbar Tayyabkhan, Executive Director at Bristol-Myers Squibb and RBM Project Lead for TransCelerate.

YOU MIGHT BE INTERESTED IN: eBook ‘Top tips from World Drug Safety US 2013’

TransCelerate was formed in 2012, and its members include AbbVie, AstraZeneca, Boehringer Ingelheim, Bristol-Myers Squibb, Eli Lilly and Company, GlaxoSmithKline, Johnson & Johnson, Pfizer, Roche, Sanofi, Astellas Pharma Inc., Biogen Idec, Braeburn Pharmaceuticals, EMD Serono, Inc. (a subsidiary of Merck KGaA, Darmstadt, Germany), Forest Research Institute (a subsidiary of Forest Laboratories, Inc.) and Onyx Pharmaceuticals.

What do you think? Do you think the recommendations will help enhance patient safety and clinical data from trials? Why not join our discussion on LinkedIn, or leave a comment below.

If you want to know more about drug safety and addressing the key challenges for safety professionals, you might be interested in attending the World Drug Safety Congress Europe 2013, 10-12 September 2013, London.

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YOU MIGHT BE INTERESTED IN:  7 universal challenges facing drug safety

> Click here to download the full press release

Shire is a Platinum Sponsor at the upcoming World Orphan Drugs Congress Asia 2013, held from 18-19 June at Grand Copthorne Waterfront Hotel, Singapore.

Despite the increasing use of patient reported outcome measures (PROMs) across a range of clinical settings and research, there are a number of important issues that remain to be addressed before we can maximise the benefits from their use in clinical trials and real world studies including, for example, the need to ensure instruments, data collection and analysis is highly credible.

There must also be demonstrable evidence that PROMs have been developed with scientific rigour with proven reliability, validity and sensitivity to detect change where change is present as well as the need to make PROM data meaningful to those who need to be informed including, nurses, clinicians, commissioners, providers and policy makers. Data must be relevant, affordable and practical to collect and not affect the delivery of care in the process.

There is also a need to know more about how best PROMs can be embedded into the decision-making process and combine the derived data with other clinical information as well as explore ways to provide feedback as insightful information to enable sound clinical decision making.

A few initiatives to get you started

1. Establish buy in from all those involved in the project. It is essential that every person involved in the process is in agreement. This will include in particular those who will be administering the PROM or who run the service in order to minimise disruption to the delivery of care process. Any potential obstacles should be resolved at this stage.
2. Understand the big picture.

• i. Determine exactly the purpose of the project. This will include defining the inputs and desired outcomes to be measured which are relevant to the patient and project in order to develop the measurement strategy.
• ii. Can the desired outcome(s) be measured?
• iii. Why these particular outcomes? Establishing the measured outcomes are essential and defining them to be ‘health status’ ‘quality of life’ or ‘health-related quality of life’ etc.
• iv. What action will follow once the PROM evidence is available and who are the key stakeholders involved?

3. Identify the practicalities. Define exactly the patient group. How will the PROM be administered? Who will undertake the analysis?
4. Selecting the appropriate PROM. This will combine a number of factors including development of the measurement strategy and a detailed and comprehensive review of the literature to identify the PROM. This is the point at which selection must be based on what the PROM is purported to measure which must be supported by a clear conceptual framework, its scientific and measurement qualities and whether it’s possible to easily interpret the scores as to really what they mean. Selection must not be based on the name alone of the PROM or because it has been used in other studies.

Ultimately, we are only scratching the surface here when it comes to the implantation of a PROM. The key comes down to this: You have to plan for measurement. It is almost impossible to measure patient reported outcomes without a clear measurement strategy defining exactly the inputs and desired outcomes.

Dr Keith Meadows is founder and director of DHP Research & Consultancy Ltd www.dhpresearch.com

Email: info@dhpresearch.com

Blog: http://thepatientoutcomesblog.com