“Gleevec can cost up $70,000 per year, while Indian generic versions cost about $2,500 a year.”
“Gleevec can cost up $70,000 per year, while Indian generic versions cost about $2,500 a year.”
Importantísimo fallo de la Suprema Corte de India, negando a Novartis el derecho a registrar una patente reverdecida (a través del evergreening)
Indian Supreme Court Delivers Verdict in Novartis Case
Decision safeguards access to affordable medicines and prevents abusive patenting of medicines
“Bioética y Derechos Humanos en América Latina: Parte I Bioética y Derechos Humanos”Coordinadores/Docentes: Dr. Juan Carlos Tealdi y Dra. María Luisa PfeifferProfesores interdisciplinarios: Dr. Miguel Chávez- Dr. Rodolfo Di Sarli- Dra. Patricia Digilio- Dr. Francisco Maglio- Dr. Mario Pecheny- Abogado César San Juan.Comienzo: a confirmarFin: a confirmarDías de Cursada: martesHorario: 14 a 17hs.Duración/Créditos: 32hs./2Sede: Santiago del Estero 1029 Aula: 110Inscripción: próximamenteVer Programa
Esto aparece al final de la carta de Huber, pero ha sido ya publicado en varias revistas. Esta prohibición de investigar se opone a la ciencia, y como dicen los entomólogos que la redactaron los “inhibe de cumplir con su rol en protección del bien público”.
Letter from 26 University Entomologists to EPA (2008)
The following statement has been submitted by 26 leading corn insect scientists working at public research institutions located in 16 corn producing states. All of the scientists have been active participants of the Regional Research Project NCCC-46 “Development, Optimization, and Delivery of Management Strategies for Rootworms and Other Below-ground Insect Pests of Maize” and/or related projects with corn insect pests. The names of the scientists have been withheld because virtually all of us require cooperation from industry at some level to conduct our research.
“Technology/stewardship agreements required for the purchase of genetically modified seed explicitly prohibit research. These agreements inhibit public scientists from pursuing their mandated role on behalf of the public good unless the research is approved by industry. As a result of restricted access, no truly independent research can be legally conducted on many critical questions regarding the technology, its performance, its management implications, IRM, and its interactions with insect biology. Consequently, data flowing to an EPA Scientific Advisory Panel from the public sector is unduly limited.”
Don M. Huber, Professor Emeritus of Purdue University writes a letter to Vilsack regarding his concerns about glyphosate.
This cover letter is provided to explain the reasoning and concerns that were conveyed in a letter which I sent to Secretary of Agriculture, Thomas Vilsack on January 17, 2011 (Attachment 1). The letter was not intended for public distribution; however, the letter was ‘leaked’ and subsequently posted on the internet from which it soon became public knowledge world-wide. Once it was widely distributed, I gave permission for subsequent postings in order to keep it consistent. My busy meeting and travel schedule has delayed getting further information on this matter out publicly to the many individuals who have requested it. The scientific data on this newly recognized organism is being prepared for formal publication.
I wrote the letter to Secretary Vilsack for a very simple reason: we are experiencing a large number of problems in production agriculture in the U.S. that appear to be intensified and sometimes directly related to genetically engineered (GMO) crops, and/or the products they were engineered to tolerate – especially those related to glyphosate (the active chemical in Roundup® herbicide and generic versions of this herbicide). We have witnessed a deterioration in the plant health of corn, soybean, wheat and other crops recently with unexplained epidemics of sudden death syndrome of soybean (SDS), Goss’ wilt of corn, and take-all of small grain crops the last two years. At the same time, there has been an increasing frequency of previously unexplained animal (cattle, pig, horse, poultry) infertility and spontaneous abortions. These situations are threatening the economic viability of both crop and animal producers.
Incidence of high infertility and spontaneous abortions in the various animal species is becoming more common. Often, all previously known causes of these conditions can be ruled out as factors for these particular farm operations (Attachment 2). Detailed examination for the newly recognized organism has shown its presence in all of the cases examined to date. Koch’s postulates have been completed for animals to verify the cause/effect relationship with this newly culturable organism. A search for the source of animal infections revealed a high population of this newly discovered electron microscopic sized organism in soybean meal and corn products. The organism appears compatible, and probably synergistic, with other microorganisms such as Fusarium solani fsp. glycines, the cause of SDS of soybeans and also with gram positive bacteria. The organism also is in a very high population in Goss’ wilt infected corn caused by the gram positive bacterium Clavibacter michiganensis subsp. nebraskensis.
Although most corn hybrids have been genetically resistant to Goss’ wilt, preliminary research in 2010 demonstrated that the application of glyphosate herbicide, or the surfactant from glyphosate formulations, nullified this resistance and rendered them fully susceptible to this pathogen (Fig. 1). This disease was commonly observed in many Midwestern U.S. fields planted to RR corn in 2009 and 2010, while adjacent non-GMO corn had very light to no infections in spite of the high inoculum present in no-till crop residues (Figure 2). The increased Goss’ wilt in 2010 was a major contributor to the estimated almost one billion bushels of corn ‘lost’ last year (based on USDA August estimated yields and actually harvested crop reported by USDA in January) in spite of generally good harvest conditions.
Increased severity of plant diseases after glyphosate is applied (Fig. 3) is well documented and, although rarely cited, the increased disease susceptibility is the herbicidal mode of action of glyphosate (Johal andRahe,1988, 1990; Johal and Huber, 2009; Schafer et al, 2009, 2010). The loss of disease resistance in Roundup Ready® sugar beets when glyphosate was applied prompted researchers at the USDA sugar beet laboratory to include a precautionary statement in their paper, e.g. “Precautions need to be taken when certain soil-borne diseases are present if weed management for sugar beet is to include post-emergence glyphosate treatments” (Larson et al, 2006).
The loss of genetic resistance in Roundup Ready® corn hybrids to Goss’ wilt (Clavibacter michiganensis subsp. nebraskensis) (Figs. 2, 3), synergistic relationship of the newly recognized electron microscopic organism causing infertility and abortions in animals with gram+ bacteria, and high populations of the new EM organism in RR corn leaves and silage creates a concern for the deregulation of Roundup Ready® alfalfa which is productive in many areas only because of its genetic resistance to bacterial wilt caused by Clavibacter michiganensis subsp. insidiosum. This disease could make alfalfa unprofitable for production and, if the EM organism is associated with it in alfalfa as it is in corn, also unsafe for animal feed and their products such as milk for human consumption. The loss of alfalfa, the United State’s most valuable forage crop and fourth most economically important crop, could strike a mortal blow to struggling dairy and beef operations.
Extensive research has shown that this potent tool for weed management, glyphosate, is also a strong immobilizer (chelator) of essential plant nutrients to impair nutrient uptake, translocation, and physiological efficiency at only a fraction of the labeled herbicidal rate (Ekers, Ozturk, Cakmak, Zobiole, Jolly et al., 2004). Glyphosate is a powerful biocide to harm beneficial soil organisms important for nutrient recycling, N-fixation, nutrient availability, and natural disease control (Kremer & Means, Zobiole et al, Dick et al) with a resultant increase in diseases of corn, soybeans (Fig. 3), wheat and other crops. The close relationship between mineral nutrition and disease severity is well documented (Datnoff et al, 2007). These activities can have deleterious effects on plant nutrition, disease susceptibility, and nutritional quality of the crop produced.
Deleterious effects of GM crops also are vividly demonstrated in reports from livestock producers in the U.S. Although some of these reports are anecdotal because of limited analytical techniques to verify the cause, some producers have been able to resume economical operations by changing feed sources to non-GMO crops. Replicated independent research is needed in this area, especially in light of the serious toxicological concerns raised recently that show potential human and animal toxicity from very low levels of residual glyphosate in food/feed that are many times lower than permitted in U.S. food and feed products (Seralini et al., 2011). The recent Indian Supreme Court’s independent analysis and Ruling that GMO egg plant posed a significant health risk to humans needs further evaluation in the U.S. (AgroNews, 2011).
I feel I would be totally irresponsible to ignore my own research and the vast amount of published research now available that support the concerns we are seeing in production agriculture, without bringing it to the attention of the Secretary of Agriculture with a request for him to initiate the much needed independent research. Many producers can’t wait an additional 3-10 years for someone to find the funds and neutral environment to conduct such critical research (Attachment 2. Entomologists letter to EPA).
Based on the scientific evidence currently accumulating, I do not believe it is in the best interests of the agricultural producer or consuming public for regulatory agencies to approve more GMO crops, particularly Roundup Ready® alfalfa and sugar beets, until independent research can establish their productivity when predisposed to potentially severe diseases, the irrelevance of the new EM organism, and their nutritional equivalency. In my letter, I asked the Secretary to allocate the necessary resources to do this, and requested that he exercise the utmost caution in deregulating these crops until such findings resolve the concerns expressed in the letter, if they do.
Don M. Huber
Professor Emeritus, Purdue University 17/1/2011
Ver bibliografía en el link al texto original.
Esta entrevista es del 2009, pero cobra actualidad y urgencia con la publicación de
Séralini, G.-E., et al. Long term toxicity of a Roundup herbicide and a Roundup-tolerant genetically modiﬁed maize. Food Chem. Toxicol. (2012)
ABSTRACT – The health effects of a Roundup-tolerant genetically modiﬁed maize (from 11% in the diet), cultivated with or without Roundup, and Roundup alone (from 0.1 ppb in water), were studied 2 years in rats. In females, all treated groups died 2–3 times more than controls, and more rapidly. This difference was visible in 3 male groups fed GMOs. All results were hormone and sex dependent, and the pathological proﬁles were comparable. Females developed large mammary tumors almost always more often than and before controls, the pituitary was the second most disabled organ; the sex hormonal balance was modiﬁed by GMO and Roundup treatments. In treated males, liver congestions and necrosis were 2.5–5.5 times higher. This pathology was conﬁrmed by optic and transmission electron microscopy. Marked and severe kidney nephropathies were also generally 1.3–2.3 greater. Males presented 4 times more large palpable tumors than controls which occurred up to 600 days earlier. Biochemistry data conﬁrmed very signiﬁcant kidney chronic deﬁciencies; for all treatments and both sexes, 76% of the altered parameters were kidney related. These results can be explained by the non linear endocrine-disrupting effects of Roundup, but also by the overexpression of the transgene in the GMO and its metabolic consequences.
Entre las conclusiones se afirma que “The results of the study presented here clearly demonstrate that lower levels of complete agricultural glyphosate herbicide formulations, at concentrations well below ofﬁcially set safety limits, induce severe hormone-dependent mammary, hepatic and kidney disturbances.”
“Other mutagenic and metabolic effects of the edible GMO cannot be excluded. This will be the subject of future studies, including transgene and glyphosate presence in rat tissues. Reproductive and multigenerational studies will also provide novel insights into these problems. This study represents the ﬁrst detailed documentation of long-term deleterious effects arising from the consumption of a GM R-tolerant maize and of R, the most used herbicide worldwide.”
Hace muchos años que Séralini viene estudiando estos temas, y ha denunciado claramente que las semilleras no dan los datos de sus estudios al conocimiento público, los consideran confidenciales. Además tratan de bloquear la investigación. Por supuesto que gran cantidad de científicos objetan sus conclusiones, pero así se genera la ciencia. De todos modos es imprescindible recordar la necesidad de aplicar el principio de precaución.
¿Como es posible que el Ministerio de Salud argentino no haya establecido un grupo permanente de investigación sobre estos temas de contaminación por plaguicidas y salud pública? ¿Como es posible que trabajos como el que se hizo en la Sardá sobre clorados en leche materna no hayan podido completarse adecuadamente por falta de fondos para reactivos, ni hayan podido tener un seguimiento adecuado de la maduración neurológica de los niños involucrados?
Editorial del British Medical Journal que dice, entre otras cuantas cosas:
“nine of the 10 largest drug companies were bound by corporate integrity agreements under civil and criminal settlements or judgments in the United States. The corporate activity that has led to recent government investigations has involved unethical and unlawful practices that are well beyond mere administrative offences.
Whistleblowers’ and other “insider” accounts in the US typically include allegations that companies systematically planned complex marketing campaigns to increase drug sales, which involved illegal and fraudulent activities. These included active promotion of off label, or otherwise inappropriate, use of drugs, despite company knowledge that such use could seriously harm patients. “
Al referirse a la falta de medidas de castigo de gravedad apropiada el BMJ dice: “Such timid regulatory behaviour may be symptomatic of the extent to which regulators have been encouraged by governments to be responsive to the commercial interests of industry and to view large drug firms as clients whose fees increasingly fund these agencies.”
Ver Wikipedia “The Prescription Drug User Fee Act (PDUFA) was a law passed by the United States Congress in 1992 which allowed the Food and Drug Administration (FDA) to collect fees from drug manufacturers to fund the new drug approval process.”
Esta gente -bioeticistas de USA y Europa – pretende pontificar sobre corrupción, y darnos clase, cuando tiene la corrupción instalada hasta los huesos del sistema, y además legalizada, tal como lo denuncia el BMJ.
Sin ruborizarse afirman: “Dr. Ben Goldacre’s book Bad Pharma provides a one-sided and factually questionable view of the clinical trial process led by the innovative biopharmaceutical sector. The book ignores that the current clinical trial system, which is essential to the development of new medicines that save and improve lives, is scientifically rigorous, tightly regulated and working well.”
Al final manifiestan en un recuadrito:
“The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading innovative biopharmaceutical research and biotechnology companies, which are devoted to discovering and developing medicines that enable patients to live longer, healthier, and more productive lives.”
O sea que estábamos errados, no lo hacen con fines de lucro.
SPIRIT 2013 Statement: Defining Standard Protocol Items for Clinical Trials
Para leer con detenimiento.
The protocol of a clinical trial serves as the foundation for study planning, conduct, reporting, and appraisal. However, trial protocols and existing protocol guidelines vary greatly in content and quality. This article describes the systematic development and scope of SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) 2013, a guideline for the minimum content of a clinical trial protocol.
Análisis de la Salud Colectiva Ambiental de Malvinas Argentina – Córdoba
Una investigación socio-ambiental y sanitaria a través de técnicas cualitativas y relevamiento epidemiológico cuantitativo
- Cátedra de Clínica Pediátrica, Facultad de Ciencias Médicas, Universidad Nacional de Córdoba
De la investigación desarrollada se concluye que: “La vulnerabilidad social y económica de la población es las más alta en la Provincia de Córdoba y la capacidad del Estado Municipal de responder a la demanda de enfermedad es mínima. Someter a esta población a un nuevo golpe en su salud ambiental, como el que generará una enorme planta de semillas transgénicas de Monsanto en su jurisdicción no es recomendable desde el punto de vista médico, y es intolerable desde el punto de vista social.”
Medardo Ávila Vázquez | email@example.com | 351-15-5915933
Luciana Ruderman | firstname.lastname@example.org | 351-15-3724132
Se garantiza la privacidad en los ensayos clínicos y en la recolección de muestras para farmacogenómica…
Yaniv Erlich, a computer informatics and genetics researcher at the Whitehead Institute, and his colleagues published a paper in Science where they explained that Erlich and his then graduate student Melissa Gymrek had re-identified 50 of the 1000 Genomes samples. Erlich and Gymrek developed a tool that allowed them to quickly identify repeated areas of DNA on the Y chromosome and applied that technique to 10 male samples from 1000 Genomes. Comparing that information to other web-based genetic databases—from the Center for Study of Human Polymorphisms (CEPH) and the National Institute of General Medical Sciences Human Genetic Cell Repository at the Coriell Institute—provided ages, geographic location, and even family trees that enabled Erlich and Gymrek to identify 50 of the 1000 Genomes donors. In short, they could re-identify the de-identified data.
Investigadores y bioeticistas que ven con simpatía al “mercado de la salud” y cosas semejantes a menudo se quejan de que no hay que “demonizar” a la sufrida industria farmacéutica… Pareciera que es un sesgo de los bioeticistas latinoamericanos “de izquierda” o algo así, y que no está fundado en evidencias sino en prejuicios ideológicos… puede ser, pero por eso nos preguntamos:
¿ Quien dice
“Drug firms often develop products that have minor value, and neglect investing in significant innovative therapies.”
“They often behave as if they have no responsibility for how physicians prescribe or patients use drugs.”
“They sometimes promote drugs in ways that endanger the public’s health.”
“They market drugs for uses where risks outweigh the benefits.”
“They fuel inappropriate prescribing while earning income from improper drug use.”
“Worse, drug firms have slanted the information available to public officials who decide whether to authorize the sale of drugs and who monitor the risks of drugs on the market.”
“They bias the information available to physicians, hospitals, policymakers, and insurers and thereby corrupt the practice of medicine.” ?
(Tomado de “Conflicts of Interest, Institutional Corruption, and Pharma: An Agenda for Reform” by Marc Rodwin “). Lo interesante es que está ampliamente fundamentado y con una bibliografía extensiva.
Lo dicen investigadores del Edmond J. Safra Center for Ethics Research Lab de la Universidad de Harvard. Lessig, Miller, Rodwin, Light, y unos cuantos más. Comentan que su trabajo incluye, entre otras cosas, el Special Issue on Conflicts of Interest in Medicine Vol. 40, Issue 3 of The Journal of Law, Medicine & Ethics , dedicated to exploring conflicts of interest in medicine from a variety of fresh and powerful perspectives. Articles contributed by Lab Fellows and Faculty Associates include the “Introduction: Insights from a National Conference” by Aaron Kesselheim and David Orentlicher; “Effect of Financial Relationships on the Behaviors of Health Care Professionals: A Review of the Evidence” by Christopher Robertson, Susannah Rose and Aaron Kesselheim; “Conflicts of Interest and Your Physician: Psychological Processes That Cause Unexpected Changes in Behavior” by Sunita Sah; and “Conflicts of Interest, Institutional Corruption, and Pharma: An Agenda for Reform” by Marc Rodwin.
Furthermore, Professor Rodwin is currently editing a Special Issue of JLME on Institutional Corruption and Pharmaceutical Policy, scheduled for publication next year.
Entonces, le sugerimos a la gente que trabaja para las CROs, los diversos “Comités de Ética Independientes”, y a las asociaciones que apoyan/se apoyan en la industria farmacéutica que publiquen. Que publiquen papers, plenos de ejemplos concretos que desmientan las afirmaciones que hacemos, y que parece que más de uno comparte. Este blog está abierto para publicar sus refutaciones científicas, basadas en la evidencia.
Alguien que habla claro sobre el conflicto de intereses básicamente inherente a la industria farmacéutica, cuyo objetivo primario (el lucro), no es precisamente la búsqueda de conocimiento para el beneficio común. Y esto corrompe la investigación, los tratamientos, la medicina, y las instituciones, llegando hasta la OMS. Los dos trabajos de Rodwin son excelentes y se pueden bajar gratis en texto completo de SSRN, que es un portal recomendable.
Drug manufacturers face a fundamental conflict of interest. Pursuit of profit compromises their impartial assessment of their drugs’ benefits and risks. 1 Their biased evaluation can corrupt public knowledge of drugs, lead to marketing unsafe and/or ineffective drugs, and undermine rational physician prescribing. 2 Over the last century, federal regulation has mitigated but not eliminated this problem, which corrupts drug therapy and
medical practice. 3
This article explores a reform proposal that precludes bias in clinical trials used to test drugs. Simply stated, it removes all drug firm influence on the design and conduct of clinical trials used to decide whether to allow marketing of a drug. Recently advocated by several leaders in drug policy and research, this reform proposal has a long history, but was neglected for over half a century due to pharmaceutical industry opposition, and so the federal government pursued alternative strategies to counter bias, which proved ineffective. When regulation of manufacturer controlled clinical trials failed to eliminate bias, flawed practice, and fraud, public officials
increased oversight relying on the same flawed approach.
The corruption lies at the root: drug firm control over clinical trials, and so to be effective, reforms need to eliminate this problem.
Conflicts of Interest, Institutional Corruption, and Pharma: An Agenda for Reform
Marc A. Rodwin
Suffolk University Law School
October 16, 2012
Journal of Law, Medicine and Ethics, Vol. 40, p. 511, 2012
Suffolk University Law School Research Paper No. 12-40
El texto en rojo, negritas y subrayado ha sido modificado por mi, el estilo académico de Rodwin es impecable, y jamás haría eso.
Gorik Ooms a, Claire Brolan b, Natalie Eggermont a, Asbjørn Eide c, Walter Flores d, Lisa Forman e, Eric A Friedman f, Thomas Gebauer g, Lawrence O Gostin f, Peter S Hill b, Sameera Hussain h, Martin McKee i, Moses Mulumba j, Faraz Siddiqui f, Devi Sridhar k, Luc Van Leemput a, Attiya Waris l & Albrecht Jahn m
European Commissioner for Development Andris Piebalgs recently pointed out the need for “updated and modernised [Millennium Development Goals], providing decent living standards for all – a set of minimum floors below which no one should fall”.1 He added that “these ‘MDGs plus’ would provide the basic rights that every citizen on the planet should expect … with, where necessary, for the poorest countries, the support of the international community through continued overseas development assistance”.1
We concur with Commissioner Piebalgs’ demand for basic rights for all people and feel that the right to health and its imperative of narrowing health inequities should be central to the post-2015 international health agenda. We take this stand as members of Go4Health, a consortium of academics and members of civil society tasked with advising the European Commission on the international health-related goals to follow the Millennium Development Goals (MDGs). What does this mean, given that the present MDGs on maternal health, child health and infectious disease control will probably be succeeded by the goal of universal health coverage, defined by the World Health Organization (WHO) as universal coverage with needed health services and financial risk protection?2,3
First, we view an aggregate health goal such as universal health coverage as an improvement over the current set of disparate goals. Ensuring the right to health requires a comprehensive approach. Universal health coverage anchored in the right to health, while building on efforts to meet the present health-related MDGs, would raise the bar for improving health care overall.
Second, although we support making universal health care one of the post-2015 development goals, we feel that universal health coverage is not enough, as defined by WHO and typically conceived,3 to ensure the right to health. For the right to health to become a reality, policy-makers must strive for a healthy physical and social environment (e.g. safe drinking water and good sanitation, adequate nutrition and housing, safe and healthy occupational and environmental conditions and gender equality.)4 These “underlying determinants of health” are partially captured in the present MDGs and their corresponding targets, although under different goals (e.g. nutrition under MDG 1, to eradicate extreme hunger and poverty, and water and sanitation under MDG 7, to promote environmental sustainability). These determinants and many more that are needed for a sustainable healthy environment should figure prominently in the post-2015 health agenda.
Third, specifying people’s entitlements is necessary but not enough. One important reason for the failure to attain all MDGs is the ambiguity of the “shared responsibility” mentioned in Article 2 of the Millennium Declaration: “We recognize that, in addition to our separate responsibilities to our individual societies, we have a collective responsibility to uphold the principles of human dignity, equality and equity at the global level.”5 If we want “every citizen on the planet” to claim his or her right to health, the post-2015 health agenda must specify how citizens will participate in the decision-making processes surrounding their health services and their physical and social environment. Furthermore, the agenda should also explicitly describe the accountability mechanisms that will make it possible for people to claim – not beg for – additional national public resources and international assistance, if needed.
Finally, we are concerned not just about the substance of the post-2015 health goals, but also about the process of formulating them. We have entered a post-2015 frenzy, as evidenced by the appointment of a high-level panel expected to submit a report to the United Nations Secretary-General in the first half of 2013.6 Go4Health is committed to ensuring that any post-2015 health development goals are articulated in collaboration with the communities whose health is at stake. However, truly participatory consultations take time and require a continuing relationship among researchers, governments and those communities. Such an approach should be adopted to prevent goals from being formulated by policy elites after token and superficial consultations, which would be at odds with the rights that must underpin the new goals.
The PLOS Medicine Editors (2013) Getting More Generous with the Truth: Clinical Trial Reporting in 2013 and Beyond. PLoS Med 10(1): e1001379.
“The pharmaceutical industry is at a critical point in its relationship to society. Much of the early promise of the pharmaceutical industry in revolutionizing health care has not continued, with pipelines for innovative drugs drying up (including in critical areas such as neglected diseases ), an increasing number of me-too drugs flooding the market, and concern over spiraling drug costs. Trust has become an even more fundamental issue, in that many who study the output of pharmaceutical companies simply do not believe there is complete and accurate representation of the data on which study reports of clinical trials are based.”
Recomendable para leer entero. El trabajo al que se refiere está en una entrada anterior del blog.
Esta estrategia comercial se ve en la práctica con la judicialización de las demandas de pacientes por acceso a estos medicamentos en cumplimiento del indiscutible derecho a la salud. Sin embargo, debería haber un amplio debate social al respecto, ya que en algunas situaciones esto puede generar un aumento de la inequidad del sistema (ver Ferraz en Health&Human Rights. Como se puede ver en el artículo del NYT abajo, las farmacéuticas sostienen e impulsan las asociaciones de pacientes.
BEDMINSTER, N.J. — Drug companies are fond of saying that every patient counts, but in the world of orphan diseases, entire business plans are built around the idea.
That is why Andrew E. Jablonski, a 26-year-old from Lincoln, Neb., was a guest of honor this month at NPS Pharmaceuticals, a small New Jersey company that is about to begin selling Gattex, its only approved drug. Mr. Jablonski, who was born missing most of his intestines and has a condition known as short bowel syndrome, is central to the company’s mission to win over the tiny pool of patients — fewer than 5,000 nationwide — that the drug is intended to treat.
Over the last two years, NPS has carefully tended its relationship with Mr. Jablonski, helping to finance his nonprofit, the Short Bowel Syndrome Foundation, and flying company leaders to visit him in Lincoln. On Jan. 15, he met with the chief executive and strategized with marketing employees about how to promote Gattex to his social network of 1,000 patients and caregivers.
“It’s a nice, close relationship,” Mr. Jablonski said. “I’m an asset to them, as they are an asset to me.”
NPS hopes that Gattex will ultimately bring in hundreds of millions of dollars in annual sales and help establish the company in the increasingly hot orphan drug market, where companies can charge premium prices to treat overlooked diseases with little or no competition. NPS expects to charge about $300,000 a year for Gattex, a breathtaking price tag that is nonetheless on par with other ultra-orphan drugs on the market.
Development of these drugs is increasingly attractive to pharmaceutical companies, which are searching for new sources of revenue as sales of more traditional, mass-market drugs have been lost to generic competition. The orphan drug market was worth more than $50 billion in 2011 and turns out blockbusters at the same rate as the broader industry, according to a recent Thomson Reuters analysis. Pfizer opened a rare disease unit in 2010, and in 2011, Sanofi acquired the biotech company Genzyme, considered one of the pioneers of the orphan drug industry.
As orphan drugs have become increasingly popular, companies have honed their marketing techniques, knowing that they cannot afford to lose a single patient. Following in the footsteps of drug makers like Vertex, which makes the cystic fibrosis drug Kalydeco, NPS has formed close bonds with advocates like Mr. Jablonski and hired so-called patient care coordinators who will do everything from making sure that Gattex is delivered to patients’ doorsteps to writing appeal letters if an insurer refuses to pay for it.
To be credible, published research must present an unbiased, transparent, and accurate description of the study methods and findings so that readers can assess all relevant information to make informed decisions about the impact of any conclusions. Therefore, research publications should conform to universally adopted guidelines and checklists. Studies to establish whether a treatment is effective, termed randomized controlled trials (RCTs), are checked against a comprehensive set of guidelines: The robustness of trial protocols are measured through the Standard Protocol Items for Randomized Trials (SPIRIT), and the Consolidated Standards of Reporting Trials (CONSORT) statement (which was constructed and agreed by a meeting of journal editors in 1996, and has been updated over the years) includes a 25-point checklist that covers all of the key points in reporting RCTs.
Although the CONSORT statement has helped improve transparency in the reporting of the methods and findings from RCTs, the statement does not define how certain types of analyses should be conducted and which patients should be included in the analyses, for example, in an intention-to-treat analysis (in which all participants are included in the data analysis of the group to which they were assigned, whether or not they completed the intervention given to the group). So in this study, the researchers used internal company documents released in the course of litigation against the pharmaceutical company Pfizer regarding the drug gabapentin, to compare between the internal and published reports the reporting of the numbers of participants, the description of the types of analyses, and the definitions of each type of analysis. The reports involved studies of gabapentin used for medical reasons not approved for marketing by the US Food and Drug Administration, known as “off-label” uses.
The researchers identified trials sponsored by Pfizer relating to four off-label uses of gabapentin and examined the internal company protocols, statistical analysis plans, research reports, and the main publications related to each trial. The researchers then compared the numbers of participants randomized and analyzed for the main (primary) outcome and the type of analysis for efficacy and safety in both the internal research report and the trial publication. The researchers identified 21 trials, 11 of which were published RCTs that had the associated documents necessary for comparison.
The researchers found that in three out of ten trials there were differences in the internal research report and the main publication regarding the number of randomized participants. Furthermore, in six out of ten trials, the researchers were unable to compare the internal research report with the main publication for the number of participants analyzed for efficacy, because the research report either did not describe the primary outcome or did not describe the type of analysis. Overall, the researchers found that seven different types of efficacy analyses were described in the protocols, statistical analysis plans, and publications, including intention-to-treat analysis. However, the protocol or publication used six different descriptions for the intention-to-treat analysis, resulting in several important differences between the internal and published documents about the number of patients included in the analysis.
These findings from a sample of industry-sponsored trials on the off-label use of gabapentin suggest that when compared to the internal research reports, the trial publications did not always accurately reflect what was actually done in the trial. Therefore, the trial publication could not be considered to be an accurate and transparent record of the numbers of participants randomized and analyzed for efficacy. These findings support the need for further revisions of the CONSORT statement, such as including explicit statements about the criteria used to define each type of analysis and the numbers of participants excluded from each type of analysis. Further guidance is also needed to ensure consistent terminology for types of analysis. Of course, these revisions will improve reporting only if authors and journals adhere to them. These findings also highlight the need for all individual patient data to be made accessible to readers of the published article.