Trece medicamentos que cambiarán paradigmas terapéuticos (II)

 

Trece medicamentos que cambiarán paradigmas terapéuticos

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Trece medicamentos que cambiarán paradigmas terapéuticos (I)

Clarivate selecciona terapias disruptivas y con potencial superventas entre las novedades más recientes.

EDICIÓN CRISPR/CAS9

Exagamglogene autotemcel (exa-cel, de Vertex y CRISPR Therapeutics), para anemia falciforme y beta talasemia, consiguió en 2023 el hito de convertirse en la primera terapia de edición génica con CRISPR/Cas9 aprobada por las agencias.

Pero además de por su tecnología, tanto exa-cel como lovotibeglogene autotemcel (lovo-cel, de Bluebird), también para anemia falciforme, están despertando mucha expectación ya que pueden suponer la cura funcional de estas enfermedades, muy discapacitantes y con opciones limitadas de tratamiento sintomático.

También en el informe aparecen las primeras vacunas para el virus respiratorio sincitial (VRS): Abrysvo, de Pfizer, y Arexvy, de GSK. La consultora resalta la alta morbilidad que provoca este patógeno entre los bebés y los adultos mayores, y la capacidad de protección que han mostrado las vacunas.

El nuevo medicamento para la EPOC ensifentrina, un inhibidor dual de la fosfodiesterasa (PDE) 3 y 4 inhalado, se espera que reduzca las exacerbaciones sin los efectos secundarios sistémicos de los inhibidores de la PDE orales. Si se aprueba sería el primer medicamento con un nuevo mecanismo de acción para esta enfermedad en diez años.

OPCIONES MEJORADAS

Efanesoctocog alfa (Sanofi y SOBI), para hemofilia A, es la primera terapia de infusión intravenosa de reemplazo del factor VIII que se puede infundir una vez a la semana o cada 10 días, lo que supone un avance importante para la calidad de los pacientes con respecto a la infusión diaria o cada dos días. La consultora apunta que puede representar una opción atractiva para los pacientes reacios a recibir terapias novedosas, como anticuerpos monoclonales o terapia génica.

Otro avance en la dosificación del tratamiento es la versión mejorada de Eylea para la forma húmeda de la DMAE. La dosis más alta de aflibercept de Eylea HD (Bayer y Regeneron) permite espaciar su administración cada 12-16 semanas, frente a las 8 del anterior Eylea, y las 4 de Lucentis (Genentech). Una cuestión muy importante para la aceptación de los pacientes, ya que son inyecciones intravítreas.

Nefecon es la formulación patentada de liberación retardada de budenosida oral de Calliditas, Everest Medicines y Stada para nefropatía por IgA. Frente a los corticoides convencionales, ha demostrado una mayor eficacia en la reducción de la proteinuria y en la progresión de la insuficiencia renal, así como un perfil de seguridad muy superior. Clarivate prevé que su penetración en el mercado sea rápida en pacientes de alto riesgo.

Completa el listado de trece mirikizumab, el anticuerpo monoclonal de Lilly dirigido a la subunidad p19 de IL23, primero de su clase para colitis ulcerosa. Los retrasos en su lanzamiento por problemas de fabricación en Estados Unidos han hecho que este medicamento, que ya aparecía en el informe del año pasado, repita en el de este año. Destaca su perfil de eficacia y seguridad, y sería una opción especialmente atractiva para pacientes intolerantes o resistentes a los anti-TNF.

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De_ROCHE a BAYER..."es bueno" (I)

 

Bayer AG has announced the appointment of Bill Anderson as its new chief executive officer, signalling the exit of current CEO Werner Baumann, who has served the company for 35 years.

Anderson, a chemical engineer by education, brings a strong pharma background to his new role, having held various leadership positions in the life sciences industry over the past 25 years.

Most recently, he served as CEO of Roche’s pharmaceuticals division, where he led a comprehensive transformation programme resulting in a number of new product launches, significant revenue growth and greater productivity across the organisation.

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Novartis de_Roche de acciones...

Prior to his role at Roche, Anderson was chief executive officer of Genentech, and has held several senior leadership positions in general management, product development and finance at Biogen and Raychem, an American technology and electronics company.

During his time in these roles, Anderson was involved in the development and launch of 25 new medicines, including 15 blockbusters.

Anderson’s appointment was unanimously backed by the company’s board after a selection process that began in mid-2022, with chairman Professor Dr Norbert Winkeljohann describing Anderson as the “the ideal candidate to lead Bayer together with the team into a new, successful chapter at a time of a disruptive innovation cycle in biology, chemistry and artificial intelligence”.

“Bill has an outstanding track record of building strong product pipelines and turning biotech breakthroughs into products. Beyond that, he is a truly transformational leader who creates a culture that propels innovation, boosts productivity and performance, and allows people to thrive,” Winkeljohann added.

Commenting on his new role, Anderson said: “[Bayer’s] leading R&D investments in agriculture, medicines and consumer health hold the promise for additional breakthroughs. I look forward to working with the people of Bayer to accelerate innovation, increase performance, advance sustainability and unleash the full potential of the company.”

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Alzheimer: No Clinical Benefit From Drug Targeting Amyloid (Aduhelm)

 

A recent review article in the Journal of Alzheimer Disease identified 9 compounds targeting amyloid that had failed in phase 3 trials since 2018.

In January 2019, Roche, Genentech’s parent company, announced it was discontinuing 2 phase 3 trials of crenezumab in patients with mild, sporadic Alzheimer disease because preplanned interim analyses concluded that the treatment was unlikely to meet its primary end point of slowing cognitive decline.

And a year before the findings of the Colombia crenezumab trial were reported, the US Food and Drug Administration (FDA) approved aducanumab (Aduhelm), another antiamyloid monoclonal antibody, even though none of the agency’s panel of outside experts had voted “yes” when asked whether clinical trials had shown it to be effective in treating Alzheimer disease.

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Todo sobre Aduhelm en PHARMACOSERÍAS

The agency granted aducanumab “accelerated approval,” which is based on a surrogate end point—in this case, a reduction in amyloid-β plaque in the brain—that, according to the FDA, “is reasonably likely to predict a clinical benefit to patients.” Aducanumab, the only antiamyloid drug that has ever received FDA approval, is the first Alzheimer therapy designed to modify the underlying disease process and not just treat symptoms.

After the FDA approved aducanumab, the Centers for Medicare & Medicaid Services (CMS) released a national coverage policy for the therapy as well as future antiamyloid antibodies to spur the collection of more information about their safety and effectiveness.

Medicare will cover aducanumab and other such therapies granted accelerated approval based on a surrogate end point, such as amyloid reduction, only for beneficiaries participating in FDA-sanctioned randomized clinical trials to determine clinical effectiveness. Aducanumab isn’t cheap. In January, Biogen cut the drug’s wholesale acquisition cost in half, to $28 200 per year for an average-size patient (dosing is based on patients’ weight).

For monoclonal antibodies that receive FDA approval via the conventional pathway, which involves demonstrating clinical benefit, Medicare will still only cover the cost for patients in CMS-approved or NIH-supported studies.

.../...

No matter their opinion about the usefulness of targeting amyloid, dementia experts seem to agree that the complexity of Alzheimer disease calls for a multipronged treatment approach tailored to the particular patient, depending on such factors as the stage of their disease.

“I have a hard time imagining how targeting any single molecule or any single pathology is likely to yield a meaningful clinical benefit,” Gandy said.

 (Sam Gandy, MD, PhD, director of the Mount Sinai Center for Cognitive Health in New York)

Although the final answer on amyloid may not yet be in, researchers are branching out in their search for tools—both drugs and lifestyle changes—that could at least slow the development or progression of Alzheimer disease.

“A decade ago, there wasn’t much in terms of alternative targets” to amyloid, Hodes said. To illustrate his point, he noted that 5 of the 8 late-stage Alzheimer trials funded by the NIA involve antiamyloid therapies. However, Hodes said, only 13 of the 61 phase 1 or phase 2 trials receiving NIA funding target amyloid.

 (Richard Hodes, MD, director of the National Institute on Aging (NIA))

Nonamyloid therapeutic targets include other proteins, such as tau—tau neurofibrillary tangles are a hallmark of Alzheimer disease that haven’t received as much attention as amyloid plaques—TDP-43 (transactive response DNA-binding protein 43), the accumulation of which in the central nervous system is also a feature of other neurodegenerative diseases; and α-synuclein, which appears to interact with tau in neurodegenerative diseases, Hodes said. Besides proteins, other Alzheimer therapeutic targets for which the NIA is funding trials include inflammation, genetics, and vascular system changes, he said.

The NIA is also supporting 131 studies of nonpharmacological interventions focused on cognitive training, sleep, and exercise, among others, Hodes said. One NIA-funded phase 3 trial presented at the Alzheimer’s Association conference evaluated whether regular exercise could benefit people with amnesic mild cognitive impairment (MCI), which primarily affects memory and increases the risk of Alzheimer disease or related dementias.

The trial randomized 296 adults to either moderate-intensity aerobic training or low-intensity stretching, balance, and range-of-motion exercises for 18 months.

Exercise sessions took place at a YMCA 4 times a week for a total of 120 minutes to 150 minutes per week. In the first 12 months, a trainer supervised 2 sessions a week, while the other 2 were unsupervised. All exercise was unsupervised in the last 6 months. Neither group showed significant declines from baseline in the primary measure of cognitive function over 12 months, suggesting that both the moderate- and low-intensity exercise, and, possibly, the socialization participants received with it, stalled cognitive decline, researchers reported at the meeting. In contrast, cognitive function did decline over a year in similar adults with MCI who participated in a large “usual care” observational study.

Approximately 6.5 million people aged 65 years or older in the US are living with Alzheimer disease, and that number is expected to nearly double by 2050, according to the Alzheimer’s Association.

”The stakes are too high just to focus on amyloid,” Reiman said.

(Eric Reiman, executive director of the Banner Alzheimer Institute in Phoenix)

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Reputation Institute’s 2018 US Pharma RepTrak

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The Largest Study on Reputation of Pharmaceutical Brands in the US

BOSTON — June 14, 2018 — Reputation Institute (RI), the world’s leading provider of reputation measurement, monitoring and management services, today announced the company’s annual US Pharma RepTrak® rankings. Based on more than 2,608 individual ratings of pharmaceutical companies during the first quarter of 2018, the survey quantifies the emotional bond stakeholders have with pharma companies in the US, and how these connections drive supportive behavior such as the willingness to purchase a company’s products, recommend the brand, invest in or work for a company.(Más)

1. Sanofi. RepTrak Points: 74.6 
Sanofi's winning characteristics lies in its promotion of ethics and transparency, according to Reputation Institute. Sanofi has in the past year promised tolimit price increases and disclose "transparency reports" behind overall costs of its drugs. 

2. Genentech. RepTrak Points: 74.0 
A subsidary of Roche, Genentech is investing in a new way to treat Alzheimer's. It also recently made a $534 million deal with Microbiotica to research gut bacteria in hopes of developing new treatments for inflammatory bowel disease. 

3. Celgene. RepTrak Points: 72.5 
Celgene specializes in drugs for cancer and inflammatory diseases, known for its treatment of multiple myeloma. However, in February, the company hit a major setbackwith its multiple sclerosis drug. 

4. AbbVie. RepTrak Points: 72.3 
 The Humira-maker spun out of of Abbott Laboratories back in 2013. Recently, its rheumatoid arthritis drug successfully completed a late-stage trial.  
AbbVie was ranked first in last year's list, but has fallen back a few spots this year.

Ver:

Pharma RepTrak 2017: Pharma Reputation 2014-2017 (cont.) Top companies

5. Biogen. RepTrak Points: 72.0 
The company specializes in treatments for neurodegenerative, blood-based, and autoimmune diseases. Althought it hasn't made many major moves this past year, it's still a major playerwhen it comes to its neuroscience pipeline. Early this year, it sold its hemophilia drug maker company, Bioverativ, to Sanofi for $11.6 billion. 

6. Bayer. RepTrak Points: 70.7 
Best known for making aspirin, the German company is also throwing its hat in the ring to bet on gene therapies. In June, Bayer finalized its $66 billion merger with agriculture giant Monsanto, which has farmers worried. 

7. Gilead. RepTrak Points: 70.1 
Gilead has historically been focused on antiviral drugs to treat diseases like HIV and hepatitis C. But earlier this year, it just made a huge bet on an experimental gene-editing technology for cancer treatments.

8. Allergan. RepTrak Points: 69.8 
The Botox-maker has had a rocky relationship with its investors this year. It's been actively refreshing its board, and just last month, it announced its plan to sell offtwo of its non-core businesses to appease investors. In June, the company presented positive data in a glaucoma candidate and meet goals with its oral migraine drug. 

9. Novo Nordisk. RepTrak Points: 68.8 
The Danish company is known as a diabetes drug giant. 
The turbulent US market forced it tolay off 3,000 people and axe its long term growth plan. 

10. McKesson. RepTrak Points: 68.7 
McKesson distributes not only drugs, but also health IT, medical supplies and care management tools. In June, the company reduced the CEO's pay by 10% after investors revolted. 

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A Cancer Conundrum: Too Many Drug Trials, Too Few Patients / Dónde están los pacientes...?

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With the arrival of two revolutionary treatment strategies, immunotherapy and personalized medicine, cancer researchers have found new hope — and a problem that is perhaps unprecedented in medical research.

There are too many experimental cancer drugs in too many clinical trials, and not enough patients to test them on.

The logjam is caused partly by companies hoping to rush profitable new cancer drugs to market, and partly by the nature of these therapies, which can be spectacularly effective but only in select patients.

In July, an expert panel of the Food and Drug Administration recommended approval of a groundbreaking new leukemia treatment, a type of immunotherapy. Companies are scrambling to develop other drugs based on using the immune system itself to attack cancers.

Many of these experimental candidates in trials are quite similar. Yet each drug company wants to have its own proprietary version, seeing a potential windfall if it receives F.D.A. approval.

As a result, there are more than 1,000 immunotherapy trials underway, and the number keeps growing. “It’s hard to imagine we can support more than 1,000 studies,” said Dr. Daniel Chen, a vice president at Genentech, a biotechnology company.

In a commentary in the journal Nature, he and Ira Mellman, also a vice president at the company, wrote that the proliferating trials “have outstripped our progress in understanding the basic underlying science.”

“I think there is a lot of exuberant rush to market,” said Dr. Peter Bach, director of the Center for Health Policy and Outcomes at Memorial Sloan Kettering Cancer Center. “And we are squandering our most precious resource — patients.”


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Pharma execs popularity...

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Sometimes adult life sends us through a disturbing time warp. It’s much too reminiscent of high school. The cool kids’ cafeteria table (or parking lot, where I grew up) becomes the cool grown-up party at J.P. Morgan, for instance. Step into biopharma Twitter, where the snark level sometimes hits Mean Girls levels. 

But today, it’s the “best all-around” votes senior year echoed in post-high school reality. Only instead of the most popular graduating senior, these votes chose the most congenial CEOs in biopharma. 

The business-information analysts over at Owler sifted through various data streams, including its own CEO rating tool (smile, frown or in-between) to come up with a ranking of the best-liked pharma leaders. The top 10 is mostly packed with Big Pharma’s top dogs, with a couple of surprises in the mix. 

Including No. 1, Sanjay Bhatia of Laborate Pharmaceuticals, a small, privately held drugmaker in Delhi, India. His approval rating with Owler’s tool? A stunning 99 out of 100. And it’s not because he has 100 employees who all voted with a smile. Laborate has an estimated 5,180 staffers and 1,980 followers on the Owler site. 

Roche and its Genentech unit swept second and third, with Roche’s Severin Schwan in second place with a 92.9 approval rating. 
Bill Anderson at Genentech clocked an 87.7.

Switzerland-based Roche bought the share of California-based Genentech it didn’t already own back in 2009, and the Genentech side of the business has delivered some of Roche’s biggest-selling drugs in the last few years. 

AbbVie’s Richard Gonzalez comes in fourth with an 83.8 approval rating—he’s been at the helm at that company since it was spun off by Abbott Laboratories in 2013. He’s consistently among the highest-paid CEOs in the industry, and while popularity among staff isn’t among his compensation metrics, driving “employee engagement” is. 

Rounding out the top five is Shire’s Flemming Ornskov, whose most recent claim to fame is the company’s buyout of Bioverativ, the hemophilia unit spun off by Baxter last year. 

 The rest of the top 10? 
Merck’s Ken Frazier; GlaxoSmithKline’s Emma Walmsley, who’s brand-new to the Glaxo CEO post; Johnson & Johnson’s Alex Gorsky; and Bayer’s relatively new chief, Werner Baumann. Steven H. Collis, CEO of the drug distributor AmerisourceBergen, came in seventh. All of their ratings fell in the 70s. (Más)

Críticas al meeting Pharma & President Trump (I): Drug approvals / David Lazarus*

At a recent meeting with pharmaceutical-industry bigwigs, President Trump declared that “we’re going to be cutting regulations at a level that nobody’s ever seen before.” 
He also said that “we’re going to have tremendous protection for the people.” 

It’s hard to see how he can do both.

Ver:

Precios: Trump se reune con "top pharma execs".

As with Trump’s proposed elimination of consumer safeguards, environmental-protection measures and financial reforms, the reality is that if his administration proceeds with a wholesale deregulation of the drug industry, the public will be largely undefended against the aggressive and potentially dangerous predations of multibillion-dollar conglomerates. 

“We need regulations to make sure first and foremost that there’s drug safety and efficacy,” said Michael Santoro, a management professor at Santa Clara University and co-editor of the book “Ethics and the Pharmaceutical Industry.” 

What Trump is basically proposing is less testing of drugs before they’re made available to patients. 

“The pharmaceutical industry is a business and wants to bring drugs to the market quickly,” Santoro said. “That’s why you need the Food and Drug Administration to look at the medical and scientific evidence to make sure that speeding up the process doesn’t harm the public.” 

In some cases, experts say, faster drug approvals may be warranted. It now can take years for a new drug to obtain the FDA’s go-ahead. For a terminally ill cancer patient, say, that can be a death sentence. 

Sam Peltzman, a professor emeritus of economics at the University of Chicago, noted that the more testing that’s conducted, “the more stuff that can come to light that can prevent harm.” But he said “anything that speeds up the drug-approval process will have net positive effects.”That remains to be seen. My colleague Melody Petersen reported on a $7,800-a-month cancer drug called Tarceva. Under pressure from Genentech and patient advocates, the FDA expanded use of the drug despite little evidence it worked. Patients suffered as a result. 

The trick is finding the right balance of speed and safety, and it’s fair to wait and see what the Trump administration comes up with. He hasn’t yet even named an FDA commissioner, except to say that he has a “fantastic person” in mind. 

Another change sought by drugmakers, however, is intended solely to boost profits. It would greatly expand the industry’s ability to pitch prescription meds directly to consumers. 

 This is a terrible idea. (Más)

(*) David Lazarus is an American business and consumer columnist for the Los Angeles Times

Y si en 2017 quieres un nuevo empleo...

Tan solo J&J (26º) y Genentech (32º) aparecen en el Glassdoor Top 50
  
If you're looking for a new job, here's a good place to start. 

 Career website Glassdoor Inc. has released its annual report on the best places to work based on employees who elected to anonymously submit a company review on its website over a 12-month time frame. This year's winner, Bain & Company Inc., has been in the top five since the list's inception in 2009 and has been No. 1 three times. 

Employees cite a great salary, benefits, mentorship, and a culture that promotes employee engagement, according to Scott Dobroski, Glassdoor community expert. .(Más)

Creatividad: MM&M Awards 2016/Best Professional Sales Tool

GOLD 
Havas Life Metro and Genentech 
Interactive MOA (Gazyva) 

Selling to oncologists is tough. Many of them are weary of personal visits from sales representatives and their sales aids. To break through and drive a high level of physician interaction, this team created an interactive self-guided digital storytelling experience to explain the unique mechanism of action of Gazyva. 

The Gazyva mechanism of action animation requires the viewing doctor to make some sort of hand movement on an iPad to move the story forward. For example, when the doctor physically traces the path of the antibody therapeutic, it triggers a reaction against a cancer cell in the animation. Swiping and other gestures activate visual and auditory reward mechanisms once certain operations are completed. (Ver)

“A rich, immersive approach to MOA articulation,” one judge noted. “The self-guided storytelling and interactivity are quite impressive.” 

Side-by-side views differentiate Gazyva features from its competitors'. An agency representative noted that story nuances that might otherwise go unnoticed were more easily integrated into the narrative because the user is immersed in the visually appealing and interactive world of the therapeutic. 

 Metrics were unavailable at submission date, but the team reported positive response from sales reps, as well as oncologists and their staffs.

SILVER  
Partners+simons and B. Braun Medical 
Secondhand Chemo 

A complete presentation platform overhaul resulted in a 36% increase in 2015 sales of the OnGuard Closed System Drug Transfer Device. Improvements included real-time customization, interactive and simplified digital assets, and features that significantly reduce the administrative burden on sales reps. Judges applauded the aid's content, interactivity, and closed-loop functionality.

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Creatividad: MM&M Awards 2016/Best Professional Print Campaign

Creatividad: MM&M Awards 2016/Best Multicultural Campaign

GOLD 
HealthWork (powered by BBDO and CDMi) and Genentech 
Sister Pact 

After The New York Times reported that a disproportionately high percentage of African-American women in Memphis die from breast cancer, Genentech made it a priority to help. 

 The core campaign concept — making a pact with a friend to get a mammogram — taps into existing trusted relationships to drive both awareness and participation. Local influencers helped reach out to the targeted audience of African-American women aged 35 to 60. 

 The campaign creative features local women and their friends realistically filmed together in their hometown environment. Execution included ads (digital, print, broadcast, and out of home), local events, a call center, and Sisterpact.com.

SILVER 
XL Alliance and Valeant Pharmaceuticals 
 No More Hair, More You (Clear+Brilliant Pélo Laser) 

The concept of ­shedding unwanted hair and labels was used to introduce millennials and the LGBTQ community to the Clear+Brilliant Pélo hair-removal laser. 
The campaign, which features various humorous and arresting hairy elements, helped drive more than $1 million in sales in four months. Judges found the effort smart and funny. (Ver)

Ver anterior: 
Creatividad: MM&M Awards 2016/Best Professional Sales Tool

TAPUR: Investigación farmacéutica de ASCO...

AINHOA IRIBERRI
Madrid Actualizado:02/06/2015

La principal asociación de médicos especialistas en cáncer del mundo, la Sociedad Americana de Oncología Clínica (ASCO), dio este lunes una patada al modelo tradicional de investigación sobre nuevos tratamientos frente al cáncer y anunció la puesta en marcha del primer ensayo clínico global para la búsqueda de nuevos datos de la llamada medicina personalizada o de precisión, como la bautizó el presidente estadounidense Barack Obama el pasado mes de enero.

The announcement came at the annual meeting of the American Society of Clinical Oncology, which is launching a similar study called TAPUR, in which five pharmaceutical companies so far have agreed to provide a total of 13 genetically targeted drugs already on the market. The intent is to determine “what happens to patients who have their tumors sequenced and are treated with a targeted drug” that isn’t specifically approved for their cancer, said Richard L. Schilsky, ASCO’s chief medical officer.  (Más)

El estudio TAPUR (siglas en inglés de Registro de Utilización y Utilidad de Agentes Dirigidos) fue presentado en el congreso anual de ASCO por la plana mayor de la organización y quiere dotar de oficialidad y utilidad a una práctica común en los hospitales de todo el mundo: el uso fuera de indicación de fármacos dirigidos a una mutación específica. 

En la actualidad, cuando se descubre uno de estos fármacos personalizados se sigue un proceso estandarizado: las autoridades sanitarias lo aprueban para la indicación para la que ha resultado eficaz. 

Peter Paul Yu

En paralelo, es fácil que otros estudios demuestren que la misma mutación presente en el cáncer X (para el que se ha aprobado el fármaco) está también presente en el cáncer Y. Así, los oncólogos más informados podrán probar el fármaco fuera de indicación, aunque a menudo es el paciente el que ha de pagar los carísimos tratamientos. "Los oncólogos usan a menudo terapias aprobadas para una indicación específica para tratar a personas con otros tipos de cáncer avanzados, pero es raro que todos aprendamos de esa experiencia de forma que se beneficien más pacientes", explicó el presidente de ASCO, Peter Paul Yu. 

Al final, los pacientes que se benefician de esos potenciales nuevos usos son una minoría, apenas el 3% de enfermos, los que participan en ensayos clínicos. Y los que dejan de beneficiarse son precisamente los más necesitados, aquellos para los que no existen opciones terapéuticas. 

Ese será justo el perfil del participante de TAPUR, que comenzará a seleccionar pacientes antes de fin de año. Son tres los desafíos a los que pretende responder este estudio: encontrar nuevas formas de probar fármacos según las características moleculares del tumor (y no dónde está situado este); aprender de todos y cada uno de los pacientes y, por último, recopilar datos con nuevas herramientas no utilizadas hasta la fecha. 

Barreras 

Los enfermos que participen en TAPUR podrán vencer varias barreras. La primera, por supuesto, disponer de un tratamiento; la segunda, que se les haga un perfil genómico. Una vez llevado a cabo este análisis y siempre que se detecte una anomalía genética sobre la que algún fármaco ya descubierto actúe, los pacientes superarán un escollo importante: no tendrán problemas para conseguir el medicamento, ya que cinco laboratorios (Astra -Zeneca, Bristol Myers Squibb, Eli Lilly Co., Genentech y Pfizer) se han comprometido con ASCO y aportarán sus fármacos de forma gratuita. 

Richard Schilsky

A partir de ahí son todo expectativas, como recordó el director médico de ASCO, Richard Schilsky. "Hay muchos condicionantes", apuntó. Sobre el papel, parece incontestable que las cosas van a mejorar: los pacientes recibirán moléculas dirigidas que no hubieran recibido antes, los médicos tendrán a su alcance los resultados de los test moleculares, guías sobre recomendaciones terapéuticas según los mismos, acceso a medicamentos sin tener que participar en ensayos específicos de la industria y, sobre todo, datos clínicos sobre los usos fuera de indicación. "Si no funciona, habrá que desarrollar otras estrategias", comentó Schilsky, resaltando la caducidad del modelo tradicional de investigación clínica. 

El estudio TAPUR medirá la respuesta objetiva al tratamiento como primer parámetro, aunque también obtendrá datos sobre supervivencia libre de enfermedad, supervivencia general, duración del tratamiento y toxicidad del mismo. Incluirá a pacientes con cualquier tipo de tumor sólido avanzado, mieloma múltiple o linfoma no Hodgkin de células B para los que no haya tratamientos disponibles. (Más)

Palabras: "budget-busting drugs"

 


"Sovaldi, the first of the so called 

‘budget-busting’ drugs. 

The hepatitis C treatment’s $84,000 list price..."

"budget-busting drugs"

Que revientan presupuestos...

Evaluatepharma Budget-Busters

 Click sobre imagen para ampliar

"Sovaldi and other high-priced drugs such as Lucentis, an eye medication made by Genentech that can cost as much as $2,000 per dose, are part of an expected wave of breakthroughs in new, expensive pharmaceutical treatments for a variety of diseases and chronic conditions." (Ver)

The price of Gilead Sciences' breakthrough drug Sovaldi has drawn all of the attention this year. By pricing it at $85,000 for a 12-week course, Gilead Sciences has been pilloried by politicians, payers and providers. But as EvaluatePharma points out, at that price Sovaldi barely makes it into the top 20 most expensive drugs, number 19 to be exact, just above Novartis' Gleevec and just below GlaxoSmithKline's Mekinist. Sovaldi is pricey, but if you want to talk about really pricey, then look at the 10 most expensive. The least of those, Celgene's Revlimid, ran $128,666 last year, the numbers ninjas at EvaluatePharma determined. The most expensive, Soliris, costs nearly $537,000.(Más)

Ver también:

ROCHE: Con Kadcyla al "Club de los $1.000 day"

Novartis & Roche: De res Aviditatis (Codicia)...Avastin & Lucentis

Huérfano y biotecnológico, perfil de los medicamentos más caros