FDA Approves Orenitram™ (treprostinil) Extended-Release Tablets for the Treatment of Pulmonary Arterial Hypertension

SILVER SPRING, Md., Dec. 20, 2013 /PRNewswire/ -- United Therapeutics Corporation (UTHR) announced today that the United States Food and Drug Administration (FDA) has approved Orenitram (treprostinil) Extended-Release Tablets for the treatment of pulmonary arterial hypertension (PAH) in WHO Group I patients to improve exercise capacity.

"This approval marks the first time that the FDA has approved an orally administered prostacyclin analogue for any disease – and our fifth approval from the FDA for treatment of PAH – supporting our mission of providing a wider choice of PAH therapies for physicians and patients," said Roger Jeffs, Ph.D., United Therapeutics' President and Chief Operating Officer.  "We are grateful for the FDA's thorough review and will continue to build clinical support for the use of Orenitram."

The primary efficacy study, FREEDOM-M, demonstrated that patients receiving Orenitram twice daily (BID) improved their median six-minute walk distance (6MWD) by +23 meters [p=0.013] as compared to patients receiving only placebo.  As the sole vasodilator, the effect of Orenitram on exercise is small and Orenitram has not been shown to add to other vasodilator therapy.  Two other Phase 3 studies (FREEDOM-C and FREEDOM-C²) did not demonstrate a benefit in exercise with median 6MWD at Week 16 (11 meters [p=0.072] and 10 meters [p=0.089], respectively). 

The most common side effects reported in the clinical studies with Orenitram (placebo-corrected incidence >10%) are headache, nausea, and diarrhea.

Orenitram is dosed twice a day with food, but the total daily dose can be divided and given three times daily with food. Orenitram is available in four strengths:  0.125 mg, 0.25 mg, 1 mg and 2.5 mg.  The dose of Orenitram should be increased as tolerated to achieve optimal clinical response.  The maximum dose is determined by tolerability.

Conference Call

United Therapeutics will host a half hour teleconference on December 23, 2013, at 9:00 a.m. Eastern Time. The teleconference is accessible by dialing 1-877-351-5881, with international callers dialing 1-970-315-0533. A rebroadcast of the teleconference will be available for one week and can be accessed by dialing 1-855-859-2056, with international callers dialing 1-404-537-3406, and using conference code: 29125503.

This teleconference is also being webcast and can be accessed via United Therapeutics' website at http://ir.unither.com/events.cfm.

About Orenitram

Orenitram is an extended-release tablet for oral administration used to treat PAH, a life-threatening disease that constricts the flow of blood through the pulmonary vasculature. Orenitram contains the same active ingredient (treprostinil) as Remodulin^® (treprostinil) Injection and Tyvaso^® (treprostinil) Inhalation Solution.

Orenitram is indicated for the treatment of PAH (WHO Group 1) to improve exercise capacity.  The primary study that established efficacy (FREEDOM-M) included predominately patients with WHO functional class II-III symptoms and etiologies of idiopathic or heritable PAH (75%) or PAH associated with connective tissue disease (19%).  When used as the sole vasodilator, the effect of Orenitram on exercise is about 10% of the deficit, and the effect, if any, on a background of another vasodilator is probably less than this.  Orenitram is probably most useful to replace subcutaneous, intravenous, or inhaled treprostinil, but this use has not been studied.

Important Safety Information for Orenitram

• Orenitram is contraindicated for patients with severe hepatic impairment (Child Pugh Class C).
• Abrupt discontinuation or sudden large reductions in dosage of Orenitram may result in worsening of PAH symptoms.
• Orenitram inhibits platelet aggregation and increases the risk of bleeding, particularly among patients receiving anticoagulants.
• Orenitram should not be taken with alcohol as release of treprostinil from the tablet may occur at a faster rate than intended.
• The Orenitram tablet shell does not dissolve.  In patients with diverticulosis (blind-end pouches), Orenitram tablets can lodge in a diverticulum.
• Concomitant administration of Orenitram with diuretics, antihypertensive agents or other vasodilators increases the risk of symptomatic hypotension.
• There is a marked increase in the systemic exposure to treprostinil in hepatically impaired patients.
• Co-administration of Orenitram and the CYP2C8 enzyme inhibitor gemfibrozil increases exposure to treprostinil, therefore, Orenitram dosage reduction may be necessary in these patients.
• In the 12-week placebo-controlled monotherapy study, adverse reactions with rates at least 5% higher on Orenitram than on placebo included headache, diarrhea, nausea, flushing, pain in jaw, pain in extremity, hypokalemia, and abdominal discomfort.

For full patient information and full prescribing information, visit:
http://www.unither.com/assets/unither/docs/OrenitramFullPrescribingInformation.PDF.

About United Therapeutics

United Therapeutics Corporation is a biotechnology company focused on the development and commercialization of unique products to address the unmet medical needs of patients with chronic and life-threatening conditions.

Forward-looking Statements

Statements included in this press release that are not historical in nature are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995.  Forward-looking statements include, among others, our intention to continue to build clinical support for the use of Orenitram.  These forward-looking statements are subject to certain risks and uncertainties, such as those described in our periodic reports filed with the Securities and Exchange Commission, that could cause actual results to differ materially from anticipated results.  Consequently, such forward-looking statements are qualified by the cautionary statements, cautionary language and risk factors set forth in our periodic reports and documents filed with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K.   We claim the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements.  We are providing this information as of December 20, 2013, and assume no obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason. [uthr-g]

ORENITRAM is a trademark of United Therapeutics Corporation.

REMODULIN and TYVASO are registered trademarks of United Therapeutics Corporation.

Takeda Announces Termination of Fasiglifam (TAK-875) Development

December 26, 2013 06:00 PM Eastern Standard Time

OSAKA, Japan--(BUSINESS WIRE)--Takeda Pharmaceutical Company Limited (Takeda) announced today that it has decided voluntarily to terminate the development activities for fasiglifam (TAK-875), an investigational treatment for type 2 diabetes, due to concerns about liver safety.

 

Patient safety is Takeda's highest priority. The company has worked with three independent panels of experts to provide for the safety of trial participants and ensure independent safety oversight for the clinical trials throughout the duration of the fasiglifam (TAK-875) Phase 3 development program.

 

The expert panels include the independent Data Monitoring Committee (DMC), a committee that oversees the fasiglifam global clinical development program, reviews the unblinded clinical data from program trials and provides continual safety oversight of trial subjects and recommendations. The DMC is comprised of clinical experts in endocrinology, cardiology and hepatology as well as a statistician. The independent Liver Safety Evaluation Committee (LSEC) is comprised of five hepatologists with expertise in drug-induced liver injury. While remaining blinded to treatment information, the LSEC regularly evaluates data on liver enzymes elevations and adjudicates cases that impacted the liver. In addition, an independent Executive Committee (EC) provides additional oversight for the fasiglifam (TAK-875) cardiovascular outcomes trial.

 

After careful consideration of the data emerging from all the clinical trials and in consultation with these panels, the company has reached the conclusion that, on balance, the benefits of treating patients with fasiglifam (TAK-875) do not outweigh the potential risks. For this reason, Takeda has decided voluntarily to terminate the development activities for fasiglifam.

 

Takeda is in communication with trial investigators and the relevant regulatory authorities regarding the company's decision, to provide them with updated and current information in compliance with local regulations. Takeda is working with trial investigators and local regulatory authorities to ensure that patients who participated in the fasiglifam (TAK-875) trials are transitioned to appropriate therapies and ensure that trial participants receive appropriate care. Patients enrolled in the fasiglifam (TAK-875) clinical trials are urged to consult their study investigators to address any questions, and before making any changes to their medication. For additional information, please visit www.takeda.com.

 

Takeda remains committed to the development of novel treatments for diabetes, a disease which represents a growing health burden for people worldwide.

 

About Takeda Pharmaceutical Company Limited

 

Located in Osaka, Japan, Takeda is a research-based global company with its main focus on pharmaceuticals. As the largest pharmaceutical company in Japan and one of the global leaders of the industry, Takeda is committed to strive towards better health for people worldwide through leading innovation in medicine. Additional information about Takeda is available through its corporate website, www.takeda.com.

 

This press release contains forward-looking statements. Forward-looking statements include statements regarding Takeda's plans, outlook, strategies, results for the future, and other statements that are not descriptions of historical facts. Forward-looking statements may be identified by the use of forward-looking words such as "may," "believe," "will," "expect," "project," "estimate," "should," "anticipate," "plan," "assume," "continue," "seek," "pro forma," "potential," "target," "forecast," "guidance," "outlook" or "intend" or other similar words or expressions of the negative thereof. Forward-looking statements are based on estimates and assumptions made by management that are believed to be reasonable, though they are inherently uncertain and difficult to predict. Investors are cautioned not to unduly rely on such forward-looking statements.

 

Forward-looking statements involve risks and uncertainties that could cause actual results or experience to differ materially from that expressed or implied by the forward-looking statements. Some of these risks and uncertainties include, but are not limited to, (1) the economic circumstances surrounding Takeda's business, including general economic conditions in Japan, the United States and worldwide; (2) competitive pressures and developments; (3) applicable laws and regulations; (4) the success or failure of product development programs; (5) actions of regulatory authorities and the timing thereof; (6) changes in exchange rates; (7) claims or concerns regarding the safety or efficacy of marketed products or product candidates in development; and (8) integration activities with acquired companies.

 

The forward-looking statements contained in this press release speak only as of the date of this press release, and Takeda undertakes no obligation to revise or update any forward-looking statements to reflect new information, future events or circumstances after the date of the forward-looking statement. If Takeda does update or correct one or more of these statements, investors and others should not conclude that Takeda will make additional updates or corrections.

German Merck is among the companies fishing in Iran for more business

Pharmaceuticals are among the small number of products that Western companies can still sell to Iran under the sanctions program designed to deter its nuclear ambitions. The process, however, is complicated, lengthy and still not without risks. Novartis' ($NVS) Alcon unit acknowledged last year that federal authorities were investigating its sales there. But with the prospect of sanctions easing, drugmakers are sizing up the opportunities, and Merck KGaA and others intend to seize them.

Germany-based Merck ($MRK) told the Financial Times it is talking with Iranian companies about producing its diabetes drug Glucophage and its high-blood-pressure treatment Concor, looking for someone who can meet its quality standards. France-based Sanofi ($SNY), which licenses some cancer meds in Iran and maintains a staff of 170 there, plans to expand there in 2014. "It's a good, solid business," a spokesperson told the publication.

A final deal is not yet in place and not assured. Plenty of resistance remains in Congress to easing sanctions without a full nuclear capitulation from Iran. The provisional deal struck last month would keep the primary restrictions on Iran's oil industry and banking abilities in place, but between $6 billion and $7 billion in assets would be released. Pharmaceuticals, which have been hard for it to come by, is one category that Iran could spend the money on.

But as some drugmakers know, selling to Iran can be complicated, slow and still has risks. According to the Financial Times, Pfizer ($PFE) is still awaiting full payment on about $854,000 worth of drugs sold to Iran in 2011 which got caught up in the complex payment requirements. Novartis filed a special Iran Notice with the SEC last year indicating that its Alcon eye-care unit, which is based in Texas, is being investigated. The feds are looking at whether it violated sanctions against sales to Iran and other pariah nations. The company said a "grand-jury subpoena" asked for documents dating back to 2005.

Amarin is waiting till next year for an FDA decision on Vascepa, but investors are fine

Amarin ($AMRN) will have to wait until the new year to find out whether its fish oil pill Vascepa will be approved for a wider indication. The FDA indefinitely delayed that decision, and it's reviewing the trial design of the New Jersey-based company's ANCHOR design in the meantime. The holdup apparently didn't put off investors, with shares spiking as much as 20% Friday morning

Amarin's ($AMRN) plodding effort to get Vascepa approved for a wider indication just got longer as the FDA delayed its final decision on the fish oil pill, but the company's backers are staying optimistic, and shares jumped as much as 20% on Friday morning.

The company is looking to expand the label of its cholesterol-fighting omega-3 drug, and the FDA was due to make a final ruling Friday. The delay, however, makes procedural sense: In October, the agency backed out of its support for the design of Amarin's ANCHOR study, eliciting an appeal from the company. Regulators would be loath to make a final decision on a drug without first weighing in on its supporting data, and the agency told Amarin it will rule on the study design appeal by Jan. 14, the company said.

Just when the FDA will make a decision on Amarin's supplemental drug application remains unknown, and the company notes in its announcement that "there can be no assurance that Amarin will be successful in this effort."

Investors were much cheerier, however, sending Amarin's now-pilloried shares up in premarket trading. Still, the company has plummeted by about 75% since October, when an FDA panel cast serious doubt on its odds of winning approval to sell Vascepa to patients with less-severe high triglycerides.

The ensuing consternation led Amarin to slash its payroll and may have spelled the demise of CEO Joe Zakrzewski, who will walk out the door Jan. 1 in what the company is calling a "retirement."

Actelion gets second boost for Opsumit with EU approval

Actelion is granted Marketing Authorisation for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH) by the European Commission

-1 of 3- 20 Dec 2013 16:35:00 UTC  *DJ Actelion is granted Marketing Authorisation for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH) by the European Commission

   December 20, 2013 11:35 ET (16:35 GMT)- - 11 35 AM EST 12-20-13
-2 of 3- 20 Dec 2013 16:35:00 UTC  Press Release: Actelion is granted Marketing Authorisation for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH) by the European Commission
Actelion Pharmaceuticals Ltd / Actelion is granted Marketing Authorisation for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH) by the European Commission . Processed and transmitted by Nasdaq OMX Corporate Solutions. The issuer is solely responsible for the content of this announcement.
-- Approval granted by European Commission on 20 December 2013
-- Opsumit approved as monotherapy or in combination with another PAH therapy for the long-term treatment of WHO Functional Class II to III PAH patients
-- First EU launch planned in Germany in February 2014
ALLSCHWIL, SWITZERLAND - 20 December 2013 - Actelion Ltd (SIX: ATLN) today announced that Opsumit(R) (macitentan), a novel dual endothelin receptor antagonist (ERA), has been granted marketing authorisation for the long-term treatment of PAH in the EU by the European Commission. Opsumit, as monotherapy or in combination, is indicated for the longterm treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III.
Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease.
Macitentan 10mg is also described as reducing the risk of PAH related death or hospitalization for PAH up to end of treatment (EOT) by 50% (HR 0.50; 97.5% CI: 0.34 to 0.75; logrank p < 0.0001). Macitentan 10mg improved quality of life assessed by the SF-36 questionnaire.
Dr Nazzareno Galiè from the Institute of Cardiology, University of Bologna, Bologna, Italy spoke of the impact of the availability of Opsumit, "We are all very pleased with the approval of Opsumit in Europe. For the first time we can offer patients a therapy that has demonstrated improvement in long term clinical outcome showing a significant effect in naive patients and patients who are already on a specific PAH treatment."
The EU label was based in part on data from the landmark Phase III SERAPHIN study which was published in the New England Journal of Medicine in August 2013, the SERAPHIN study demonstrated that treatment with macitentan 10 mg resulted in a 45% risk reduction (hazard ratio [HR] 0.55; 97.5% CI: 0.39 to 0.76; p < 0.0001) of the composite morbidity-mortality endpoint up to (EOT) when compared to placebo.[1]
Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: "We are delighted with today's announcement as we believe that Opsumit represents a major step forward for the management of PAH. Our company strategy of sustaining and growing our PAH franchise has yet again been bolstered by this approval. This achievement marks a significant moment for the PAH community in Europe as the first and only PAH treatment with proven long-term efficacy, from controlled clinical trials in PAH, and will now offer hope of a better future to those living with this disease. Actelion is now working to make this important medicine available to patients around European markets in the coming months."
The most commonly reported adverse drug reactions are nasopharyngitis (14.0%), headache (13.6%) and anaemia (13.2%). The majority of adverse reactions are mild to moderate in intensity.
Opsumit was approved by the FDA on 18 October 2013 and by Health Canada in November 2013. It is also undergoing regulatory assessment in other countries including Switzerland.

Genocea brings 2nd vaccine to clinic to rival $4B-a-year Prevnar

Genocea Biosciences, which three months ago announced what it called “unprecedented” results in a trial of its herpes vaccine, today began an early-stage trial of a second potential vaccine based on the same disease-fighting method.

The Cambridge biotech has been focused on fighting infectious diseases through stimulation of T cells in the body since it was founded in 2007. CEO Chip Clark says the approach is superior to existing vaccines (which are based on the so-called B-cell response) in two ways: It can potentially fight all strains of a disease, rather than a select few, and can kill infected cells even before they make their way into the bloodstream and potentially start to make a patient sick.

That approach has resulted in a potential vaccine for herpes simplex virus type 2, and the results from its first-ever human trial were announced in September. The study found that the vaccine reduced viral-shedding, which often results in the disease emerging in lesions.

It has also been behind $73 million in investment to date from backers including Polaris Venture Partners, Lux Capital Management, Johnson & Johnson Development Corp., Skyline Ventures, Cycad Group, Auriga Partners, the Bill & Melinda Gates Foundation, MP Healthcare Ventures, and Morningside.

Today, it’s led to the trial of a potential vaccine against pneumococcus, a disease of the nose and throat which can lead to pneumonia, meningitis and sepsis, and kills between 500,000 and 1 million children every year. The company has begin a a Phase 1 clinical study with GEN-004, which Clark intends to be the first vaccine that would protect against all 90 strains of the disease.

Clark said that the current market-leading vaccine on the market is Prevnar, a $4 billion-a-year drug marketed by Pfizer. (One of the members of Genocea's board, George Sibor, is the former chief scientific officer of Wyeth Vaccines where he lead the development of Prevnar) Clark says the vaccine is “phenomenal” in its effectiveness against 13 of the most important strains of the disease, but says “there is evidence that the 80-plus strains that are not covered by the vaccine are increasing.”

The Phase 1 study will involve about 90 healthy adult volunteers, and will test the safety and effectiveness of GEN-004 in a range of doses. The company expects results in the second quarter of 2014.

Genocea begins a PhI trial of its rival to Pfizer's Prevnar

The huge sales generated by Prevnar 13 mean Pfizer ($PFE) appears to have the pneumococcal vaccine sector sewn up, but Genocea Biosciences sees an opportunity to disrupt the monopoly. This week, the Cambridge, MA-based biotech began a Phase I clinical trial of the vaccine it thinks can unseat Prevnar.

Genocea developed the vaccine, currently called GEN-004, using the antigen discovery technology that spawned its lead candidate, herpes simplex vaccine GEN-003. It is this approach that gives Genocea the chutzpah to take on Pfizer's blockbuster. While the latest version of Prevnar protects against 13 strains of pneumococcus, Genocea is aiming to confer immunity against all forms of the bacteria. Prevnar 7 and 13 have had a huge effect on disease, but the 80 strains not covered by the vaccines are still causing some cases.

A paper published in PLOS ONE in September found that the introduction of Prevnar 7 coincided with a rise in cases of invasive pneumococcal disease caused by the 80 strains not covered by the vaccine. Pfizer subsequently expanded protection by introducing Prevnar 13, but Genocea thinks there is still a need for a more comprehensive vaccine that will stop the impact of strain replacement. "In a way, Prevnar is a victim of its own success," Genocea CEO Chip Clark told FierceVaccines.

Genocea, a 2008 FierceBiotech Fierce 15 company, has heavy hitters in its corner. George Siber, who was chief scientific officer at Wyeth when Prevnar 7 was in development, sits on Genocea's board of directors. Siber sees potential in using a T cell-directed approach to create a universal pneumococcal vaccine that will combat the bacteria in the nasopharynx, the part of the body where it may evolve. The theory is now being put to the test in a Phase I trial of 90 healthy volunteers. Data is due to be presented in the second quarter of 2014.

Genocea pitches $75M IPO to back T-cell vaccine pipeline work Read more: Genocea pitches $75M IPO to back T-cell vaccine pipeline work - FierceBiotech http://www.fiercebiotech.com/story/genocea-pitches-75m-ipo-back-t-cell-vaccine-pipeline-work/2013-12-24#ixzz2oPoojbUi Subscribe at FierceBiotech

Genocea Biosciences is throwing its hat in the growing IPO ring. The biotech has laid out plans to raise $75 million to help fund its work on a pair of early-stage T-cell vaccines aimed at herpes and all strains of the bacteria pneumococcus.

Genocea has built a development platform that hunts up ideal antigens for spurring a T-cell attack on a pathogen. Its work has attracted the high-profile support of the Bill & Melinda Gates Foundation and may have applications in cancer immunotherapy as well.

The Cambridge, MA-based Genocea launched its Phase I study of its new pneumococcus vaccine just days ago, explaining to FierceVaccines that the biotech thinks it can do better than Pfizer's ($PFE) blockbuster Prevnar 13, which guards against 13 strains of the bacteria. Genocea says it has a more comprehensive approach.

Last fall, Genocea--a 2008 Fierce 15 company--landed a $30 million round, with the Gates Foundation joining CVF, Polaris Venture Partners, Lux Capital, SR One, Johnson & Johnson Development Corp., Skyline Ventures, Cycad Group, Auriga Partners, MP Healthcare Ventures and Morningside in bringing its total raise to $76 million.

This year has seen the biggest burst of biotech IPOs in a decade, a welcome turnaround from the drought years that led up to 2013. But in recent months, the market has seen more stumbles as a growing number of developers try to make the leap. Genocea will be among the first to test the waters in 2014.

Ariad storms back with an FDA nod to get Iclusig back on the U.S. market

Losing more than $2.5 billion in market value didn't kill Ariad Pharmaceuticals ($ARIA), and now the biotech is set to relaunch the once-spurned cancer drug Iclusig in the U.S. as the FDA has approved a new label and indication for the company's only product.

Ariad surged more than 30% on the news, announcing it would reintroduce the leukemia treatment by mid-January now that the FDA has OK'd the drug for T315I-positive patients and amended the label to include warnings about Iclusig's risk of blood clotting and heart failure. Those same safety concerns prompted the FDA to ask Ariad to take its drug off the market in October, the heaviest blow in a downward spiral that blasted the company's shares by more than 70%. But that's all in the past, CEO Harvey Berger said.

"We are back on track," Berger told Bloomberg. "We'll enter 2014 largely where we were as we started off the fall in September."

"Largely" being a relative term, of course. Ariad's hellish October led it to lay off about 160 U.S. employees in an effort to save $26 million, and while the company plans to piece together another U.S. sales force, it won't have the same marketing heft as before, Berger said. Furthermore, while the European Medicines Agency hasn't ordered Iclusig off the market, the group's Pharmacovigilance Risk Assessment Committee has reopened the books on the drug's safety profile, putting Ariad's overseas sales in jeopardy.

Still, Iclusig's exit from the tomb is undoubtedly good news for Ariad, as many analysts speculated it might take a year to win over the FDA if it ever happened at all. The company's shares leapt as high as $7.74 on the Friday announcement, by far their highest mark since an October freefall.

"In less than two months of suspending marketing and commercial distribution of Iclusig in the U.S., we addressed the issues raised by the FDA and now are able to market and distribute Iclusig again in the U.S.," Berger said in a statement.

Farmaci per il diabete hanno effetti diversi sul cuore degli uomini e delle donne

Secondo quanto suggerito da un nuovo studio pubblicato sul numero di dicembre dell’American Journal of Physiology - Heart and Circulatory Physiology, alcuni farmaci per il diabete ampiamente utilizzati hanno effetti diversi sul cuore degli uomini e delle donne. Lo studio ha considerato tre trattamenti comunemente prescritti per il diabete di tipo 2: metformina, metformina più rosiglitazone  e metformina più un tipo di olio di pesce.

I ricercatori hanno esaminato la maniera in cui tre trattamenti comunemente prescritti per il diabete di tipo 2 hanno influenzato 78 pazienti che sono stati divisi in tre gruppi. Un gruppo ha assunto metformina da sola, il secondo gruppo ha assunto metformina più rosiglitazone  e il terzo gruppo ha preso metformina più un tipo di olio di pesce.
La Metformina riduce la produzione di zucchero nel sangue dal fegato e migliora la sensibilità all'insulina. Rosiglitazone migliora la sensibilità all'insulina e rimuove gli acidi grassi liberi dal sangue. L’olio di pesce abbassa i livelli ematici dei trigliceridi.

I ricercatori hanno concluso che i farmaci hanno effetti molto diversi e talvolta opposti sui cuori degli uomini e delle donne, anche se i farmaci controllano lo zucchero nel sangue altrettanto bene in entrambi i sessi. Lo studio compare nel numero di dicembre dell’American Journal of Physiology - Heart and Circulatory Physiology.

Anche se la metformina ha avuto effetti positivi sul cuore nelle donne, ha indotto il metabolismo del cuore degli uomini a bruciare meno zucchero e più grassi. La combustione cronica del grasso da parte del cuore può risultare in cambiamenti nocivi che possono portare a insufficienza cardiaca, hanno detto i ricercatori della Scuola di Medicina dell'Università di Washington a St. Louis.
L’Assunzione di rosiglitazone o lovaza con metformina sembrava ridurre alcuni degli effetti negativi sul cuore della metformina assunta da sola negli uomini. L’assunzione di rosiglitazone in aggiunta a metformina ha ulteriormente migliorato il metabolismo del cuore delle donne, rispetto alla metformina da sola.

L'aggiunta dell’olio di pesce alla metformina non ha avuto un forte effetto in entrambi i casi per gli uomini o le donne, hanno aggiunto i ricercatori.
"Il nostro studio suggerisce che abbiamo bisogno di definire meglio quali terapie sono ottimali per le donne con diabete e quali sono ottimali per gli uomini ", ha detto il Dott. Robert Gropler, professore di radiologia. Lo studio non ha tuttavia dimostrato un legame di causa-effetto tra le combinazioni di farmaci e i cambiamenti cardiaci. Ha mostrato solo una associazione.

FDA approva prime versioni generiche del farmaco antidepressivo duloxetina

La staunitense Food and Drug Administration ha approvato lo scorso 11 dicembre le prime versioni generiche di
duloxetina (nella formulazione capsule a rilascio ritardato), un farmaco usato per trattare la depressione.
"Gli operatori sanitari ed i pazienti possono essere sicuri che questi farmaci generici approvati dalla FDA hanno soddisfatto i nostri rigorosi standard", ha detto Kathleen Uhl, MD, direttore dell’Ufficio Farmaci Generici del Centro di Valutazione dei farmaci e Ricerca della FDA. I farmaci generici offrono un maggiore accesso alle cure sanitarie per molte persone."
La depressione è caratterizzata da sintomi che interferiscono con la capacità di una persona di lavorare, dormire, studiare, mangiare e godere di attività una volta piacevoli. Gli episodi di depressione spesso si ripetono per tutta la vita di una persona. Segni e sintomi della depressione includono: umore depresso, perdita di interesse nelle attività abituali, significativo cambiamento di peso o di appetito, insonnia o sonno eccessivo (ipersonnia), agitazione / stimolazione (agitazione psicomotoria), aumento della fatica, sentimenti di colpa o di inutilità, rallentato pensare o alterata concentrazione e tentativi di suicidio o pensieri di suicidio.
La Duloxetina e gli altri farmaci antidepressivi hanno un un’avvertenza sulla confezione che descrive l'aumento del rischio di pensieri e comportamenti suicidi durante il trattamento iniziale nei bambini, adolescenti e giovani adulti tra i 18 ei 24 anni. L'avviso dice anche che i dati non mostrano questo aumento del rischio in pazienti di età superiore a 24 anni, e che i pazienti di 65 anni che assumono antidepressivi hanno un ridotto rischio di pensieri e comportamenti suicidari. L’avvertenza riporta che la depressione e altri gravi disturbi psichiatrici sono le più importanti cause di suicidio, e che è necessario un attento monitoraggio dei pazienti che iniziano il trattamento con questi farmaci. La duloxetina deve essere dispensata unitamente ad una guida che descrive le informazioni importanti sugli usi e sui rischi associati al farmaco.
I farmaci generici approvati dalla FDA hanno la stessa alta qualità ed efficacia dei farmaci “di marca”. Anche i siti produttivi e di confezionamento dei farmaci equivalenti devono passare gli stessi controlli di qualità, allo stesso modo dei farmaci di marca.

Bristol-Myers bails out of 'Diabetes Inc.' in $4.1B sell-out to AstraZeneca

When Bristol-Myers Squibb got out of the diabetes development business last month, the logical follow-up question was this: Will the company bag diabetes sales, too? 

The answer is yes. Bristol-Myers ($BMY) is selling out of its 6-year-old diabetes partnership with AstraZeneca ($AZN) for up to $4.1 billion.

For a Big Pharma, the turnabout was speedy. As recently as last summer, Bristol-Myers was fully committed to its diabetes business. That's when the company engineered a major expansion of the partnership, with the $5.3 billion buyout of diabetes specialist Amylin Pharmaceuticals.

AstraZeneca then shelled out $3.4 billion, bringing Amylin and its products--Byetta (exanatide) and its longer-acting cousin Bydureon--into the joint venture. Early this year, the two partners brought their diabetes marketing teams together in one location that AstraZeneca CEO Pascal Soriot called "Diabetes Inc."

Now, the British drugmaker will take Diabetes Inc. forward on its own. AstraZeneca will pay $2.7 billion up front, plus another $1.4 billion if the diabetes business hits sales and development targets. Bristol-Myers will also collect royalties through 2025.

With diabetes growing quickly worldwide, the opportunity is enormous. But the business is not without its challenges. Competition in the market is fierce, with longtime specialist Novo Nordisk ($NVO) and Big Pharma rivals Sanofi ($SNY) and Merck ($MRK) all hunting after market share. In fact, as The Wall Street Journal notes, AstraZeneca booked a $1.7 billion charge today, thanks to Bydureon's floundering sales. Onglyza (saxagliptin), its DPP-4 inhibitor, has scrabbled for a hold in the market but remains far outsold by Merck's Januvia (sitagliptin).

Meanwhile, the partnership's newest product, dapagliflozin, has suffered from a delay at FDA, which declined to approve it earlier this year. The drug won European approval soon after--under the brand name Forxiga--but ramping up sales in the EU isn't a quick proposition, thanks to each country's own reimbursement decisions. Indeed, the two partners last week pulled Forxiga in Germanyafter hitting an impasse in pricing negotiations.

Dapagliflozin won backing from an FDA advisory panel last week, though, so things are looking up for the drug. And AstraZeneca is obviously confident enough of success with it and the rest of its lineup to put up $2.7 billion for the chance.

Soriot sees diabetes as one of AstraZeneca's best chances for growth. Since he took the reins last fall, Soriot has been buying in drug candidates right and left but has done few deals that will yield immediate sales. Taking control of the diabetes venture could do just that; as the WSJ points out, Bristol booked $1.2 billion in sales from the partnership's four main products for the first 9 months of the year, while AstraZeneca posted $546 million.

And as Soriot said in a statement, AstraZeneca figures it can leverage its expertise in emerging markets to funnel its diabetes treatments to the fast-growing numbers of patients there. "Today's announcement reinforces AstraZeneca's long-term commitment to diabetes, a core strategic area for us and an important platform for returning AstraZeneca to growth," Soriot said. "I would like to extend a warm welcome to the Bristol-Myers Squibb people who are due to join us."

So, just what happens to the Diabetes Inc. employees? AstraZeneca says that about 4,100 of the Bristol-Myers workers dedicated to diabetes will eventually move over. That includes workers from Amylin, 400 of whom lost their jobs earlier this year. 

Actelion will pay $407M in damages for its buyout to protect Tracleer

Drugmakers have often been accused of trying to thwart competition with arrangements like pay-for-delay deals or aggressive patent litigation to protect their key drugs. But Swiss drugmaker Actelion ($ATLN) went even further than that, a court ruled, buying out a drug company and then canceling a development deal in an effort to protect its only significant product at the time, pulmonary arterial hypertension (PAH) drug Tracleer.

A state appeals court in San Francisco has upheld a $407 million jury award against Actelion and in favor of Asahi Kasei Pharma of Japan, according to SFGate.com. Asahi had developed Fasudil for treating PAH, and it was a potential threat to Tracleer, Actelion's foundational product. Tracleer delivered 87% of Actelion's $1.84 billion in revenue last year.

In 2006, Asahi struck a deal with Bay Area biotech CoTherix to develop oral and inhaled versions of Fasudil and take it to regulators in the U.S. and Europe for approval. Five months later, however, Actelion scooped up CoTherix for $400 million and canceled the arrangement with Japanese company. Asahi sued.

According to SFGate.com, Actelion claimed it killed the deal because it had determined Fasudil was not safe. A jury in 2011 found otherwise and awarded Asahi $546 million, which was later reduced to $377 million for the company to cough up and $30 million from Actelion executives.

It is not the only time that Actelion's protective nature has led to litigation. The drugmaker has been sued by generic drug companies for refusing to provide samples of Tracleer for them to work with. But Actelion is now moving beyond Tracleer. The FDA in October approved Opsumit, Actelion's new PAH treatment and a follow-up to Tracleer that is expected to be a blockbuster.

Jazz will pay $1B for Italy's Gentium, sweeping up another specialty drugmaker

Jazz Pharmaceuticals ($JAZZ), the Ireland-based company known for its narcolepsy drug Xyrem, is often the subject of takeover talk. Because of its specialty drug portfolio and low-tax domicile, analysts often see it as a potential target. But the drugmaker has kept up a steady drumbeat of mostly smaller deals itself to expand its line of specialty drugs. Today it jumped the $1 billion deal fence with a deal to buy Italy-based Gentium ($GENT) along with its EU-approved orphan drug, Defitelio.

Jazz and Gentium inked the price at $57 a share, which is only a 2% premium, but as the Associated Press points out, Gentium shares have quadrupled this year, reaching an all-time high last week of $59.25. Boards for both companies have approved the deal. Gentium specializes in drugs to treat rare diseases. In October, Defitelio was approved by EU regulators for treating obstructions in the small veins of the liver for patients having stem cell transplants.

"Because Defitelio is already approved in the EU, the acquisition would add a new orphan product that has potential for short- and long-term revenue generation, high growth and expansion of our multi-national commercial platform," Jazz CEO Bruce Cozadd said in a statement.

Jazz, which had been based-in Palo Alto, CA, in 2011 agreed to merge with Azur Pharma and move to Azur's home base in Ireland. Its Dublin address has made some analysts see it as a takeover target. In July, Allegan, MI-based Perrigo ($PRGO), a maker of generic and OTC products, agreed to buy Ireland-based Elan in a deal valued at $8.6 billion, saying that having Elan's Irish domicile would save the combined company $150 million annually, mostly from lower taxes. The Azur deal also expanded Jazz' portfolio, getting Azur's women's health and urology products and orphan disease product Gastrocom. In February, Jazz agreed to pay $120 million for the worldwide rights for a drug from Lexington, MA-based Concert Pharmaceuticals that has the same active ingredient as Xyrem but is expected to have less burdensome dosing.

Ariad returns Iclusig to market but with more restrictions and smaller patient pool

Ariad Pharmaceuticals ($ARIA) is back in the market with Iclusig. The FDA is allowing the Cambridge, MA-based company to again sell the leukemia drug, but with tighter restrictions to face. Ariad has a lot of ground to regain to return to its former glory.

While CEO Harvey Berger said the company is starting 2014 where it left off, that is not entirely accurate. Iclusig is now approved for a smaller patient population. The company also has post-marketing requirements including a trial testing other doses of the drug.

And as for the size of the company, he said it would add some sales reps, but not nearly the 160 that it let go in November after the FDA had it yank the drug from the market because of blood clot risks, Bloomberg reports. Those 160 represented 40% of the company's U.S. workforce, and Ariad expected to save about $26 million through 2014 with the cuts. At the time, it did not touch its EU sales group because it had not limited use of the drug. But since then, the EU has added tighter restrictions as well.

The company also saw its market cap plunge after questions first arose in the fall. Its shares were up as much as 31% to $7.25 in mid-morning trading, but that is off from about $18.25 a share in September.

The FDA is now allowing Iclusig to be used for adult patients with a mutation called T315I and for patients for whom no other tyrosine-kinase inhibitors are indicated. Previously, it could be for adult patients when that therapy didn't work or was intolerable. Under the old label, the company said 640 patients were taking the drug. The FDA has so far approved on a case-by-case basis 350 who would qualify.

It has been a rollercoaster ride for Ariad and Iclusig, a drug the FDA granted accelerated approval to less than a year ago, approving it to treat both patients with chronic myeloid leukemia who have stopped responding to other drugs and patients with Philadelphia chromosome-positive acute lymphoblastic leukemia. The company quickly rolled the drug out, putting a $115,000-a-year price tag on it, and touted how the drug had been brought to market in 5 years, turning Ariad into a commercial oncology company in the process. Analysts believed there was a blockbuster in the making.

A prosperous new year for pharma? Let's check the tea leaves

As usual, the biopharma year is ending with a bang, as companies scramble to wrap up pending business before the books close. Plenty going on in the present to occupy the mind. And as a long-range type of business--governed by patent periods and extended R&D timelines--this industry is as likely to be thinking about 2017 as next week.

We'd like to turn your attention past the flurry of today, but before the years of lifecycle management ahead. To 2014, a.k.a. next year. A mere two weeks from now, it stretches out into the dimly lit future, with four quarterly reports, 12 monthly goals, and, if you're fortunate, one year-end bonus, amount as yet determined.

We can't predict your incentive pay, of course. We can, however, hazard a few predictions about the forces--positive and not so much--shaping the industry in 2014.

Most troublesome for drugmakers is the growing pricing pressure--including private insurers in the U.S. Once counted upon for the pricing freedom drugmakers lack in most other world markets, the U.S. system is changing. In the wake of the mergers between Express Scripts ($ESRX) and Medco Health Solutions, and CVS ($CVS) and Caremark, pharmacy benefits managers (PBMs) find themselves with more power. And they're using it. CVS Caremark last year rolled out some strict formulary restrictions on a handful of drugs, as something of a test case for bigger moves down the line.

But the big blow came this year, from Express Scripts, when the PBM introduced its 2014 National Formulary. For the first time, that formulary specifically excludes a list of drugs, including some commonly used products--and some brand-new drugs Big Pharma hopes will vault it past the patent cliff. Pfizer's ($PFE) rheumatoid arthritis drug Xeljanz, GlaxoSmithKline's ($GSK) respiratory med Breo Ellipta, Johnson & Johnson's ($JNJ) anti-inflammatories Simponi and Stelara all fall in that latter category. Since then, an Express Scripts executive told Bloomberg that the PBM will be tough on the new crop of pricey hepatitis C drugs. Gilead Sciences' ($GILD) Sovaldi just launched at an $80,000-per-course price, and Gilead is about to follow up with a combo pill that could be even more expensive. Express Scripts will be looking to cut a deal and plans to compare the different treatments head-to-head to make sure it's getting the best value for its buck.

And it's not just the U.S., of course; Australia is tracking patients using Bristol-Myers Squibb's ($BMY) melanoma treatment Yervoy to gauge its value for the $110,000-a-year list price. And then there's the newly strict Germany, where unfavorable pricing prompted Bristol and AstraZeneca ($AZN) to yank their new diabetes treatment Forxiga. And arguably, the most dramatic form of pricing pressure can be seen in China, where the government is probing most Big Pharma companies for potential bribery, most notably GSK. Various on-the-ground sources have said that price cuts could ease the scrutiny.

While these pricing battles rage, Pfizer, Novartis ($NVS), and to a lesser extent, Glaxo and Bristol-Myers are taking a hard look at their businesses to see where they might unlock value, free up cash for stock buybacks, and refocus their attention. Pfizer, of course, has already jettisoned a few units peripheral to its core business; its successful spinoff of the animal health business Zoetis ($ZTS) wrapped up earlier this year. But the pharma behemoth took another step in July that people will be watching closely: It split its commercial operations into three separate businesses with separate financial reporting to give investors an eye on their value. Pfizer will unveil the first of those numbers with first-quarter results.

Inspired, Glaxo is creating a new unit encompassing its older brands, and it, too, will get its own line item in the company's financial results. Meanwhile, Novartis launched a strategic review after its 2013 annual meeting, and it is now mulling over some unit sales--with animal health first on its list. The Swiss drugmaker already hived off a small segment of its diagnostics business, and it intends to shed other businesses it considers poor bets for growth. Units that aren't market leaders now will either build up through M&A and joint ventures, or ship out. That review will continue into the new year, with action sure to follow. 

It's no coincidence that these companies also are buying back their shares to support the stock and keep investors happy. The cash is also helping to fund dividends. Glaxo sold off a couple of its drinks brands and several thrombosis drugs, raising more than $4 billion, funding some recent deals, as well as share buybacks. Pfizer already granted a boon to investors with the Zoetis shares that effected its spinoff. And Bristol-Myers, looking to focus more tightly on a core of several therapeutic areas, bowed out of diabetes research and sold its share of its diabetes joint venture to partner AstraZeneca.

On the ground, sales reps are feeling ongoing tremors across the pharma landscape. While medical schools and teaching hospitals crack down on their site visits--and soon may bar reps altogether--physician practices continue to raise barriers. Speaking fees for physicians, a staple in the pharma-promotion diet, will get more scrutiny in 2014 as the Sunshine Act forces disclosure of all payments and gifts to U.S. doctors. And while marketing experts don't expect the rest of Big Pharma to follow suit anytime soon, GlaxoSmithKline is moving to remake its marketing strategies, rolling out a new compensation system for reps worldwide and nixing most speaking fees and other physician payments.

Meanwhile, the iPads that reps now tote on sales calls could soon replace many of them altogether, as the share of doctors who prefer electronic detailing grows. And these days, those doctors are more likely to own their own iPads, where they can review drug presentations whenever they like. The sales-rep jobs that remain--and remain unchanged--are fewer, too, as the move to specialty drugs leaves droves of primary-care sales people out of jobs. Eli Lilly ($LLY) was 2013's prime example, with layoffs meant to ease blockbuster sales losses as the antidepressant faces generic competition. While the sweeping layoffs of several years ago appear to be past, Big Pharma remains in cost-cutting mode, and more job cuts are likely to come in 2014.

Hiring will continue in some places--in emerging markets, namely, where drugmakers have been staffing up for several years. But these days, companies are handling developing countries with more care. India's intellectual property crackdown continues, with compulsory licenses threatened on several cancer drugs. Bayer went so far as to give up pursuing patent protection for its breast cancer blockbuster Herceptin, and Indian drugmaker Biocon immediately pledged to get a biosimilar version to market posthaste. China's corruption scandal has roped in Sanofi ($SNY), Novartis, Eli Lilly and others--in addition to GSK--showing how politics can throw up obstacles quickly and unexpectedly. After all, payments to doctors have till now been common in China, among domestic and foreign companies alike.

And now, the FDA is following up on its promise to beef up local inspections in developing countries, with agency staffers setting up new bases in India, and soon, China. Some manufacturers that teamed up with multinational pharma companies years ago have found themselves in hot water for manufacturing violations as a result.

Most drugmakers aren't deterred, however. Glaxo, at the center of the Chinese scandal, is spending billions amping up its stake in emerging-markets subsidiaries, while Sanofi continues its march not only in the biggies like India and China, but smaller countries like Turkey and Saudi Arabia. Smaller drugmakers are getting in on the act, too; CFR Pharmaceuticals' ill-fated buyout of South Africa's Adcock Ingram is as much about manufacturing capacity for feeding emerging markets as it is about access to Adcock's drug portfolio and distribution. Just over the past 10 days, the small Hungarian drugmaker Richter announced a joint venture in Malaysia and a deal for Mexico's DNA Pharmaceuticals. Emerging markets will remain the world's fastest-growing, so navigating new obstacles is just a reality of today's drug business.

On the bright side, the FDA has ushered another big crop of drugs into the market, and several of them are already on their way to blockbuster status. Biogen Idec's ($BIIB) multiple sclerosis pill Tecfidera, Roche's ($RHHBY) breakthrough breast cancer drug Kadcyla, and now Gilead's Sovaldi show that real treatment advances can still deliver big sales. Whatever the vagaries of the pharma market, that's a perennial promise. 

What are your goals and challenges for 2014? Let us know. And tell us what we've missed. We have a lot of ground to cover next year, too. -- Tracy Staton 

XII RAPPORTO SULLE POLITICHE DELLA CRONICITÀ DI CITTADINANZATTIVA

Creato Venerdì, 13 Dicembre 2013

Tra costi elevati e difficoltà in ambito lavorativo, curasi non è più permesso. Presentato il XII Rapporto sulle politiche della cronicità di Cittadinanzattiva

Avere una o più patologie croniche o rare, o accudire una persona malata, è diventato oggi un “lusso” che non ci si può più permettere, perché i costi diretti ed indiretti della malattia risultano insostenibili per un numero crescente di pazienti e di famiglie. E l’estremo risultato è non solo non curarsi nella maniera adeguata, ma addirittura “nascondere” la propria patologia in alcuni contesti, fra cui quello lavorativo. 
Sul piano sociosanitario, emerge una assistenza, soprattutto a livello di accesso ai farmaci, a macchia di leopardo, con regioni più avanti e altre che stentano a assicurare anche i LEA, mentre i tagli incidono maggiormente sull’assistenza domiciliare e sulla riabilitazione.
A descrivere questa situazione è il XII Rapporto nazionale sulle politiche della cronicità, dal titolo “Permesso di cura”, presentato oggi a Roma dal Coordinamento nazionale delle Associazioni dei malati cronici (CnAMC) di Cittadinanzattiva. 
“Ritardare o rinunciare alle cure necessarie, perdere il posto di lavoro, confrontarsi con la crisi dei redditi familiari e con le discriminazioni regionali nell’accesso alle prestazioni socio sanitarie è ciò che vivono sulla propria pelle i cittadini grazie ad anni di politiche di disinvestimento del Welfare e di erosione dei diritti. Non possiamo accettare che per “fare cassa” si continui a smantellare il SSN o peggio ancora a svendere i diritti dei cittadini alla salute, al lavoro e all’inclusione sociale”, ad affermarlo Tonino Aceti, coordinatore nazionale del Tribunale per i diritti del malato e responsabile del CnAMC di Cittadinanzattiva. ”Chiediamo al Governo e al Parlamento un’azione concreta, a partire dalla Legge di Stabilità in discussione, eliminando l’insopportabile misura prevista dalla L. 214/2011 e dal nuovo regolamento ISEE secondo cui i trattamenti assistenziali come indennità di invalidità civile e di accompagnamento sono considerati “fonti di reddito” e quindi da considerare nel computo dei redditi familiari. Chiediamo inoltre al Governo e alle Regioni di avviare un confronto anche con le Associazioni di cittadini e di pazienti sia sul Patto per la Salute, sia sulla prossima Spending review, che rappresentano le vere partite per il nostro Servizio Sanitario Nazionale. Non vogliamo infatti correre il rischio che queste misure possano comportare un’ulteriore compressione di tutele e di diritti.
Ricordiamo al Governo che i cittadini hanno già dato tanto e sono invece ancora in attesa di ricevere quanto è stato previsto e promesso anche da leggi. Pensiamo solo all’aggiornamento dei Livelli Essenziali di Assistenza, al palo da oltre 10 anni, e che dovrebbe prevedere, tra l’altro, la revisione del Nomenclatore tariffario delle protesi e degli ausili e degli elenchi delle patologie croniche e rare esenti“

L’84% delle associazioni dichiara che i pazienti non riescono a conciliare l’orario di lavoro con le esigenze di cura ed assistenza, al punto che nel 63% dei casi hanno ricevuto segnalazioni di licenziamenti o mancato rinnovo del rapporto lavorativo per le persone con patologie croniche e invalidanti e nel 41% dei casi per i familiari che li assistono.  Il 60% ha riscontrato difficoltà nella concessione dei permessi retribuiti, il 45% nella concessione del congedo retribuito di due anni; il 49% evita di prendere sul lavoro permessi per cura, il 43% nasconde la propria patologia e il 40% si accontenta di eseguire un lavoro non adatto alla propria condizione lavorativa. 
L’assistenza sociosanitaria costa e non si può rischiare di perdere il posto di lavoro: il 54% ritiene troppo pesante o oneroso il carico assistenziale non garantito dal Servizio sanitario nazionale. Si spendono in media 1585 euro all’anno per tutto ciò che serve alla cosiddetta prevenzione terziaria (diete particolari, attività fisica, dispositivi e tutto ciò che è utile per evitare le complicanze), più di 1.000 euro per visite ed esami a domicilio, o ancora in media 3711 euro l’anno per adattare la propria abitazione alle esigenze di cura. Chi non può pagare, in una percentuale che arriva anche all’80% di chi è in cura, rinuncia alla riabilitazione, al monitoraggio della patologia, ad acquistare i farmaci non dispensati,  alla badante, all’acquisto di protesi e ausili non passati dal servizio sanitario nazionale.
Spese medie annuali a carico del paziente

Badante	9.082 euro
Retta strutture residenziali o semiresidenziali	7.390 euro
Adattamento della abitazione	3.711 euro
Prevenzione terziaria (diete, att.fisica)	1.585 euro
Supporto psicologico	1.247 euro
Assistenza domiciliare non coperta dal SSN	1.070 euro
Parafarmaci (creme, integr.alimentari, pomate)	901 euro
Dispositivi medici (assorbenti, cateteri, siringhe, sacche)	737 euro
Farmaci non rimborsati	650 euro
Visite ed esami in intramoenia o nel privato	Oltre 600 euro
Protesi ed ausili	537 euro

Fonte: XII Rapporto CnAMC-Cittadinanzattiva

La prevenzione, eterna cenerentola
Il 53% delle Associazioni non ritiene sufficiente la prevenzione primaria (corretti stili di vita); il 60% considera inadeguata o del tutto carente la prevenzione secondaria (interventi per una diagnosi precoce e per la riduzione del danno). Per la prevenzione terziaria (relativa alle complicanze) sale al 64% la percentuale delle Associazioni che ritengono non si faccia. 
Anche chi si impegna personalmente nella prevenzione, riconoscendone l’importanza, ha difficoltà ad accedere alle visite specialistiche o esami diagnostici necessari nel 66% dei casi e l’89% è, quindi, costretto a sostenere costi privati per accedere a queste prestazioni.
La diagnosi troppo spesso in ritardo
Il 75% delle Associazioni dichiara di aver ravvisato ritardi diagnostici nella propria patologia di riferimento. Il sospetto diagnostico viene formulato generalmente dallo specialista di riferimento (67%) e solo nel 20% dei casi dal Medico di Medicina Generale che si interfaccia con lo specialista solo per il 59% delle associazioni.
Si può attendere, quindi, otto anni in media per una diagnosi di endometriosi o cinque per la diagnosi di Lupus Eritematoso Sistemico. C’è addirittura chi ha atteso 33 anni per la diagnosi della sindrome di Bechet. Le cose non vanno meglio per i tempi medi: c’è chi attende al massimo un anno, ma anche chi in media attende dai due ai sei anni. In generale dipende dal medico che incontri o dal trovarsi al Nord o al Sud.
Invalidità civile, l’odissea continua
Crescono le difficoltà per vedersi riconosciuti l’handicap grave (L 104/92, +44% rispetto al 2011)  e il contrassegno per invalidi (+21% rispetto al 2011), così come aumentano le difficoltà di accesso alla invalidità (+16%). Il 68% delle associazioni, a distanza di oltre due anni dall’entrata in vigore della nuova legge, non riscontra né semplificazione né riduzione nei tempi per il riconoscimento: il 65% afferma che i propri associati sono costretti a sottoporsi a doppia visita di accertamento, presso la ASL e l’INPS, anche perché il medico INPS continua a non essere integrato nella commissione di accertamento nel 45% dei casi. Le convocazioni a visita di verifica avvengono in maniera non regolare per il 41% delle associazioni, attraverso sms (59%), lettera semplice (47%) o addirittura messaggi lasciati in segreteria (12%). 
Una Italia a più velocità
L’assistenza farmaceutica, quella protesica, così come l’assistenza domiciliare e la riabilitazione sono erogati nel nostro Paese a macchia di leopardo. Anche i percorsi diagnostici terapeutici  e i registri di patologia (che indicano il numero di pazienti, suddivisi per patologia e regione di residenza) sono poco diffusi e segnalati principalmente al Nord.
Il 61% delle Associazioni dichiara di avere difficoltà di accesso all’assistenza farmaceutica in alcune regioni. Le principali criticità riguardano i tempi eccessivamente lunghi per l’autorizzazione all’immissione in commercio da parte dell’AIFA (50%), il costo dei farmaci non rimborsati dal SSN in fascia C (44%), le limitazioni da parte dell’Aziende ospedaliere o dalle ASL per motivi di budget ed i tempi di inserimento dei farmaci nei Prontuari regionali diversi da regione a regione (41%). Il 39% delle Associazioni, ancora, ha riscontrato l’interruzione o il mancato accesso a terapie perché particolarmente costose.
L’assistenza domiciliare integrata è adeguata alle esigenze di cura solo in alcune regioni (44%).Il principale problema in questo ambito è l’assenza di un supporto psicologico (41%) seguito dalla mancanza di alcune figure professionali (38%) e da un numero di ore di assistenza insufficienti (29%).
Anche la riabilitazione risente fortemente delle differenze regionali. Infatti risulta adeguata, ma solo in alcune regioni per il 65% delle Associazioni. La principale criticità riguarda i tempi di attesa incompatibili e la mancanza di posti letto e strutture (77%). Particolarmente critica la durata del ciclo riabilitativo, considerata inadeguata dal 73% delle Associazioni.
Le principali proposte:

• prevedere all’interno del Patto per la salute 2013-2015 in discussione l’impegno a realizzare un Piano di azione nazionale sulle patologie croniche;
• approvare il Piano nazionale sulle malattie rare 2013-2016;
• istituire i Registri nazionali di patologia, per ciascuna di esse;
• implementare lo sviluppo di PDTA nazionali in grado di garantire livelli uniformi di assistenza socio-sanitaria su tutto il territorio nazionale;
• garantire un accesso equo, tempestivo ed uniforme alle terapie farmacologiche, nonché la partecipazione delle Organizzazioni civiche e dei pazienti ai processi decisionali nazionali e regionali inerenti  l’assistenza farmaceutica