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