House com­mit­tee weighs drug pric­ing pro­pos­als

The House Ways and Means Com­mit­tee on Tues­day heard tes­ti­mo­ny on ways the US could ad­dress the ris­ing cost of pre­scrip­tion drugs.

The hear­ing fol­lows two re­cent Con­gres­sion­al hear­ings on drug pric­ing held by the House Over­sight Com­mit­tee and Sen­ate Fi­nance Com­mit­tee late last month and pre­cedes a much-an­tic­i­pat­ed Sen­ate Fi­nance Com­mit­tee hear­ing on 26 Feb­ru­ary where the CEOs of drug­mak­ers Ab­b­Vie, Bris­tol-My­ers Squibb, John­son & John­son, Mer­ck, Pfiz­er and Sanofi are ex­pect­ed to tes­ti­fy.

Ris­ing pre­scrip­tion drug prices have been a fierce­ly de­bat­ed po­lit­i­cal is­sue in re­cent years, with both De­moc­rats and Re­pub­li­cans in Con­gress and the Trump ad­min­is­tra­tion all pitch­ing their own so­lu­tions, in­clud­ing al­low­ing pre­scrip­tion drug im­por­ta­tion from Cana­da, fur­ther Medicare Part D ne­go­ti­a­tions, set­ting in­ter­na­tion­al ref­er­ence pric­ing and re­duc­ing bar­ri­ers to com­pe­ti­tion.

The De­part­ment of Health and Hu­man Ser­vices al­so re­cent­ly pro­posed a rule to elim­i­nate pro­tec­tions for re­bates paid to phar­ma­cy ben­e­fit man­agers (PBMs), which the ad­min­is­tra­tion says would low­er costs for con­sumers.

While drug­mak­ers, in­clud­ing Eli Lil­ly and No­var­tis, have backed the pro­pos­al, the Phar­ma­ceu­ti­cal Care Man­age­ment As­so­ci­a­tion, the trade group rep­re­sent­ing PBMs, has ar­gued that PBMs help low­er costs by ne­go­ti­at­ing re­bates.

Dur­ing the hear­ing, Ways and Means Com­mit­tee Chair­man Richard Neal (D-MA) cit­ed the fin­ger point­ing be­tween the var­i­ous ac­tors in­volved in pre­scrip­tion drug spend­ing.

Rep. Richard Neal

“Drug com­pa­nies point to the PBMs, who point to the in­sur­ance com­pa­nies, who point to the hos­pi­tals. The one group that is not the prob­lem but is the biggest vic­tim is in­deed the pa­tients,” he said, adding that Con­gress will need to take a “mul­ti-pronged” ap­proach to ad­dress high pre­scrip­tion drug spend­ing in­volv­ing changes at the US Food and Drug Ad­min­is­tra­tion, Cen­ters for Medicare & Med­ic­aid Ser­vices (CMS) and po­ten­tial­ly changes to the tax code.

Neal, along with rank­ing mem­ber Kevin Brady (R-TX), said they are com­mit­ted to work­ing to­geth­er “to take mean­ing­ful ac­tion to low­er the cost of pre­scrip­tion drugs in the US health care sys­tem.”

How­ev­er, de­spite the promise to work to­geth­er, mem­bers of the two par­ties are not ful­ly aligned on what steps to take.

One area of dis­agree­ment is over whether Medicare should be al­lowed to ne­go­ti­ate drug prices in its Part B and Part D pro­grams.

While ad­vo­cat­ing for Medicare Part B and Part D re­forms, Brady dis­missed the no­tion that the gov­ern­ment should ne­go­ti­ate drug prices.

Rep. Kevin Brady

“When Wash­ing­ton ne­go­ti­ates in gov­ern­ment-run health­care pro­grams, tax­pay­ers of­ten end up bear­ing the cost while Amer­i­cans can be de­nied ac­cess to the most in­no­v­a­tive break­through med­i­cines avail­able to oth­ers in the pri­vate mar­ket,” he said.

In­stead, Brady ar­gued that “em­pow­er­ing pa­tients to choose the most af­ford­able med­i­cines for them and elim­i­nat­ing the in­cen­tives in Medicare that re­ward bad ac­tors and lead to high prices” would be a more ef­fec­tive so­lu­tion, re­fer­ring Medicare’s per­cent­age-based re­im­burse­ment struc­ture which some ar­gue in­cen­tivizes pre­scrib­ing more ex­pen­sive drugs.

Rachel Sachs

Mark Miller, ex­ec­u­tive vice pres­i­dent of health care at Arnold Ven­tures, told the com­mit­tee that mov­ing to a flat fee-per-pre­scrip­tion mod­el for Medicare Part B could help to curb some spend­ing.

Both Miller and Rachel Sachs, as­so­ciate pro­fes­sor of law at Wash­ing­ton Uni­ver­si­ty in St. Louis, al­so said that the US could save mon­ey on pre­scrip­tion drugs by ty­ing their prices to an in­ter­na­tion­al ref­er­ence list, as the Trump ad­min­is­tra­tion re­cent­ly pro­posed.

First pub­lished here and syn­di­cat­ed in part­ner­ship with RAPS.

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Step­ping on Roche's toes, Mer­ck cuts in­to SCLC niche with third-line Keytru­da OK

In the in­creas­ing­ly crowd­ed check­point race, small cell lung can­cer has been a rare area where Roche, a sec­ond run­ner-up, has a lead over the en­trenched lead­ers Mer­ck and Bris­tol-My­ers Squibb. But Mer­ck is fi­nal­ly mak­ing some head­way in that di­rec­tion with the lat­est ap­proval for its PD-1 star.

The lat­est green light en­dors­es Keytru­da in the third-line treat­ment of metasta­t­ic SCLC, where it would be giv­en to pa­tients whose dis­ease ei­ther don’t re­spond to or re­lapse af­ter chemother­a­py, which would have fol­lowed at least one pri­or line of ther­a­py.

Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.