Opin­ion: US and EU re­views of blood clots and J&J’s Covid vac­cine show why a trans­par­ent process mat­ters

When the CDC’s top ad­vi­so­ry com­mit­tee met last week to re­view whether a hand­ful of very rare but po­ten­tial­ly fa­tal blood clots with low platelets might be linked to J&J’s Covid-19 vac­cine, the as­sump­tion was that the com­mit­tee would, at the very least, fol­low its own agen­da and vote on whether to wait for more da­ta, or make in­ter­im age- or risk-based rec­om­men­da­tions for the vac­cine.

In­stead, the com­mit­tee did nei­ther, punt­ing its vote un­til this Fri­day when it can hold a longer dis­cus­sion with the lat­est da­ta. None of the ACIP mem­bers raised ma­jor con­cerns with the de­ci­sion, or lack there­of, de­spite some calls to not pause the vac­cine in­def­i­nite­ly.

But the in­ac­tion al­so raised con­cerns about whether the CDC and FDA were shirk­ing their re­spon­si­bil­i­ties and un­nec­es­sar­i­ly de­lay­ing J&J vac­ci­na­tions as the race to slow the spread of the coro­n­avirus con­tin­ues.

In con­trast, the Eu­ro­pean Med­i­cines Agency’s safe­ty com­mit­tee quick­ly con­clud­ed its re­view of the blood clots and the J&J vac­cine on Tues­day, de­cid­ing the vac­cine’s ben­e­fits out­weigh the risks, while adding a warn­ing to the vac­cine’s la­bel, even as no one in Eu­rope has re­ceived the vac­cine yet. Fol­low­ing the com­mit­tee’s an­nounce­ment, J&J said it would re­sume ship­ments of the vac­cine.

At first blush, the EMA, which just went through a sim­i­lar re­view with As­traZeneca’s vac­cine and came to a sim­i­lar con­clu­sion, seems to have been the more proac­tive reg­u­la­tor. The EMA made a quick de­ci­sion on the avail­able da­ta and re-start­ed the process for get­ting more shots in­to arms.

But be­yond the head­line and fi­nal de­ci­sion lies a very dif­fer­ent sto­ry.

In the case of the CDC’s com­mit­tee, any mem­ber of the pub­lic (any­where in the world) could turn on a com­put­er and watch the pro­ceed­ings, lis­ten to the se­ri­ous­ness of the de­tails, hear the ar­gu­ments and see the slides that each of the com­mit­tee mem­bers re­view. The pub­lic even has an op­por­tu­ni­ty to weigh in at the CDC meet­ing, al­though it’s un­clear how those com­ments are ac­tu­al­ly used.

The ACIP de­ci­sion to de­lay ac­tion and wait for more da­ta on the blood clots may seem like an al­most neg­li­gent re­sponse, es­pe­cial­ly giv­en the EMA’s snap de­ci­sion, when time is of the essence. But the risk cal­cu­la­tions for the two re­gions are very dif­fer­ent. The US has more of the Pfiz­er and Mod­er­na Covid vac­cines than the EU to fill the gap left by the J&J pause. The US al­so has about 10 mil­lion dos­es of the J&J vac­cine wait­ing on shelves so if the FDA and CDC of­fer a green­light, those dos­es will im­me­di­ate­ly be ad­min­is­tered.

Al­so, the EMA’s safe­ty com­mit­tee com­plet­ed its analy­sis of the J&J and As­traZeneca vac­cine da­ta be­hind closed doors, on­ly of­fer­ing a peek in­to what oc­curred via lim­it­ed press re­leas­es and press con­fer­ences that usu­al­ly lead to as many new ques­tions as an­swers.

Pe­ter Ar­lett

For in­stance, at each of the EMA press con­fer­ences on the vac­cine la­bel up­dates, Pe­ter Ar­lett, the head of phar­ma­covig­i­lance at the EMA, has un­veiled new fig­ures on the cas­es of blood clots with low platelets for each of the vac­cines au­tho­rized in the EU.

But in do­ing so, Ar­lett fails to of­fer any fur­ther in­for­ma­tion on how many to­tal dos­es of the vac­cines have been ad­min­is­tered or where these new cas­es oc­curred. At least twice now, ei­ther Ar­lett or some­one else at the EMA has had to cor­rect or clar­i­fy what the num­bers mean, or where the cas­es did or did not oc­cur.

If the EMA is go­ing to re­lease these num­bers, why not put them in a press re­lease and ful­ly ex­plain them? In­stead, the agency leaves it to the pub­lic to de­cide on what they might mean, which is con­cern­ing as they are not ap­ples-to-ap­ples com­par­isons. Pfiz­er see­ing 25 cas­es of the blood clots and low platelets ver­sus J&J see­ing 8 cas­es are not com­pa­ra­ble con­sid­er­ing how many tens of mil­lions of more dos­es of the Pfiz­er vac­cine have been ad­min­is­tered.

In oth­er in­stances (e.g. re­leas­ing clin­i­cal tri­al re­sults), the EMA has tak­en a lead­ing role in show­ing the world how trans­paren­cy works. But in this case, even with the FDA and CDC tak­ing ex­tra days to make its de­ci­sion, and even if the de­ci­sion is to re­strict the use of the J&J vac­cine to a spe­cif­ic pop­u­la­tion, the im­por­tance of trans­paren­cy is clear.

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In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

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Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

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As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

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The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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In­no­force opens new man­u­fac­tur­ing site in Chi­na

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But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

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Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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