With the emer­gence of re­al-world ev­i­dence as a promis­ing way to cap­ture da­ta points out­side of clin­i­cal tri­als and speed up drug de­vel­op­ment, con­tract re­search or­ga­ni­za­tions — the stal­warts of tra­di­tion­al tri­al ex­e­cu­tion — are of­ten lead­ing the charge in adopt­ing new tech­nol­o­gy. Parex­el, a lead­ing play­er in the field, is step­ping up that com­mit­ment through a deal with Data­vant.

Jamie Mac­don­ald

Spawned by Vivek Ra­maswamy’s start­up fac­to­ry Roivant and led by Travis May, Data­vant has po­si­tioned it­self as a key ag­gre­ga­tor link­ing up the dis­parate datasets that ex­ist in health­care, of­ten in si­los. In the new part­ner­ship, its soft­ware will be in­te­grat­ed in­to Parex­el’s work­flow such that all the da­ta from their stud­ies will be con­nect­ed to re­al-world da­ta such as elec­tron­ic health records, claims and di­ag­nos­tics, as well as in­for­ma­tion re­lat­ing to ge­nomics, wear­able de­vices, so­cioe­co­nom­ic and be­hav­ioral da­ta and oth­ers.

All the da­ta gen­er­at­ed in the process are al­so quick­ly de-iden­ti­fied — one of Data­vant’s key pitch­es amid in­creas­ing con­cerns about pa­tient pri­va­cy.

“(O)ne of the most sig­nif­i­cant bar­ri­ers to gen­er­at­ing re­al-world ev­i­dence is over­com­ing lim­i­ta­tions of sin­gle da­ta sources,” Parex­el CEO Jamie Mac­don­ald said in a state­ment. “Part­ner­ing with Data­vant has pro­vid­ed Parex­el the abil­i­ty to link dis­parate da­ta sets to dri­ve the com­plex analy­ses nec­es­sary for pro­vid­ing in­no­v­a­tive sci­en­tif­ic and clin­i­cal da­ta strate­gies.”

Travis May

Mac­don­ald be­gan work­ing with Data­vant last Sep­tem­ber, a few months af­ter he took the helm. Af­ter ham­mer­ing out how best to in­cor­po­rate Data­vant’s tech in­to Parex­el’s way of de­sign­ing and con­duct­ing tri­als, the duo has agreed to for­mal­ly ex­pand the re­la­tion­ship in­to a mul­ti-year strate­gic pact.

The use of re­al-world ev­i­dence has gained steam as bio­phar­ma com­pa­nies look to trim the cost of clin­i­cal de­vel­op­ment and ad­vo­cate for a sort of pa­tient-cen­tric nar­ra­tive in which their day-to-day ex­pe­ri­ence with ther­a­pies can be tak­en in­to reg­u­la­to­ry ac­count. The FDA kick-start­ed its own pi­lot projects to eval­u­ate whether RWE can help pre­dict the re­sults of clin­i­cal tri­als that have yet to wrap up. The dis­cus­sion is still lim­it­ed to drugs al­ready on the mar­ket — for which de­vel­op­ers might be seek­ing la­bel changes — and ques­tions about da­ta qual­i­ty stan­dards re­main, but the mo­men­tum is there to cre­ate new ways to build a fran­chise treat­ment.

So­cial im­age: Jamie Mac­don­ald, Travis May

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.