Com­pas­sion­ate Use/Ex­pand­ed Ac­cess pro­grams: Three steps to im­prove their im­ple­men­ta­tion for the ben­e­fit of all

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to Amber Tong.

Com­pas­sion­ate Use or Ex­pand­ed Ac­cess is a treat­ment op­tion for pa­tients in des­per­ate need which can be in­de­pen­dent­ly re­quest­ed by a treat­ing physi­cian, pro­vid­ing ac­cess to lo­cal­ly un­li­censed med­ica­tions when all ap­proved treat­ment op­tions have been ex­haust­ed and en­roll­ment in a clin­i­cal tri­al is not pos­si­ble. There is no doubt about the hu­man val­ue of these pro­grams for pa­tients with no oth­er op­tion, and it’s al­so in­creas­ing­ly clear just how ben­e­fi­cial they can be for the en­tire health care ecosys­tem in ad­dress­ing the un­met med­ical needs of pa­tients. But many stake­hold­ers, from gov­ern­ments, health­care in­sti­tu­tions to phar­ma com­pa­nies, are not set up to make the best use of com­pas­sion­ate use. The time to change that is now.

I have been pas­sion­ate about com­pas­sion­ate use pro­grams for many years, but two re­cent events have crys­tal­lized their im­por­tance for me — in­di­cat­ing that we have reached a piv­otal mo­ment where the full po­ten­tial of these pro­grams is there for all to see, if the col­lec­tive will is there to grasp it.

First, in April, the FDA ap­proved a ther­a­py for a rare dis­ease based sole­ly on re­al-world da­ta ob­tained from pa­tients who were part of a com­pas­sion­ate use pro­gram. This was un­prece­dent­ed and shows just how cru­cial da­ta col­lec­tion in com­pas­sion­ate use has be­come to ac­cel­er­ate ap­proval of much-need­ed ther­a­pies, es­pe­cial­ly in rare dis­eases where there are of­ten none or very few treat­ment op­tions.

Sec­ond, my col­leagues and I in­ves­ti­gat­ed fac­tors as­so­ci­at­ed with com­pas­sion­ate use re­quests re­ceived at No­var­tis over a three-year pe­ri­od. The da­ta pub­lished in the Jour­nal of Amer­i­can Med­ical As­so­ci­a­tion (JA­MA) Health Fo­rum showed that of the 31,711 com­pas­sion­ate use re­quests from 110coun­tries, 87% were from high-in­come coun­tries. An even larg­er pro­por­tion – 94% – came from coun­tries with com­pas­sion­ate use reg­u­la­tions made pub­licly avail­able on the in­ter­net, and al­most all (96%) came from coun­tries with a high lev­el of clin­i­cal tri­al ac­tiv­i­ty. While these da­ta clear­ly re­veal vast dis­par­i­ties at the macro­eco­nom­ic lev­el, I be­lieve they al­so shine a light on the path for­ward — for gov­ern­ments, reg­u­la­tors and phar­ma alike.

As a phar­ma ex­ec­u­tive, I am most­ly fo­cused on what com­pa­nies can do to op­ti­mize their com­pas­sion­ate use pro­grams. I’ve been in­volved in this space for well over a decade, and the evo­lu­tion dur­ing this pe­ri­od has been phe­nom­e­nal. At No­var­tis alone, we now re­ceive on av­er­age about 10,000 re­quests every year, with an ap­proval rate of around 95%. We have gar­nered sig­nif­i­cant ex­pe­ri­ence and un­der­stand­ing over time, which we have used to push the bound­aries, pave the path for­ward and im­prove our pro­grams in the fol­low­ing ways:

Find the will be­fore the way

First, there must be a gen­uine de­sire to im­ple­ment and sup­port com­pas­sion­ate use pro­grams — and it must start at the top. Health­care com­pa­nies have a re­spon­si­bil­i­ty to en­able ac­cess to their in­no­v­a­tive med­i­cines to the pa­tients who need them most. At No­var­tis, we are com­mit­ted to help­ing en­sure that our med­i­cines are ac­ces­si­ble to as many pa­tients as pos­si­ble, ir­re­spec­tive of where they come from. We im­ple­ment com­pas­sion­ate use pro­grams be­cause it’s the right thing to do.

Ow­ing to the na­ture of the reg­u­la­to­ry and re­im­burse­ment process, there is of­ten a gap of sev­er­al months to years be­tween gen­er­a­tion of ro­bust ef­fi­ca­cy and safe­ty da­ta in tri­als and lo­cal mar­ket­ing au­tho­riza­tion with sub­se­quent lo­cal pa­tient ac­cess. Com­pas­sion­ate use pro­grams en­able the pro­vi­sion of cut­ting-edge ther­a­pies to those who des­per­ate­ly need them dur­ing this win­dow. For com­pa­nies, these pro­grams al­so pro­vide op­por­tu­ni­ties for the col­lec­tion of re­al-world da­ta from a broad­er pa­tient pop­u­la­tion be­yond the clin­i­cal tri­al, and an op­por­tu­ni­ty to en­gage with pa­tient com­mu­ni­ties, of­ten in­valu­able in rare dis­eases.

I be­lieve one of the main rea­sons for our high rate of com­pas­sion­ate use ap­provals is be­cause there’s an un­der­pin­ning phi­los­o­phy where the de­fault is to say ‘yes’, un­less there’s a jus­ti­fied med­ical or sci­en­tif­ic ra­tio­nale not to. Hav­ing this pa­tient-cen­tric mind­set in place and the will­ing­ness to de­ploy re­sources in this area is es­sen­tial.

In­still a ro­bust com­pa­ny pol­i­cy en­abled by an end-to-end re­quest sys­tem

A com­mit­ment to com­pas­sion­ate use means hav­ing the poli­cies, end-to-end process­es, and sys­tems in place to man­age re­quests in a prompt, fair and ef­fi­cient man­ner. The US 21st Cen­tu­ry Cures Act has since 2017 re­quired that phar­ma com­pa­nies de­vel­op­ing in­ves­ti­ga­tion­al drugs (in­clud­ing bi­o­log­ics) make their poli­cies re­gard­ing eval­u­at­ing and re­spond­ing to re­quests read­i­ly and pub­licly avail­able, e.g. on com­pa­ny web­sites.

A cen­tral­ized pol­i­cy and gov­er­nance pro­vide a sol­id foun­da­tion. We have a ded­i­cat­ed group in place act­ing as a Cen­ter of Ex­cel­lence, which has holis­tic over­sight and en­sures all el­e­ments are in place. This al­so in­cludes in­cor­po­rat­ing ad­e­quate guardrails in­to the process, the han­dling of ex­cep­tions, ad­dress­ing ini­tial pe­di­atric use and dos­ing ques­tions (e.g. man­aged with our in­te­grat­ed safe­ty as­sess­ment board), and the use of an ex­ter­nal in­de­pen­dent bioethics ad­vi­so­ry com­mit­tee (IBAC) for se­lect­ed eth­i­cal chal­lenges re­lat­ed to com­pas­sion­ate use.

With the in­creas­ing use of ge­net­ic test­ing and evo­lu­tion of tar­get­ed ther­a­pies, it is im­por­tant to con­sid­er how in­di­vid­ual pa­tient re­quests will be han­dled, es­pe­cial­ly for in­di­ca­tions where the com­pa­ny has no ac­tive or on­go­ing de­vel­op­ment pro­gram.

It’s al­so es­sen­tial to build a user-friend­ly com­pas­sion­ate use re­quest sys­tem — any dif­fi­cul­ties ac­cess­ing or us­ing the sys­tem, or lim­i­ta­tions to who can use it, are like­ly to de­ter treat­ing physi­cians and hin­der the re­ceipt of re­quests.

We launched an on­line re­quest sys­tem in De­cem­ber 2019 to stream­line re­quest man­age­ment. It’s sim­ple and can be used by physi­cians any­where in the world. Treat­ing physi­cians can sub­mit and han­dle a re­quest through any desk­top or mo­bile de­vice. In gen­er­al, it en­ables a speedy turn­around time — most re­view out­comes are pro­vid­ed with­in five work­ing days, en­sur­ing the pa­tient and treat­ing physi­cian do not lose a lot of time.

Part­ner with oth­ers and share your find­ings

We be­lieve open­ness and part­ner­ships are vi­tal pieces of the puz­zle. The da­ta from our JA­MA analy­sis was lim­it­ed to the ex­pe­ri­ence of a sin­gle com­pa­ny, and re­sults may dif­fer across oth­er or­ga­ni­za­tions. In light of this, we would wel­come sim­i­lar da­ta analy­sis from our in­dus­try peers and reg­u­la­tors to help in­form and en­rich the over­all knowl­edge base in this space.

There are on­go­ing best-prac­tice col­lab­o­ra­tions be­tween mul­ti­ple stake­hold­ers — in­clud­ing phar­ma com­pa­nies, pa­tient groups, reg­u­la­tors, gov­ern­ments, health­care pro­fes­sion­als, ven­dors and acad­e­mia — to en­sure that pa­tient ac­cess needs in com­pas­sion­ate use are ad­dressed holis­ti­cal­ly. It is im­por­tant to be a part of these con­ver­sa­tions and col­lab­o­ra­tions to en­sure di­verse per­spec­tives and ex­pe­ri­ences are con­sid­ered in the de­vel­op­ment of so­lu­tions to help ad­dress pa­tient ac­cess needs glob­al­ly.

There has been so much progress al­ready, but there’s still a lot that could be done to im­prove the im­ple­men­ta­tion of com­pas­sion­ate use pro­grams around the world. The best way for­ward is to­geth­er, for the ul­ti­mate ben­e­fit of pa­tients.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.

Andrew Phillips, Nexo Therapeutics CEO

Scoop: Ver­sant, NEA launch new biotech helmed by ex-CEO of pro­tein de­grad­er C4 Ther­a­peu­tics

Long-time biotech venture firms Versant and New Enterprise Associates are backing a new startup run by former C4 Therapeutics chief executive Andrew Phillips.

The fledgling biotech has raised at least $30 million so far, according to paperwork filed with the SEC this week. The round could balloon to $60 million.

Phillips, who left protein degradation startup C4 in 2020 to be a managing director at Cormorant Asset Management, is running the show of the new venture as president, the SEC filing outlines. He also served as interim CEO of Cormorant-backed and Hansoh Pharmaceutical-partnered Blossom Bioscience last year.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.