Happy New Year!
Welcome back. As we kick-off the season for transitions, renewed commitments, and overcrowded gyms (we swear we’re getting back on the wagon on that one), we ask a series of questions:
Check out our perspectives for these questions and more in The Week That Was!
h EXPANDED ACCESS: ARE YOU READY?
If the answer to that question is “no,” then put this high atop your New Year’s Resolutions list. Why? The 21st Century Cures Act was signed over the holidays and has many implications for drug makers, including a provision requiring them to make their Expanded Access Policy (EAP) “readily available.” If you are asking “what is an expanded access policy?”, let us assist you. Expanded access, also called “compassionate use,” provides a legal pathway for patients to gain access to investigational drugs for serious or terminal diseases or conditions. Under the newly signed Cures Act which champions the need to be pro-patient and pro-transparency, pharmaceuticals must disclose whether or not they will offer expanded access programs. However, the Cures Act does not require that companies make an investigational medicine available through an EAP. In fact, it explicitly states that there is no guarantee of access, and says companies can change policies at any time. But just last week, a Washington Post column by a member of the Right to Try movement – a campaign that calls for restricting the FDA’s oversight of expanded access programs − reminded us of the deep sensitivity of EAP. The piece eulogized a Right to Try advocate who lived longer with ALS because of his access to an experimental therapy.
If you are a developer of gene therapies, ultra-rare disease therapies or innovative oncology medicines – you should be paying attention to Right to Try (RTT). In 2016, the RTT movement successfully had thirty-some states pass model legislation supporting the right to try. While many of these bills are unenforceable, it creates confusion as to what rights patients have to an investigational medicine. Ensuring your policies and communications are clear, caring and in compliance with the law — should be a priority.
REPEAL AND ¯\_(ツ)_/¯
“Repeal and replace with something TERRIFIC,” was a major campaign promise by President-elect Trump. But, what he will repeal and replace the ACA with has become the hot question on Capitol Hill. Congressional Republicans have presented multiple plans to replace the Affordable Care Act, but have not yet settled on one set of policy proposals to substitute for the healthcare overhaul. Speaker Paul Ryan was questioned by journalists at a press conference last week about when the Republican party will have a clear plan for replacement. But with Senators like Bob Corker (R-TN) and Susan Collins (R-ME) signaling their desire to have a solid plan before repealing the ACA, the concept of “repeal and delay” has emerged as a potential reality. On the other hand, Senator Rand Paul tweeted over the weekend that the PEOTUS supports his plan that replacement must coincide with repeal. Depending on the timeline of a replacement, many Americans – – especially those who are covered through the Medicaid expansion or the individual insurance market – -could be left unsure about their coverage status. It also means that pharmaceutical companies may have to account for patients with even greater cost burdens beyond 2017, while at the same time, many patients may be left with inadequate coverage relative to their needs.
The timeframe for the ACA’s replacement is still anyone’s guess… taking certain elements out while keeping just the popular provisions is the equivalent of taking the foundational pieces out of a Jenga tower. In the meantime, millions of patients have opted into state exchanges that have high premiums, high deductibles and high coinsurance. For the biopharmaceutical industry, this means that patients will be more exposed to the costs of medicines on the road ahead – an important point to consider as you evaluate your 2017 patient assistance charities and copay programs.
SPINRAZA PRICING SENDS PRESS SPINNING
Biogen’s spinal muscular atrophy (SMA) drug, Spinraza, which won approval just before Christmas, is getting lots of attention for its recently released list price. The cost: $750,000 for the first year of use and $375,000 for every subsequent year thereafter. The reaction: serious sticker shock. While higher list prices are not unusual in the world of ultra-orphan drugs, the case of Spinraza raises important lessons that manufacturers should take note of for future pricing disclosures. Biogen’s pricing surprised many investors – with some claiming it was 333% higher than the experts expected. Other policy onlookers questioned whether Biogen opened Pandora’s Box with the ultra-rare designation, because the SMA patient population is in the thousands versus the typical hundreds. Reporters at outlets from Forbes to Fox questioned whether the PEOTUS would tweet about the drug’s price. The Spinraza spin cycle aligns with our proprietary data which shows a steep uptick on media stories on rare disease drug pricing over the last five years.
The response to Sprinraza’s pricing shines the spotlight on several issues. First, Biogen did not disclose the list price for Spinraza, nor did it provide thorough context for the therapy’s cost in its approval press release – creating a void of information for others to fill. Additionally, the company missed an opportunity to explain how the maintenance doses after discounts will indeed be in closer alignment to analyst expectations. Under the pressure of real-time decision-making, it can be challenging to provide this context when the clock is ticking. But to mitigate negative scrutiny surrounding pricing disclosures, you must be the first to report on the price of your drug… because if you don’t, someone like an analyst, patient or media outlet will happily do it for you.
Until next week,
– The Issues Management Practice @inVentiv Health PR
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