A new first-of-its-kind therapy is launching onto the U.S. marketplace with a costly price tag — and value-based deals with a handful of health insurers that should help with patient access while assuring them that they are paying for value, AIS Health reported.
On Aug. 10, the FDA approved Alnylam Pharmaceuticals, Inc.'s Onpattro (patisiran) for the treatment of adults with polyneuropathy caused by hereditary transthyretin-mediated (hATTR) amyloidosis. It is the first drug the agency has approved for this condition, as well as the first in a new class of drugs called small interfering ribonucleic acid (siRNA) treatment.
As knowledge around biomarkers grows, more drugs are coming onto the market with FDA-approved labels indicating their use with a particular diagnostic test. With most manufacturers nowadays choosing not to develop their own companion diagnostic but rather to partner with an outside firm, pharma companies need to be able to incorporate a diagnostic strategy into their drug development approach, AIS Health reported.
Previously some companies would develop both the drug and the diagnostic, but the number that now do so has declined. According to Amit Agarwal at Deloitte Consulting LLP, "diagnostic is a very different margin and business model than the pharmaceutical industry….The economics of it differ pretty dramatically."
Continuing the Trump administration's spate of actions aimed at lowering prescription drug costs, the FDA recently unveiled the 11-step "Biosimilar Action Plan" designed to help get more biosimilar drugs to the market that can compete with pricey biologics, AIS Health reported.
While the FDA has approved 11 biosimilars, only three are currently being marketed in the U.S., FDA Commissioner Scott Gottlieb said. "Competition is, for the most part, anemic," he concluded, adding that an FDA analysis showed Americans could have saved $4.5 billion in 2017 if all the approved biosimilars were available to consumers.
CMS recently denied a waiver request by Massachusetts to allow its Medicaid program, MassHealth, to use a closed formulary that excludes certain drugs. But the agency, in its June 27 decision, left the door open on the state's pursuit of tighter drug controls, AIS Health reported.
"The Massachusetts proposal brought to the table a new approach to increase its leverage to get lower drug prices, but an approach that left many unanswered questions," says Jack Hoadley, Ph.D., a research professor emeritus in Georgetown University’s Health Policy Institute. "Its rejection by CMS should not stop states from continuing to seek new ways to achieve lower prices. The key is how to balance maintaining access to needed drugs with tools to get lower prices."
For safety concerns, the FDA can require pharmacy manufacturers to have a Risk Evaluation and Mitigation Strategy (REMS) for a therapy in order to manage those risks while still allowing people to have access to it. Although the programs may present challenges to many stakeholders, manufacturers in particular may use them to their advantage, AIS Health reported.
One of the top challenges that manufacturers may grapple with is the "increasing complexity of developing and administering" REMS, says Glenn Carroll, principal at Deloitte. "Many of the REMS being developed today are more rigorous to implement than what we’ve seen in the past."
The FDA has multiple pathways for companies to get a review of promising drugs for conditions sorely in need of a treatment earlier in their development process than is typical, based on a surrogate endpoint. Following the FDA's approval of the first three gene therapies last year, the agency recently indicated that it will issue guidance on these products, including their potential approval based on surrogate measures. Yet this accelerated pathway holds challenges for manufacturers and payers, AIS Health reported.
The FDA has multiple programs that provide accelerated drug development and review: fast track designation, accelerated approval, breakthrough therapy designation and regenerative medicine advanced therapy (RMAT) designation, which was established in December 2016. The agency in November 2017 released draft guidance on applying the RMAT designation, which some gene therapies may qualify for.
The Trump administration has touted competition as one way to bring down drug prices. With this goal in mind, the FDA recently released a pair of draft guidance documents aimed at helping generics get onto the market sooner while still upholding the focus on safety, AIS Health reported.
One of the documents, titled Development of a Shared System Risk Evaluation and Mitigation Strategy (REMS) (83 Fed. Reg. 25468, June 1, 2018), offers recommendations for developing a shared system REMS for multiple drug products. The other, titled Waivers of the Single, Shared System REMS Requirement (83 Fed. Reg. 25465, June 1, 2018), describes situations in which the agency will waive the requirement that a generic drug and its reference product use a single, shared system (SSS) REMS with Elements to Assure Safe Use.
As more oncology immunotherapies enter the U.S. market, manufacturers are working to effectively compete and gain market share, according to AIS Health.
Stephen Cichy at Monarch Specialty Group, LLC, points out that the definition of cancer is changing. The increased use of predictive biomarkers is leading to “an increase in the number of personalized medicines that can specifically target unique cancer populations.”
A concerted effort is underway to develop hepatitis A and B vaccines and vaccines against human papilloma virus (HPV) to prevent the incidence of certain types of cancers, according to AIS Health .
Though the return on investment for any given vaccine-related venture is anything but assured, vaccine franchises are becoming a viable source of revenue for many companies. The overall world vaccines market is predicted to grow from $34 billion in 2017 to $49 billion by 2022, according to an August 2017 study from market-research company MarketsandMarkets.
The rise of CAR-T and other gene therapies, newly identified oncology targets and next-generation sequencing will shape the pipeline for 2018.
With the first CAR T-cell therapy approved, this new form of immunotherapy has a bright future. Yet drug companies also need to respond to worrisome side effects, such as severe cytokine release syndrome.