As we look back over the past year, the COVID-19 pandemic has been top of mind for many involved in pharma commercialization and coverage. Less than a year after scientists sequenced the COVID genome, the FDA granted emergency use authorization to vaccines from Pfizer Inc./BioNTech SE and Moderna, Inc. in December — an unprecedented time frame for vaccine development, testing and rollout. Shortly thereafter, vaccinations started to be administered. But that wasn’t the only pharma industry story of 2020. AIS Health spoke with a variety of industry experts on various trends over the past year.
What were some of the most notable developments within the pharmaceutical industry in 2020, and why?
Dan Danielson, R.Ph., senior director of the access experience team at PRECISIONvalue: “Well, the vaccines and COVID have consumed most of the oxygen in the room. Accommodating to the new reality for the industry has been a challenge. How do you maintain contact with your customers in a socially distanced environment? How do you replace or adapt when you can’t be face to face? How do you make the most of your engagements?
“Precision did surveys to understand what payers were looking for in terms of information and how they wanted to receive that info. The short story is that while the kinds of information that payers desired didn’t change much, we did find that folks in the account manager (AM) role and medical/health economics roles had the most influence with customers. People in these roles could help channel customers to alternate sources of information, like social media or websites with managed access (passwords or access codes provided by the AM or MSL [i.e., medical science liaison]).”
Mike DeLone, principal and national sector leader for life sciences at Deloitte Consulting LLP: “Life sciences companies have
completely changed the way vaccines are developed.…The speed at which the vaccines were developed, approved and administered has been remarkable. We have also seen significant advancements in testing, logistics, supply chain, clinical trials and manufacturing. The knowledge that researchers and scientists have accumulated over the past year will create a foundation for advances that will unfold over the months, years and decades ahead. Also notable is the remarkable pivot that all industries, including life sciences, made to transform the work and innovation they do every day to a virtual workplace. The learning of this year, in all regards, will continue to transform how medicines are made and distributed in the future.”
Could you comment on the COVID-19 pandemic and its impact over the past year?
Danielson: “I don’t think you [can] underestimate the economic and societal impact that COVID-19 has. In the U.S., the overall economy has contracted from the pre-pandemic period, with consummate contraction in GDP [i.e., gross domestic product] and job losses on a scale most of us have not seen before.
“Lost jobs meant changes in insurance for many people. Most appear to have been able to transition from their former employers’ coverage to that of their spouse’s employer, those eligible may have joined Medicaid, [and] others lost coverage altogether. The hardest-hit sectors were, and are still, made up of lower-wage workers, many of which did not have employer-sponsored insurance and may have already been on Medicaid or already uninsured.
“Access to health care has been greatly changed; virtual medicine visits peaked during the months of the pandemic. In-office visits are conducted with full PPE [i.e., personal protective equipment]. Access to medications shifted as well. Mail-order shares increased as people locked down; at retail, 90-day supplies of prescriptions became common.
“Clinical trials either slowed down enrollments or delayed their start dates until 2021.”
DeLone: “While it’s quickly become an overused phrase, the pandemic has truly created a new normal — one that has seen life sciences companies pour more resources into science and technologies to achieve important breakthroughs and create novel partnerships. I’m feeling more optimistic than ever that science is progressing and that, because of the amazing work happening in the industry during the past year, we’ll be better prepared for the next event that has the potential to have as large an impact as COVID-19 has. While the pandemic has been a difficult and painful experience for so many, I believe that COVID has also acted as a light shining on a better way to work, collaborate and innovate.”
David Lassen, Pharm.D., chief clinical officer at Prime Therapeutics LLC: “The COVID-19 pandemic has created increased speed to market for scientific breakthroughs and vaccinations, owing to new cross-functional partnerships (government working with the private sector), new regulations and coordination of federal agencies (FDA, CMS, CDC) working to improve both the effectiveness and efficiency of therapeutic drug evaluations.
“The emergence of telehealth and home offices are here to stay. The marketplace has successfully shifted to a remote environment for many in the health care sector. This will create greater efficiencies for the provision of care through home diagnostics, digital therapies and wearables becoming more in demand as we migrate to a more mature telehealth environment.
“We’ve seen a heightened demand to reduce member and provider points of friction in health care. The care continuum will demand new ways of prescribing medications and treating patients with less administrative burdens, such as utilization management. Traditional approaches will be replaced by emerging capabilities to provide more information to the prescriber at the point of care — enabling transparency in drug costs, alternatives, benefit design and coverage limitations — to decrease the need for downstream actions necessary to achieve coverage for treatment.”
Could you comment on the race to find a vaccine?
Danielson: “The fact that the FDA has authorized these two vaccines that went from bench science to production in less than a year, while still heeding the needs for patient safety, is an amazing achievement.
“mRNA [i.e., messenger RNA] technology is at the core of the Pfizer and Moderna vaccines. That tech has been used in other therapy areas. For example, [Alnylam Pharmaceuticals, Inc.’s] Onpattro for polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adults is called a ‘gene silencing’ therapy in that it interferes with the production of mutated TTR proteins” (SMA 9/18, p. 11). “Similarly, the upcoming drug inclisiran (Leqvio) [from Novartis Pharmaceuticals Corp.] for management of LDL cholesterol…interferes with the production of PCSK9 proteins, essentially mimicking a gene knockout seen in people who have very low LDL cholesterol in their bloodstream.
“Instead of being a gene silencer like the two examples, in the vaccines the tech is used to cause the body to produce a protein found on the SARS-COV-2 virus (the spike protein). That protein is expressed on the cell surface, recognized as foreign to the body and stimulates an immune response.
“Seriously, this is wicked cool science.”
DeLone: “The development and approval of the COVID-19 vaccine has been nothing short of remarkable. While the speed has been unprecedented, stakeholders from across the globe will really need to collaborate to make sure these vaccines are available and accepted. I think there are still several questions that the industry is looking to answer now that the vaccine is being distributed, including how pricing will be determined and who will pay for it. In addition to the race to create a vaccine is the race to distribute and supply the vaccine to the world — almost as daunting/challenging (but not quite) as [challenging as] the development of the vaccine itself.”
Lassen: “The pace with which COVID-19 vaccine candidates have been developed is unprecedented. We saw the approval and launch of the Pfizer and Moderna vaccines in December 2020. Other manufacturers’ (e.g., AstraZeneca, J&J) vaccines will likely come to market in 2021. Most of the vaccine candidates will require two doses; however, a single-dose vaccine is also in development. Launching a single-dose vaccine could have an advantage in the marketplace especially if it offers similar or better safety and efficacy compared to the dual dose vaccines.
“Overall, the available efficacy data of the vaccines is very encouraging; however, certain key information will be unknown upon launch. This includes the durability of response and effectiveness in children. We, as a PBM, have engaged with manufacturers and regulatory agencies who in turn have been transparent with their data and development plan. Helping to manage the distribution channel and ensuring patients get the second dose of vaccine will be an important challenge for 2021.”
What were the most notable FDA approvals, and why?
Danielson: “For me the approval of [Seagen Inc.’s] Tukysa for metastatic HER2 positive breast cancer and [Immunomedics, Inc.’s] Trodelvy for triple negative breast cancer were big ones. I have two sisters-in-law who are breast cancer survivors, so they are kind of personal.
“In migraine, rimegepant [Biohaven Pharmaceuticals, Inc.’s Nurtec], an oral CGRP for acute migraine, was approved early in the year. It reminds us of the still unanswered question of how to manage chronic and episodic migraine: Do you use the same MOA [i.e., mechanism of action] for prevention as you do for treatment? Or should you use different MOAs?”
Lassen: “There were many approvals for rare conditions that previously had limited or no treatment options. These include:
- “Two new agents, [Roche’s] Enspryng and [Viela Bio’s] Uplizna, to treat neuromyelitis optica spectrum disorder (NMOSD). These agents will compete with Soliris [from Alexion Pharmaceuticals, Inc.] and off-label Rituxan [from Roche Group member Genentech USA, Inc. and Biogen] and at a lower cost compared to Soliris.
- “An orally administered drug for spinal muscular atrophy (SMA), Evrysdi [from Genentech], which will compete with [Biogen’s] Spinraza in this space” (SMA 10/5/20, p. 5).
- “Palforzia [from Aimmune Therapeutics, Inc.] was approved as the first medication for the mitigation of allergic reactions, including anaphylaxis that may occur with accidental exposure to peanut. It is approved for use in patients aged 4 to 17 with a confirmed diagnosis of peanut allergy. Prior to this approval, allergist-controlled desensitization was the only option available for patients with peanut allergy.
- “Zokinvy [from Eiger BioPharmaceuticals] is the first treatment FDA approved for Hutchinson-Gilford progeria syndrome and processing-
deficient progeroid laminopathies — ultra-rare genetic diseases that cause premature aging and death. Compared to untreated patients, Zokinvy reduces the risk of mortality in Hutchinson-Gilford progeria syndrome.
- “Oxlumo [from Alnylam] is the first FDA-approved treatment for primary hyperoxaluria type 1 (PH1). It is the only therapy that lowers harmful oxalate levels that cause the progression of PH1 disease” (see story, p. 6).
- “Imcivree [from Rhythm Pharmaceuticals, Inc.] is the first FDA-approved treatment for chronic weight management of obesity due to POMC, PCSK1 or LEPR deficiency. In Phase III trials, treatment with Imcivree resulted in 80% of patients with obesity due to POMC or PCSK1 deficiency achieving greater than 10% weight loss and 45.5% of patients with obesity due to LEPR deficiency achieving greater than 10% weight loss after one year of treatment.
- “Orladeyo [from BioCryst Pharmaceuticals, Inc.] is the first oral medication for the prevention of hereditary angioedema in adults and pediatric patients 12 years and older. The advantage of an oral option comes with the drawback of lesser efficacy than the self-administered subcutaneous injections including [Takeda Pharmaceutical Company Ltd.’s] Takhzyro and [CSL Behring GmbH’s] Haegarda.”
In addition, Gilead Sciences, Inc.’s “Veklury (remdesivir) was an expedited approval for COVID-19 treatment. This is used for in-patient treatment; thus, the majority of the claims will be seen on the medical benefit. However, it is notable based on the speed to market sparked by the current pandemic. And [Lundbeck’s] Vyepti for migraine prevention.”
Could you comment on merger and acquisition (M&A) activity that occurred in 2020 and has the potential to impact the pharma
Danielson: “In terms of M&A, I have some concerns about the vertical integration of insurers, PBMs, specialty pharmacies and provider groups. While full integration will take time, the idea of such a silo, potentially swaying access to medications to particular outlets or even driving therapy selection in a much more enforceable manner than just a health system formulary or treatment protocol, raises some concerns.
“My concerns generally revolve primarily around transparency in decision-making and access as well as influence on providers. In some ways this is a logical extension of the group model HMO but on a vastly larger scale, with wider implications and potential for unintended consequences for health care in the U.S. These are the same concerns I have about socialized/national health care. At this point, I just don’t know what trade-offs will be required as we ‘defragment’ the health care environment in the U.S. That said, I am hopeful that the benefits outweigh the risks.”
DeLone: “There was a very bullish outlook for the M&A pipeline in January of 2020, and while things slowed from March to May due to the pandemic, they really picked back up by July. Valuations stayed reasonably robust this year. We also saw companies doing a lot of tracking and sensing through new mechanisms to monitor for potential deals.”
Is there anything I’ve neglected to ask about that you’d like to add?
DeLone: “We should all be proud to be part of an industry that, coming from the very bottom of the list in terms of public opinion/sentiment, has stepped up, collaborated [and] brought the best scientific minds to bear to help find a path forward in arguably the most challenging health/pandemic of our lifetime.
“I think this momentum, from a year that we all are eager to have done, will carry forward towards a better industry moving forward.”