1. Congress


Energy and Commerce Subcommittee Schedules 340B Hearing

Earlier this week, House Energy & Commerce Oversight Subcommittee announced a hearing date has been set to discuss the 340B drug discount program on July 18. Hearing witnesses include Captain Krista Pedley, director of the Office of Pharmacy Affairs at the Health Resources and Services Administration; Debbie Draper, director of health care at the Government Accountability Office; and Erin Bliss, assistant inspector general with the Office of Evaluation and Inspections at the HHS Office of Inspector General. The hearing follows E&C Chairman Greg Walden (R-OR) and health subcommittee Chairman Mike Burgess’s (R-TX) letter addressing concern about program and lack of oversight.

Chairman Walden and Burgess’s letter can be found by clicking here.

For more information on the hearing, click here.

House Passes FDA User Fee Bill

On July 12, the FDA Reauthorization Act of 2017 passed the House floor by voice vote. The bill, H.R. 2430, seeks to extend user fee programs that fund the agency’s drug and medical device reviews for five years. The bill now heads to the Senate, and Senate HELP Chairman Lamar Alexander (R-TN) has said they plan to vote on the bill before the end of the month as it was “pre conferenced.” If the bill does not pass, August 1 the FDA will be required to notify employees of layoffs.

To view the FDA Reauthorization Act of 2017, please click here.

White House Raises Issues with House-Passed FDA User Fee Bill

The White House once again urged Congress to require drug and device companies to pay for the full cost of their FDA reviews, in a statement on the FDA Reauthorization Act, H.R. 2430, passed by the House Wednesday.

The administration has proposed that FDA receive more than $2 billion in medical product industry fees in fiscal year 2018. This would allow the government to cut FDA’s taxpayer financing, but still receive the same amount of money overall. But lawmakers and the industries opted not to change the fee amounts negotiated by the Obama administration, which would impose an additional cost on the drug and device industries.

The White House also took issue with portions of the bill it said would provide additional marketing exclusivity to manufacturers, saying that this would make exclusivity unpredictable and decrease competition. The administration also has a number of technical requests, including adjusting the foreign drug facility inspection fee.

The statement put out by the White House the day after the House had passed the bill did not threaten a veto. The Senate still needs to vote on the user fee package and is expected to do so before the August recess.

NIH Gets Appropriations Increase

The House Appropriations Labor-HHS Subcommittee released a bill earlier this week giving NIH a $1.1 billion raise for FY 2018. This would provide NIH with $35.2 billion next year and is $8.6 billion over what administration officials had requested. The bill also contains language mandating NIH reimburse grantee research institutions for facilities and other costs. This also goes against White House requests to reduce university funding for overhead expenses. Alzheimer’s research, the Cancer Moonshot and regenerative medicine also received substantial funding.

To view the bill’s language, click here.

FDA Funding Approved in House Appropriations Subcommittee

On July 12, the House Appropriations Subcommittee for Agriculture, Rural Development, Food and Drug Administration, and Related Agencies approved an increase of almost $500 million in annual funding for the FDA. The bill gives the FDA $5.1 billion for FY 2018 with $2.8 billion from taxpayer revenue and the rest from industry user fees. The approval comes after a tense hearing where Rep. Chris Stewart’s (R-UT) amendment to remove the FDA ban on drug compounding in doctor’s offices, but not pharmacies, was opposed by committee leaders who are looking for alternative avenues to address the issue.

To view the bill’s language, click here.

Home Infusion Legislation Introduced

House Ways & Means Health Subcommittee Chairman Pat Tiberi (R-OH) and Rep. Bill Pascrell (D-NJ) introduced legislation to create a transitional pay structure for home infusion services that would bridge a gap in reimbursement created by policies in the 21st Century Cures Act. The National Home Infusion Association says the bill’s introduction is an important step in getting the gap resolved.

The 21st Century Cures Act includes provisions meant to fix what the HHS Office of Inspector General said was a flawed benchmark for determining payment for those drugs. The Cures Act moved reimbursement from an average wholesale prices-based formula to the average sales price-based formula used for most other Part B drugs—ASP + 6 percent. That shift went into effect at the beginning of the year. However, a second provision in the bill, which provides reimbursement for the professional services associated with infusing the drugs, doesn’t go into effect until 2021. This created a reimbursement payment gap. The Medicare Part B Home Infusion Services Temporary Transitional Payment Act (H.R. 3161) creates a new transitional payment structure for providers of home infusion services before the 21st Century Cures Act provision takes effect in 2021.

Ways and Means Committee Reports Two Bills on Medicare

On July 14, the Ways and Means Committee approved two bills focused on improving Medicare.

Medicare Part B Improvement Act of 2017 (H.R. 3178), introduced by Ways and Means Committee Chairman Kevin Brady (R-TX) and Ranking Member Richard Neal (D-MA), makes a series of targeted improvements to Medicare Part B programs, including expanding access to in-home treatments for patients. The legislation includes several bipartisan ideas from members of the committee, including policies reflected in the following bills:

  • H.R. 3161, introduced by Health Subcommittee Chairman Pat Tiberi (R-OH) and Rep. Bill Pascrell (D-NJ), which creates a transition payment for home infusion therapies for Medicare beneficiaries to ensure there is no gap in care. To view the bill, click here.
  • H.R. 3172, introduced by Chairman Brady and Rep. Doris Matsui (D-CA), which extends a successful pilot program that allows Medicare beneficiaries with weakened immune systems to receive care in their homes. To view the bill, click here.
  • H.R. 3171, introduced by Reps. Mike Bishop (R-MI) and Mike Thompson (D-CA), which protects access to orthotics and prosthetics for Medicare beneficiaries who need them. To view the bill, click here.
  • H.R. 3166, introduced by Reps. Lynn Jenkins (R-KS) and John Lewis (D-GA), which improves the accreditation process for dialysis facilities so Medicare beneficiaries with chronic kidney disease living in rural communities can more easily access the treatments they need. To view the bill, click here.
  • H.R. 3164, introduced by Reps. Diane Black (R-TN), Suzan DelBene (D-WA), Mike Thompson and Pat Meehan (R-PA), which expands the use of telehealth technologies for Medicare beneficiaries receiving dialysis in their homes. To view the bill, click here.
  • H.R. 3173, introduced by Reps. Kenny Marchant (R-TX) and Ron Kind (D-WI), which puts into law existing regulations to modernize Medicare’s physician self-referral laws, known as “Stark laws.” To view the bill, click here.

Legislation to reauthorize Medicare Special Needs Plans (H.R. 3168), introduced by Health Subcommittee Chairman Tiberi and Ranking Member Sander Levin (D-MI), extends and strengthens Medicare Special Needs Plans (SNPs) to increase efficiency and improve access to high-quality health care for elderly patients living in poverty or with a chronic illness.

To view the markup, click here.


Senators Ask If the FDA Needs Additional Authority to Speed Up Recalls

On June 29, twelve senators sent a letter to FDA Commissioner Scott Gottlieb requesting information on the recent recall of faulty detection tests. The letter asked what additional authorization the agency needed and if the faulty tests were part of an expedited approval process. The senators asked why the FDA only acted in June when the issues had been identified three years ago and how the FDA coordinates with the Centers for Disease Control and Prevention; they also raised concerns that Magellan appeared to provide faulty information.

The letter comes after the FDA issued an alert in May on inaccurate lead detection results from Magellan and issued a recall in June over concerns that the test may not accurately represent lead levels in blood.

The letter can be found by clicking here.

Senate Releases New Bill to Repeal and Replace

On July 13, the Senate leadership released a revised version of the Better Care Reconciliation Act, legislation to repeal and replace the Affordable Care Act. The plan is to move the motion to proceed—a parliamentary step needed before beginning debate on the bill this week. It is unclear however if there are enough votes to pass the motion to proceed.

Revisions include:

  1. Out-of-Pocket Costs: An additional $70 billion is dedicated to driving state-based reforms, which could include help with driving down premiums through cost-sharing, health savings accounts (HSA) and other innovative ideas to help pay for health care costs. This is in addition to the $112 billion in funding already in the original bill.
  2. Health Savings Accounts: A provision has been included in the bill that would, for the first time, allow people to use their HSAs to pay for their premiums. This is a policy that the Joint Committee on Taxation says will increase health care coverage.
  3. Funding to Combat the Opioid Epidemic: An additional $45 billion is provided for substance abuse treatment and recovery.
  4. Catastrophic Plans, Higher Deductible Plans and Tax Credits: Individuals who enroll in catastrophic plans would be eligible for the tax credit so long as they meet other tax credit eligibility requirements. The ACA prohibited individuals enrolled in catastrophic plans from receiving a tax credit. Anyone in the individual market would be allowed to purchase a lower-premium health insurance plan with their federal tax credit assistance. These plans are higher deductible plans that cover three primary care visits a year and have federal protections that limit an individual’s out-of-pocket costs.
  5. Tax Revisions: The new draft bill will not include any changes from current law to the net investment income tax, the additional Medicare Health Insurance (HI) tax or the remuneration tax on executive compensation for certain health insurance executives.
  6. Medicaid Revisions: (a) the new draft discussion changes the DSH calculation from per Medicaid enrollee to per uninsured; (b) states may apply for a waiver for the purpose of continuing and/or improving home and community-based services for aged, blind and disabled populations; (c) if a public health emergency is declared, state medical assistance expenditures in a particular part of the state will not be counted toward the per capita caps or block grant allocations for the declared period of the emergency; and (d) states can use block grant option to allow states to also add expand Medicaid.
  7. Higher-Risk Individuals: Creates a fund for the purpose of making payments to specified health insurance issuers for the associated costs of covering high-risk individuals enrolled in the qualified health plans on the Affordable Care Act’s individual exchange. In order to qualify for such funds, an issuer must offer sufficient minimum coverage on the exchange that remains subject to Title 1 mandates. Offering such coverage would enable the issuer to also provide coverage off the exchange that would be exempt from certain Title 1 mandates.

To view the bill’s updated language, click here.

2. Administration

New Regulatory Scheme for Regenerative Medicine to Be Unveiled in September

On July 7, FDA Commissioner Scott Gottlieb signaled that the FDA will reveal a framework on how the FDA will regulate regenerative medicine through a succession of guidance reports that will make agency policies more efficient. The reports are aimed at helping manufacturers demonstrate safety and effectiveness. Further, the FDA’s biologics center is executing the new regenerative medicine advanced therapy (RMAT) designation to speed up development and the approval process for medical products and bring down costs. Gottlieb also noted that the FDA has approved four RMAT designation requests, which aims to speed certain cell therapies, engineering products and combination products to market.

CMS and Treasury Approve Alaska’s 1332 State Innovation Waiver

On July 11, the Treasury Department and Centers for Medicare & Medicaid Services (CMS) approved Alaska’s 1332 State Innovation Waiver application, which seeks to implement the Alaska Reinsurance Program (ARP) in future years in order to stabilize the individual health care market. This waiver allows the state to implement the ARP for the next five plan years, until Dec. 31, 2022. The ARP is a state-operated program that covers claims in the individual market for people with identified costly conditions, in order to stabilize premiums. The state believes the ARP will bring premiums down by 20 percent in 2018.

For more information about the Alaska 1332 waiver, click here.

Medicare Hospital Trust Fund Does Not Trigger IPAB

Medicare’s hospital trust fund is expected to run out of money in 2029, a year later than what trustees predicted in 2016, because national health spending continues to grow at a slower rate, the program’s trustees said in a new report published July 13. Medicare’s trustees predict that the program’s costs will grow from 3.6 percent of GDP to 5.6 percent of GDP by 2041, driven largely by enrollment. Projected spending rates are still lower than Medicare’s target level, meaning the Independent Payment Advisory Board (IPAB) created by the Affordable Care Act will remain dormant. IPAB was created to provide Congress with Medicare cuts should Medicare spending hit certain levels.

The trustees last year had warned that IPAB would be triggered this year for the first time since Obamacare’s passage. However, the new report states they anticipate that the panel won’t be activated until 2021. That would give lawmakers critical of the payment board significantly more time to eliminate it before it takes effect.

DOJ Cracks Down on Fraudulent Opioid Treatment Programs

On July 13, Attorney General Jeff Session announced the Justice Department would be enforcing sanctions against fraudulent opioid treatment programs. The action will charge 412 people, including 56 physicians, with defrauding the federal government of $1.3 billion. This announcement follows the settlement the Justice Department made with Mallinckrodt Pharmaceuticals, an opioid manufacturer. Mallinckrodt Pharmaceuticals has agreed to pay $35 million to settle charges that it failed to notify the Drug Enforcement Agency (DEA) of suspicious drug orders from 2008 to 2011.

To view the Justice Department’s press release, click here.

3. Courts

House v. Price: Republicans and DOJ Ask to Reject Democratic AGs’ Move to Intervene

Earlier this week, House Republicans and the Department of Justice filed separately to ask the D.C. Court of Appeals to reject a motion to intervene in the ACA cost-sharing reduction case, House v. Price. The motion was submitted by Democratic attorneys-general. House Republicans have argued that the attorneys-general lack standing. The Justice Department argues that the motion is speculative and that pending legislation in the Senate would fund CSRs and the case would be moot. In House v. Price , GOP Republicans charged the Obama administration for illegally providing cost-sharing payments without congressional appropriation. The GOP asked the appeals court to put aside the case after President Trump’s election, to consider alternative paths.

Suit Alleges CA Medicaid Payment Rates Violate Civil Rights

The Mexican American Legal Defense and Educational Fund, the Civil Rights Education and Enforcement Center and law firm Feinberg, Jackson, Worthman & Wasow have filed suit against the state of California for low Medicaid payment rates that violate civil rights of the 14 million enrolled in the program. The suit filed on July 12 asserts that Medi-Cal patients are not able to access care because of poor provider reimbursement rates and that rates have declined as Latino enrollment has climbed. Just last month, California lawmakers approved a budget deal that gives doctors and dentists who treat Medicaid patients a raise of $325 million and $180 million, respectively.

To view the complaint, click here.

To view California’s budget bill, click here.

4. Regulations Open for Comment

CMS Issues Proposed Revision Requirements for Long-Term Care Facilities’ Arbitration Agreements

On June 5, CMS issued proposed revisions to arbitration agreement requirements for long-term care facilities. The proposed revisions would help strengthen transparency in the arbitration process, reduce unnecessary provider burden and support residents’ rights to make informed decisions about important aspects of their health care.

The Reform of Requirements for Long-Term Care Facilities Final Rule, published on Oct. 4, 2016, listed the requirements facilities need to follow if they choose to ask residents to sign agreements for binding arbitration. The final rule also prohibited predispute agreements for binding arbitration. The American Health Care Association and a group of nursing homes sued for preliminary and permanent injunction to stop CMS from enforcing that requirement. The court granted a preliminary injunction on Nov. 7, 2016. After that decision, CMS reviewed and reconsidered the arbitration requirements in the 2016 Final Rule.

The proposed rule focuses on the transparency surrounding the arbitration process and includes the following proposals:

  • The prohibition on predispute binding arbitration agreements is removed.
  • All agreements for binding arbitration must be in plain language.
  • If signing the agreement for binding arbitration is a condition of admission into the facility, the language of the agreement must be in plain writing and in the admissions contract.
  • The agreement must be explained to the resident and his or her representative in a form and manner they understand, including that it must be in a language they understand.
  • The resident must acknowledge that he or she understands the agreement.
  • The agreement must not contain any language that prohibits or discourages the resident or anyone else from communicating with federal, state or local officials, including federal and state surveyors, other federal or state health department employees, or representatives of the State Long-Term Care Ombudsman.
  • If a facility resolves a dispute with a resident through arbitration, it must retain a copy of the signed agreement for binding arbitration and the arbitrator’s final decision so it can be inspected by CMS or its designee.
  • The facility must post a notice regarding its use of binding arbitration in an area that is visible to both residents and visitors.

This proposed rule is scheduled to be published in the Federal Register on June 8, 2017, and comments are due by Aug. 7, 2017. For more information, click here.

CMS Proposes MACRA Rule

On June 19, CMS issued a proposed rule that would make changes in the second year of the Quality Payment Program as required by the Medicare Access and CHIP Reauthorization Act of 2015 (MACRA).

The 1,058-page rule continues the “pick-your-pace” option in year two of the program, letting doctors report a limited amount of quality data to be exempted from Medicare’s penalties.

CMS creates a “virtual group” reporting option, allowing doctors to pool the information on how they care for patients and be subjected to Medicare’s quality payment scheme.

CMS is also increasing the minimum number of patients doctors can treat before being subject to the program’s Merit-based Incentive Payment System. It establishes more flexibility for doctors who see limited numbers of patients face to face or in a hospital. For 2017, roughly 800,000 clinicians were exempt from the MIPS program.

CMS will not require doctors to use 2015 certified EHRs next year, as it had ordered during the Obama administration. However, clinicians are offered bonuses for using new versions of the software. Medicare also will delay for another year judging doctors for how much they spend for treating patients.

Comments on the rule are due no later than 5 p.m. on Aug. 21, 2017. For a fact sheet on the proposed rule, click here.

CMS Proposes 2018 Policy and Payment Rate Changes for End-Stage Renal Disease Facilities

On June 29, the Centers for Medicare & Medicaid Services (CMS) issued a proposed rule that would update payment policies for the End-Stage Renal Disease (ESRD) Prospective Payment System (PPS). The rule covers payment rates for renal dialysis services, including updates to acute kidney injury (AKI), furnished to beneficiaries on or after Jan. 1, 2018.

The ESRD Quality Incentive Program (QIP) proposed changes are for payment years 2019, 2020 and 2021, and a number of key dialysis data methodologies and quality measures. The proposed rule also requests comment on how to include individuals with acute kidney injury in the ESRD Quality Improvement Program.

In addition to the proposed rule, CMS is releasing a request for information to welcome continued feedback on the Medicare program. CMS is committed to maintaining flexibility and efficiency throughout Medicare. Through transparency, flexibility, program simplification and innovation, CMS aims to transform the Medicare program and promote the availability of high-value and efficiently provided care for its beneficiaries.

Comments are due no later than 5 p.m. on Aug. 28, 2017.

For a fact sheet on the proposed rule, please click here.

For the ESRD proposed rule (CMS 1674-P), please click here.

CMS Proposes 2018 Policy and Rate Changes for Hospital Outpatient, Ambulatory Surgical Center Payment Systems

The Centers for Medicare & Medicaid Services (CMS) on July 13, issued a proposed rule that updates payment rates and policy changes in the Hospital Outpatient Prospective Payment System (OPPS) and Ambulatory Surgical Center (ASC) Payment System.

Among the provisions in this rule, CMS is proposing to change the payment rate for certain Medicare Part B drugs purchased by hospitals through the 340B program. The proposed rule also requests comment on how CMS can best implement the proposal to pass savings on to beneficiaries and providers, and to allow seniors to save money on their drug costs. The 340B Drug Pricing Program allows certain hospitals and other health care providers to purchase drugs and biologicals (other than vaccines) that are administered in a hospital outpatient department from drug manufacturers at discounted prices.

The proposed rule also includes a provision to address rural hospitals recruiting physicians by placing a two-year moratorium on the direct supervision requirement currently in place at rural hospitals and critical access hospitals.

In addition, CMS is releasing within the proposed rule a request for information to welcome continued feedback on flexibilities and efficiencies in the Medicare program.

Comments are due 5 p.m. Sept. 11, 2017.

To view a fact sheet on the proposed rule, click here.

To view the proposed rule, click here.

CMS Proposes 2018 Payment and Policy Updates for the Physician Fee Schedule

The Centers for Medicare & Medicaid Services (CMS) on July 13 issued a proposed rule that would update Medicare payment and policies for doctors and other clinicians who treat Medicare patients in calendar year (CY) 2018. This proposed rule seeks public comment on reducing administrative burdens for providing patient care, including visits, care management and telehealth services. The rule takes steps to better align incentives and provide clinicians with a smoother transition to the new Merit-based Incentive Payment System under the Quality Payment Program (QPP). The rule also attempts to encourage fairer competition between hospitals and physician practices by promoting greater payment alignment, and it would improve the payment for office-based behavioral health services that are often the therapy and counseling services used to treat opioid addiction and other substance use disorders. In addition, the proposed rule makes additional proposals to implement the Center for Medicare & Medicaid Innovation’s Medicare Diabetes Prevention Program expanded model starting in 2018.

In addition to the proposed rule, CMS is releasing a request for information to welcome continued feedback on the Medicare program. CMS is committed to maintaining flexibility and efficiency throughout Medicare. Through transparency, flexibility, program simplification and innovation, CMS aims to transform the Medicare program and promote the availability of high-value and efficiently provided care for its beneficiaries. This will inform the discussion on future regulatory action related to the Physician Fee Schedule. Comments are due by 5 p.m. on Sept. 11, 2017.

For a fact sheet on the proposed rule, click here.

To view the proposed rule, click here.

5. Reports

Hospital Value-Based Purchasing: CMS Should Take Steps to Ensure Lower-Quality Hospitals Do Not Qualify for Bonuses

Through the Hospital Value-Based Purchasing Program, hospitals that participate in Medicare receive bonuses or penalties, depending on their performance on a set of quality and efficiency measures.

Despite the program’s intention to reward hospitals that provide high-quality care at a lower cost, GAO found that some hospitals with low quality scores received bonuses because they had relatively high efficiency scores.

To ensure that lower-quality hospitals do not receive these bonuses, GAO recommended that the Centers for Medicare & Medicaid Services revise the methodology this program uses to calculate total performance scores.

To read the report, click here.

Gulf War Illness: Improvements Needed for VA to Better Understand, Process and Communicate Decisions on Claims

The Department of Veterans Affairs (VA) completed processing about 11,400 Gulf War Illness (GWI) claims in fiscal year 2015, which was more than double the 4,800 claims processed in fiscal year 2010. GWI is a collective term for certain medical conditions among veterans who have served in Southwest Asia since 1990. Symptoms of GWI can include joint pain, gastrointestinal problems, fatigue and neurological problems. On average, GWI claims have twice as many medical issues per claim as other disability claims, and take four months longer to complete. During fiscal years 2010 through 2015, the most recent data available at the time of our review, approval rates for GWI claims were about three times lower than for all other claimed disabilities. Several factors may contribute to lower approval rates, including that—according to VA—GWI claims are not always well understood by VA staff, and veterans sometimes file for benefits without medical records to adequately support their claim.

VA’s ability to accurately process GWI claims is hampered by inadequate training, and its decision letters for denied claims do not communicate key information to veterans. VA claims rating staff often rely on VA medical examiners to assess a veteran’s disability before a decision can be made on a claim. VA medical examiners told GAO that conducting Gulf War general medical exams is challenging because of the range of symptoms that could qualify as GWI. VA has developed elective GWI training for its medical examiners, but only 10 percent of examiners had taken the training as of February 2017. Federal internal control standards call for adequate training for staff so they can correctly carry out an agency’s procedures. Medical examiners who do not take this GWI-specific training may not be able to provide information to VA staff to correctly decide whether to grant a veteran’s claim. Once a determination is made, VA regulations also require clear explanations to veterans regarding claim decisions. GAO found that decision letters for GWI claims do not always include key information on why the claim was denied.

The VA considers research when adding to the list of conditions it associates with Gulf War service, but it does not have a plan to develop a uniformly used case definition of GWI. In 2010, VA added nine infectious diseases to the list of GWI-related conditions. VA advisory groups noted, however, that researchers face obstacles in conducting GWI research, including the lack of a single case definition of the illness for research and treatment purposes. In its 2015 Gulf War Research Strategic Plan, VA included an objective to develop a single case definition, but an official told GAO that VA had no action plan in place to achieve it. Without a plan to achieve a single case definition, research on and treatment for GWI may continue to progress slowly.

To read the report, click here.

Individual Insurance Market Performance in Early 2017

On July 10, the Kaiser Family Foundation released a study that shows the Obamacare markets are firming up and health plans are regaining profitability. The first quarter analysis shows that claims of slow growth for medical expenses contributed to financial improvements and hospitalization data shows the risk pool may not be getting progressively sicker. Nevertheless, Kaiser says there is uncertainty regarding future cost-sharing subsidies and enforcement of the Obamacare mandate could raise premiums. Senior Vice President Larry Levitt said that “loss ratios will definitely rise throughout the year. Individual market plans tend to have a pretty big deductible.”

Read the full study here.

Investigational New Drugs: FDA Has Taken Steps to Improve the Expanded Access Program but Should Further Clarify How Adverse Events Data Are Used

A new GAO report says the FDA should be clearer with drug and medical device companies about how safety events in patients using experimental medicines outside clinical trials could impact ultimate FDA approval. Greater clarity from FDA may lead more companies to be comfortable providing patients with such drugs. Overall, the GAO report praised FDA for taking steps to improve the expanded access process, highlighting a new website and easier forms for patients and doctors to request experimental medicines.

However, the GAO found only one document the FDA uses to communicate with industry that referenced how it will use adverse event data from expanded access programs. And the GAO felt that document was vague. Manufacturers told the GAO the lack of clarity can impact their decision on patient access. Companies are concerned that a safety event could mean the FDA halts the clinical trials with the drug—delaying the treatment’s development and FDA approval.

The GAO did note that FDA recently released new data showing that drugs used in compassionate use situations actually had better odds of approval. The study found no instance in which expanded access led to a drug’s being denied approval.

The GAO also looked at other steps state governments and private groups have taken to expand access to experimental medicines. It highlights two concerns with state “right-to-try” laws. First, the laws that allow patients to request drugs outside the FDA program don’t guarantee them access because they don’t compel manufacturers to provide them. Second, the laws might give patients false hope.

To view the report, click here.